Trial Outcomes & Findings for Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (NCT NCT03340675)
NCT ID: NCT03340675
Last Updated: 2026-01-26
Results Overview
Percentage of subjects with one or more treatment emergent adverse event
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
46 participants
Primary outcome timeframe
Baseline through 12 months
Results posted on
2026-01-26
Participant Flow
Patients were randomized 1:1:1 to receive either high dose ifetroban, low dose ifetroban, or matching placebo daily for 52 weeks.
46 participants were randomized into the study
Participant milestones
| Measure |
High Dose Ifetroban
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Double Blind
STARTED
|
18
|
12
|
11
|
|
Double Blind
COMPLETED
|
13
|
9
|
8
|
|
Double Blind
NOT COMPLETED
|
5
|
3
|
3
|
|
Open Label Extension
STARTED
|
13
|
9
|
8
|
|
Open Label Extension
COMPLETED
|
10
|
7
|
8
|
|
Open Label Extension
NOT COMPLETED
|
3
|
2
|
0
|
Reasons for withdrawal
| Measure |
High Dose Ifetroban
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Double Blind
Adverse Event
|
3
|
1
|
1
|
|
Double Blind
Lost to Follow-up
|
0
|
0
|
1
|
|
Double Blind
Withdrawal by Subject
|
1
|
2
|
1
|
|
Double Blind
Protocol Violation
|
1
|
0
|
0
|
Baseline Characteristics
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Baseline characteristics by cohort
| Measure |
High Dose Ifetroban
n=18 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=12 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=11 Participants
Oral placebo daily for 52 weeks
|
Total
n=41 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
18.2 Years
STANDARD_DEVIATION 5.9 • n=25 Participants
|
17.3 Years
STANDARD_DEVIATION 5.8 • n=25 Participants
|
14.7 Years
STANDARD_DEVIATION 4.9 • n=50 Participants
|
17.0 Years
STANDARD_DEVIATION 5.7 • n=152 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=152 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=25 Participants
|
12 Participants
n=25 Participants
|
11 Participants
n=50 Participants
|
41 Participants
n=152 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=152 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
1 Participants
n=152 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=152 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=152 Participants
|
|
Race (NIH/OMB)
White
|
16 Participants
n=25 Participants
|
12 Participants
n=25 Participants
|
10 Participants
n=50 Participants
|
38 Participants
n=152 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
1 Participants
n=152 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
1 Participants
n=50 Participants
|
1 Participants
n=152 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
3 Participants
n=50 Participants
|
4 Participants
n=152 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
17 Participants
n=25 Participants
|
12 Participants
n=25 Participants
|
8 Participants
n=50 Participants
|
37 Participants
n=152 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=152 Participants
|
|
Weight
|
54.3 kg
STANDARD_DEVIATION 17.9 • n=25 Participants
|
53.2 kg
STANDARD_DEVIATION 20.7 • n=25 Participants
|
42.4 kg
STANDARD_DEVIATION 16.9 • n=50 Participants
|
50.8 kg
STANDARD_DEVIATION 18.8 • n=152 Participants
|
|
BMI (kg/m^2)
< 18.5 (Underweight)
|
3 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
3 Participants
n=50 Participants
|
6 Participants
n=152 Participants
|
|
BMI (kg/m^2)
18.5 - 24.9 (Normal weight)
|
7 Participants
n=25 Participants
|
7 Participants
n=25 Participants
|
5 Participants
n=50 Participants
|
19 Participants
n=152 Participants
|
|
BMI (kg/m^2)
25.0 - 29.9 (Overweight)
|
5 Participants
n=25 Participants
|
3 Participants
n=25 Participants
|
1 Participants
n=50 Participants
|
9 Participants
n=152 Participants
|
|
BMI (kg/m^2)
≥ 30 (Obese)
|
3 Participants
n=25 Participants
|
2 Participants
n=25 Participants
|
2 Participants
n=50 Participants
|
7 Participants
n=152 Participants
|
|
BMI Z-scores
|
0.62 Z-score
STANDARD_DEVIATION 1.4 • n=25 Participants
|
1.1 Z-score
STANDARD_DEVIATION 0.71 • n=25 Participants
|
0.15 Z-score
STANDARD_DEVIATION 2.2 • n=50 Participants
|
0.59 Z-score
