Actively Recruiting
Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.
Led by Elpida Therapeutics SPC · Updated on 2026-04-20
24
Participants Needed
2
Research Sites
321 weeks
Total Duration
On this page
Sponsors
E
Elpida Therapeutics SPC
Lead Sponsor
U
University of Texas Southwestern Medical Center
Collaborating Sponsor
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female children aged 4 months to 72 months at screening for the treatment group.
- Molecularly confirmed diagnosis of SPG50 by genetic testing showing bi-allelic pathogenic variants in the AP4M1 gene for treatment group.
- Presence of neurological dysfunction symptoms by clinical history and examination.
- Stable doses of medications like anti-spasticity, anti-seizure, behavioral, sleep medications, or nutritional support for at least 3 months before screening.
- Parent or legal guardian willing to provide informed consent.
- Ability of the subject and caregiver to travel to the study center and reside within 100 miles for 30 days after treatment.
- For control group, male and female children aged 4 to 72 months with molecularly confirmed SPG47, SPG50, or SPG52.
- Ability to comply with all protocol requirements and procedures.
You will not qualify if you...
- Loss of any of the 8 major motor milestones in the last 12 months, including sitting, crawling, standing, walking, or jumping.
- Inability to participate in clinical evaluations as determined by investigators.
- Clinically significant abnormal lab values including hemoglobin, white blood cell count, platelets, INR, liver enzymes, bilirubin, or creatinine.
- Medical conditions preventing lumbar puncture or sedation such as scoliosis or bleeding disorders.
- Documented cardiomyopathy or serious congenital heart problems.
- History of severe allergic reactions to immunosuppressive drugs or gadolinium.
- Illness or chronic treatments posing risks for gene therapy or immunosuppression.
- Contraindications for MRI or other study procedures.
- Significant AP-4 related central nervous system impairment or behavioral issues confounding study results.
- Recent or planned elective surgery within 6 months.
- Failure to obtain informed consent.
- Expected non-compliance with study procedures.
- Use of investigational drugs within 30 days before screening or planned use during the study (except gene therapy).
- Participation in another interventional clinical trial within 90 days before screening.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75025
Actively Recruiting
2
Sant Joan de Deu
Barcelona, Spain, 08950
Active, Not Recruiting
Research Team
R
Rachel Thomas
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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