Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

NCT02329769 | Terminated Phase 2 DMC

• 1 other identifier: PRO044-CLIN-02
• Study Type: interventional
• Target: 15
• Locations: 4 countries
• Sites: 5

Brief Summary

The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne muscular dystrophy; DMD; BioMarin; PRO044

Outcome Measures

Primary Outcomes (2)

• Efficacy of PRO044 (composite of several measures)

  Efficacy parameters: Muscle Function * 6 Minute Walk Distance (6MWD) * North Star Ambulatory Assessment * Timed tests (10-meter walk/run, rising from floor, stair climb) * DMD Functional Outcomes Questionnaire (DMD-FOS) -for ambulant subjects only * Egen Klassification - for non-ambulant subjects. Muscle strength * Pulmonary Function (Spirometry) * Handheld myometry. Exploratory: * Performance Upper Limb (PUL). * Patient Reported Outcome measure (PROM).

  After 48 weeks of treatment

• Safety and tolerability of PRO044 (treatement emergent adverse events)

  Number of subjects with 1 or more treatement emergent adverse events following SC or IV PRO044 dosing

  After 48 weeks of treatment

Secondary Outcomes (1)

• Assess the pharmacokinetics of PRO044 (composite of several measures)

  After 48 weeks of treatment

Study Arms (3)

PRO044 SC 6 mg/kg

EXPERIMENTAL

Weekly subcutaneous (SC) dosing with 6 mg/kg

Drug: PRO044 SC 6 mg/kg

PRO044 IV 6 mg/kg

EXPERIMENTAL

Weekly intravenous (IV) dosing with 6 mg/kg

Drug: PRO044 IV 6 mg/kg

PRO044 SC 9 mg/kg

EXPERIMENTAL

Weekly intravenous (IV) dosing with 9 mg/kg

Drug: PRO044 IV 9 mg/kg

Interventions

PRO044 SC 6 mg/kg DRUG

PRO044 SC 6 mg/kg

PRO044 IV 6 mg/kg DRUG

PRO044 IV 6 mg/kg

PRO044 IV 9 mg/kg DRUG

PRO044 SC 9 mg/kg

Eligibility Criteria

• Age: 9 Years - 20 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subjects previously treated with PRO044.
• Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to the dose regimen or cessation of glucocorticoids will be at the discretion of the Principle Investigator (PI) in consultation with the subject/parent and the Medical Monitor. If the subject is not on steroids, involvement in the study needs to be discussed with the medical monitor

You may not qualify if:

• Current, or history of, liver or renal disease.
• Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements.
• Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study
• Need for daytime mechanical ventilation.
• Screening aPTT above the upper limit of normal (ULN).
• Screening platelet count below the lower limit of normal (LLN).
• Use of anticoagulants, antithrombotics or antiplatelet agents.
• Use of any investigational product within 6 months prior to the start of Screening for the study.
• Current or history of drug and/or alcohol abuse.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (5)

UZ Leuven

Leuven, Belgium

Location

S.Anna Hospital

Ferrara, Italy

Location

Policlinico Universitario Agostino Gemelli

Roma, Italy

Location

Leids Universitair Medisch Centrum

Leiden, Netherlands

Location

Drottning Silvias Barn- ochungdomssjukhus

Gothenburg, Sweden

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2014
• First Posted: January 1, 2015
• Study Start: December 1, 2014
• Primary Completion: July 1, 2016
• Study Completion: August 31, 2016
• Last Updated: December 8, 2017

Record last verified: 2017-12

Locations

NL (1); IT (2); BE (1); SE (1)