PhenylketonuriaNCT00728676
Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors
An observational study of Phenylketonuria, run by Shoji Yano at 1 site in United States.
Trial summary
To validate the outcome measures and the tolerability of Kuvan treatment in the improvement of behavioral symptoms in 10 selected adults with Phenylketonuria (PKU) with or without mental retardation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Phenylketonuria
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2008to May 2010
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Individuals with PKU followed at our clinic
Who may be able to join
- Biochemical confirmation of PKU,
- Measurable maladaptive behavior at baseline,
- Signed informed consent,
- Not pregnant or lactating,
- Females on birth control if applicable,
- Willing to comply.
Who cannot join
- Subjects or care provider unreliable, use of investigational products within 30 days prior to enrollment,
- Pregnant or lactating,
- Concurrent disease or condition that would interfere with study participation or safety, ie multiple psychotropic meds, any condition the PI renders the subject at high risk, on drugs that inhibit folate synthesis,
- Concurrent use of levodopa.
Trial locations and contacts
1 site in 1 country.
United States1 site
LAC+USC Medical Center
Los Angeles, California, 90033
About this study
Official title: Pilot Study to Evaluate the Effects of Kuvan on Adult Individuals With Phenylketonuria With Measurable Maladaptive Behaviors
To validate the outcome measures and the tolerability of Kuvan treatment in the improvement of behavioral symptoms in 10 selected adults with Phenylketonuria (PKU) with or without mental retardation.
- Conditions
- Phenylketonuria
- Keywords
- PKU,, Phe,, PAH,, Kuvan,, Trp,, Tyr
- Medical subject headings
- Phenylketonurias
What is being measured
The primary outcome is the main question the study is designed to answer.
Pattern of change from baseline to 6 and 12 months in Vineland scale standard scores, evaluated with a repeated measure ANOVA approach.
Measured: One year
Secondary outcomes (1)
Correlation and regression methods (non-linear if appropriate) to examine the relationship between changes in behavior and in amino acid profiles.
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2008 (estimated)
- Study startAug 2008
- Primary completionMar 2010
- Study completionMay 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shoji Yano
- Sponsor type
- University, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Responsible party
- Sponsor-investigator, Shoji Yano, University of Southern California
- Investigators
- Shoji Yano, MD, Ph.D., Principal investigator, University of Southern California
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00728676
- Sponsor's protocol ID
- BioMarin-300
- Status last verified
- Jan 2013
- First submitted
- 30 Jul 2008
- Last updated
- 18 Jan 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study to Evaluate the Effects of Kuvan on Adult Individuals With Phenylketonuria With Measurable Maladaptive Behaviors
- ClinicalTrials.gov ID
- NCT00728676
- Acronym / sponsor ID
- BioMarin-300
- Conditions
- Phenylketonuria
- Keywords
- PKU,, Phe,, PAH,, Kuvan,, Trp,, Tyr
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Shoji YanoUniversity, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Study start
- Aug 2008
- Primary completion
- Mar 2010 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 6 Aug 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To validate the outcome measures and the tolerability of Kuvan treatment in the improvement of behavioral symptoms in 10 selected adults with Phenylketonuria (PKU) with or without mental retardation.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Individuals with PKU followed at our clinic
Key inclusion criteria (6 listed)
- Biochemical confirmation of PKU,
- Measurable maladaptive behavior at baseline,
- Signed informed consent,
- Not pregnant or lactating,
- Females on birth control if applicable,
Key exclusion criteria (4 listed)
- Subjects or care provider unreliable, use of investigational products within 30 days prior to enrollment,
- Pregnant or lactating,
- Concurrent disease or condition that would interfere with study participation or safety, ie multiple psychotropic meds, any condition the PI renders the subject at high risk, on drugs that inhibit folate synthesis,
- Concurrent use of levodopa.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Pattern of change from baseline to 6 and 12 months in Vineland scale standard scores, evaluated with a repeated measure ANOVA approach.
Time frame: One year
1 secondary outcome
Correlation and regression methods (non-linear if appropriate) to examine the relationship between changes in behavior and in amino acid profiles.
Time frame: one year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shoji Yano, MD, Ph.D. principal investigator
University of Southern California
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Phenylketonuria, run by Shoji Yano at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pattern of change from baseline to 6 and 12 months in Vineland scale standard scores, evaluated with a repeated measure ANOVA approach. (time frame: One year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jan 2013.