Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
Recruiting
Phase 1
Phase 2
Interventional Study
Spinal Muscular Atrophy Type 3
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 2 and older
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 21 (estimated)
- Sponsor
- GeneCradle Inc · Industry
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About This Trial
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* ≥2 years of age on the day of signing the informed consent form;
* Genetic and clinical diagnosis of type 3 SMA with bi-allelic deletion of SMN1 of 5qSMA;
* Hammersmith Functional Motor Scale - Expanded (HFMSE) score is between 10 and 54 at screening;
* Female patients of chil…
Contacts