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Angelman Syndrome Natural History Study-FAST UK

A Monocentric, Prospective, Longitudinal and Observational Natural History Study for Patients With Angelman Syndrome in the United Kingdom: Natural History - Foundation for Angelman Syndrome Therapeutics (FAST) United Kingdom (UK)

NCT05100810•University of Oxford

View on ClinicalTrials.gov

Summary

The goal of this study is to conduct a prospective, longitudinal assessment of the natural clinical progression of Angelman syndrome (AS) in children and adults. This will be performed by acquiring baseline measurements, and developing effective outcome measures and diagnostic tools for the syndrome, to prepare the healthcare system for forthcoming clinical trials.

Detailed Description

This study is being conducted in anticipation of several candidate therapies which are approaching clinical readiness for Angelman syndrome. This study will comprehensively evaluate the natural clinical progression of the disease using scales and questionnaires for the assessment of motor function and global development, motor measuring devices (ActiMyo), and by collecting sleep and seizure diaries. In addition, proteomic analysis and electroencephalography (EEG) recordings will be collected to identify biomarkers which will indicate improvements in disease outcome following treatment.

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•For the candidate participants affected by AS:
•Genetically confirmed diagnosis of AS
•0-99 years
•Male or Female
•Obtained consent forms and/or record of consultation by the carers.
•Male or Female
•\>18 years
•Legal carer of the patient diagnosed with AS
•Willingness to follow study procedures, as assessed by the research team
•Willingness to sign the consent form
+1 more criteria

Exclusion Criteria

•The participant affected by AS may not enter the study if there is any comorbidity (\*) that could potentially affect the results of the study. This will be subject to the clinical judgement of the Chief Investigator (CI) and/or the Principal Investigator (PI). Participants of ongoing (interventional) clinical trials that assess the efficacy of potential treatments will be excluded as assessments need to be done on the basis that represent the natural progression of AS.
•(\*) This includes any confirmed chronic or acute condition or disease affecting any system(s), which could interfere with the results of the study and/or the compliance with the study procedures.

Locations (1)

University of Oxford

Oxford, OXON, United Kingdom

Key Dates

Start Date

November 1, 2021

Primary Completion Date

December 31, 2024

Completion Date

December 31, 2024

Last Updated

February 28, 2024

Enrollment

ESTIMATED participants

Conditions

Angelman Syndrome

Interventions

Natural History Study

OTHER

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

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COMPLETEDPHASE2

A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

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COMPLETEDPHASE1

A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 28, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Angelman Syndrome Natural History Study-FAST UK

Inclusion Criteria

Exclusion Criteria

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

A Study in Adults and Adolescents With Angelman Syndrome (STARS)

A Study of OV101 in Individuals With Angelman Syndrome (AS)

Study on the Brain Network of Angelman Syndrome