N/A
Completed N=90
Dietary Supplements for the Treatment of Angelman Syndrome
Angelman Syndrome · nervous system diseases
Source: ClinicalTrials.gov NCT00348933 ↗
Enrolled (actual)
90
Serious AEs
53.3%
Results posted
Sep 2012
Primary outcomePrimary: Average Change in Functioning in Specific Areas of Development, Including Speech and Communications Skills, Cognitive Abilities and Daily Living Skills — 5.7; 4.7; 5.8; 3.7 units on a scale
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Average Change in Functioning in Specific Areas of Development, Including Speech and Communications Skills, Cognitive Abilities and Daily Living Skills |
5.7; 4.7; 5.8; 3.7; 4.7; 2.0 | — |
| SECONDARY Change in Levels of Betaine, Creatine, Dimethylglycine, Guanidinoacetate, Homocysteine, and Methionine. |
206.9; 83.3; 93.7; 0.11; 2.3; 5.5 | — |
| SECONDARY Change in RBC Folate |
77 | — |
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT00348933). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.