NCT03785964 · STUDY IN CONTEXT

Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF)

Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

Progression will be determined radiographically using RECIST v1.1 (Eisenhauer, 2009) or clinically as assessed by the investigator. Clinical progression is defined as the onset or worsening of symptoms resulting in a global deterioration of health status causing the permanent discontinuation from study treatment and the initiation of emergent treatment (e.g., radiotherapy, surgery, or systemic therapy including chemotherapy or tyrosine kinase inhibitors) for DT/AF. Events of clinical progression will be adjudicated by an independent blinded central Endpoint Adjudication Committee (EAC) which will qualify events of clinical progression for inclusion in the PFS endpoint prior to study unblinding according to an EAC Review Charter.

Intent-to-Treat Population (All Randomized)

→

secondary · POSTED

Objective response rate (ORR) is defined as the proportion of participants having a confirmed Best Overall Response (BOR) of CR or PR by central reader using RECIST v1.1 criteria. Responses obtained up until progression, or last evaluable assessment in the absence of progression, will be included in the assessment of ORR. However, any responses, which occurred after a new anticancer therapy was received, will not be included. ORR is presented by percentages of responders.

Intent-to-Treat Population (All Randomized)

→

secondary · POSTED

The Brief Pain Inventory consists of 9 questions and utilizes a 11-point Numerical pain Rating Scale from 0-10 measuring severity from "no pain" to "pain as bad as you can imagine," with a 24-hour recall period. Average Pain Intensity is calculated as the 7-day average (when results on at least 4 days for a VISIT are available) of Brief Pain Inventory Question #3 - Worst Pain in last 24 hours. A lower score indicates greater pain relief. The minimum and maximum of the actual score are (0, 8) for Nirogacestat and (0,9) for Placebo, respectively. A positive change from Baseline value indicates worsening of Average Pain Intensity and a negative change from Baseline value indicates improvement of Average Pain Intensity. The minimum and maximum of change from baseline score are (-7, 3) for Nirogacestat and (-5, 5) for Placebo, respectively.

Intent-to-Treat Population

→

secondary · POSTED

The DEsmoid Tumor Symptom Scale is an 11-point, numeric rating scale from 0 to 10 to measure severity from "none" to "as bad as you can imagine," with a 24-hour recall period. The Total Symptom Score is calculated as the mean of Pain items (Items 1-3) as a single score, then a mean of this with items 4-7). Weekly summary scores will be created by averaging the daily scores over the 7 days period prior to each visit. A weekly score will be derived only if 4 or more out of 7 days period have non-missing scores. The weekly summary score will be used in analyses. If no weekly summary score is calculable, the participant will have data considered as missing at that visit. Higher scores represent worse symptom severity. The minimum and maximum of the actual score are (0,7) for Nirogacestat and (0,10) for Placebo. A positive change from Baseline value indicated worsening of symptoms. The minimum and maximum of change from baseline are (-6,1) for Nirogacestat and (-4,5) for Placebo.

Intent-to-Treat Population

→

secondary · POSTED

The items are evaluated on a 5-point Likert Scale ranging from "none of the time" to "all of the time" to measure frequency, with a 7-day recall period. The Physical Function Domain Score are calculated as the average Item 01 Moving, Item 02 Reaching (Freq), Item 06 Vigorous Activity, Item 7 Moderate Activity, and Item 08 Accomplished Less. Higher scores represent worst impact severity. The minimum and maximum of the actual score are (1, 5) for Nirogacestat and (1,5) for Placebo, respectively. A positive change from baseline value indicates worsening impact and a negative change from baseline value indicates improvement in impact. The minimum and maximum of change from baseline score are (-3, 0) for Nirogacestat and (-1, 2) for Placebo, respectively.

