TriangleNCT04173494 · OUTCOME
Change From Baseline in Physical Function Score as Assessed by Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 10b at Week 24
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
- Measure
- LEAST_SQUARES_MEAN
- Unit
- Scores on a scale
- Interval / dispersion
- Standard Error
- Time frame
- Baseline and Week 24
What was measured
PROMIS Physical Function Short Form 10b consists of 14 questions; each with a 5-point response. PROMIS short form assesses self-reported capability of a participant rather than actual performance of physical activities. This includes functioning of one's upper extremities (dexterity), lower extremities (walking or mobility), and central regions (neck, back) as well as instrumental activities of daily living, such as running errands. Participants scored each response on a scale from 1 (unable to do) to 5 (without any difficulty, or not at all). Total possible range of scores was 14 to 70, with higher scores corresponding to a greater physical function ability. An increase in score from Baseline indicated an improvement in physical function ability and a decrease in score from Baseline indicated a reduction in physical function ability. Baseline was last assessment done before or on the day of first dose date. Change from Baseline was defined as post-Baseline value minus Baseline value.
Analysis population: ITT Analysis Set. Only those participants with data available at specified time points were analyzed.
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 | 89 |
| DAN 300 mg BID + Placebo | 32 |
Reported measurements
Source class 1
| Group | Value | Spread | Lower | Upper | Comment |
|---|---|---|---|---|---|
| MMB 200 mg QD + Placebo | 1.19 | 0.77 | Not reported | Not reported | Not reported |
| DAN 300 mg BID + Placebo | -0.11 | 1.21 | Not reported | Not reported | Not reported |
Source statistical analyses
- ci Lower Limit
- -1.49
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 4.11
- group Ids
- OG000
- OG001
- non Inferiority Type
- OTHER
- p Value
- 0.3570
- param Type
- Least Squares Mean Difference
- param Value
- 1.31
- statistical Method
- MMRM
Complete source fields
- analyses
- ci Lower Limit
- -1.49
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 4.11
- group Ids
- OG000
- OG001
- non Inferiority Type
- OTHER
- p Value
- 0.3570
- param Type
- Least Squares Mean Difference
- param Value
- 1.31
- statistical Method
- MMRM
- classes
- categories
- measurements
- group Id
- OG000
- spread
- 0.77
- value
- 1.19
- group Id
- OG001
- spread
- 1.21
- value
- -0.11
- denoms
- counts
- group Id
- OG000
- value
- 89
- group Id
- OG001
- value
- 32
- units
- Participants
- description
- PROMIS Physical Function Short Form 10b consists of 14 questions; each with a 5-point response. PROMIS short form assesses self-reported capability of a participant rather than actual performance of physical activities. This includes functioning of one's upper extremities (dexterity), lower extremities (walking or mobility), and central regions (neck, back) as well as instrumental activities of daily living, such as running errands. Participants scored each response on a scale from 1 (unable to do) to 5 (without any difficulty, or not at all). Total possible range of scores was 14 to 70, with higher scores corresponding to a greater physical function ability. An increase in score from Baseline indicated an improvement in physical function ability and a decrease in score from Baseline indicated a reduction in physical function ability. Baseline was last assessment done before or on the day of first dose date. Change from Baseline was defined as post-Baseline value minus Baseline value.
- dispersion Type
- Standard Error
- 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
- LEAST_SQUARES_MEAN
- population Description
- ITT Analysis Set. Only those participants with data available at specified time points were analyzed.
- reporting Status
- POSTED
- time Frame
- Baseline and Week 24
- title
- Change From Baseline in Physical Function Score as Assessed by Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 10b at Week 24
- type
- SECONDARY
- unit Of Measure
- Scores on a scale
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Preserved source evidence · Not medical advice