Sapropterin as a Treatment for Autistic Disorder

This study has been completed.
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Glen R. Elliott, The Children's Health Council
ClinicalTrials.gov Identifier:
NCT00850070
First received: February 20, 2009
Last updated: January 27, 2014
Last verified: January 2014
Results First Received: August 31, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Autistic Disorder
Interventions: Drug: sapropterin
Drug: Placebo

  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
Recruitment spanned from 4/09 to 6/11. A variety of methods were used to advertise the study, but most successful recruitment was word-of-mouth from study participants.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Few participants were excluded. Most common exclusions were failure to meet inclusion criteria, mostly absence of an autism diagnosis or cognitive functioning below the required minimum.

Reporting Groups
  Description
Sapropterin tetrahydrobiopterin (BH4); dosage was 20mg/kg/day administered once per day orally in tablet form. Pills could be crushed and mixed with a variety of food substances (e.g., liquids or solids).
Placebo sugar pill; matched identical tablet, dosage was 20mg/kg/day administered once per day orally in tablet form. Pills could be crushed and mixed with a variety of food substances (e.g., liquids or solids).

Participant Flow:   Overall Study
    Sapropterin     Placebo  
STARTED     23     23  
COMPLETED     20     22  
NOT COMPLETED     3     1  
Lack of Efficacy                 1                 1  
Adverse Event                 2                 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.
No text entered.

Reporting Groups
  Description
Sapropterin tetrahydrobiopterin (BH4)
Placebo sugar pill
Total Total of all reporting groups

Baseline Measures
    Sapropterin     Placebo     Total  
Number of Participants  
[units: participants]
  23     23     46  
Age  
[units: participants]
     
<=18 years     23     23     46  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Age  
[units: years]
Mean ± Standard Deviation
  5  ± 1     5  ± 1     5  ± 1  
Gender  
[units: participants]
     
Female     3     5     8  
Male     20     18     38  
Region of Enrollment  
[units: participants]
     
United States     23     23     46  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Clinical Global Impression -- Improvement (CGI-I) Scale   [ Time Frame: Weekly for 4 weeks, then monthly, with 16-week end point. Primary outcome assessment used two time points, baseline and 16 weeks. ]

2.  Primary:   Clinical Global Impression -- Severity (CGI-S) Scale   [ Time Frame: Baseline, 8 weeks, and 16 weeks. Primary outcome assessment used 2 time points, baseline and 16 weeks. ]

3.  Secondary:   Preschool Language Scale-Fourth Edition (PLS-4). Assesses Expressive and Receptive Language Skills in Ages Birth Through 6 Years, 11 Months.   [ Time Frame: Primary outcome assessment examined the difference in scores between baseline and week 16. ]

4.  Secondary:   Vineland Adaptive Behavior Scale-II.   [ Time Frame: Primary outcome assessment used two time points, baseline and 16 weeks. ]

5.  Secondary:   Aberrant Behavior Checklist (ABC) - Inappropriate Speech   [ Time Frame: Primary outcome assessment used two time points, baseline and 16 weeks. ]

6.  Secondary:   Social Responsiveness Scale (SRS)   [ Time Frame: Primary outcome assessment used two time points, baseline and 16 weeks. ]

7.  Secondary:   Children's Yale Brown Obsessive Compulsive Scale (C-YBOCS)   [ Time Frame: Baseline, 8 weeks, and 16 weeks ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

8.  Secondary:   Connor's Preschool ADHD Questionnaire   [ Time Frame: Baseline, 8 weeks, and 16 weeks ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

9.  Secondary:   Adverse Events Scale   [ Time Frame: Every 1-2 weeks for 16 weeks ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   Yes

10.  Secondary:   Parent Global Assessment (PGA) Scale   [ Time Frame: Baseline, 8 weeks, and 16 weeks ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Limitations and Caveats
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
No text entered.  


Results Point of Contact:  
Name/Title: Glen R. Elliott, Ph.D., M.D.
Organization: The Children's Health Council
phone: 650.688.3649
e-mail: gelliott@chconline.org


Publications of Results:

Responsible Party: Glen R. Elliott, The Children's Health Council
ClinicalTrials.gov Identifier: NCT00850070     History of Changes
Other Study ID Numbers: CHC-0901
Study First Received: February 20, 2009
Results First Received: August 31, 2012
Last Updated: January 27, 2014
Health Authority: United States: Institutional Review Board