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

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ClinicalTrials.gov Identifier: NCT00829439
Recruitment Status : Completed
First Posted : January 27, 2009
Results First Posted : November 21, 2016
Last Update Posted : November 21, 2016
Sponsor:
Information provided by (Responsible Party):
Wen-Hann Tan, Boston Children’s Hospital

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Angelman Syndrome
Intervention Drug: Levodopa/Carbidopa (4:1)
Enrollment 16
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Levodopa/Carbidopa 2 mg/kg/Day Levodopa / Carbidopa 5 mg/kg/Day Levodopa / Carbidopa 10 mg/kg/Day Levodopa / Carbidopa 15 mg/kg/Day
Hide Arm/Group Description Levodopa at 2 mg/kg/day in 3 divided doses Levodopa 5 mg/kg/day in 3 divided doses Levodopa 10 mg/kg/day in 3 divided doses Levodopa / Carbidopa 15 mg/kg/day in 3 divided doses
Period Title: Overall Study
Started 3 3 6 4
Completed 3 3 5 4
Not Completed 0 0 1 0
Reason Not Completed
Adverse Event             0             0             1             0
Arm/Group Title Levodopa/Carbidopa
Hide Arm/Group Description

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.

Levodopa/Carbidopa (4:1): 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.

Overall Number of Baseline Participants 16
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
<=18 years
16
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
Female
10
  62.5%
Male
6
  37.5%
1.Primary Outcome
Title Maximum Dose of Levodopa/Carbidopa That Can be Tolerated (Without Any Dose Limiting Toxicity) by at Least 3 Subjects.
Hide Description [Not Specified]
Time Frame 1 week
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Levodopa/Carbidopa
Hide Arm/Group Description:

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.

Levodopa/Carbidopa (4:1): 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.

Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: mg/kg/day
15
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Levodopa/Carbidopa
Hide Arm/Group Description

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.

Levodopa/Carbidopa (4:1): 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.

All-Cause Mortality
Levodopa/Carbidopa
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Levodopa/Carbidopa
Affected / at Risk (%) # Events
Total   0/16 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Levodopa/Carbidopa
Affected / at Risk (%) # Events
Total   5/16 (31.25%)    
Gastrointestinal disorders   
Vomiting  2/16 (12.50%)  2
Nervous system disorders   
Shaky or Unsteady on feet  3/16 (18.75%)  3
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Wen-Hann Tan
Organization: Boston Children's Hospital
Phone: 617-355 6394
Responsible Party: Wen-Hann Tan, Boston Children’s Hospital
ClinicalTrials.gov Identifier: NCT00829439     History of Changes
Other Study ID Numbers: 08-10-0490
First Submitted: January 26, 2009
First Posted: January 27, 2009
Results First Submitted: August 5, 2016
Results First Posted: November 21, 2016
Last Update Posted: November 21, 2016