Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05293184




Registration number
NCT05293184
Ethics application status
Date submitted
27/06/2017
Date registered
24/03/2022
Date last updated
23/02/2024

Titles & IDs
Public title
The Global Angelman Syndrome Registry
Scientific title
The Global Angelman Syndrome Registry
Secondary ID [1] 0 0
RG-16-078-AM01
Universal Trial Number (UTN)
Trial acronym
GASR
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
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Observational study only

Individuals with Angelman Syndrome - Individuals from birth to adulthood with Angelman Syndrome


Other interventions: Observational study only
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Gather longitudinal data on individuals living with Angelman Syndrome
Timepoint [1] 0 0
70 years (lifespan)

Eligibility
Key inclusion criteria
- Diagnosis of Angelman Syndrome
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
-

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/09/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2099
Actual
Sample size
Target
5000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Queensland University of Technology - Brisbane
Recruitment postcode(s) [1] 0 0
4000 - Brisbane

Funding & Sponsors
Primary sponsor type
Other
Name
Foundation for Angelman Syndrome Therapeutics, Australia
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Queensland University of Technology
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The Global Angelman Syndrome Registry is an online patient organisation driven registry to
collect information about the natural history of children and adults with Angelman Syndrome.
The registry will facilitate 1) recruitment for clinical trials into therapies and
interventions to benefit participants with Angelman Syndrome and their families, and 2)
advancement of research and best standards of care for Angelman Syndrome.

The registry is currently available in English, Spanish, Traditional Chinese, Italian,
Polish, Hindi, and Brazilian Portuguese.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05293184
Trial related presentations / publications
Napier KR, Tones M, Simons C, Heussler H, Hunter AA, Cross M, Bellgard MI. A web-based, patient driven registry for Angelman syndrome: the global Angelman syndrome registry. Orphanet J Rare Dis. 2017 Aug 1;12(1):134. doi: 10.1186/s13023-017-0686-1.
Tones M, Cross M, Simons C, Napier KR, Hunter A, Bellgard MI, Heussler H. Research protocol: The initiation, design and establishment of the Global Angelman Syndrome Registry. J Intellect Disabil Res. 2018 May;62(5):431-443. doi: 10.1111/jir.12482.
Tones M, Zeps N, Wyborn Y, Smith A, Barrero RA, Heussler H, Cross M, McGree J, Bellgard M. Does the registry speak your language? A case study of the Global Angelman Syndrome Registry. Orphanet J Rare Dis. 2023 Oct 19;18(1):330. doi: 10.1186/s13023-023-02904-1.
Public notes

Contacts
Principal investigator
Name 0 0
Helen (Honey) Heussler, MBBS, FRACP DM
Address 0 0
The University of Queensland
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Megan Tones, PhD
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
curator@angelmanregistry.info
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05293184