Intent-to-Treat Population

→

secondary · POSTED

The EORTC Quality of Life Questionnaire-Core 30 version 3.0 was used with a 7-day recall period. It consists of 30 questions with all items scored 1 ("not at all") to 4 ("very much") except for the 2 items contributing to the global health status/QoL, which are scored 1 ("very poor") to 7 ("excellent"). The instrument yields the following scales:5 functional scales, 3 symptom scales, and a global health status/quality of life scale. A high score for the global health status/QoL represents a high QoL. The minimum and maximum of the actual score are (33, 100) for Nirogacestat and (8,92) for Placebo, respectively. A positive change from baseline indicated improvement of global health status and a negative change from baseline value indicated worsening of global health status. The minimum and maximum of change from baseline score are (-58, 67) for Nirogacestat and (-67, 42) for Placebo, respectively.

Intent-to-Treat Population

→

Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

Brief Pain Inventory · Change From Baseline at Cycle 10 in the Brief Pain Inventory (BPI) Average Pain Intensity (API) Score.

Score direction: Not stated by the study. Direction is never inferred from an instrument name.

release id: clinical-indications-draft-1
disease state id: ds-desmoid-fibromatosis
nct id: NCT03785964
qol key: qol-0002
source section id: NCT03785964-om-0002
source outcome index: 2
instrument: Brief Pain Inventory
instrument basis: matched 'brief pain inventory' in the study's own wording
scale title: Change From Baseline at Cycle 10 in the Brief Pain Inventory (BPI) Average Pain Intensity (API) Score.
measure definition: The Brief Pain Inventory consists of 9 questions and utilizes a 11-point Numerical pain Rating Scale from 0-10 measuring severity from "no pain" to "pain as bad as you can imagine," with a 24-hour recall period. Average Pain Intensity is calculated as the 7-day average (when results on at least 4 days for a VISIT are available) of Brief Pain Inventory Question #3 - Worst Pain in last 24 hours. A lower score indicates greater pain relief. The minimum and maximum of the actual score are (0, 8) for Nirogacestat and (0,9) for Placebo, respectively. A positive change from Baseline value indicates worsening of Average Pain Intensity and a negative change from Baseline value indicates improvement of Average Pain Intensity. The minimum and maximum of change from baseline score are (-7, 3) for Nirogacestat and (-5, 5) for Placebo, respectively.
time frame: Daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role: secondary
reporting status: POSTED
higher is better: Source null
direction quote: Source null
population note: Intent-to-Treat Population
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z

Open the exact result →

Patient-reported symptom or function measure · Change From Baseline at Cycle 10 in the GOunder/Desmoid Tumor Research Tumor Foundation (DTRF) DEsmoid Tumor Symptom Scale (DTSS) - Total Score

Score direction: Lower is better, according to the source. Direction is never inferred from an instrument name.

release id: clinical-indications-draft-1
disease state id: ds-desmoid-fibromatosis
nct id: NCT03785964
qol key: qol-0003
source section id: NCT03785964-om-0003
source outcome index: 3
instrument: Patient-reported symptom or function measure
instrument basis: matched 'symptom score' in the study's own wording
scale title: Change From Baseline at Cycle 10 in the GOunder/Desmoid Tumor Research Tumor Foundation (DTRF) DEsmoid Tumor Symptom Scale (DTSS) - Total Score
measure definition: The DEsmoid Tumor Symptom Scale is an 11-point, numeric rating scale from 0 to 10 to measure severity from "none" to "as bad as you can imagine," with a 24-hour recall period. The Total Symptom Score is calculated as the mean of Pain items (Items 1-3) as a single score, then a mean of this with items 4-7). Weekly summary scores will be created by averaging the daily scores over the 7 days period prior to each visit. A weekly score will be derived only if 4 or more out of 7 days period have non-missing scores. The weekly summary score will be used in analyses. If no weekly summary score is calculable, the participant will have data considered as missing at that visit. Higher scores represent worse symptom severity. The minimum and maximum of the actual score are (0,7) for Nirogacestat and (0,10) for Placebo. A positive change from Baseline value indicated worsening of symptoms. The minimum and maximum of change from baseline are (-6,1) for Nirogacestat and (-4,5) for Placebo.
time frame: Daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role: secondary
reporting status: POSTED
higher is better: 0
direction quote: Higher scores represent worse
population note: Intent-to-Treat Population
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z

Open the exact result →

EORTC QLQ · Change From Baseline at Cycle10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Global Health Status/Quality of Life (GHS/QoL) Scale.

