Trial · NCT00850070
Sapropterin as a Treatment for Autistic Disorder
NCT00850070, Phase 2, Completed, The Children's Health Council; results first posted 2014-02-28
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ConductScience Library. Sapropterin as a Treatment for Autistic Disorder. Trials, record NCT00850070. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00850070
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Sapropterin as a Treatment for Autistic Disorder
- NCT number
- NCT00850070
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 46 participants, actual
- Lead sponsor
- The Children's Health Council
- Conditions
- Autistic Disorder
- Interventions
- sapropterin, Placebo
- First posted
- 2009-02-24
- Last updated
- 2018-05-01
- Results first posted
- 2014-02-28
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Clinical Global Impression -- Improvement (CGI-I) Scale | Weekly for 4 weeks, then monthly, with 16-week end point. Primary outcome assessment used two time points, baseline and 16 weeks. | The CGI-I assessed the number of participants showing much or very much improvement on the CGI-I scale. This is a summary judgment made by a trained clinician based on observed and reported behaviors of the child compared to baseline. It is a 7-point scale from very much worse (1) to very much improved (7). Chi-square analyses were used to assess change in CHI-I scores (by group, post-test). Mixed-effects regression models via SPSS MIXED determined the main effects attributed to differences by group (BH4 and placebo), time (treated as categorical at levels baseline, 8 weeks, and 16 weeks) and the group-by-time interaction. We used random intercept and trend modeling that accounts for each individual's initial level of symptom severity/functioning and rate of change/time |
| Primary | Clinical Global Impression -- Severity (CGI-S) Scale | Baseline, 8 weeks, and 16 weeks. Primary outcome assessment used 2 time points, baseline and 16 weeks. | The CGI-S assessed the number of participants with improved severity illness on the CGI-S scale. This is a summary judgment made by a trained clinician of symptom severity. It is a 7-point scale that rates the severity of the patient's illness at time of assessment with 1 - normal, not at all, to 7 - extremely ill. Mixed-effects regression models via SPSS MIXED determined the main effects attributed to differences by group (BH4 and placebo), time (treated as categorical at levels baseline, 8 weeks, and 16 weeks) and the group-by-time interaction. We used random intercept and trend modeling that accounts for each individual's initial level of symptom severity/functioning and rate of change/time |
| Secondary | Preschool Language Scale-Fourth Edition (PLS-4). Assesses Expressive and Receptive Language Skills in Ages Birth Through 6 Years, 11 Months. | Primary outcome assessment examined the difference in scores between baseline and week 16. | Measures expressive \& receptive language and total scores in ages birth to 6 years 11 months. The scales generate raw, standard, and age-equivalent scores; raw scores for the total scale were selected for use in this study. Total is average of subscales. Minimum raw score = 0, maximum = 130. Higher scores indicate better language abilities. Mixed-effects regression models via SPSS MIXED determined the main effects attributed to differences by group (BH4 and placebo), time (treated as categorical at levels baseline, 8 weeks, and 16 weeks) and the group-by-time interaction. For the outcome effect, the difference between baseline and 16 weeks was determined as an indicator for change. |
| Secondary | Vineland Adaptive Behavior Scale-II. | Primary outcome assessment used two time points, baseline and 16 weeks. | the Vineland-2 is a semi-structured interview designed to assess communicatino, daily living, socialization and motor skills. The Vineland-2 is comprised of a total Adaptive Composite scale; we chose to use 10 subscales that specifically address functional domains relevant for a young ASD sample - Receptive Communication, Expressive Communication, Personal Daily Living Skills, Domestic Daily Living Skills, Community Daily Living Skills, Interpersonal Relations, Play Skills, Coping Skills, Gross Motor Skills, Fine Motor Skills. Scales generate raw or sum, V-, and age-equivalent scores; raw scores were selected for use in the study. Raw score ranges from 0 to 108 depending on the scale. Total raw scale range is from 0 to 766. Subscale scores are averaged to create the total adaptive behavior composite. Higher subscale scores indicate more skills. Difference between baseline and week 16 was used as an indicator of change. |