STANDARD_DEVIATION 1.6 • n=152 Participants
|
|
Ambulatory
Yes
|
8 Participants
n=25 Participants
|
4 Participants
n=25 Participants
|
4 Participants
n=50 Participants
|
16 Participants
n=152 Participants
|
|
Ambulatory
No
|
10 Participants
n=25 Participants
|
8 Participants
n=25 Participants
|
7 Participants
n=50 Participants
|
25 Participants
n=152 Participants
|
|
Ventilatory Support
Yes
|
2 Participants
n=25 Participants
|
2 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
4 Participants
n=152 Participants
|
|
Ventilatory Support
No
|
16 Participants
n=25 Participants
|
10 Participants
n=25 Participants
|
11 Participants
n=50 Participants
|
37 Participants
n=152 Participants
|
|
Stage of DMD
Early (LVEF > 45%)
|
14 Participants
n=25 Participants
|
11 Participants
n=25 Participants
|
11 Participants
n=50 Participants
|
36 Participants
n=152 Participants
|
|
Stage of DMD
Late (LVEF 35 - 45%)
|
4 Participants
n=25 Participants
|
1 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
5 Participants
n=152 Participants
|
|
Background DMD Therapy
No background therapy
|
2 Participants
n=25 Participants
|
1 Participants
n=25 Participants
|
1 Participants
n=50 Participants
|
4 Participants
n=152 Participants
|
|
Background DMD Therapy
Steroids only
|
11 Participants
n=25 Participants
|
6 Participants
n=25 Participants
|
6 Participants
n=50 Participants
|
23 Participants
n=152 Participants
|
|
Background DMD Therapy
ASO only
|
0 Participants
n=25 Participants
|
0 Participants
n=25 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=152 Participants
|
|
Background DMD Therapy
Steroids + ASO
|
5 Participants
n=25 Participants
|
5 Participants
n=25 Participants
|
4 Participants
n=50 Participants
|
14 Participants
n=152 Participants
|
PRIMARY outcome
Timeframe: Baseline through 12 monthsPercentage of subjects with one or more treatment emergent adverse event
Outcome measures
| Measure |
High Dose Ifetroban
n=18 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=12 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=11 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Incidence of Treatment-Emergent Adverse Events
Any TEAE
|
14 Participants
|
9 Participants
|
10 Participants
|
SECONDARY outcome
Timeframe: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post-dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7Population: Fewer participants are represented here due to missing assessments for this measure.
Plasma ifetroban and its acyl glucuronide metabolite were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7. Area under the curve until the last measurement was calculated for the plasma concentration versus time curves for ifetroban and its acyl glucuronide metabolite after administration of assigned oral ifetroban dose
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=8 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Pharmacokinetics Area Under the Curve
Ifetroban
|
3196 h*ng/mL
Standard Deviation 2485
|
761 h*ng/mL
Standard Deviation 585
|
—
|
|
Pharmacokinetics Area Under the Curve
Ifetroban Glucuronide
|
45116 h*ng/mL
Standard Deviation 30211
|
13789 h*ng/mL
Standard Deviation 9700
|
—
|
SECONDARY outcome
Timeframe: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7Population: Fewer participants are represented here due to missing assessments for this measure.
Plasma ifetroban and its acyl glucuronide metabolite were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7 to determine the maximum plasma concentration of ifetroban and its acyl glucuronide metabolite after administration of assigned oral ifetroban dose
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=8 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Pharmacokinetics Maximum Serum Concentration (Cmax)
Ifetroban
|
1560 ng/mL
Standard Deviation 2028
|
272 ng/mL
Standard Deviation 300
|
—
|
|
Pharmacokinetics Maximum Serum Concentration (Cmax)
Ifetroban Glucuronide
|
9787 ng/mL
Standard Deviation 7148
|
2276 ng/mL
Standard Deviation 1955
|
—
|
SECONDARY outcome
Timeframe: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7Population: Fewer participants are represented here due to missing assessments for this measure.
