TriangleNCT03785964 · OUTCOME
Change From Baseline in the GOunder/Desmoid Tumor Research Foundation (DTRF) DEsmoid Tumor Impact Scale (DTIS) - Physical Functioning Domain Score
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
- Measure
- LEAST_SQUARES_MEAN
- Unit
- score on a scale
- Interval / dispersion
- 95% Confidence Interval
- Time frame
- On the last day of every cycle (each cycle is 28 days) through study completion, average of 2 years.
What was measured
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.
Analysis population: Intent-to-Treat Population
Groups in this outcome
Double-Blind Phase - Nirogacestat
Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet
Double-Blind Phase - Placebo
Placebo by mouth, twice daily Placebo Oral Tablet: Sugar pill manufactured to mimic nirogacestat 50 mg tablet
| Group | Source count |
|---|---|
| Double-Blind Phase - Nirogacestat | 70 |
| Double-Blind Phase - Placebo | 72 |
Reported measurements
Source class 1
| Group | Value | Spread | Lower | Upper | Comment |
|---|---|---|---|---|---|
| Double-Blind Phase - Nirogacestat | -0.613 | Not reported | -0.808 | -0.418 | Not reported |
| Double-Blind Phase - Placebo | 0.094 | Not reported | -0.113 | 0.300 | Not reported |
Source statistical analyses
- group Description
- Mixed model with repeated measures (MMRM) with treatment and visit as factors, Baseline score and primary tumor location (intra-abdominal or extra-abdominal) as covariates, included baseline by visit and treatment by visit interactions. Only participants with a Baseline and at least one post-baseline score were included in the analysis. 39 and 28 participants contributed to this analysis at Cycle 10 from Nirogacestat and Placebo respectively.
- group Ids
- OG000
- OG001
- non Inferiority Type
- SUPERIORITY
- p Value
- <0.001
- statistical Comment
- An unstructured covariance structure was used and degrees of freedom were estimated using the Kenward-Roger approximation.
- statistical Method
- Mixed Models Analysis
Complete source fields
- analyses
- group Description
- Mixed model with repeated measures (MMRM) with treatment and visit as factors, Baseline score and primary tumor location (intra-abdominal or extra-abdominal) as covariates, included baseline by visit and treatment by visit interactions. Only participants with a Baseline and at least one post-baseline score were included in the analysis. 39 and 28 participants contributed to this analysis at Cycle 10 from Nirogacestat and Placebo respectively.
- group Ids
- OG000
- OG001
- non Inferiority Type
- SUPERIORITY
- p Value
- <0.001
- statistical Comment
- An unstructured covariance structure was used and degrees of freedom were estimated using the Kenward-Roger approximation.
- statistical Method
- Mixed Models Analysis
- classes
- categories
- measurements
- group Id
- OG000
- lower Limit
- -0.808
- upper Limit
- -0.418
- value
- -0.613
- group Id
- OG001
- lower Limit
- -0.113
- upper Limit
- 0.300
- value
- 0.094
- denoms
- counts
- group Id
- OG000
- value
- 70
- group Id
- OG001
- value
- 72
- units
- Participants
- description
- 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.
- dispersion Type
- 95% Confidence Interval
- groups
- description
- Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet
- id
- OG000
- title
- Double-Blind Phase - Nirogacestat
- description
- Placebo by mouth, twice daily Placebo Oral Tablet: Sugar pill manufactured to mimic nirogacestat 50 mg tablet
- id
- OG001
- title
- Double-Blind Phase - Placebo
- param Type
- LEAST_SQUARES_MEAN
- population Description
- Intent-to-Treat Population
- reporting Status
- POSTED
- time Frame
- On the last day of every cycle (each cycle is 28 days) through study completion, average of 2 years.
- title
- Change From Baseline in the GOunder/Desmoid Tumor Research Foundation (DTRF) DEsmoid Tumor Impact Scale (DTIS) - Physical Functioning Domain Score
- type
- SECONDARY
- unit Of Measure
- score on a scale
Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0
Preserved source evidence · Not medical advice