| Secondary | Children's Yale Brown Obsessive Compulsive Scale (C-YBOCS) | Baseline, 8 weeks, and 16 weeks | The C-YBOCS is a scale is designed to rate the severity of obsessive and compulsive symptoms in children and adolescents, ages 6 to 17 years. It can be administered by a clinican or trained interviewer in a semi-structured fashion. In general, the ratings depend on the child's and parent's report; however, the final rating is based on the clinical judgement of the interviewer. Rate the characteristics of each item over the prior week up until, and including, the time of the interview. Scores should reflect the average of each item for the entire week, unless otherwise specified. |
| Secondary | Connor's Preschool ADHD Questionnaire | Baseline, 8 weeks, and 16 weeks | Conners Early Childhood, addresses child behavior for ages 2 years to 6 years with a variety of scales, including an ADHD subdomain. |
| Secondary | Adverse Events Scale | Every 1-2 weeks for 16 weeks | This was not a standardized scale but a set of questions that was asked of each family - some standard and others open ended. |
| Secondary | Aberrant Behavior Checklist (ABC) - Inappropriate Speech | Primary outcome assessment used two time points, baseline and 16 weeks. | Subscale assessing echolalia \& other odd speech. Higher subscale scores indicate more symptoms. 4 items comprise the subscale, with range of scores from 0-4. Total score range on this subscale is 0 to 16. Scores are averaged to compute overall score. Difference in scores between baseline and week 16 were used as indicator of change. |
| Secondary | Social Responsiveness Scale (SRS) | Primary outcome assessment used two time points, baseline and 16 weeks. | The SRS is a 65-item scale used to measure the severity of symptoms in ASD as they occur in natural social settings. The SRS is comprised of 1 Total scale and 5 subscales that generate raw scores that can be converted to standard T-scores (with mean of 50 and standard deviation of 10) for gender and rater type; standard scores were selected for use in this study. A total T-score of 76 or higher is considered severe and strongly associated with a clinical diagnosis of autistic disorder. A t-score of 60-75 is in the mild to moderate range and considered typical for children with mild or 'high-functioning' ASD, while a T-score of 59 or less suggests an absence of ASD symptoms. A total raw score of \>75 were associated with a sensitivity value of .85 and a specificity value of .75 for ASD. Difference in scores between baseline and week 16 were used as an indicator of change. |
| Secondary | Parent Global Assessment (PGA) Scale | Baseline, 8 weeks, and 16 weeks | This is a measure where parents rate their impression of their child's improvement, in a global manner. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Clinical Global Impression -- Improvement (CGI-I) Scale | Weekly for 4 weeks, then monthly, with 16-week end point. Primary outcome assessment used two time points, baseline and 16 weeks. | This was an Intent-to-Treat Analysis with Last Observation Carried Forward.Analyses looked at percentage of children who improved (showing much or very much improved ratings) at 16-week time frame. | participants | NUMBER |
| PRIMARY | Clinical Global Impression -- Severity (CGI-S) Scale | Baseline, 8 weeks, and 16 weeks. Primary outcome assessment used 2 time points, baseline and 16 weeks. | This was an Intent-to-Treat Analysis with Last Observation Carried Forward.Analyses looked at number of children who improved (from markedly, severely or extremely ill to markedly, mildly or no illness) at 16-week time frame. | participants | NUMBER |
| SECONDARY | Preschool Language Scale-Fourth Edition (PLS-4). Assesses Expressive and Receptive Language Skills in Ages Birth Through 6 Years, 11 Months. | Primary outcome assessment examined the difference in scores between baseline and week 16. | Intent-to-treat analysis; all subjects included. Difference in scores between baseline and 16 weeks was used as treatment outcome. | units on a scale | MEAN |
| SECONDARY | Vineland Adaptive Behavior Scale-II. | Primary outcome assessment used two time points, baseline and 16 weeks. | Intent-to-treat analysis; all subjects included. Includes mean at 16-week outcome. | units on a scale | MEAN |
| SECONDARY | Children's Yale Brown Obsessive Compulsive Scale (C-YBOCS) | Baseline, 8 weeks, and 16 weeks | The data was not analyzed secondary to lack of significant findings in the primary outcome measure. Also, limited data collected secondary to the age range of the children we saw.The data cannot now be provided as the research team has since disbanded and it is not possible to reanalyze the data at this time. | ||
