Angelman Syndrome Natural History Study
NCT04507997 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 300
Last updated 2025-04-03
Summary
The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be useful for future clinical trials.
Conditions
Sponsors & Collaborators
-
University of California, San Diego
collaborator OTHER -
Angelman Syndrome Biomarker & Outcome Measure Consortium (A-BOM)
collaborator UNKNOWN -
Food and Drug Administration (FDA)
collaborator FED -
Angelman Syndrome Foundation Canada
collaborator UNKNOWN -
Foundation for Angelman Syndrome Therapeutics Canada
collaborator UNKNOWN -
Foundation for Angelman Syndrome Therapeutics
collaborator UNKNOWN -
Angelman Syndrome Foundation, Inc.
collaborator OTHER - lead OTHER
Principal Investigators
-
Wen-Hann Tan, BMBS · Boston Children's Hospital
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2018-08-01
- Primary Completion
- 2031-04-30
- Completion
- 2031-04-30
Countries
- United States
- Canada
Study Locations
More Related Trials
-
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
NCT03882918 ·Status: TERMINATED ·Phase: PHASE3
-
The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)
NCT05705674 ·Status: RECRUITING
-
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
NCT06914609 ·Status: RECRUITING ·Phase: PHASE3
-
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
NCT06617429 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
Natural History Study of Children With Metachromatic Leukodystrophy
NCT01963650 ·Status: TERMINATED
-
Natural History Study of Patients With Canavan Disease (CANinform Study)
NCT04126005 ·Status: COMPLETED
-
Long-term Extension of GTX-102 in Angelman Syndrome
NCT06415344 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3
-
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
NCT07157254 ·Status: RECRUITING ·Phase: PHASE2
-
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
NCT02996305 ·Status: COMPLETED ·Phase: PHASE2
-
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
NCT06151600 ·Status: RECRUITING
-
Natural History of Netherton Syndrome
NCT05902663 ·Status: TERMINATED
-
A Study of OV101 in Individuals With Angelman Syndrome (AS)
NCT04106557 ·Status: COMPLETED ·Phase: PHASE3
-
Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 Deficiency
NCT05050669 ·Status: COMPLETED
-
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.
NCT05630066 ·Status: COMPLETED ·Phase: PHASE2
-
Natural History of Rett Syndrome & Related Disorders
NCT02738281 ·Status: COMPLETED
-
STXBP1 and SYNGAP1 Related Disorders Natural History Study
NCT06555965 ·Status: RECRUITING
-
Late-Onset Treatment Study Extension Protocol
NCT00455195 ·Status: COMPLETED ·Phase: PHASE4
-
Clinical Utility of Prenatal Whole Exome Sequencing
NCT03482141 ·Status: COMPLETED ·Phase: NA
-
ASXL-Related Disorders Natural History Study
NCT03303716 ·Status: RECRUITING
-
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
NCT04880356 ·Status: RECRUITING
-
Natural History Study of Serious Medical Events in PWS
NCT03718416 ·Status: COMPLETED
-
An Observational Study in Children and Adults With Stargardt Disease
NCT06591806 ·Status: RECRUITING
-
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
NCT07181837 ·Status: RECRUITING ·Phase: PHASE1/PHASE2
-
An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy
NCT07222371 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2
-
A Natural History Study of Sanfilippo Syndrome Type D
NCT05648851 ·Status: COMPLETED