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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	ClinicalTrials.gov
Last refreshed on:	19 October 2017
Main ID:	NCT00688844
Date of registration:	29/05/2008
Prospective Registration:	Yes
Primary sponsor:	Emory University
Public title:	Nutritional and Neurotransmitter Changes in PKU Subjects on BH4 BH4&PKU
Scientific title:	Baseline Evaluation and Long-term Follow-up of Nutritional Status and Neurotransmitter Concentrations in Phenylketonuria Patients Initiating Treatment With Sapropterin Dihydrochloride (KuvanTM), a Tetrahydrobiopterin Analog.
Date of first enrolment:	October 2008
Target sample size:	58
Recruitment status:	Completed
URL:	https://clinicaltrials.gov/show/NCT00688844
Study type:	Observational
Study design:
Phase:	N/A

Countries of recruitment
United States

Contacts

Name:	Rani H Singh, PhD, RD
Address:
Telephone:
Email:
Affiliation:	Emory University Department of Human Genetics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosed with PKU

- ability to provide informed consent (or have legal guardian who can provide informed
consent)

- at least 4 years of age

- planning on trying BH4 treatment

Exclusion Criteria:

- Pregnant

- unable to provide informed consent

- less than 4 years of age

- currently taking BH4

Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Phenylketonuria

Intervention(s)

Drug: KuvanTM Therapy

Primary Outcome(s)

Change From Baseline in Bone Mineral Density (BMD) at 12 Months [Time Frame: Baseline and 12 months]

Change From Baseline in BMI at 12 Months [Time Frame: Baseline and 12 months]

Change From Baseline in Percent (%) Lean Mass at 12 Months [Time Frame: Baseline and 12 months]

Change From Baseline in Total Dietary Protein Intake at 12 Months [Time Frame: Baseline and 12 months]

Change From Baseline in Phenylalanine Intake at 12 Months [Time Frame: Baseline and 12 months]

Change From Baseline in Plasma Phenylalanine at 12 Months [Time Frame: Baseline and 12 months]

Change From Baseline in Percent (%) Fat Mass at 12 Months [Time Frame: Baseline and 12 months]

Secondary Outcome(s)

Secondary ID(s)

IRB00007828

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

BioMarin Pharmaceutical

Atlanta Clinical and Translational Science Institute

Ethics review

Results

Results available:	Yes
Date Posted:	20/08/2014
Date Completed:
URL:	https://clinicaltrials.gov/ct2/show/results/NCT00688844

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