Trial Outcomes & Findings for Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome (NCT NCT00829439)

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

16 participants

Primary outcome timeframe

1 week

Results posted on

2016-11-21

Participant milestones
Measure	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
Overall Study STARTED	3	3	6	4
Overall Study COMPLETED	3	3	5	4
Overall Study NOT COMPLETED	0	0	1	0

Reasons for withdrawal
Measure	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
Overall Study Adverse Event	0	0	1	0

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

Baseline characteristics by cohort
Measure	Levodopa/Carbidopa n=16 Participants 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.
Age, Categorical <=18 years	16 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants

Timeframe: 1 week

Outcome measures
Measure	Levodopa/Carbidopa n=15 Participants 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.
Maximum Dose of Levodopa/Carbidopa That Can be Tolerated (Without Any Dose Limiting Toxicity) by at Least 3 Subjects.	15 mg/kg/day

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Adverse event data not reported

Other adverse events
Measure	Levodopa/Carbidopa n=16 participants at risk 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.
Nervous system disorders Shaky or Unsteady on feet	18.8% 3/16 • Number of events 3
Gastrointestinal disorders Vomiting	12.5% 2/16 • Number of events 2

Boston Children's Hospital

Phone: 617-355 6394