Dietary Supplements for the Treatment of Angelman Syndrome

• Status: Completed
• Trial end date: 2010-02-01
• Target enrollment: 0
• Participant gender: All

Summary

Angelman syndrome (AS) is a complex genetic disorder that affects the nervous system. The purpose of this study is to determine the effectiveness of certain dietary supplements in treating the symptoms of AS.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of California, San Diego
• Collaborators: Baylor College of Medicine; Boston Children's Hospital; Boston Children’s Hospital; Greenwood Genetic Center; Rady Children's Hospital, San Diego; Rare Diseases Clinical Research Network
• Treatments: Betaine; Hydroxocobalamin; Vitamin B 12; Vitamin B Complex; Vitamins

Criteria

Inclusion Criteria:

• Diagnosis of AS

• In stable condition with relatively good control of seizures

• Willing to comply with treatment, study visit schedule, and study assessments

• Willing to take oral or G-tube medication

• Willing to be contacted monthly during the course of the study

• Parent or guardian willing to provide informed consent

Exclusion Criteria:

• History of liver or kidney disease

• Currently being treated for a serious acute illness

• Known hypersensitivity to any of the study drugs

• Received high-dose folate drug treatment in the 12 months prior to study entry

• Other significant medical problems, including those involving the liver, kidney, or heart

• Other comorbidities, genetic disorders, or extreme prematurity; children with autism are not excluded