Plasma ifetroban and its acyl glucuronide metabolite were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7 to determine the time to maximum plasma concentration of ifetroban and its acyl glucuronide metabolite following administration of assigned oral ifetroban dose
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=8 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Pharmacokinetics Time to Reach Cmax (Tmax) Concentration
Ifetroban
|
0.71 h
Standard Deviation 0.24
|
1.09 h
Standard Deviation 1.2
|
—
|
|
Pharmacokinetics Time to Reach Cmax (Tmax) Concentration
Ifetroban Glucuronide
|
2.81 h
Standard Deviation 6.43
|
1.89 h
Standard Deviation 2.47
|
—
|
SECONDARY outcome
Timeframe: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7Population: Fewer participants are represented here due to missing assessments for this measure.
Plasma ifetroban were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7. The terminal elimination half-life was calculated from the plasma concentration versus time curves for ifetroban following administration of assigned oral ifetroban dose
Outcome measures
| Measure |
High Dose Ifetroban
n=7 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=4 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Pharmacokinetics Plasma Terminal Half-life Concentration
|
12.96 h
Standard Deviation 5.73
|
8.99 h
Standard Deviation 4.6
|
—
|
SECONDARY outcome
Timeframe: Baseline visit and Month 12 visitPopulation: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
Change from baseline at month 12 in left ventricular ejection fraction
Outcome measures
| Measure |
High Dose Ifetroban
n=12 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=7 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=6 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in Left Ventricular Ejection Fraction
|
1.8 Percentage of LVEF
Standard Deviation 5.44
|
0 Percentage of LVEF
Standard Deviation 5.16
|
-1.5 Percentage of LVEF
Standard Deviation 3.27
|
SECONDARY outcome
Timeframe: Baseline through month 12Population: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
Change from baseline at month 12 in Forced Expiratory Volume in 1 second
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=9 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=7 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in FEV1
|
-0.05 Liters
Standard Deviation 0.233
|
-0.11 Liters
Standard Deviation 0.294
|
0.01 Liters
Standard Deviation 0.169
|
SECONDARY outcome
Timeframe: Baseline through month 12Population: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
Change from baseline at month 12 in percent predicted Forced Expiratory Volume in 1 second
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=9 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=7 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in Percent Predicted FEV1
|
-1.92 Percent
Standard Deviation 10.67
|
-7.89 Percent
Standard Deviation 9.226
|
-3.29 Percent
Standard Deviation 6.775
|
SECONDARY outcome
Timeframe: Baseline through month 12Population: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
Change from baseline at month 12 in Forced Vital Capacity
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=9 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=7 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in FVC
|
-0.08 Liters
Standard Deviation 0.291
|
-0.07 Liters
Standard Deviation 0.378
|
-0.08 Liters
Standard Deviation 0.291
|
SECONDARY outcome
Timeframe: Baseline through month 12Population: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
Change from baseline at month 12 in percent predicted Forced Vital Capacity
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=9 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=7 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in Percent Predicted FVC
|
-3.31 Percent
Standard Deviation 10.491
|
-6.11 Percent
Standard Deviation 11.050
|
-8.14 Percent
Standard Deviation 15.700
|
SECONDARY outcome
Timeframe: Baseline through month 12Population: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
Change from baseline at month 12 in maximal expiratory pressure
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=8 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=6 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in Maximal Expiratory Pressure
|
-1.69 cm of water
Standard Deviation 8.625
|
2.00 cm of water
Standard Deviation 16.466
|
-16.50 cm of water
Standard Deviation 25.774
|
SECONDARY outcome
Timeframe: Baseline through month 12Population: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
Change from baseline at month 12 in maximal inspiratory pressure
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=8 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=6 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in Maximal Inspiratory Pressure
