Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome

NCT03235037 | Completed DMC

• 1 other identifier: IRB PRO00004116
• Study Type: interventional
• Target: 10
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to evaluate the safety and effectiveness (how well a drug works) of Carbidopa/levodopa (Sinemet) in individuals with Angelman syndrome. Sinemet is a medication that helps to raise dopamine levels (a chemical that signals nerve cells) in the brain and central nervous system. There is evidence that dopamine concentrations may be abnormal in patients with Angelman syndrome. This study is investigating whether Sinemet helps motor control, intellectual function and the achievement of developmental milestones in people with Angelman syndrome

Conditions

Angelman Syndrome

Outcome Measures

Primary Outcomes (3)

• Improvement in motor control determined through composite measure- Tremor

  Documented improvement in motor control including tremor using SARA Scale ( Short Ataxia Rating Assesment)

  2 years

• Improvement in motor control determined through composite measure-Ataxia

  Documented improvement in motor control including ataxia, using SARA Scale ( Short Ataxia Rating Assesment)

  2 years

• Improvement in motor control determined through composite measure- SARA score

  Documented improvement in SARA score

  2 years

Secondary Outcomes (1)

• Improvement in development determined through composite measure-Psychoeducational Testing

  2 years

Study Arms (1)

Sinemet

OTHER

The target dose of Sinemet is 2-10 mg per kilogram per day of levodopa. The initial dose will be determined by your weight and age and will start at a low dosage level, 1 mg per kilogram per day. The dose will be re-evaluated after the first 2 weeks and may be adjusted at each visit depending on your response to the drug.

Drug: Levodopa

Interventions

Levodopa DRUG

The target dose of Sinemet is 2-10 mg per kilogram per day of levodopa. The initial dose will be determined by your weight and age and will start at a low dosage level, 1 mg per kilogram per day. The dose will be re-evaluated after the first 2 weeks and may be adjusted at each visit depending on your response to the drug.

Also known as: Sinemet

Sinemet

Eligibility Criteria

• Age: 18 Months - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosis of Angelman syndrome confirmed by molecular genetic testing.
• Age between 18 months and 16 years of age
• Absence of any contraindication to the use of Sinemet as determined by the PI

You may not qualify if:

• Intractable epilepsy not responsive to anticonvulsive therapy in the patient with this syndrome.
• History of prior drug intolerances/drug hypersensitivity to any agent that may be similar to L Dopa .
• Progressively deteriorating EEG pattern.

Sponsors & Collaborators

• Bennett Lavenstein: lead

MeSH Terms

Conditions

Angelman Syndrome

Interventions

Levodopa; 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

Study Officials

• Bennett Lavenstein, MD

  Children's National Health System, Department of Neurology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: MD

Model Details: This is an open label study to assess the motor and cognitive and developmental effects of carbidopa/levodopa when administered orally in doses of 1 -10mg per kg per day. This study will recruit and monitor patients over a 2 year period.

Study Record Dates

• First Submitted: July 28, 2017
• First Posted: August 1, 2017
• Study Start: November 26, 2013
• Primary Completion: February 1, 2019
• Study Completion: February 1, 2019
• Last Updated: April 22, 2019

Record last verified: 2019-04