| SECONDARY | Connor's Preschool ADHD Questionnaire | Baseline, 8 weeks, and 16 weeks | Data was not analyzed secondary to lack of significant findings in primary outcome measures and limited data collected on this instrument. The data cannot now be provided as the research team has since disbanded and it is not possible to reanalyze the data at this time. | ||
| SECONDARY | Adverse Events Scale | Every 1-2 weeks for 16 weeks | We did not specifically analyze this data but instead use it as a guide to determine if a drug should be discontinued for a particular child. | ||
| SECONDARY | Aberrant Behavior Checklist (ABC) - Inappropriate Speech | Primary outcome assessment used two time points, baseline and 16 weeks. | Intent to treat analysis | units on a scale | MEAN |
| SECONDARY | Social Responsiveness Scale (SRS) | Primary outcome assessment used two time points, baseline and 16 weeks. | Intent-to-treat analysis; all subjects included. Includes mean at 16-week outcome. | units on a scale | MEAN |
| SECONDARY | Parent Global Assessment (PGA) Scale | Baseline, 8 weeks, and 16 weeks | Data was not analyzed secondary to lack of significant findings in primary outcome measures and limited data collected on this instrument. The data cannot now be provided as the research team has since disbanded and it is not possible to reanalyze the data at this time. |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Clinical Global Impression -- Improvement (CGI-I) Scale | Sapropterin | 5 | participants | ||
| Clinical Global Impression -- Improvement (CGI-I) Scale | Placebo | 3 | participants | ||
| Clinical Global Impression -- Severity (CGI-S) Scale | Sapropterin | 3 | participants | ||
| Clinical Global Impression -- Severity (CGI-S) Scale | Placebo | 1 | participants | ||
| Preschool Language Scale-Fourth Edition (PLS-4). Assesses Expressive and Receptive Language Skills in Ages Birth Through 6 Years, 11 Months. | Sapropterin | 84 | Standard Deviation 29 | units on a scale | |
| Preschool Language Scale-Fourth Edition (PLS-4). Assesses Expressive and Receptive Language Skills in Ages Birth Through 6 Years, 11 Months. | Placebo | 60 | Standard Deviation 25 | units on a scale | |
| Vineland Adaptive Behavior Scale-II. | Sapropterin | 344.76 | Standard Deviation 50.0 | units on a scale | |
| Vineland Adaptive Behavior Scale-II. | Placebo | 294.9 | Standard Deviation 70.1 | units on a scale | |
| Aberrant Behavior Checklist (ABC) - Inappropriate Speech | Sapropterin | 2.6 | Standard Deviation 1.9 | units on a scale | |
| Aberrant Behavior Checklist (ABC) - Inappropriate Speech | Placebo | 3.9 | Standard Deviation 3.6 | units on a scale |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sapropterin | 23 | |
| Overall Study | STARTED | Placebo | 23 | |
| Overall Study | COMPLETED | Sapropterin | 20 | |
| Overall Study | COMPLETED | Placebo | 22 | |
| Overall Study | NOT COMPLETED | Sapropterin | 3 | |
| Overall Study | NOT COMPLETED | Placebo | 1 |
Source ClinicalTrials.gov
Adverse events14
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | irritablity | Psychiatric disorders | Sapropterin | 5 | 23 | |
| other | irritablity | Psychiatric disorders | Placebo | 4 | 23 | |
| other | difficulty sleeping | Nervous system disorders | Sapropterin | 2 | 23 | |
| other | difficulty sleeping | Nervous system disorders | Placebo | 4 | 23 | |
| other | Change in Bowel Habits | Gastrointestinal disorders | Sapropterin | 0 | 23 | |
| other | Change in Bowel Habits | Gastrointestinal disorders | Placebo | 4 | 23 | |
| other | repetitive behaviors | Nervous system disorders | Sapropterin | 1 | 23 | |
| other | repetitive behaviors | Nervous system disorders | Placebo | 2 | 23 | |
| other | hyperactivity | Nervous system disorders | Sapropterin | 2 | 23 | |
| other | hyperactivity | Nervous system disorders | Placebo | 1 | 23 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sapropterin | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Sapropterin | 10 | 23 |
| Total, other adverse events | Placebo | 14 | 23 |
| Total, serious adverse events | Sapropterin | 2 | 23 |
| Total, serious adverse events | Placebo | 1 | 23 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 23782126 | RESULT |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Sapropterin vs Placebo: Clinical Global Impression -- Severity (CGI-S) Scale | Trial comparisons |
| Sapropterin vs Placebo: Clinical Global Impression -- Improvement (CGI-I) Scale | Trial comparisons |
| Sapropterin vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00850070
GET https://conductscience.org/api/trials/v1/NCT00850070/registered