Score direction: Not stated by the study. Direction is never inferred from an instrument name.

release id: clinical-indications-draft-1
disease state id: ds-desmoid-fibromatosis
nct id: NCT03785964
qol key: qol-0005
source section id: NCT03785964-om-0005
source outcome index: 5
instrument: EORTC QLQ
instrument basis: matched 'eortc' in the study's own wording
scale title: Change From Baseline at Cycle10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Global Health Status/Quality of Life (GHS/QoL) Scale.
measure definition: The EORTC Quality of Life Questionnaire-Core 30 version 3.0 was used with a 7-day recall period. It consists of 30 questions with all items scored 1 ("not at all") to 4 ("very much") except for the 2 items contributing to the global health status/QoL, which are scored 1 ("very poor") to 7 ("excellent"). The instrument yields the following scales:5 functional scales, 3 symptom scales, and a global health status/quality of life scale. A high score for the global health status/QoL represents a high QoL. The minimum and maximum of the actual score are (33, 100) for Nirogacestat and (8,92) for Placebo, respectively. A positive change from baseline indicated improvement of global health status and a negative change from baseline value indicated worsening of global health status. The minimum and maximum of change from baseline score are (-58, 67) for Nirogacestat and (-67, 42) for Placebo, respectively.
time frame: Last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role: secondary
reporting status: POSTED
higher is better: Source null
direction quote: Source null
population note: Intent-to-Treat Population
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z

Open the exact result →

EORTC QLQ · Change From Baseline at Cycle 10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Physical Functioning

Score direction: Not stated by the study. Direction is never inferred from an instrument name.

release id: clinical-indications-draft-1
disease state id: ds-desmoid-fibromatosis
nct id: NCT03785964
qol key: qol-0006
source section id: NCT03785964-om-0006
source outcome index: 6
instrument: EORTC QLQ
instrument basis: matched 'eortc' in the study's own wording
scale title: Change From Baseline at Cycle 10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Physical Functioning
measure definition: The EORTC Quality of Life Questionnaire-Core 30 version 3.0 was used with a 7-day recall period. It consists of 30 questions with all items scored 1 ("not at all") to 4 ("very much") except for the 2 items contributing to the global health status/QoL, which are scored 1 ("very poor") to 7 ("excellent"). The instrument yields the following scales:5 functional scales, 3 symptom scales, and a global health status/quality of life scale. A high score for a Physical functional scale represents a high/healthy level of functioning. The minimum and maximum of the actual score are (27, 100) for Nirogacestat and (7,100) for Placebo, respectively. A positive change from baseline indicated improvement of global health status and a negative change from baseline value indicated worsening of physical functioning scores. The minimum and maximum of change from baseline score are (-7, 40) for Nirogacestat and (-40, 27) for Placebo, respectively.
time frame: Last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role: secondary
reporting status: POSTED
higher is better: Source null
direction quote: Source null
population note: Intent-to-Treat Population
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z

Open the exact result →

EORTC QLQ · Change From Baseline at Cycle 10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Role Functioning.

Score direction: Not stated by the study. Direction is never inferred from an instrument name.

release id: clinical-indications-draft-1
disease state id: ds-desmoid-fibromatosis
nct id: NCT03785964
qol key: qol-0007
source section id: NCT03785964-om-0007
source outcome index: 7
instrument: EORTC QLQ
instrument basis: matched 'eortc' in the study's own wording
scale title: Change From Baseline at Cycle 10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Role Functioning.
measure definition: The EORTC Quality of Life Questionnaire-Core 30 version 3.0 was used with a 7-day recall period. It consists of 30 questions with all items scored 1 ("not at all") to 4 ("very much") except for the 2 items contributing to the global health status/QoL, which are scored 1 ("very poor") to 7 ("excellent"). The instrument yields the following scales:5 functional scales, 3 symptom scales, and a global health status/quality of life scale. A positive change from baseline indicated improvement of global health status and a negative change from baseline value indicated worsening of global health status and functioning scores. The minimum and maximum of change from baseline score are (-17, 83) for Nirogacestat and (-100, 50) for Placebo, respectively.
time frame: Last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role: secondary
reporting status: POSTED
higher is better: Source null
direction quote: Source null
population note: Intent-to-Treat Population
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z

