Pivotal Study of N-acetyl-L-leucine for CACNA1A (NCT07221292)
Effects of N-Acetyl-L-Leucine on CACNA1A Disorders: A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study
🩺 Plain-English Summary (8th-Grade Level)
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.
Who can join: Review the eligibility criteria below.
Location: Johns Hopkins University — Baltimore, Maryland
Age range: 4 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (10 Sites)
- Johns Hopkins University — Baltimore, Maryland
- Massachusetts General Hospital — Boston, Massachusetts
- The University of Texas Health (UT Health) — Houston, Texas
- Medical University of Innsbruck — Innsbruck,
- University of Cologne — Cologne,
- University of Athens — Athens,
- University of Milan — Milan,
- Bambino Gesu' Children's Research Hospital — Rome,
- University Hospital Bern Inselspital — Bern,
- University of Oxford - John Radcliffe Hospital — Oxford,
📞 Contact the Study Coordinator
Phone: +447426956369
Email: tfields@intrabio.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
Explore by condition: All CACNA1A clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.