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Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03703882
Recruitment Status : Completed
First Posted : October 12, 2018
Results First Posted : June 21, 2022
Last Update Posted : June 21, 2022
Sponsor:
Information provided by (Responsible Party):
Catabasis Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Muscular Dystrophy, Duchenne
Interventions Drug: Edasalonexent
Drug: Placebo
Enrollment 131
Recruitment Details This was a multi-center study conducted by 37 principal investigators at 37 study centers in 8 countries (United States, Canada, United Kingdom, Germany, Ireland, Israel, Sweden, and Australia.)
Pre-assignment Details A total of 151 patients were screened of which 20 failed screening. 131 patients who participated in the study included 126 randomized patients and 5 participants who were dosed siblings of previously randomized patients.
Arm/Group Title Dose 1 Placebo
Hide Arm/Group Description

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

Edasalonexent: 100 mg/kg/day

Matching placebo

Placebo: Placebo

Period Title: Overall Study
Started 88 43
Completed 85 37
Not Completed 3 6
Reason Not Completed
Adverse Event             1             0
Lost to Follow-up             0             2
Non-compliance with study drug             0             1
Physician Decision             0             1
Progressive disease             1             0
Withdrawal by parent/guardian             1             0
Starting another treatment             0             1
noncompliance with study procedures             0             1
Arm/Group Title Dose 1 Placebo Total
Hide Arm/Group Description

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

Edasalonexent: 100 mg/kg/day

Matching placebo

Placebo: Placebo

Total of all reporting groups
Overall Number of Baseline Participants 88 43 131
Hide Baseline Analysis Population Description
Safety Population: All patients who received at least 1 dose of study drug, with patients analyzed based on the actual study treatment received. This included the set of patients who were assigned the same treatment as their randomized sibling.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 88 participants 43 participants 131 participants
5.65  (1.048) 5.77  (0.995) 5.69  (1.029)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 88 participants 43 participants 131 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
88
 100.0%
43
 100.0%
131
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 88 participants 43 participants 131 participants
Hispanic or Latino
14
  15.9%
6
  14.0%
20
  15.3%
Not Hispanic or Latino
69
  78.4%
35
  81.4%
104
  79.4%
Unknown or Not Reported
5
   5.7%
2
   4.7%
7
   5.3%
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 87 participants 43 participants 130 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
3
   3.4%
2
   4.7%
5
   3.8%
Native Hawaiian or Other Pacific Islander
1
   1.1%
0
   0.0%
1
   0.8%
Black or African American
4
   4.6%
1
   2.3%
5
   3.8%
White
74
  85.1%
38
  88.4%
112
  86.2%
More than one race
3
   3.4%
1
   2.3%
4
   3.1%
Unknown or Not Reported
2
   2.3%
1
   2.3%
3
   2.3%
[1]
Measure Analysis Population Description:

Race is missing for one subject.

A patient who reported more than 1 race was categorized as multiracial.

1.Primary Outcome
Title Change From Baseline in North Star Ambulatory Assessment (NSAA)
Hide Description To assess change from baseline in North Star Ambulatory Assessment(NSAA) Total Score at Wk52. NSAA is clinician-reported outcome instrument designed to measure ambulatory function in males with Duchenne muscular dystrophy(DMD). Patients asked to perform 17 different functional activities,including 10MWT,rising from sit to stand,standing on one leg,climbing & descending a step,stand from supine, lifting the head, standing on heels, & jumping. Each function activity will be scored as0=(unable to achieve independently),scored as1=(modified method but achieves goal independent of physical assistance from another),or scored as2=(no obvious modification of activity)or "Not Scored". If NSAA test was performed & any of the individual items are scored as "not scored"(i.e, for reasons unrelated to patients physical capabilities), corresponding total score will be set to missing. Sum of 17 scores will be used to form an ordinal total score(range 0-34).Higher scores imply better functional status
Time Frame Baseline (Day 1) to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment.
Arm/Group Title Dose 1 Placebo
Hide Arm/Group Description:

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

Edasalonexent: 100 mg/kg/day

Matching placebo

Placebo: Placebo

Overall Number of Participants Analyzed 81 38
Mean (Standard Deviation)
Unit of Measure: score on a scale
-1.5  (4.41) -1.8  (3.81)
2.Secondary Outcome
Title Change From Baseline in 10-meter Walk/Run Test
Hide Description To assess the changes from baseline to Week 52 on the 10-meter walk/run test (10MWT). For timed function tests (TFTs), the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1 or 2 (from a 6-point scale). 1=Unable to walk independently 2=Unable to walk independently but can walk with knee-ankle foot orthoses or support from a person 3=Highly adapted wide based lordotic gait. Cannot increase walking speed 4=Moderately adapted gait. Can pick up speed but cannot run 5=Able to pick up speed, but runs with a double stance phase, i.e. cannot achieve both feet off the ground 6=Runs and gets both feet off the ground (with no double stance phase)
Time Frame Baseline (Day 1) to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment.
Arm/Group Title Dose 1 Placebo
Hide Arm/Group Description:

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

Edasalonexent: 100 mg/kg/day

Matching placebo

Placebo: Placebo

Overall Number of Participants Analyzed 81 38
Mean (Standard Deviation)
Unit of Measure: score on a scale
-0.0058  (0.03010) -0.0093  (0.02538)
3.Secondary Outcome
Title Change From Baseline in Time to Stand From Supine
Hide Description To assess the change from baseline in the stand from supine speed at Week 52. For timed function tests (TFTs) , the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1 or 2 (from a 6-point scale). 1 = Unable to stand from supine, even with use of a chair, 2 = Assisted Gowers - requires furniture for assist in arising from supine to full upright posture (no time to be recorded) 3=Rolls over, stands up with both hands "climbing up" the legs to achieve full upright posture 4=Rolls over, stands up with 1 hand support on leg 5=Rolls to the side and stands up with one or both hands on the floor to start to rise but does not touch legs 6=Stands up without rolling over or using hands on legs or floor
Time Frame Baseline (Day 1) to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment.
Arm/Group Title Dose 1 Placebo
Hide Arm/Group Description:

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

Edasalonexent: 100 mg/kg/day

Matching placebo

Placebo: Placebo

Overall Number of Participants Analyzed 81 38
Mean (Standard Deviation)
Unit of Measure: score on a scale
-0.0389  (0.06728) -0.0459  (0.06171)
4.Secondary Outcome
Title Change From Baseline in 4-stair Climb
Hide Description To assess the change from baseline to Week 52 on the 4-Stair Climb. For timed function tests (TFTs) , the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1(from a 6-point scale)1=Unable to climb 4 standard stairs(no time recorded) 2=Climbs 4 standard stairs "marking time"(climbs one foot at a time, with both feet on a step before moving to next step), uses both arms on one or both handrails or uses 1 handrail and the other arm pushes on the leg 3=Climbs 4 standard stairs "marking time", using one arm on one handrail or one hand pushing on leg or body 4=Climbs 4 standard stairs "marking time", not needing handrail and not using hands to push on leg 5=Climbs 4 standard stairs alternating feet, needs handrail/s for support or uses arms to push on the leg or body 6=Climbs 4 standard stairs alternating feet, not needing handrail support or using arm to push on the leg
Time Frame Baseline (Day 1) to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment).
Arm/Group Title Dose 1 Placebo
Hide Arm/Group Description:

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

Edasalonexent: 100 mg/kg/day

Matching placebo

Placebo: Placebo

Overall Number of Participants Analyzed 81 38
Mean (Standard Deviation)
Unit of Measure: score on a scale
-0.0220  (0.08920) -0.0392  (0.07352)
5.Secondary Outcome
Title Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Hide Description Adverse events that occurred from the time of the administration of the first dose of investigational product (IP) through the end of the safety follow-up were considered treatment-emergent AEs (TEAEs). Serious adverse event (SAE).
Time Frame Up to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population: All patients who received at least 1 dose of study drug, with patients analyzed based on the actual study treatment received. This included the set of patients who were assigned the same treatment as their randomized sibling.
Arm/Group Title Dose 1 Placebo
Hide Arm/Group Description:

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

Edasalonexent: 100 mg/kg/day

Matching placebo

Placebo: Placebo

Overall Number of Participants Analyzed 88 43
Measure Type: Number
Unit of Measure: participants
Treatment Emergent Adverse Event (TEAEs) 85 41
Related Treatment Emergent Adverse Event (TEAEs) 61 14
Serious TEAE 1 1
Serious Related TEAEs 0 0
TEAEs Leading to Study Discontinuation 1 0
Treatment-Related TEAEs Leading to Study Discontinuation 1 0
Time Frame Up to Week 52
Adverse Event Reporting Description Subjects with more than one AE of the same system organ class (SOC) / preferred term (PT) were counted only once for that SOC / PT.
 