Open the exact result →

Full draft applicability and source-link record

trial

release id: clinical-indications-draft-1
disease state id: ds-desmoid-fibromatosis
nct id: NCT03785964
trial label: Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF)
applicability: narrower
applicability note: Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z
provenance class: regulator-cited
discovery note: Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
outcome Count: 6
harms Count: 2
qol Count: 5

outcomes

release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0000
source section id
NCT03785964-om-0000
source outcome index
0
endpoint
Progression Free Survival (PFS) Defined as the Time From Randomization Until Date of Assessment of Progression or Death by Any Cause.
endpoint definition
Progression will be determined radiographically using RECIST v1.1 (Eisenhauer, 2009) or clinically as assessed by the investigator. Clinical progression is defined as the onset or worsening of symptoms resulting in a global deterioration of health status causing the permanent discontinuation from study treatment and the initiation of emergent treatment (e.g., radiotherapy, surgery, or systemic therapy including chemotherapy or tyrosine kinase inhibitors) for DT/AF. Events of clinical progression will be adjudicated by an independent blinded central Endpoint Adjudication Committee (EAC) which will qualify events of clinical progression for inclusion in the PFS endpoint prior to study unblinding according to an EAC Review Charter.
time origin
On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, assessed up to approximately 2 years
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
Intent-to-Treat Population (All Randomized)
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0001
source section id
NCT03785964-om-0001
source outcome index
1
endpoint
Objective Response Rate Using RECIST Version 1.1 Criteria.
endpoint definition
Objective response rate (ORR) is defined as the proportion of participants having a confirmed Best Overall Response (BOR) of CR or PR by central reader using RECIST v1.1 criteria. Responses obtained up until progression, or last evaluable assessment in the absence of progression, will be included in the assessment of ORR. However, any responses, which occurred after a new anticancer therapy was received, will not be included. ORR is presented by percentages of responders.
time origin
On the first day of every 3 cycles (each cycle is 28 days) through study completion, an average of 2 years
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Intent-to-Treat Population (All Randomized)
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0002
source section id
NCT03785964-om-0002
source outcome index
2
endpoint
Change From Baseline at Cycle 10 in the Brief Pain Inventory (BPI) Average Pain Intensity (API) Score.
endpoint definition
The Brief Pain Inventory consists of 9 questions and utilizes a 11-point Numerical pain Rating Scale from 0-10 measuring severity from "no pain" to "pain as bad as you can imagine," with a 24-hour recall period. Average Pain Intensity is calculated as the 7-day average (when results on at least 4 days for a VISIT are available) of Brief Pain Inventory Question #3 - Worst Pain in last 24 hours. A lower score indicates greater pain relief. The minimum and maximum of the actual score are (0, 8) for Nirogacestat and (0,9) for Placebo, respectively. A positive change from Baseline value indicates worsening of Average Pain Intensity and a negative change from Baseline value indicates improvement of Average Pain Intensity. The minimum and maximum of change from baseline score are (-7, 3) for Nirogacestat and (-5, 5) for Placebo, respectively.
time origin
Daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Intent-to-Treat Population
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0003
source section id
NCT03785964-om-0003
source outcome index
3
endpoint
Change From Baseline at Cycle 10 in the GOunder/Desmoid Tumor Research Tumor Foundation (DTRF) DEsmoid Tumor Symptom Scale (DTSS) - Total Score
endpoint definition
The DEsmoid Tumor Symptom Scale is an 11-point, numeric rating scale from 0 to 10 to measure severity from "none" to "as bad as you can imagine," with a 24-hour recall period. The Total Symptom Score is calculated as the mean of Pain items (Items 1-3) as a single score, then a mean of this with items 4-7). Weekly summary scores will be created by averaging the daily scores over the 7 days period prior to each visit. A weekly score will be derived only if 4 or more out of 7 days period have non-missing scores. The weekly summary score will be used in analyses. If no weekly summary score is calculable, the participant will have data considered as missing at that visit. Higher scores represent worse symptom severity. The minimum and maximum of the actual score are (0,7) for Nirogacestat and (0,10) for Placebo. A positive change from Baseline value indicated worsening of symptoms. The minimum and maximum of change from baseline are (-6,1) for Nirogacestat and (-4,5) for Placebo.
time origin
Daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Intent-to-Treat Population
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0004
source section id
NCT03785964-om-0004
source outcome index
4
endpoint
Change From Baseline in the GOunder/Desmoid Tumor Research Foundation (DTRF) DEsmoid Tumor Impact Scale (DTIS) - Physical Functioning Domain Score
endpoint definition
The items are evaluated on a 5-point Likert Scale ranging from "none of the time" to "all of the time" to measure frequency, with a 7-day recall period. The Physical Function Domain Score are calculated as the average Item 01 Moving, Item 02 Reaching (Freq), Item 06 Vigorous Activity, Item 7 Moderate Activity, and Item 08 Accomplished Less. Higher scores represent worst impact severity. The minimum and maximum of the actual score are (1, 5) for Nirogacestat and (1,5) for Placebo, respectively. A positive change from baseline value indicates worsening impact and a negative change from baseline value indicates improvement in impact. The minimum and maximum of change from baseline score are (-3, 0) for Nirogacestat and (-1, 2) for Placebo, respectively.
time origin
On the last day of every cycle (each cycle is 28 days) through study completion, average of 2 years.
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Intent-to-Treat Population
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0005
source section id
NCT03785964-om-0005
source outcome index
5
endpoint
Change From Baseline at Cycle10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Global Health Status/Quality of Life (GHS/QoL) Scale.
endpoint definition
The EORTC Quality of Life Questionnaire-Core 30 version 3.0 was used with a 7-day recall period. It consists of 30 questions with all items scored 1 ("not at all") to 4 ("very much") except for the 2 items contributing to the global health status/QoL, which are scored 1 ("very poor") to 7 ("excellent"). The instrument yields the following scales:5 functional scales, 3 symptom scales, and a global health status/quality of life scale. A high score for the global health status/QoL represents a high QoL. The minimum and maximum of the actual score are (33, 100) for Nirogacestat and (8,92) for Placebo, respectively. A positive change from baseline indicated improvement of global health status and a negative change from baseline value indicated worsening of global health status. The minimum and maximum of change from baseline score are (-58, 67) for Nirogacestat and (-67, 42) for Placebo, respectively.
time origin
Last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Intent-to-Treat Population
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

