Actively Recruiting
Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
Led by Elpida Therapeutics SPC · Updated on 2024-10-08
4
Participants Needed
1
Research Sites
397 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
MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss. Outcomes will evaluate the safety and tolerability of a single dose of MELPIDA, which will be measured by the treatment-associated adverse events (AEs) and serious adverse events (SAEs). Secondarily, the trial will explore efficacy in terms of disease burden assessments.
CONDITIONS
Official Title
Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 4 months to 10 years old
- Confirmed SPG50 diagnosis by genetic testing showing pathogenic variants in the AP4M1 gene or clinical features consistent with SPG50 including neurologic dysfunction
- Parent or legal guardian able and willing to provide written informed consent
- Able to follow all study procedures
- Ability to stand for more than 5 seconds OR take 5 steps independently or with a walker OR have a Modified Ashworth Scale score of 2 or below in the ankles
You will not qualify if you...
- Unable to participate in study procedures as determined by the investigator
- Medical condition preventing lumbar puncture or use of anesthetics
- History of bleeding disorder or contraindication to lumbar puncture
- Cannot be safely sedated according to anesthesiologist
- Active infection at time of dosing
- Concomitant illness or chronic drug treatment posing risks for gene therapy
- Unable to undergo MRI or other required procedures
- Significant non-SPG50 related central nervous system impairment or behavioral disturbances
- Received investigational drug within 30 days before screening or plans to receive one during study
- Currently enrolled in another interventional clinical trial
- Contraindication to MELPIDA or immune suppression medications used
- Abnormal laboratory values including elevated liver enzymes, creatinine ≥1.5 mg/dL, hemoglobin <6 or >20 g/dL, or white blood cell count >20,000 per cmm prior to gene therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Children's Medical Center Dallas
Dallas, Texas, United States, 75235
Actively Recruiting
Research Team
S
Sydney Cooper, MSc
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here