A phase II, open-label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy

Phase 2

Completed

• Conditions: Duchenne disease; Duchenne muscular dystrophy; 10028302

• Registration Number: NL-OMON41314
• Lead Sponsor: Prosensa Therapeutics B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 3

Inclusion Criteria

1. Subjects previously treated with PRO044 in the PRO044-CLIN-01 study.;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 or cessation of glucocorticoids will be at the
discretion of the 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 or during participation in the study.
9. Current or history of drug and/or alcohol abuse.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Safety, tolerability and efficacy</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Pharmacokinetic profile</p><br>