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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02329769
• Other study ID #: PRO044-CLIN-02
• Status: Terminated
• Phase: Phase 2
• First received: December 22, 2014
• Last updated: December 6, 2017
• Start date: December 2014
• Est. completion date: August 31, 2016

Study information

• Verified date: December 2017
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

A Phase II, open-label, extesion study. Following a Screening period of up to one month, subjects previously treated with PRO044, and eligible for enrolment in PRO044-CLIN-02, will be allocated to one of three groups to receive either 6 mg/kg or 9 mg/kg PRO044 weekly by IV infusion or 6 mg/kg weekly by SC injection for 48 weeks.

Safety and tolerability, pharmacokinetics (PK), pharmacodynamic (PD) and efficacy assessments will be conducted at regular intervals throughout the study.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Est. completion date: August 31, 2016
• Est. primary completion date: July 1, 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 9 Years to 20 Years

Eligibility

Inclusion Criteria:

1. Subjects previously treated with PRO044.

2. 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

Exclusion Criteria:

1. Current, or history of, liver or renal disease.

2. Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements.

3. Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study

4. Need for daytime mechanical ventilation.

5. Screening aPTT above the upper limit of normal (ULN).

6. Screening platelet count below the lower limit of normal (LLN).

7. Use of anticoagulants, antithrombotics or antiplatelet agents.

8. Use of any investigational product within 6 months prior to the start of Screening for the study.

9. Current or history of drug and/or alcohol abuse.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• PRO044 SC 6 mg/kg

• PRO044 IV 6 mg/kg

• PRO044 IV 9 mg/kg

Locations

Country	Name	City	State
Belgium	UZ Leuven	Leuven
Italy	S.Anna Hospital	Ferrara
Italy	Policlinico Universitario Agostino Gemelli	Roma
Netherlands	Leids Universitair Medisch Centrum	Leiden
Sweden	Drottning Silvias Barn- ochungdomssjukhus	Goteborg

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

Belgium,  Italy,  Netherlands,  Sweden, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	Assess the pharmacokinetics of PRO044 (composite of several measures)	Pharmacokinetic parameters: t ½; AUC: 0-24h, 0-8 (where applicable); Cmax; tmax; CL (for IV subjects) or CL/F (for SC subjects); PRO044 concentrations in muscle tissue.	After 48 weeks of treatment