Phase 3 Study of MELPIDA for Hereditary Spastic Paraplegia Type 50 (SPG50)
This study is testing a gene therapy called MELPIDA for children with Hereditary Spastic Paraplegia Type 50 (SPG50). SPG50 is a genetic condition that affects movement. MELPIDA is designed to deliver a working copy of the AP4M1 gene, which is faulty in people with SPG50. The study will give MELPIDA as a single injection into the fluid around the spinal cord. Researchers will look at how well MELPIDA helps with motor skills, like sitting, standing, and walking, over 156 weeks (about 3 years). To join, you must be between 4 months and 72 months old and have a confirmed diagnosis of SPG50 through genetic testing. This study is currently unclear about its recruitment status.
- Study design
- This is an open-label Phase 3 study, meaning both you and the study team will know you are receiving MELPIDA. It plans to enroll 24 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for 156 weeks (about 3 years) to assess motor function.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.
At a glance
Conditions
NCT06692712
Where you'd take part
This study runs at 2 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
University of Texas Southwestern Medical Center
Dallas, Texasno site contact published
Recruiting
Sant Joan de Deu
Barcelona, Spainno site contact published
Active, not recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Gross Motor Function Measure (GMFM-88) Defined Major Milestones156 weeks
Change in total percent score of the 8 Major Motor Milestone Scores from baseline in treated group compared to change in total percent score of the 8 Major Motor Milestone Scores from baseline in untreated controls 1. #24: Sit on mat: Maintain, arms free, 3 seconds 2. #38: Prone: Creeps forward 1.8m (6') 3. #52: On the floor: Pulls to stand at large bench 4. #67: Standing: 2 hands held: walks forward 10 steps 5. #69: Standing: Walks forward 10 steps 6. #84: Standing: Holding 1 rail: walks up 4 steps, holding 1 rail, alternating feet 7. #85: Standing: Holding 1 rail: walks down 4 steps, holding 1 rail, alternating feet 8. #88: Standing on 15cm (6") step: Jumps off, both feet simultaneously