A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
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- STATUS
- Recruiting
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- participants needed
- 20
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- sponsor
- GeneTX Biotherapeutics, LLC
Summary
This is a Phase 1/2, open-label, multiple-dose, dose-escalating study to evaluate the safety, tolerability, and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102 in pediatric patients with Angelman syndrome. Approximately 20 patients (male and female) 4 and 17 years of age with a genetically confirmed diagnosis of full maternal UBE3A gene deletion (within 15q11.2-q13 region) will be enrolled.
Description
This is a Phase 1/2, open-label, multiple-dose, dose-escalating study to evaluate the safety, tolerability, and plasma and CSF concentrations of GTX-102 in pediatric patients with AS.
The study includes screening, baseline, treatment, and safety follow-up periods. Eligible patients who meet all of the inclusion criteria and none of the exclusion criteria and who successfully complete all screening and baseline assessments will be assigned to one of 5 sequential dose cohorts. GTX-102 will be administered by intrathecal (IT) injection via lumbar puncture (LP) at Baseline followed by 3 subsequent IT injections on study Days 30, 58 and 86. The procedure for IT administration of GTX-102 will performed by a clinician with experience in LP and anesthetic care will be directed by experienced anesthesiologists with a focus on patient safety and comfort. The total duration of study for each patient is anticipated to be approximately 6 months. An open-label extension (OLE) study will be conducted under a separate protocol where patients can receive continued treatment with GTX-102.
Details
| Condition | Angelman Syndrome |
|---|---|
| Age | 4-17 years |
| Treatment | GTX-102 |
| Clinical Study Identifier | NCT04259281 |
| Sponsor | GeneTX Biotherapeutics, LLC |
| Last Modified on | 19 February 2024 |
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
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