arms

release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0000
arm key
OG000
arm label
Double-Blind Phase - Nirogacestat
regimen
Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet
analyzed count
70
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0000#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0000
arm key
OG001
arm label
Double-Blind Phase - Placebo
regimen
Placebo by mouth, twice daily Placebo Oral Tablet: Sugar pill manufactured to mimic nirogacestat 50 mg tablet
analyzed count
72
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0000#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0001
arm key
OG000
arm label
Double-Blind Phase - Nirogacestat
regimen
Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet
analyzed count
70
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0001#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0001
arm key
OG001
arm label
Double-Blind Phase - Placebo
regimen
Placebo by mouth, twice daily Placebo Oral Tablet: Sugar pill manufactured to mimic nirogacestat 50 mg tablet
analyzed count
72
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0001#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0002
arm key
OG000
arm label
Double-Blind Phase - Nirogacestat
regimen
Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet
analyzed count
70
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0002#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0002
arm key
OG001
arm label
Double-Blind Phase - Placebo
regimen
Placebo by mouth, twice daily Placebo Oral Tablet: Sugar pill manufactured to mimic nirogacestat 50 mg tablet
analyzed count
72
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0002#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0003
arm key
OG000
arm label
Double-Blind Phase - Nirogacestat
regimen
Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet
analyzed count
70
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0003#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0003
arm key
OG001
arm label
Double-Blind Phase - Placebo
regimen
Placebo by mouth, twice daily Placebo Oral Tablet: Sugar pill manufactured to mimic nirogacestat 50 mg tablet
analyzed count
72
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0003#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0004
arm key
OG000
arm label
Double-Blind Phase - Nirogacestat
regimen
Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet
analyzed count
70
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0004#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0004
arm key
OG001
arm label
Double-Blind Phase - Placebo
regimen
Placebo by mouth, twice daily Placebo Oral Tablet: Sugar pill manufactured to mimic nirogacestat 50 mg tablet
analyzed count
72
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0004#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0005
arm key
OG000
arm label
Double-Blind Phase - Nirogacestat
regimen
Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet
analyzed count
70
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0005#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
outcome key
om-0005
arm key
OG001
arm label
Double-Blind Phase - Placebo
regimen
Placebo by mouth, twice daily Placebo Oral Tablet: Sugar pill manufactured to mimic nirogacestat 50 mg tablet
analyzed count
72
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03785964-om-0005#groups/OG001

