Muscular Dystrophy, DuchennePhase 3PolarisDMDNCT03703882
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
A Phase 3 study testing Edasalonexent in people with Muscular Dystrophy, Duchenne, run by Catabasis Pharmaceuticals at 40 sites in 8 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 7 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- What is being tested?
- Edasalonexent
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 131 participants
- Where
- 40 sitesin 8 countries
- Study dates
- 2 Oct 2018to 22 Sep 2020
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 7 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
- Diagnosis of DMD based on a clinical phenotype with increased serum creatine kinase (CK) and documentation of mutation(s) in the dystrophin gene known to be associated with a DMD phenotype
- Able to perform stand from supine without assistance in ≤ 10 seconds
- Able to perform the 10MWT and 4-stair climb
- Followed by a doctor or medical professional who coordinates Duchenne care on a regular basis and willingness to disclose patient's study participation with medical professionals
Who cannot join
- Use of corticosteroids within 24 weeks prior to Day 1; use of inhaled, intranasal, and topical corticosteroids is permitted
- Use of another investigational drug, idebenone, or dystrophin-focused therapy within 4 weeks. Exception: Patients who have received at least 24 weeks of a stable dose of eteplirsen prior to Day 1, and expected to continue treatment, will be eligible
- Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, warfarin, phenytoin, S mephenytoin, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus, tacrolimus, or paclitaxel
- Use of human growth hormone within 3 months prior to Day 1
- Other prior or ongoing significant medical conditions
Trial locations and contacts
40 sites in 8 countries.
40 sites in 8 countries
United States24 sites
University of Michigan
Ann Arbor, Michigan, 48109
Rare Disease Research, LLC
Atlanta, Georgia, 30318
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Johns Hopkins School of Medicine
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
Rush University Children's Hospital
Chicago, Illinois, 60612
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
MetroHealth Medical Center
Cleveland, Ohio, 44109
University of Kansas Medical Center
Fairway, Kansas, 66205
Cook Children's Medical Center
Fort Worth, Texas, 76104
University of Iowa Children's Hospital
Iowa City, Iowa, 52242
Las Vegas Clinic
Las Vegas, Nevada, 89145
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital of Los Angeles
Los Angeles, California, 90027
University of Minnesota
Minneapolis, Minnesota, 55455
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510
Nemours Children's Hospital
Orlando, Florida, 32827
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Shriners Hospitals for Children
Portland, Oregon, 97239
Children's Hospital of Richmond at VCU
Richmond, Virginia, 23298
UC Davis
Sacramento, California, 95817
University of Utah
Salt Lake City, Utah, 84112
University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
Canada4 sites
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
London Health Sciences Centre - Children's Hospital
London, Ontario, N6A 4G5
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
United Kingdom4 sites
Bristol Children's Hospital
Bristol, BS2 8AE
Evelina Children's Hospital
London, SE1 7EU
Great Ormond Street Hospital (GOSH)
London, WC1N 3JH
Royal Manchester Children's Hospital
Manchester, M13 9WL
Australia3 sites
Royal Children's Hospital
Parkville, Victoria, 3052
Children's Health Queensland Children's Hospital and Health Service
South Brisbane, Queensland, 4101
The Children's Hospital at Westmead
Westmead, New South Wales, 2145
Germany2 sites
University of Hamburg
Hamburg, 20246
University of Munich
Munich, 80337
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
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.
Read the detailed description
The study includes a 52-week, randomized, double-blind, placebo-controlled period, followed by a 2-week follow- up. Approximately 125 boys with DMD will be enrolled in this trial, with 2 boys receiving edasalonexent for every 1 boy receiving placebo.
Following completion of the treatment period, patients may elect to continue in a separate open-label extension study.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- Edasalonexent, Placebo
- Keywords
- Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases, Duchenne muscular dystrophy, DMD, dystrophin, dystrophy, Duchenne
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EdasalonexentDrug
Also called Edasa, CAT-1004
100 mg/kg/day
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Dose 1 | Experimental | Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day. Receives: Drug: Edasalonexent |
| Placebo | Placebo comparator | Matching placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in North Star Ambulatory Assessment (NSAA)
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
Measured: Baseline (Day 1) to Week 52
Secondary outcomes (4)
Change From Baseline in 10-meter Walk/Run Test
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)
Measured: Baseline (Day 1) to Week 52
Change From Baseline in Time to Stand From Supine
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
Measured: Baseline (Day 1) to Week 52
Change From Baseline in 4-stair Climb
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
Measured: Baseline (Day 1) to Week 52
Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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).
