Trial Outcomes & Findings for Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders (NCT NCT01141595)

Results Overview

Change in the raw score from baseline of the Preschool Language Scales over the 16 week period. The raw score was measured at baseline, 8 weeks and 16 weeks after starting treatment and the change over the 16 week period from baseline to the end of the study was calculated. There was no imputed data and the analysis was as treated. The raw score ranged from 0 to 130. Higher scores indicate better performance.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

10 participants

Primary outcome timeframe

16 weeks

Results posted on

2014-04-01

Participant Flow

All patients were recruited from a medical clinic specalizing in neurodevelopmental disorders

No significant events

Participant milestones

Participant milestones
Measure	Kuvan® Patients will be instructed to take 20 mg/kg/day of Kuvan® orally dissolved in 4 - 8oz. of water or apple juice with breakfast.
Overall Study STARTED	10
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Kuvan® n=10 Participants Patients will be instructed to take 20 mg/kg/day of Kuvan® orally dissolved in 4 - 8oz. of water or apple juice with breakfast.
Age, Categorical <=18 years	10 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Age, Continuous	5.0 years STANDARD_DEVIATION 1.2 • n=99 Participants
Sex: Female, Male Female	1 Participants n=99 Participants
Sex: Female, Male Male	9 Participants n=99 Participants
Region of Enrollment United States	10 participants n=99 Participants

PRIMARY outcome

Timeframe: 16 weeks

Population: The number of participants with data available that completed at least the 16 week assessment.

Change in the raw score from baseline of the Preschool Language Scales over the 16 week period. The raw score was measured at baseline, 8 weeks and 16 weeks after starting treatment and the change over the 16 week period from baseline to the end of the study was calculated. There was no imputed data and the analysis was as treated. The raw score ranged from 0 to 130. Higher scores indicate better performance.

Outcome measures

Outcome measures
Measure	Kuvan® n=8 Participants Patients will be instructed to take 20 mg/kg/day of Kuvan® orally dissolved in 4 - 8oz. of water or apple juice with breakfast.
Preschool Language Scales	7.6 Raw score units / 16 weeks Standard Deviation 8.4

Adverse Events

Kuvan®

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Kuvan® n=10 participants at risk Patients will be instructed to take 20 mg/kg/day of Kuvan® orally dissolved in 4 - 8oz. of water or apple juice with breakfast.
Psychiatric disorders Irritability	10.0% 1/10 • Number of events 1
Psychiatric disorders Excitement	10.0% 1/10 • Number of events 1
Gastrointestinal disorders Upset stomach	10.0% 1/10 • Number of events 1
General disorders Insomnia	10.0% 1/10 • Number of events 1

Additional Information

Richard Frye

Arkansas Children's Hospital Reseach Insitute

Phone: 501-364-5465

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place