harms

release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
harm key
EG000
source section id
NCT03785964-ae
event group key
EG000
event group label
Double-Blind Phase - Nirogacestat
serious affected
14
serious at risk
69
other affected
69
other at risk
69
frequency threshold
5
collection period
All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment, an average of 1 years and 4 months and up to 2 years and 10 months. Medical occurrences that begin before the start of study treatment but after obtaining informed consent will be recorded as AEs.
event definition
One patient randomized to Nirogacestat arm, discontinued prior to receiving any study drug.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
harm key
EG001
source section id
NCT03785964-ae
event group key
EG001
event group label
Double-Blind Phase - Placebo
serious affected
8
serious at risk
72
other affected
69
other at risk
72
frequency threshold
5
collection period
All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment, an average of 1 years and 4 months and up to 2 years and 10 months. Medical occurrences that begin before the start of study treatment but after obtaining informed consent will be recorded as AEs.
event definition
One patient randomized to Nirogacestat arm, discontinued prior to receiving any study drug.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

quality of life

release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
qol key
qol-0002
source section id
NCT03785964-om-0002
source outcome index
2
instrument
Brief Pain Inventory
instrument basis
matched 'brief pain inventory' in the study's own wording
scale title
Change From Baseline at Cycle 10 in the Brief Pain Inventory (BPI) Average Pain Intensity (API) Score.
measure definition
The Brief Pain Inventory consists of 9 questions and utilizes a 11-point Numerical pain Rating Scale from 0-10 measuring severity from "no pain" to "pain as bad as you can imagine," with a 24-hour recall period. Average Pain Intensity is calculated as the 7-day average (when results on at least 4 days for a VISIT are available) of Brief Pain Inventory Question #3 - Worst Pain in last 24 hours. A lower score indicates greater pain relief. The minimum and maximum of the actual score are (0, 8) for Nirogacestat and (0,9) for Placebo, respectively. A positive change from Baseline value indicates worsening of Average Pain Intensity and a negative change from Baseline value indicates improvement of Average Pain Intensity. The minimum and maximum of change from baseline score are (-7, 3) for Nirogacestat and (-5, 5) for Placebo, respectively.
time frame
Daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
Intent-to-Treat Population
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
qol key
qol-0003
source section id
NCT03785964-om-0003
source outcome index
3
instrument
Patient-reported symptom or function measure
instrument basis
matched 'symptom score' in the study's own wording
scale title
Change From Baseline at Cycle 10 in the GOunder/Desmoid Tumor Research Tumor Foundation (DTRF) DEsmoid Tumor Symptom Scale (DTSS) - Total Score
measure definition
The DEsmoid Tumor Symptom Scale is an 11-point, numeric rating scale from 0 to 10 to measure severity from "none" to "as bad as you can imagine," with a 24-hour recall period. The Total Symptom Score is calculated as the mean of Pain items (Items 1-3) as a single score, then a mean of this with items 4-7). Weekly summary scores will be created by averaging the daily scores over the 7 days period prior to each visit. A weekly score will be derived only if 4 or more out of 7 days period have non-missing scores. The weekly summary score will be used in analyses. If no weekly summary score is calculable, the participant will have data considered as missing at that visit. Higher scores represent worse symptom severity. The minimum and maximum of the actual score are (0,7) for Nirogacestat and (0,10) for Placebo. A positive change from Baseline value indicated worsening of symptoms. The minimum and maximum of change from baseline are (-6,1) for Nirogacestat and (-4,5) for Placebo.
time frame
Daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role
secondary
reporting status
POSTED