|
-6.00 cm of water
Standard Deviation 7.000
|
-9.13 cm of water
Standard Deviation 8.935
|
1.17 cm of water
Standard Deviation 20.14
|
SECONDARY outcome
Timeframe: Baseline through month 12Population: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
Change from baseline at month 12 in peak expiratory flow rate
Outcome measures
| Measure |
High Dose Ifetroban
n=13 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=6 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=6 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in Peak Expiratory Flow Rate
|
0.09 Liters/second
Standard Deviation 0.643
|
-0.19 Liters/second
Standard Deviation 0.414
|
0.51 Liters/second
Standard Deviation 0.844
|
SECONDARY outcome
Timeframe: Baseline through month 12Population: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
Change from baseline at month 12 in percent predicted peak expiratory flow rate
Outcome measures
| Measure |
High Dose Ifetroban
n=12 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=6 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=5 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in Percent Predicted Peak Expiratory Flow Rate
|
-3.58 Percent
Standard Deviation 15.395
|
-3.83 Percent
Standard Deviation 11.250
|
2.40 Percent
Standard Deviation 20.477
|
SECONDARY outcome
Timeframe: Baseline through 12 monthsPopulation: Differences in number of participants analyzed is due to missing evaluations at one or more timepoint
The Pediatric Quality of Life Inventory (PedQL) questionnaire measures core dimensions of health as delineated by the World Health Organization. The core module includes 23 items comprising 14 dimensions. The Gastrointestinal module includes 74 items comprising 14 dimensions. The Neuromuscular module includes 25 items comprising 3 dimensions. Each item in the modules is measured on a 5-point Likert scale of 0-4 with 0 indicating 'never' and 4 indicating 'almost always'. The Likert scores for each subscale are reversed scored and linearly transform to a 0-100 scale with 0=100, 1-75, 2=50, 5=25, and 4=0. A higher score indicates better health-related quality of life. Subscale scores are averaged to generate the total score reported with a range of 0-100. Results are presented as change from baseline at 12 months. The questionnaire was completed by the participant and the parent/guardian of the participant.
Outcome measures
| Measure |
High Dose Ifetroban
n=14 Participants
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=10 Participants
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=9 Participants
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Change From Baseline in Quality-of-life
Generic Core Module (Participant Response)
|
-0.06 Scores on a scale
Standard Deviation 15.9
|
4.39 Scores on a scale
Standard Deviation 11.5
|
-4.03 Scores on a scale
Standard Deviation 13.1
|
|
Change From Baseline in Quality-of-life
Generic Core Module (Parent Response)
|
-7.62 Scores on a scale
Standard Deviation 14.3
|
0.43 Scores on a scale
Standard Deviation 19.6
|
-11.4 Scores on a scale
Standard Deviation 21.5
|
|
Change From Baseline in Quality-of-life
Gastrointestinal Module (Participant Response)
|
-7.08 Scores on a scale
Standard Deviation 22.7
|
4.08 Scores on a scale
Standard Deviation 6.45
|
-2.03 Scores on a scale
Standard Deviation 10.7
|
|
Change From Baseline in Quality-of-life
Gastrointestinal Module (Parent Response)
|
4.20 Scores on a scale
Standard Deviation 6.59
|
-0.03 Scores on a scale
Standard Deviation 7.62
|
-5.57 Scores on a scale
Standard Deviation 4.95
|
|
Change From Baseline in Quality-of-life
Neuromuscular Module (Participant Response)
|
1.31 Scores on a scale
Standard Deviation 10.4
|
3.49 Scores on a scale
Standard Deviation 13.0
|
1.93 Scores on a scale
Standard Deviation 12.0
|
|
Change From Baseline in Quality-of-life
Neuromuscular Module (Parent Response)
|
7.77 Scores on a scale
Standard Deviation 8.01
|
2.00 Scores on a scale
Standard Deviation 6.74
|
-6.57 Scores on a scale
Standard Deviation 10.3
|
Adverse Events
High Dose Ifetroban
Serious events: 3 serious events
Other events: 14 other events
Deaths: 0 deaths
Low Dose Ifetroban
Serious events: 2 serious events
Other events: 9 other events
Deaths: 0 deaths
Placebo
Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
High Dose Ifetroban
n=18 participants at risk
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=12 participants at risk
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=11 participants at risk
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Nervous system disorders
Cerebrovascular accident
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Gastrointestinal disorders
