Angelman Syndrome Clinical Trial
Official title:
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)
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.
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. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT05293184 -
The Global Angelman Syndrome Registry
|
||
Completed |
NCT00004351 -
Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes
|
N/A | |
Enrolling by invitation |
NCT06139172 -
Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities
|
N/A | |
Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
Recruiting |
NCT06229769 -
Natural History Study for Patients With Angelman Syndrome
|
||
Enrolling by invitation |
NCT03836300 -
Parent and Infant Inter(X)Action Intervention (PIXI)
|
N/A | |
Completed |
NCT03235037 -
Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome
|
N/A | |
Completed |
NCT03644693 -
Nutritional Formulation for Angelman Syndrome
|
N/A | |
Completed |
NCT03358823 -
Study on the Brain Network of Angelman Syndrome
|
||
Recruiting |
NCT06337383 -
Study of the Prevalence of Autistic Traits in Angelman Syndrome
|
||
Recruiting |
NCT05945576 -
IDMet (RaDiCo Cohort) (RaDiCo-IDMet)
|
||
Completed |
NCT02670694 -
Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW
|
N/A | |
Recruiting |
NCT04768803 -
Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or Obesity
|
||
Recruiting |
NCT05630066 -
Study to Investigate the Pharmacokinetics 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.
|
Phase 2 | |
Completed |
NCT00348933 -
Dietary Supplements for the Treatment of Angelman Syndrome
|
N/A | |
Recruiting |
NCT05011851 -
An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
|
Phase 2 | |
Terminated |
NCT03882918 -
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
|
Phase 3 | |
Completed |
NCT02996305 -
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
|
Phase 2 | |
Completed |
NCT02056665 -
Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
|
Phase 2 | |
Completed |
NCT01281475 -
A Trial of Levodopa in Angelman Syndrome
|
Phase 2/Phase 3 |