Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome

NCT00829439 | Completed Phase 1 Results DMC

• 1 other identifier: 08-10-0490
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief Summary

This study is designed to determine the highest dose of levodopa/carbidopa that can be tolerated without any serious side effects by children with Angelman syndrome. It has been hypothesized that levodopa may lead to an improvement in the neurodevelopment and abnormal movements (e.g. tremors) in children with Angelman syndrome. Data from this study will be used to design a phase II trial to determine the efficacy of levodopa in treating children with Angelman syndrome.

Conditions

Angelman Syndrome

Keywords

Angelman syndrome; Levodopa; Carbidopa; L-dopa

Outcome Measures

Primary Outcomes (1)

• Maximum Dose of Levodopa/Carbidopa That Can be Tolerated (Without Any Dose Limiting Toxicity) by at Least 3 Subjects.

  1 week

Study Arms (1)

Levodopa/Carbidopa

EXPERIMENTAL

Other Names: Sinemet L-dopa Dosages are based on levodopa. Each cohort of 3 subjects will be placed on an increasing dose of levodopa (2, 5, 10, and 15 mg/kg/day) for 1 week, provided subjects in the preceding cohort tolerated the lower dose. Levodopa/Carbidopa is a combined formulation that will be dispensed as capsules. It should be taken 3 times a day.

Drug: Levodopa/Carbidopa (4:1)

Interventions

Levodopa/Carbidopa (4:1) DRUG

Dosages are based on levodopa. Each cohort of 3 subjects will be placed on an increasing dose of levodopa (2, 5, 10, and 15 mg/kg/day) for 1 week, provided subjects in the preceding cohort tolerated the lower dose. Levodopa/Carbidopa is a combined formulation that will be dispensed as capsules. It should be taken 3 times a day.

Also known as: Sinemet, L-dopa

Levodopa/Carbidopa

Eligibility Criteria

• Age: 4 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Angelman syndrome, confirmed by molecular testing
• Must be willing to come for research visit on 2 days, exactly 1 week apart

You may not qualify if:

• On levodopa, carbidopa, or any dopamine agonists in the 2 weeks prior to participation
• Other medical conditions that may be associated with developmental or cognitive delays
• More than 2 clinical seizures per month
• Used monoamine oxidase (MAO) inhibitors within the last 2 weeks
• Used phenytoin within the last 2 weeks
• Used phenothiazines, butyrophenones, and thioxanthenes within last 2 weeks
• Hypersensitive to levodopa or carbidopa
• Cardiovascular disease or instability
• Respiratory diseases, including asthma, emphysema, chronic cough, and shortness of breath
• Liver disease
• Stomach or intestinal ulcers
• Kidney disease
• Hematological problems, including anemia, leucopenia, and thrombocytopenia
• Used investigational drugs/interventions within the past three months

Sponsors & Collaborators

• Boston Children's Hospital: lead

Study Sites (1)

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Angelman Syndrome

Interventions

Levodopa; Carbidopa; carbidopa, levodopa drug combination

Condition Hierarchy (Ancestors)

Movement Disorders; Central Nervous System Diseases; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders

Intervention Hierarchy (Ancestors)

Dihydroxyphenylalanine; Catecholamines; Amines; Organic Chemicals; Catechols; Phenols; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Tyrosine; Methyldopa; Hydrazines

Results Point of Contact

• Title: Wen-Hann Tan
• Organization: Boston Children's Hospital

wen-hann.tan@childrens.harvard.edu

617-355 6394

Study Officials

• Wen-Hann Tan, BMBS

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Attending Physician in Genetics

Study Record Dates

• First Submitted: January 26, 2009
• First Posted: January 27, 2009
• Study Start: January 1, 2009
• Primary Completion: June 1, 2010
• Study Completion: June 1, 2010
• Last Updated: November 21, 2016
• Results First Posted: November 21, 2016

Record last verified: 2016-09

Locations

US (1)