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Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy (MoveDMD®)

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ClinicalTrials.gov Identifier: NCT02439216
Recruitment Status : Active, not recruiting
First Posted : May 8, 2015
Last Update Posted : January 31, 2018
Sponsor:
Information provided by (Responsible Party):
Catabasis Pharmaceuticals

Brief Summary:

The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from ≥4 to <8 years of age will be enrolled.

Edasalonexent is an orally administered small molecule targeted to inhibit activated NF-κB, a molecule that is activated from infancy in DMD and which is central to causing muscle damage and preventing muscle regeneration. Data on magnetic resonance imaging of the lower and upper leg muscles, physical function (including timed function tests) and muscle strength will be studied.


Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Drug: Edasalonexent Drug: Placebo Phase 1 Phase 2

Detailed Description:

Part A was a 1-week, open-label study to assess safety, tolerability, pharmacokinetics and biomarkers for three dose levels of edasalonexent and is now complete.

Part B was a randomized, double-blind, placebo-controlled, multiple dose study to evaluate the safety, efficacy, PK, and PD of edasalonexent over 12 weeks. Patients who participated in Part A also participated in Part B, along with newly enrolled patients. Patients received either edasalonexent 67 mg/kg/day, edasalonexent 100 mg/kg/day, or placebo in Part B. Part B is now complete.

Following completion of Part B, patients receive edasalonexent for 112 weeks in Part C, the open-label portion of the MoveDMD study. Patients on the 67 mg/kg/day treatment moved to the 100 mg/kg/day treatment. Patients on the 100 mg/kg/day treatment remained on the 100 mg/kg/day treatment.

If clinically indicated, concomitant treatment with eteplirsen (Exondys 51™) may be acceptable in patients with amenable gene mutations during Part C after the patient has been exposed to edasalonexent for 6 months.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 31 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Study of Edasalonexent (CAT-1004) in Pediatric Patients With Duchenne Muscular Dystrophy
Study Start Date : April 2016
Actual Primary Completion Date : January 12, 2017
Estimated Study Completion Date : February 2019


Arm Intervention/treatment
Experimental: Dose 1
Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day
Drug: Edasalonexent
Other Names:
  • CAT-1004
  • Edasa

Experimental: Dose 2
Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day
Drug: Edasalonexent
Other Names:
  • CAT-1004
  • Edasa

Placebo Comparator: Placebo
Matching placebo
Drug: Placebo



Primary Outcome Measures :
  1. Safety and tolerability (Adverse Events) [ Time Frame: 12 Weeks ]
  2. Muscle composition and inflammation as measured by MRI [ Time Frame: 12 Weeks ]

Secondary Outcome Measures :
  1. Physical function, muscle strength, and parent/proxy reported physical functioning/quality of life [ Time Frame: 12 Weeks ]
  2. Edasalonexent PK and PD measures [ Time Frame: 12 Weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 7 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Written informed consent from parent or legal guardian prior to participation and, for patients who are 7 years of age, written assent from patient
  • Diagnosis of DMD based on a clinical phenotype with increased serum CK and the presence of a mutation in the dystrophin gene known to be associated with a DMD phenotype
  • Ability to walk independently (assistive devices are permitted)
  • Adequate immunization for influenza and varicella

Exclusion Criteria:

  • Use of corticosteroids within prior 6 months of treatment initiation or planning to initiate steroid therapy within the next 6 months
  • Other prior or ongoing significant medical conditions
  • Exposure to another investigational drug (such as eteplirsen or idebenone) within 28 days prior to start of study treatment or ongoing participation in any other therapeutic clinical trial

    • Note: There are separate criteria for patients who participated in Part A versus newly enrolling patients. New patients must meet all of the Part A entry criteria to participate in Part B.

Patients who participated in Part A must meet the following criteria to participate in Part B:

  • Completed Part A
  • Continue to meet all of the Part A entry criteria, including an absence of safety concerns (however, patients may be ≥8 years of age)

There are no entry criteria for Part C; all patients who complete Part B will automatically continue in Part C


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02439216


Locations
United States, California
Los Angeles, California, United States, 90095
United States, Florida
Gainesville, Florida, United States, 32610
Orlando, Florida, United States, 32827
United States, Oregon
Portland, Oregon, United States, 97239
United States, Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Catabasis Pharmaceuticals

Responsible Party: Catabasis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02439216     History of Changes
Other Study ID Numbers: CAT-1004-201
First Posted: May 8, 2015    Key Record Dates
Last Update Posted: January 31, 2018
Last Verified: January 2018

Keywords provided by Catabasis Pharmaceuticals:
Muscular Dystrophies
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases
Duchenne muscular dystrophy
DMD
dystrophin
dystrophy
Duchenne

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked