TriangleNCT04173494 · OUTCOME
Total Symptom Score (TSS) Response Rate at Week 24
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
- Measure
- NUMBER
- Unit
- Percentage of participants
- Interval / dispersion
- 95% Confidence Interval
- Time frame
- Baseline and Week 24
What was measured
Myelofibrosis Symptom Assessment Form (MFSAF) TSS version (v) 4.0 response rate was defined as percentage of participants with a \>= 50 percent (%) reduction from Baseline in mean MFSAF TSS over consecutive 28-day period immediately before end of Week 24. TSS response rate was measured using MFSAF v4.0. MFSAF v4.0 comprises 7 domains representing 7 most relevant symptoms of myelofibrosis (MF) identified through existing participant and clinician-based evidence: fatigue,night sweats,pruritus,abdominal discomfort,pain under left ribs,early satietyand bone pain. Participants scored each symptom domain using an 11-point numeric rating scale ranging from 0(absent) to 10(worst imaginable). The MFSAF TSS was calculated as sum of scores of 7 domains for a possible range of scores of 0 to 70, with a higher TSS corresponding to more severe symptoms. A reduction from Baseline corresponded to a lessening of MF symptoms. Baseline was the last assessment done before or on the day of first dose date.
Analysis population: Measured in the Intent-To-Treat (ITT) Analysis Set, which included all randomized participants.
Groups in this outcome
MMB 200 mg QD + Placebo
Participants were randomized to receive 200 milligrams (mg) of MMB orally QD and a DAN-placebo orally twice daily (BID) during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants continued to receive 200 mg of MMB orally QD during the open-label extended treatment period. Participants who discontinued treatment prior to Week 24 due to splenic progression or other reasons, but completed assessments, were unblinded. Participants who discontinued MMB during the randomized treatment period were not eligible to receive MMB in the open-label extended treatment period. Participants who were randomized to MMB during the randomized treatment period with confirmed splenic progression could not proceed to the open-label extended treatment period.
DAN 300 mg BID + Placebo
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period.
| Group | Source count |
|---|---|
| MMB 200 mg QD + Placebo | 130 |
| DAN 300 mg BID + Placebo | 65 |
Reported measurements
Source class 1
| Group | Value | Spread | Lower | Upper | Comment |
|---|---|---|---|---|---|
| MMB 200 mg QD + Placebo | 24.62 | Not reported | 17.49 | 32.94 | Not reported |
| DAN 300 mg BID + Placebo | 9.23 | Not reported | 3.46 | 19.02 | Not reported |
Source statistical analyses
- ci Lower Limit
- 5.54
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 25.81
- group Ids
- OG000
- OG001
- non Inferiority Type
- SUPERIORITY
- p Value
- 0.0095
- param Type
- Stratified Cochran-Mantel-Haenszel
- param Value
- 15.67
- statistical Method
- Cochran-Mantel-Haenszel
Complete source fields
- analyses
- ci Lower Limit
- 5.54
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 25.81
- group Ids
- OG000
- OG001
- non Inferiority Type
- SUPERIORITY
- p Value
- 0.0095
- param Type
- Stratified Cochran-Mantel-Haenszel
- param Value
- 15.67
- statistical Method
- Cochran-Mantel-Haenszel
- classes
- categories
- measurements
- group Id
- OG000
- lower Limit
- 17.49
- upper Limit
- 32.94
- value
- 24.62
- group Id
- OG001
- lower Limit
- 3.46
- upper Limit
- 19.02
- value
- 9.23
- denoms
- counts
- group Id
- OG000
- value
- 130
- group Id
- OG001
- value
- 65
- units
- Participants
- description
- Myelofibrosis Symptom Assessment Form (MFSAF) TSS version (v) 4.0 response rate was defined as percentage of participants with a \>= 50 percent (%) reduction from Baseline in mean MFSAF TSS over consecutive 28-day period immediately before end of Week 24. TSS response rate was measured using MFSAF v4.0. MFSAF v4.0 comprises 7 domains representing 7 most relevant symptoms of myelofibrosis (MF) identified through existing participant and clinician-based evidence: fatigue,night sweats,pruritus,abdominal discomfort,pain under left ribs,early satietyand bone pain. Participants scored each symptom domain using an 11-point numeric rating scale ranging from 0(absent) to 10(worst imaginable). The MFSAF TSS was calculated as sum of scores of 7 domains for a possible range of scores of 0 to 70, with a higher TSS corresponding to more severe symptoms. A reduction from Baseline corresponded to a lessening of MF symptoms. Baseline was the last assessment done before or on the day of first dose date.
- dispersion Type
- 95% Confidence Interval
- groups
- description
- Participants were randomized to receive 200 milligrams (mg) of MMB orally QD and a DAN-placebo orally twice daily (BID) during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants continued to receive 200 mg of MMB orally QD during the open-label extended treatment period. Participants who discontinued treatment prior to Week 24 due to splenic progression or other reasons, but completed assessments, were unblinded. Participants who discontinued MMB during the randomized treatment period were not eligible to receive MMB in the open-label extended treatment period. Participants who were randomized to MMB during the randomized treatment period with confirmed splenic progression could not proceed to the open-label extended treatment period.
- id
- OG000
- title
- MMB 200 mg QD + Placebo
- description
- Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period.
- id
- OG001
- title
- DAN 300 mg BID + Placebo
- param Type
- NUMBER
- population Description
- Measured in the Intent-To-Treat (ITT) Analysis Set, which included all randomized participants.
- reporting Status
- POSTED
- time Frame
- Baseline and Week 24
- title
- Total Symptom Score (TSS) Response Rate at Week 24
- type
- PRIMARY
- unit Of Measure
- Percentage of participants
Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0
Preserved source evidence · Not medical advice