|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
12 December 2020 |
|
Main ID: |
NCT02530905 |
|
Date of registration:
|
10/08/2015 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
|
|
Scientific title:
|
A Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping |
|
Date of first enrolment:
|
October 14, 2015 |
|
Target sample size:
|
12 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT02530905 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
|
|
Phase:
|
Phase 1
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Medical Director |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Sarepta Therapeutics, Inc. |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Genotypically confirmed DMD (amenable to exon 45 skipping).
- Stable cardiac and pulmonary function.
- Limited or no ambulation.
- On a stable dose of oral corticosteroids for at least 24 weeks OR has not received
corticosteroids for at least 24 weeks.
Exclusion Criteria:
- Current or previous treatment with the experimental agents SMT C1100 (BMN-195) or
PRO045.
- Other experimental treatment in the past 12 weeks.
- If on cardiac medication, must be on a stable dose for the past 12 weeks.
- Major surgery within the past 3 months.
Other inclusion/exclusion criteria apply.
Age minimum:
7 Years
Age maximum:
21 Years
Gender:
Male
|
|
Health Condition(s) or Problem(s) studied
|
|
Duchenne Muscular Dystrophy
|
|
Intervention(s)
|
|
Drug: Placebo
|
|
Drug: SRP-4045
|
|
Primary Outcome(s)
|
|
Incidence of adverse events
[Time Frame: Approximately 12 Weeks (double-blind dose titration)]
|
|
Secondary Outcome(s)
|
|
Drug concentration in plasma
[Time Frame: Approximately 12 Weeks (double-blind dose titration)]
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|