Dietary Supplements for the Treatment of Angelman Syndrome

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.

Conditions

• Angelman Syndrome
• Nervous System Diseases

Interventions

DRUG

Betaine

100-200 mg per kg per day by mouth with a maximum of 6 grams divided in two daily doses

DRUG

Creatine

200 mg per kg per day with a daily maximum of 5 grams divided in two daily doses

DRUG

Metafolin

0.5 mg per kg per day by mouth with a maximum of 8 milligrams divided in two daily doses

DRUG

Vitamin B12

1 mg by mouth per day for all weights and ages

Sponsors & Collaborators

• Baylor College of Medicine

  collaborator OTHER

• Rady Children's Hospital, San Diego

  collaborator OTHER

• Boston Children's Hospital

  collaborator OTHER

• Greenwood Genetic Center

  collaborator OTHER

• Rare Diseases Clinical Research Network

  collaborator NETWORK

• University of California, San Diego

  lead OTHER

Principal Investigators

• Arthur L. Beaudet, MD · Department of Molecular and Human Genetics, Baylor College of Medicine

• Carlos A. Bacino, MD · Department of Molecular and Human Genetics, Baylor College of Medicine

• Wen-Hann Tan, BMBS · Harvard Medical School, Children's Hospital Boston

• Lynne M. Bird, MD · Division of Dysmorphology/Genetics, Children's Hospital San Diego, Department of Pediatrics, University of California, San Diego

• Steven A. Skinner, MD · Greenwood Genetic Center

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

1 Day

Max Age

5 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2006-07-31

Primary Completion

2010-02-28

Completion

2010-02-28

Countries

• United States

Study Locations