Clinical Trials Logo
  1. Home
  2. Angelman Syndrome
  3. NCT04259281

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

Angelman Syndrome Clinical Trial

Official title:
A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)

Clinical Trial Summary

The primary objective of the study is to evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to participants with Angelman Syndrome (AS).

Clinical Trial Description

This is a Phase 1/2, open-label, multiple-dose, study to evaluate the safety, tolerability, and plasma and CSF concentrations of GTX-102 in pediatric participants with AS. The study includes a Loading phase followed by a Maintenance phase. Participants may continue on GTX-102 during the Maintenance phase of the study until GTX-102 is commercially available, intolerable toxicity occurs, the parent/legal guardian withdraws consent, the participant enrolls in another experimental study, or this study is terminated. This study was previously posted by GeneTX Biotherapeutics, LLC and was transferred to Ultragenyx in July 2022. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04259281
Study type Interventional
Source Ultragenyx Pharmaceutical Inc
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date February 24, 2020
Completion date December 2025
View Details
See also
  Status Clinical Trial Phase
Recruiting NCT05293184 - The Global Angelman Syndrome Registry
Completed NCT00004351 - Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes N/A
Enrolling by invitation NCT06139172 - Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities N/A
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT06229769 - Natural History Study for Patients With Angelman Syndrome
Enrolling by invitation NCT03836300 - Parent and Infant Inter(X)Action Intervention (PIXI) N/A
Completed NCT03235037 - Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome N/A
Recruiting NCT05100810 - Angelman Syndrome Natural History Study-FAST UK
Completed NCT03644693 - Nutritional Formulation for Angelman Syndrome N/A
Completed NCT03358823 - Study on the Brain Network of Angelman Syndrome
Recruiting NCT06337383 - Study of the Prevalence of Autistic Traits in Angelman Syndrome
Recruiting NCT05945576 - IDMet (RaDiCo Cohort) (RaDiCo-IDMet)
Completed NCT02670694 - Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW N/A
Recruiting NCT04768803 - Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or Obesity
Recruiting NCT05630066 - Study to Investigate the Pharmacokinetics and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype. Phase 2
Completed NCT00348933 - Dietary Supplements for the Treatment of Angelman Syndrome N/A
Recruiting NCT05011851 - An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome Phase 2
Terminated NCT03882918 - An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome Phase 3
Completed NCT02996305 - A Study in Adults and Adolescents With Angelman Syndrome (STARS) Phase 2
Completed NCT02056665 - Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome Phase 2