Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
๐ฉบ Protocol Summary
This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.
Primary Research Facility: Liverpool Hospital (Liverpool, New South Wales)
๐ Basic Study Criteria & Logistics Checklist
0 of 3 CheckedReview these 3 basic logistical and protocol criteria extracted from the public NIH record. (This is a preliminary logistical checklist, not an official clinical eligibility determination).
๐ Primary Site Map
Site: Liverpool Hospital โ Liverpool, New South Wales
๐ All Participating Trial Locations (35 Sites)
Multi-Center TrialThis protocol is enrolling participants across multiple clinical centers:
๐ Print Doctor Discussion Sheet
Preview and print a 1-page summary with 5 tailored questions for your physician.
๐ฐ Clinical Trial Costs & Insurance Coverage
Coverage for clinical trial participation varies depending on your health insurance policy, state legislation, and whether specific procedures are investigational research or routine medical care. Under federal law (Affordable Care Act ยง 2709), health insurers generally cannot drop coverage or refuse to pay for routine patient care costs incurred in approved clinical trials.
๐ก Always ask the study coordinator for the trial's official Informed Consent Form, which outlines all sponsor-funded research items versus procedures billed to your insurance.