Oesophageal obstruction
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Infections and infestations
Pneumonia
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Metabolism and nutrition disorders
Hypervolaemia
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
9.1%
1/11 • From initiation of treatment through study completion, up to 1 year
|
|
Metabolism and nutrition disorders
Dehydration
|
5.6%
1/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
5.6%
1/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
9.1%
1/11 • From initiation of treatment through study completion, up to 1 year
|
|
Injury, poisoning and procedural complications
Hip fracture
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
9.1%
1/11 • From initiation of treatment through study completion, up to 1 year
|
Other adverse events
| Measure |
High Dose Ifetroban
n=18 participants at risk
Oral ifetroban, 150 mg or 300 mg daily for 52 weeks (Subjects weighing \< 35 kg received 150 mg or ≥ 35 kg received 300 mg ifetroban)
|
Low Dose Ifetroban
n=12 participants at risk
Oral ifetroban, 50 mg or 100 mg daily for 52 weeks (Subjects weighing \< 35 kg received 50 mg or ≥ 35 kg received 100 mg ifetroban)
|
Placebo
n=11 participants at risk
Oral placebo daily for 52 weeks
|
|---|---|---|---|
|
Infections and infestations
COVID-19
|
22.2%
4/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
18.2%
2/11 • From initiation of treatment through study completion, up to 1 year
|
|
Infections and infestations
Nasopharyngitis
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
18.2%
2/11 • From initiation of treatment through study completion, up to 1 year
|
|
Infections and infestations
Pneumonia
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Injury, poisoning and procedural complications
Contusion
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Injury, poisoning and procedural complications
Fall
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Injury, poisoning and procedural complications
Foot fracture
|
5.6%
1/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.6%
1/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
18.2%
2/11 • From initiation of treatment through study completion, up to 1 year
|
|
Musculoskeletal and connective tissue disorders
Muscle contracture
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Nervous system disorders
Headache
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
27.3%
3/11 • From initiation of treatment through study completion, up to 1 year
|
|
Nervous system disorders
Dizziness
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Investigations
Ejection fraction decreased
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Skin and subcutaneous tissue disorders
Rash
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
9.1%
1/11 • From initiation of treatment through study completion, up to 1 year
|
|
Psychiatric disorders
Anxiety
|
5.6%
1/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Endocrine disorders
Delayed puberty
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
18.2%
2/11 • From initiation of treatment through study completion, up to 1 year
|
|
General disorders
Fatigue
|
5.6%
1/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
9.1%
1/11 • From initiation of treatment through study completion, up to 1 year
|
|
Gastrointestinal disorders
Nausea
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
16.7%
2/12 • From initiation of treatment through study completion, up to 1 year
|
27.3%
3/11 • From initiation of treatment through study completion, up to 1 year
|
|
Gastrointestinal disorders
Vomiting
|
16.7%
3/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
18.2%
2/11 • From initiation of treatment through study completion, up to 1 year
|
|
Gastrointestinal disorders
Diarrhoea
|
11.1%
2/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
9.1%
1/11 • From initiation of treatment through study completion, up to 1 year
|
|
Gastrointestinal disorders
Constipation
|
5.6%
1/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
9.1%
1/11 • From initiation of treatment through study completion, up to 1 year
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/12 • From initiation of treatment through study completion, up to 1 year
|
18.2%
2/11 • From initiation of treatment through study completion, up to 1 year
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.6%
1/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
|
Gastrointestinal disorders
Oesophageal obstruction
|
0.00%
0/18 • From initiation of treatment through study completion, up to 1 year
|
8.3%
1/12 • From initiation of treatment through study completion, up to 1 year
|
0.00%
0/11 • From initiation of treatment through study completion, up to 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place