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

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)

  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
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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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 ]

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  Serious Adverse Events

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  Other Adverse Events

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  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
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