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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Wen-Hann Tan, Boston Children's Hospital
ClinicalTrials.gov Identifier:
NCT00829439
First received: January 26, 2009
Last updated: September 29, 2016
Last verified: September 2016
Results First Received: August 5, 2016  
Study Type: Interventional
Study Design: Endpoint Classification: Safety Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Angelman Syndrome
Intervention: Drug: Levodopa/Carbidopa (4:1)

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Levodopa/Carbidopa 2 mg/kg/Day Levodopa at 2 mg/kg/day in 3 divided doses
Levodopa / Carbidopa 5 mg/kg/Day Levodopa 5 mg/kg/day in 3 divided doses
Levodopa / Carbidopa 10 mg/kg/Day Levodopa 10 mg/kg/day in 3 divided doses
Levodopa / Carbidopa 15 mg/kg/Day Levodopa / Carbidopa 15 mg/kg/day in 3 divided doses

Participant Flow:   Overall Study
    Levodopa/Carbidopa 2 mg/kg/Day   Levodopa / Carbidopa 5 mg/kg/Day   Levodopa / Carbidopa 10 mg/kg/Day   Levodopa / Carbidopa 15 mg/kg/Day
STARTED   3   3   6   4 
COMPLETED   3   3   5   4 
NOT COMPLETED   0   0   1   0 
Adverse Event                0                0                1                0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Levodopa/Carbidopa

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.


Baseline Measures
   Levodopa/Carbidopa 
Overall Participants Analyzed 
[Units: Participants]
 16 
Age 
[Units: Participants]
 
<=18 years   16 
Between 18 and 65 years   0 
>=65 years   0 
Gender 
[Units: Participants]
 
Female   10 
Male   6 


  Outcome Measures

1.  Primary:   Maximum Dose of Levodopa/Carbidopa That Can be Tolerated (Without Any Dose Limiting Toxicity) by at Least 3 Subjects.   [ Time Frame: 1 week ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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
e-mail: wen-hann.tan@childrens.harvard.edu



Responsible Party: Wen-Hann Tan, Boston Children's Hospital
ClinicalTrials.gov Identifier: NCT00829439     History of Changes
Other Study ID Numbers: 08-10-0490
Study First Received: January 26, 2009
Results First Received: August 5, 2016
Last Updated: September 29, 2016
Health Authority: United States: Food and Drug Administration