higher is better
0
direction quote
Higher scores represent worse
population note
Intent-to-Treat Population
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
qol key
qol-0005
source section id
NCT03785964-om-0005
source outcome index
5
instrument
EORTC QLQ
instrument basis
matched 'eortc' in the study's own wording
scale title
Change From Baseline at Cycle10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Global Health Status/Quality of Life (GHS/QoL) Scale.
measure definition
The EORTC Quality of Life Questionnaire-Core 30 version 3.0 was used with a 7-day recall period. It consists of 30 questions with all items scored 1 ("not at all") to 4 ("very much") except for the 2 items contributing to the global health status/QoL, which are scored 1 ("very poor") to 7 ("excellent"). The instrument yields the following scales:5 functional scales, 3 symptom scales, and a global health status/quality of life scale. A high score for the global health status/QoL represents a high QoL. The minimum and maximum of the actual score are (33, 100) for Nirogacestat and (8,92) for Placebo, respectively. A positive change from baseline indicated improvement of global health status and a negative change from baseline value indicated worsening of global health status. The minimum and maximum of change from baseline score are (-58, 67) for Nirogacestat and (-67, 42) for Placebo, respectively.
time frame
Last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
Intent-to-Treat Population
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
qol key
qol-0006
source section id
NCT03785964-om-0006
source outcome index
6
instrument
EORTC QLQ
instrument basis
matched 'eortc' in the study's own wording
scale title
Change From Baseline at Cycle 10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Physical Functioning
measure definition
The EORTC Quality of Life Questionnaire-Core 30 version 3.0 was used with a 7-day recall period. It consists of 30 questions with all items scored 1 ("not at all") to 4 ("very much") except for the 2 items contributing to the global health status/QoL, which are scored 1 ("very poor") to 7 ("excellent"). The instrument yields the following scales:5 functional scales, 3 symptom scales, and a global health status/quality of life scale. A high score for a Physical functional scale represents a high/healthy level of functioning. The minimum and maximum of the actual score are (27, 100) for Nirogacestat and (7,100) for Placebo, respectively. A positive change from baseline indicated improvement of global health status and a negative change from baseline value indicated worsening of physical functioning scores. The minimum and maximum of change from baseline score are (-7, 40) for Nirogacestat and (-40, 27) for Placebo, respectively.
time frame
Last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
Intent-to-Treat Population
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-desmoid-fibromatosis
nct id
NCT03785964
qol key
qol-0007
source section id
NCT03785964-om-0007
source outcome index
7
instrument
EORTC QLQ
instrument basis
matched 'eortc' in the study's own wording
scale title
Change From Baseline at Cycle 10 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Role Functioning.
measure definition
The EORTC Quality of Life Questionnaire-Core 30 version 3.0 was used with a 7-day recall period. It consists of 30 questions with all items scored 1 ("not at all") to 4 ("very much") except for the 2 items contributing to the global health status/QoL, which are scored 1 ("very poor") to 7 ("excellent"). The instrument yields the following scales:5 functional scales, 3 symptom scales, and a global health status/quality of life scale. A positive change from baseline indicated improvement of global health status and a negative change from baseline value indicated worsening of global health status and functioning scores. The minimum and maximum of change from baseline score are (-17, 83) for Nirogacestat and (-100, 50) for Placebo, respectively.
time frame
Last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
Intent-to-Treat Population
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

setting source links

Preserved source evidence · Not medical advice