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NF-kB Inhibitor

Dose 1 for Duchenne Muscular Dystrophy (PolarisDMD Trial)

Phase 3
Waitlist Available
Research Sponsored by Catabasis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline (day 1) to week 52
Awards & highlights

Summary

The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from 4-7 years of age (up to 8th birthday) will be enrolled. Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is the key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in DMD.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline (day 1) to week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day 1) to week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change From Baseline in North Star Ambulatory Assessment (NSAA)
Secondary outcome measures
Change From Baseline in 10-meter Walk/Run Test
Change From Baseline in 4-stair Climb
Change From Baseline in Time to Stand From Supine
+1 more
Other outcome measures
Change from baseline in muscle strength testing assessed by knee extension and elbow flexion
Change from baseline in parent/proxy reported physical functioning/quality of life assessed by the Pediatric Outcome Data Collection Instrument (PODCI) Questionnaire
Change from baseline in the Performance of Upper Limb (PUL) Scale to assess upper limb function

Side effects data

From 2020 Phase 3 trial • 131 Patients • NCT03703882
61%
Diarrhoea
33%
Vomiting
23%
Rash
22%
Nasopharyngitis
20%
Upper respiratory tract infection
19%
Pyrexia
17%
Abdominal pain upper
16%
Fall
15%
Cough
14%
Headache
11%
Influenza
11%
Muscle spasms
8%
Abdominal pain
8%
Ear infection
8%
Nausea
7%
Constipation
7%
Contusion
7%
Fatigue
7%
Pain in extremity
7%
Epistaxis
7%
Decreased appetite
5%
Oropharyngeal pain
5%
Pharyngitis streptococcal
5%
Abdominal discomfort
3%
Toothache
3%
Dizziness
3%
Ear pain
3%
Non-cardiac chest pain
3%
Gastroenteritis
3%
Ligament sprain
3%
Rhinorrhoea
3%
Pollakiuria
3%
Gastroenteritis viral
3%
Impetigo
3%
Tonsillitis
3%
Sinus congestion
2%
Attention deficit hyperactivity disorder
2%
Arthropod bite
2%
Dental caries
2%
Conjunctivitis
2%
Faeces soft
2%
Chromaturia
2%
Lower respiratory tract infection
1%
Weight increased
1%
Gastroenteritis norovirus
1%
Back pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Dose 1
Placebo

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Dose 1Experimental Treatment1 Intervention
Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.
Group II: PlaceboPlacebo Group1 Intervention
Matching placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Edasalonexent
2016
Completed Phase 3
~170

Find a Location

Who is running the clinical trial?

Catabasis PharmaceuticalsLead Sponsor
13 Previous Clinical Trials
850 Total Patients Enrolled
Joanne M Donovan, Chief Medical Officer, MD, PhDStudy ChairCatabasis Pharmaceuticals
~19 spots leftby Sep 2025
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