Phenylketonuria Clinical Trial
Official title:
Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy
Prospective study to compare the bone mineral density in adults with HPA on KUVAN™ therapy to those not on therapy. The investigators hypothesize that after one year of KUVAN™ therapy, there will be an improvement in their bone mineral density.
Status | Terminated |
Enrollment | 6 |
Est. completion date | August 2012 |
Est. primary completion date | August 2012 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | Both |
Age group | 18 Years to 50 Years |
Eligibility |
Inclusion Criteria: - Diagnosis of hyperphenylalaninemia at birth - Age between 18 and 50 years - Participated in study HSC-MS-110-0262 Exclusion Criteria: - Peri-menopausal and menopausal women will be excluded because this is a time of increased bone loss related to hormonal mediated factors. - Patients taking bisphosphonates because it alters bone density. Therefore, bone mineral density would reflect the biphosphonate intervention rather than their true status. - Pregnant women due to the hazard of radiation exposure during a DXA scan. In addition women who have been pregnant or who have breastfed within one year of study enrollment will be excluded because these are periods of rapid bone loss which would not reflect the entity under study but would serve to confound the data. |
Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | The University of Texas Health Science Center at Houston | Houston | Texas |
Lead Sponsor | Collaborator |
---|---|
The University of Texas Health Science Center, Houston | BioMarin Pharmaceutical |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Bone Mineral Density | A DXA scan will be conducted one year after Kuvan therapy is initiated. | 1 year after initiation of Kuvan therapy | No |
Secondary | Plasma Amino Acid Profile | Evaluation of levels of plasma amino acids. | every three months up to 1 year | No |
Secondary | Diet Analysis | Subjects will provide a 3 day diet record for every plasma amino acid evaluation. Diets will be analyzed to determine phenylalanine, protein, calories, fat, vitamins and minerals. | every 3 months up to 1 year | No |
Secondary | Plasma Phenylalanine Levels | Plasma phenylalanine levels will be monitored to determine effectiveness of Kuvan therapy. | weekly for 6 weeks, then at least every three months up to 1 year | No |
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