A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
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ClinicalTrials.gov Identifier: NCT04259281 |
Recruitment Status :
Active, not recruiting
First Posted : February 6, 2020
Last Update Posted : May 3, 2024
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Tracking Information | |||||
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First Submitted Date ICMJE | January 31, 2020 | ||||
First Posted Date ICMJE | February 6, 2020 | ||||
Last Update Posted Date | May 3, 2024 | ||||
Actual Study Start Date ICMJE | February 24, 2020 | ||||
Estimated Primary Completion Date | December 2025 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), Adverse Events of Special Interest (AESIs), AEs Leading to Discontinuation and Severity of AEs [ Time Frame: Up to Day 337 ] | ||||
Original Primary Outcome Measures ICMJE |
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Change History | |||||
Current Secondary Outcome Measures ICMJE |
Pharmacokinetics of GTX-102 over time [ Time Frame: Up to Day 337 ] Maximum drug concentration (Cmax)
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Original Secondary Outcome Measures ICMJE |
Pharmacokinetics of GTX-102 [ Time Frame: Day 2, Day 3, Day 4, Day 30, Day 58, Day 86, Day 128 (end of study) ] Maximum drug concentration (Cmax)
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures |
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Descriptive Information | |||||
Brief Title ICMJE | A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome | ||||
Official Title ICMJE | 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) | ||||
Brief 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). | ||||
Detailed 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. |
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Angelman Syndrome | ||||
Intervention ICMJE | Drug: GTX-102
antisense oligonucleotide
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Study Arms ICMJE |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Actual Enrollment ICMJE |
74 | ||||
Original Estimated Enrollment ICMJE |
20 | ||||
Estimated Study Completion Date ICMJE | December 2025 | ||||
Estimated Primary Completion Date | December 2025 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 4 Years to 17 Years (Child) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Australia, Canada, France, Germany, Israel, Spain, United Kingdom, United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT04259281 | ||||
Other Study ID Numbers ICMJE | GTX-102-001 2021-001793-36 ( EudraCT Number ) |
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Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Ultragenyx Pharmaceutical Inc | ||||
Original Responsible Party | GeneTX Biotherapeutics, LLC | ||||
Current Study Sponsor ICMJE | Ultragenyx Pharmaceutical Inc | ||||
Original Study Sponsor ICMJE | GeneTX Biotherapeutics, LLC | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | Ultragenyx Pharmaceutical Inc | ||||
Verification Date | May 2024 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |