Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

NCT02439216 | Completed Phase 1 Results DMC

• 1 other identifier: CAT-1004-201
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 5

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.

Conditions

Muscular Dystrophy, Duchenne

Keywords

Muscular Dystrophies; Musculoskeletal Diseases; Nervous System Diseases; Neuromuscular Diseases; Duchenne muscular dystrophy; DMD; dystrophin; dystrophy; Duchenne

Outcome Measures

Primary Outcomes (1)

• Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B

  The LLC5-T2 calculated from the unweighted average of the T2 relaxation times of all 5 lower leg muscles for each patient at each evaluation (the medial gastrocnemius, peroneals, soleus, tibialis anterior, and tibialis posterior muscles). Increases in LLC5-T2 relaxation time indicate muscle damage, inflammation, edema, and fat infiltration and are highly correlated with muscle fat

  Baseline to Week 12

Secondary Outcomes (4)

• Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C

  Baseline to Week 12

• Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C

  Baseline to Week 12

• Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C

  Baseline to Week 12

• Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).

  Screening to Week 152

Study Arms (3)

Dose 1

EXPERIMENTAL

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Drug: Edasalonexent

Dose 2

EXPERIMENTAL

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

Drug: Edasalonexent

Placebo

PLACEBO COMPARATOR

Matching placebo

Drug: Placebo

Interventions

Edasalonexent DRUG

Also known as: CAT-1004, Edasa

Dose 1; Dose 2

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 4 Years - 7 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Catabasis Pharmaceuticals: lead

Study Sites (5)

Unknown Facility

Los Angeles, California, 90095, United States

Location

Unknown Facility

Gainesville, Florida, 32610, United States

Location

Unknown Facility

Orlando, Florida, 32827, United States

Location

Unknown Facility

Portland, Oregon, 97239, United States

Location

Unknown Facility

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies; Musculoskeletal Diseases; Nervous System Diseases; Neuromuscular Diseases

Interventions

edasalonexent

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Andrew Nichols, PhD - Chief Scientific Officer
• Organization: Astria Therapeutics, Inc

anichols@astriatx.com

617-349-1971

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 29, 2015
• First Posted: May 8, 2015
• Study Start: April 1, 2016
• Primary Completion: January 12, 2017
• Study Completion: August 1, 2019
• Last Updated: September 23, 2022
• Results First Posted: September 23, 2022

Record last verified: 2022-09

Locations

US (5)