Trial · NCT00943579
Open-Label Extension Study of Kuvan for Autism
NCT00943579, Phase 2, Phase 3, Completed, The Children's Health Council; results first posted 2013-07-04
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ConductScience Library. Open-Label Extension Study of Kuvan for Autism. Trials, record NCT00943579. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00943579
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label Extension Study of Kuvan for Autism
- NCT number
- NCT00943579
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 41 participants, actual
- Lead sponsor
- The Children's Health Council
- Conditions
- Autistic Disorder
- Interventions
- Kuvan®
- First posted
- 2009-07-22
- Last updated
- 2018-05-02
- Results first posted
- 2013-07-04
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Clinical Global Impressions Scale | 16 weeks | 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 (1) very much improved, (2) much improved, (3) minimally improved (4) no change, (5) minimally worse, (6) much worse and (7) very much worse. Chi-square analyses were used to assess change in CHI-I scores (by group, post-test)Mixed-effects regression models 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. The mixed-effects models accounted for each participant's outcome data at each time point. The mixed-effects regression model is robust to data dependency that occurs with the repeated assessments of individuals over time \& can handle missing data. 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 | Vineland Adaptive Behavior Scale, 2nd Edition | Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902). | The Vineland-2 is semi-structured interview designed to communication, 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. The scales generate raw or sum, V-, and age-equivalent scores; raw scores were selected for use in this study. Higher subscale scores indicate more skills. Raw scores can range from 0 to 766 for the overall adaptive behavior composite. Subscales are combined to form the overall Adaptive Behavior Composite, which is essentially a weighted average of the various subscales combined. |
| Secondary | Children's Yale Brown Obsessive Compulsive Scale | Weeks 8 & 16 | 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 | Parental Global Assessment | Weeks 8 & 16 | this is a measure of parents impression of improvement. |
| Secondary | Preschool Language Scale, 4th Edition (PLS-4) | Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902). | Measures expressive \& receptive language and total scores in ages 0 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 raw scores indicate better language skills. 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. The mixed-effects models accounted for each participant's outcome data at each time point. We used random intercept \& trend modeling that accounts for each individual's initial level of symptom severity/functioning \& rate of change/time |
| Secondary | Connor's Preschool ADHD Questionnaire | Weeks 8 & 16 | This is a measure of behavioral symptomatology in children 2-6 years of age. The ADHD scale is one subdomain. |
| Secondary | Aberrant Behavior Checklist (ABC) | Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902). | This is a 58-item informant-based, factor-analyzed scale comprised of a total scale and 5 subscales that generate raw scores. Scores based on a likert scale ranging from 0-3 where 0 is not a problem to 3 where the problem is severe. Subscales include: Irritability, Social Withdrawal, Stereotypic Behaviors, Hyperactivity and Inappropriate Speech. Total maximum score is 174. Higher subscale scores indicate more symptoms. Scores are totaled to compute subscale scores. 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. The mixed-effects models accounted for each participant's outcome data at each time point. 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 | Adverse Events Reporting | Cummulative throughout study | This is not a standardized measure but instead a set of questions, both closed and open ended, asked of families about their child's response to the medication. Used for determining whether treatment needed to be discontinued. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Clinical Global Impressions Scale | 16 weeks | The number of participants analyzed included those who completed the open label extension of this study. | # participants much - very much improved | NUMBER |
| SECONDARY | Vineland Adaptive Behavior Scale, 2nd Edition | Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902). | All participants who completed the open label extension were analyzed. | units on a scale | MEAN |
| SECONDARY | Children's Yale Brown Obsessive Compulsive Scale | Weeks 8 & 16 | The data was not analyzed secondary to lack of significant findings in primary outcome measures and limited data collected on this measure. 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 | Parental Global Assessment | Weeks 8 & 16 | the data were not analyzed secondary to lack of findings in primary outcome measure as well as the nature of an open label study. 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 | Preschool Language Scale, 4th Edition (PLS-4) | Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902). | All participants completed the open label extension were analyzed in the study. | units on a scale | MEAN |
| SECONDARY | Connor's Preschool ADHD Questionnaire | Weeks 8 & 16 | Data was not analyzed secondary to lack of significant findings in primary outcome measure and reduced number of completed questionnaires. 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 | Aberrant Behavior Checklist (ABC) | Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902). | all participants who completed the open label extension were analyzed. | units on a scale | MEAN |
| SECONDARY | Adverse Events Reporting | Cummulative throughout study | Data were not specifically analyzed but used instead to determine whether treatment needed to be discontinued. 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 group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Clinical Global Impressions Scale | Kuvan Following Placebo | 3 | # participants much - very much improved | ||
| Clinical Global Impressions Scale | Kuvan Following Active Treatment | 3 | # participants much - very much improved | ||
| Vineland Adaptive Behavior Scale, 2nd Edition | Kuvan Following Placebo | 275 | Standard Error 10.77 | units on a scale | |
| Vineland Adaptive Behavior Scale, 2nd Edition | Kuvan Following Active Treatment | 321 | Standard Error 10.78 | units on a scale | |
| Preschool Language Scale, 4th Edition (PLS-4) | Kuvan Following Placebo | 57.24 | Standard Error 5.48 | units on a scale | |
| Preschool Language Scale, 4th Edition (PLS-4) | Kuvan Following Active Treatment | 77.83 | Standard Error 5.27 | units on a scale | |
| Aberrant Behavior Checklist (ABC) | Kuvan Following Placebo | 16.84 | Standard Error 1.68 | units on a scale | |
| Aberrant Behavior Checklist (ABC) | Kuvan Following Active Treatment | 9.70 | Standard Error 1.73 | units on a scale |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Kuvan Following Placebo | 21 | |
| Overall Study | STARTED | Kuvan Following Active Treatment | 20 | |
| Overall Study | COMPLETED | Kuvan Following Placebo | 15 | |
| Overall Study | COMPLETED | Kuvan Following Active Treatment | 15 | |
| Overall Study | NOT COMPLETED | Kuvan Following Placebo | 6 | |
| Overall Study | NOT COMPLETED | Kuvan Following Active Treatment | 5 |
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 | Changes in bowel movements | Gastrointestinal disorders | Kuvan Following Placebo | 5 | 21 | |
| other | Changes in bowel movements | Gastrointestinal disorders | Kuvan Following Active Treatment | 5 | 20 | |
| other | Irritability | Psychiatric disorders | Kuvan Following Placebo | 3 | 21 | |
| other | Irritability | Psychiatric disorders | Kuvan Following Active Treatment | 4 | 20 | |
| other | Difficulty Sleeping | Nervous system disorders | Kuvan Following Placebo | 6 | 21 | |
| other | Difficulty Sleeping | Nervous system disorders | Kuvan Following Active Treatment | 5 | 20 | |
| other | Rash | Skin and subcutaneous tissue disorders | Kuvan Following Placebo | 1 | 21 | |
| other | Rash | Skin and subcutaneous tissue disorders | Kuvan Following Active Treatment | 0 | 20 | |
| other | Hyperactivity | Nervous system disorders | Kuvan Following Placebo | 3 | 21 | |
| other | Hyperactivity | Nervous system disorders | Kuvan Following Active Treatment | 0 | 20 |
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 | Kuvan Following Placebo | ||
| Total, all-cause mortality | Kuvan Following Active Treatment | ||
| Total, other adverse events | Kuvan Following Placebo | 14 | 21 |
| Total, other adverse events | Kuvan Following Active Treatment | 13 | 20 |
| Total, serious adverse events | Kuvan Following Placebo | 0 | 21 |
| Total, serious adverse events | Kuvan Following Active Treatment | 0 | 20 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Kuvan Following Active Treatment vs Kuvan Following Placebo: serious adverse events |
Source 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.
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