Measured: Up to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2018
- Study start2 Oct 2018
- Primary completion22 Sep 2020
- Study completion22 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular Dystrophy, DuchenneStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 131 participants (actual)
Sponsor and investigators
- Lead sponsor
- Catabasis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Joanne M Donovan, Chief Medical Officer, MD, PhD, Study chair, Catabasis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03703882
- Sponsor's protocol ID
- CAT-1004-301
- Acronym
- PolarisDMD
- Status last verified
- Jun 2022
- First submitted
- 8 Oct 2018
- Last updated
- 21 Jun 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Finkel RS, McDonald CM, Lee Sweeney H, Finanger E, Neil Knierbein E, Wagner KR, Mathews KD, Marks W, Statland J, Nance J, McMillan HJ, McCullagh G, Tian C, Ryan MM, O'Rourke D, Muller-Felber W, Tulinius M, Burnette WB, Nguyen CT, Vijayakumar K, Johannsen J, Phan HC, Eagle M, MacDougall J, Mancini M, Donovan JM; (For the PolarisDMD Study Group). A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial. J Neuromuscul Dis. 2021;8(5):769-784. doi: 10.3233/JND-210689.
PubMed 34120912
Study at a glance
From the registry record, last updated by the sponsor on 21 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03703882
- Acronym / sponsor ID
- PolarisDMD / CAT-1004-301
- Conditions
- Muscular Dystrophy, Duchenne
- Keywords
- Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases, Duchenne muscular dystrophy, DMD, dystrophin, dystrophy, Duchenne
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 131 participants (actual)
- Lead sponsor
- Catabasis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 2 Oct 2018 (actual)
- Primary completion
- 22 Sep 2020 (actual)
- Study completion
- 22 Sep 2020 (actual)
- First posted
- 12 Oct 2018 (actual)
- Results
- Posted 21 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The study includes a 52-week, randomized, double-blind, placebo-controlled period, followed by a 2-week follow- up. Approximately 125 boys with DMD will be enrolled in this trial, with 2 boys receiving edasalonexent for every 1 boy receiving placebo.
Following completion of the treatment period, patients may elect to continue in a separate open-label extension study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dose 1 | Experimental | Drug: EdasalonexentEdasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day. |
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo |
Who may qualify
- Ages
- 4 to 7 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
- Diagnosis of DMD based on a clinical phenotype with increased serum creatine kinase (CK) and documentation of mutation(s) in the dystrophin gene known to be associated with a DMD phenotype
- Able to perform stand from supine without assistance in ≤ 10 seconds
- Able to perform the 10MWT and 4-stair climb
- Followed by a doctor or medical professional who coordinates Duchenne care on a regular basis and willingness to disclose patient's study participation with medical professionals
Key exclusion criteria (5 listed)
- Use of corticosteroids within 24 weeks prior to Day 1; use of inhaled, intranasal, and topical corticosteroids is permitted
- Use of another investigational drug, idebenone, or dystrophin-focused therapy within 4 weeks. Exception: Patients who have received at least 24 weeks of a stable dose of eteplirsen prior to Day 1, and expected to continue treatment, will be eligible
- Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, warfarin, phenytoin, S mephenytoin, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus, tacrolimus, or paclitaxel
- Use of human growth hormone within 3 months prior to Day 1
- Other prior or ongoing significant medical conditions
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EdasalonexentAlso: Edasa, CAT-1004100 mg/kg/day | Drug | Dose 1 |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
Change From Baseline in North Star Ambulatory Assessment (NSAA)
Time frame: Baseline (Day 1) to Week 52
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
4 secondary outcomes
Change From Baseline in 10-meter Walk/Run Test
Time frame: Baseline (Day 1) to Week 52
Change From Baseline in Time to Stand From Supine
Time frame: Baseline (Day 1) to Week 52
Change From Baseline in 4-stair Climb
Time frame: Baseline (Day 1) to Week 52
Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Up to Week 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jun 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34120912Finkel RS, McDonald CM, Lee Sweeney H, Finanger E, Neil Knierbein E, Wagner KR, Mathews KD, Marks W, Statland J, Nance J, McMillan HJ, McCullagh G, Tian C, Ryan MM, O'Rourke D, Muller-Felber W, Tulinius M, Burnette WB, Nguyen CT, Vijayakumar K, Johannsen J, Phan HC, Eagle M, MacDougall J, Mancini M, Donovan JM; (For the PolarisDMD Study Group). A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial. J Neuromuscul Dis. 2021;8(5):769-784. doi: 10.3233/JND-210689.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joanne M Donovan, Chief Medical Officer, MD, PhD study chair
Catabasis Pharmaceuticals
40 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Edasalonexent in people with Muscular Dystrophy, Duchenne, run by Catabasis Pharmaceuticals at 40 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in United States, Canada, United Kingdom, Australia, Germany and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Jun 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in North Star Ambulatory Assessment (NSAA) (time frame: Baseline (Day 1) to Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03703882, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jun 2022.