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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06415344




Registration number
NCT06415344
Ethics application status
Date submitted
10/05/2024
Date registered
16/05/2024
Date last updated
15/11/2024

Titles & IDs
Public title
Long-term Extension of GTX-102 in Angelman Syndrome
Scientific title
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome
Secondary ID [1] 0 0
2024-510917-14-00
Secondary ID [2] 0 0
GTX-102-CL302
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Angelman Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GTX-102

Experimental: GTX-102 - Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule.


Treatment: Drugs: GTX-102
Antisense Oligonucleotide
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
5 Years
Secondary outcome [1] 0 0
Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score
Timepoint [1] 0 0
Month 0, 5 Years

Eligibility
Key inclusion criteria
* Signed informed consent from parent(s) or legal guardian(s).
* Prior participation in a clinical trial with GTX-102; the timing for the roll-over into this study from a prior GTX-102 study is based on the Investigator and Ultragenyx Medical Monitor's approval.
* 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.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/07/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/02/2029
Actual
Sample size
Target
75
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [2] 0 0
Austin Health - Heidelberg
Recruitment hospital [3] 0 0
The Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment postcode(s) [2] 0 0
3084 - Heidelberg
Recruitment postcode(s) [3] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
Canada
State/province [6] 0 0
Alberta
Country [7] 0 0
Canada
State/province [7] 0 0
British Columbia
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
France
State/province [10] 0 0
Marseille
Country [11] 0 0
France
State/province [11] 0 0
Paris
Country [12] 0 0
Germany
State/province [12] 0 0
Hamburg
Country [13] 0 0
Germany
State/province [13] 0 0
Leipzig
Country [14] 0 0
Israel
State/province [14] 0 0
Tel HaShomer
Country [15] 0 0
Spain
State/province [15] 0 0
Majadahonda
Country [16] 0 0
Spain
State/province [16] 0 0
Sabadell
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Cambridge
Country [18] 0 0
United Kingdom
State/province [18] 0 0
London
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ultragenyx Pharmaceutical Inc
Address
Country

Ethics approval
Ethics application status

Summary
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)
Trial website
https://clinicaltrials.gov/study/NCT06415344
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Ultragenyx Pharmaceuticals Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Patients Contact: Trial Recruitment
Address 0 0
Country 0 0
Phone 0 0
1-888-756-8657
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06415344