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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-510917-14-00 | EU Trial (CTIS) Number |
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The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
This study is a phase 3, long term extension (LTE) which is open to participants rolling over from a prior GTX-102 clinical study. The LTE study will evaluate the long-term safety and efficacy of GTX-102 in participants with AS.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GTX-102 | Experimental | Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GTX-102 | Drug | Antisense Oligonucleotide |
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| Measure | Description | Time Frame |
|---|---|---|
| Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | 5 Years |
| Measure | Description | Time Frame |
|---|---|---|
| Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score | Month 0, 5 Years |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceuticals Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Los Angeles (UCLA) | Los Angeles | California | 90095 | United States | ||
| University of California, San Diego - Rady Children's Hospital |
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| Label | URL |
|---|---|
| Ultragenyx Patient Advocacy Website | View source |
| Ultragenyx Transparency Commitment | View source |
| Ultragenyx Angelman Syndrome (AS) Research Study Opportunity |
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| San Diego |
| California |
| 92123 |
| United States |
| Rare Disease Research, LLC | Atlanta | Georgia | 30329 | United States |
| Rush University Medical Center | Chicago | Illinois | 60612 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Weill Cornell Medicine | New York | New York | 10021 | United States |
| Queensland Children's Hospital | South Brisbane | Queensland | 4101 | Australia |
| The Royal Children's Hospital | Melbourne | Victoria | 3052 | Australia |
| MAGIC Clinic Ltd | Calgary | Alberta | T2E 7Z4 | Canada |
| British Columbia Children's Hospital | Vancouver | British Columbia | V6H 3V4 | Canada |
| Childrens Hospital London Health Sciences Centre | London | Ontario | N6A 5W9 | Canada |
| Childrens Hospital of Eastern Ontario | Ottawa | Ontario | K1H 8L1 | Canada |
| The Research Institute of the McGill University Health Centre | Montreal | Quebec | H3G 1A4 | Canada |
| AP-HM - Hopital de la Timone | Marseille | 13385 | France |
| AP-HP Hopital Necker-Enfants Malades | Paris | 75015 | France |
| Universitaetsklinikum Hamburg-Eppendorf | Hamburg | 20246 | Germany |
| Universitaetsklinikum Leipzig | Leipzig | 04103 | Germany |
| Sheba Medical Center | Ramat Gan | 5265601 | Israel |
| Hospital Universitario Puerta de Hierro | Majadahonda | 28222 | Spain |
| Corporacio Sanitaria Parc Tauli - Hospital de Sabadell | Sabadell | 08208 | Spain |
| University of Cambridge | Cambridge | CB2 0QQ | United Kingdom |
| Great Ormond Street Hospital for Children | London | WC1N 3JH | United Kingdom |
| Oxford University Hospitals NHS Foundation Trust | Oxford | OX3 9DU | United Kingdom |
| View source |
| ID | Term |
|---|---|
| D017204 | Angelman Syndrome |
| ID | Term |
|---|---|
| D009069 | Movement Disorders |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000096803 | Imprinting Disorders |
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