Angelman Syndrome
Conditions
Brief summary
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Detailed description
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.
Interventions
DRUGGTX-102
Antisense Oligonucleotide
Sponsors
Ultragenyx Pharmaceutical Inc
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Signed informed consent from parent(s) or legal guardian(s). * Completed a final study visit in a prior clinical trial with GTX-102. If the subject has an ongoing adverse event, medical monitor approval is required to rollover. The Screening visit must occur within 6 months of the last visit in the prior GTX-102 trial. * From the time of informed consent through the end of the study and for at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102.
Exclusion criteria
* Discontinued from study or withdrew from study treatment in a prior GTX-102 trial. * History or evidence of any other medical, neurological, or psychological condition that would expose the subject to an undue risk of a significant AE or interfere with study assessments during the course of the trial as determined by the clinical judgment of the Investigator. * Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects. * Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of Treatment Emergent Adverse Events (TEAEs) | Up to 5 Years |
Secondary
| Measure | Time frame |
|---|---|
| Change from Baseline in the Bayley-4 Raw Score | Baseline, Up to 5 Years |
| Net Response in Multidomain Responder Index (MDRI) | Month 12, Month 24 |
Countries
Australia, Canada, France, Germany, Israel, Spain, United Kingdom, United States
Contacts
STUDY_DIRECTORMedical Director
Ultragenyx Pharmaceuticals Inc.
Outcome results
None listed