Arm/Group Title Dose 1 Placebo
Hide Arm/Group Description

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

Edasalonexent: 100 mg/kg/day

Matching placebo

Placebo: Placebo

All-Cause Mortality
Dose 1 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/88 (0.00%)      0/43 (0.00%)    
Hide Serious Adverse Events
Dose 1 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/88 (1.14%)      1/43 (2.33%)    
Infections and infestations     
Gastroenteritis norovirus  1  1/88 (1.14%)  1 0/43 (0.00%)  0
Bronchiolitis  1  0/88 (0.00%)  0 1/43 (2.33%)  1
1
Term from vocabulary, 21.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
Dose 1 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   85/88 (96.59%)      41/43 (95.35%)    
Ear and labyrinth disorders     
Ear pain  1  3/88 (3.41%)  0/43 (0.00%) 
Gastrointestinal disorders     
Diarrhoea  1  54/88 (61.36%)  12/43 (27.91%) 
Vomiting  1  29/88 (32.95%)  11/43 (25.58%) 
Abdominal pain upper  1  15/88 (17.05%)  9/43 (20.93%) 
Nausea  1  7/88 (7.95%)  5/43 (11.63%) 
Constipation  1  6/88 (6.82%)  3/43 (6.98%) 
Abdominal pain  1  7/88 (7.95%)  1/43 (2.33%) 
Abdominal discomfort  1  4/88 (4.55%)  1/43 (2.33%) 
Dental caries  1  2/88 (2.27%)  1/43 (2.33%) 
Faeces soft  1  2/88 (2.27%)  1/43 (2.33%) 
Toothache  1  3/88 (3.41%)  0/43 (0.00%) 
General disorders     
Pyrexia  1  17/88 (19.32%)  9/43 (20.93%) 
Fatigue  1  6/88 (6.82%)  0/43 (0.00%) 
Influenza like illness  1  0/88 (0.00%)  5/43 (11.63%) 
Non-cardiac chest pain  1  3/88 (3.41%)  0/43 (0.00%) 
Infections and infestations     
Nasopharyngitis  1  19/88 (21.59%)  9/43 (20.93%) 
Upper respiratory tract infection  1  18/88 (20.45%)  5/43 (11.63%) 
Ear infection  1  7/88 (7.95%)  5/43 (11.63%) 
Influenza  1  10/88 (11.36%)  2/43 (4.65%) 
Pharyngitis streptococcal  1  4/88 (4.55%)  4/43 (9.30%) 
Gastroenteritis  1  3/88 (3.41%)  2/43 (4.65%) 
Gastroenteritis viral  1  3/88 (3.41%)  2/43 (4.65%) 
Lower respiratory tract infection  1  2/88 (2.27%)  2/43 (4.65%) 
Conjunctivitis  1  2/88 (2.27%)  1/43 (2.33%) 
Impetigo  1  3/88 (3.41%)  0/43 (0.00%) 
Tonsillitis  1  3/88 (3.41%)  0/43 (0.00%) 
Injury, poisoning and procedural complications     
Fall  1  14/88 (15.91%)  4/43 (9.30%) 
Contusion  1  6/88 (6.82%)  2/43 (4.65%) 
Ligament sprain  1  3/88 (3.41%)  1/43 (2.33%) 
Arthropod bite  1  2/88 (2.27%)  1/43 (2.33%) 
Investigations     
Weight increased  1  1/88 (1.14%)  2/43 (4.65%) 
Metabolism and nutrition disorders     
Decreased appetite  1  6/88 (6.82%)  2/43 (4.65%) 
Musculoskeletal and connective tissue disorders     
Muscle spasms  1  10/88 (11.36%)  1/43 (2.33%) 
Pain in extremity  1  6/88 (6.82%)  5/43 (11.63%) 
Back pain  1  1/88 (1.14%)  2/43 (4.65%) 
Nervous system disorders     
Headache  1  12/88 (13.64%)  8/43 (18.60%) 
Dizziness  1  3/88 (3.41%)  1/43 (2.33%) 
Psychiatric disorders     
Attention deficit hyperactivity disorder  1  2/88 (2.27%)  1/43 (2.33%) 
Renal and urinary disorders     
Chromaturia  1  2/88 (2.27%)  2/43 (4.65%) 
Pollakiuria  1  3/88 (3.41%)  0/43 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  13/88 (14.77%)  11/43 (25.58%) 
Rhinorrhoea  1  3/88 (3.41%)  5/43 (11.63%) 
Epistaxis  1  6/88 (6.82%)  1/43 (2.33%) 
Oropharyngeal pain  1  4/88 (4.55%)  1/43 (2.33%) 
Sinus congestion  1  3/88 (3.41%)  0/43 (0.00%) 
Skin and subcutaneous tissue disorders     
Rash  1  20/88 (22.73%)  2/43 (4.65%) 
1
Term from vocabulary, 21.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Andrew Nichols, PhD - Chief Scientific Officer
Organization: Astria Therapeutics, Inc
Phone: 617-349-1971
EMail: anichols@astriatx.com
Layout table for additonal information
Responsible Party: Catabasis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03703882    
Other Study ID Numbers: CAT-1004-301
First Submitted: October 8, 2018
First Posted: October 12, 2018
Results First Submitted: January 10, 2022
Results First Posted: June 21, 2022
Last Update Posted: June 21, 2022