Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls. (NCT06692712)
Intrathecal Administration of MELPIDA (AAV9/AP4M1) For Hereditary Spastic Paraplegia Type 50 (SPG50): A Phase 3, Open-Label Trial With Matched Prospective Concurrent Control Arm
🩺 Plain-English Summary (8th-Grade Level)
Phase 3, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of MELPIDA in individuals with Hereditary Spastic Paraplegia Type 50 (SPG50).
Who can join: Review the eligibility criteria below.
Location: University of Texas Southwestern Medical Center — Dallas, Texas
Age range: 4 Months to 72 Months
🧪 Interventions in This Study
- MELPIDA (GENETIC)
📋 "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 (2 Sites)
- University of Texas Southwestern Medical Center — Dallas, Texas
- Sant Joan de Deu — Barcelona,
📞 Contact the Study Coordinator
Phone: (833) 335-7432
Email: studyinfo@elpidatx.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 Hereditary Spastic Paraplegia Type 50 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.