Phenylketonurias Clinical Trial
Official title:
A Prospective Clinical Study of Phenylketonuria (PKU)
NCT number | NCT04452513 |
Other study ID # | 307-902 |
Secondary ID | |
Status | Completed |
Phase | |
First received | |
Last updated | |
Start date | October 11, 2019 |
Est. completion date | June 2, 2022 |
Verified date | September 2022 |
Source | BioMarin Pharmaceutical |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
This is a study for adults and children ≥ 14 years old who have Phenylketonuria (PKU) with uncontrolled plasma Phe levels. No clinical intervention or study drug is provided by BioMarin in this study.
Status | Completed |
Enrollment | 32 |
Est. completion date | June 2, 2022 |
Est. primary completion date | June 2, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 14 Years and older |
Eligibility | Inclusion Criteria: - Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency - Ability and willingness to maintain dietary protein intake consistent with baseline intake - Willingness and capable per investigator opinion to comply with study procedures and requirements - Plasma Phe Levels > 600umol/L Exclusion Criteria: - Clinically significant liver dysfunction or disease - Prior treatment with gene therapy - Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study |
Country | Name | City | State |
---|---|---|---|
United States | Emory University Hospital | Atlanta | Georgia |
United States | Children's Hosptial of Colorado | Aurora | Colorado |
United States | Boston Children's Hospital | Boston | Massachusetts |
United States | Ann & Robert H. Lurie Chilren's Hospital | Chicago | Illinois |
United States | University of Florida | Gainesville | Florida |
United States | University of Texas Health Science Center at Houston | Houston | Texas |
United States | Riley Hospital for Children | Indianapolis | Indiana |
United States | University of Kentucky | Lexington | Kentucky |
United States | Morristown Medical Center | Morristown | New Jersey |
United States | UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania |
United States | Oregon Health & Science University | Portland | Oregon |
United States | Clinic for Special Children | Strasburg | Pennsylvania |
United States | University of South Florida | Tampa | Florida |
United States | Medical College of Wisconsin | Wauwatosa | Wisconsin |
Lead Sponsor | Collaborator |
---|---|
BioMarin Pharmaceutical |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in Plasma Phe Levels | through study completion, an average of 2 years | ||
Secondary | Change in dietary protein intake from medical and intact food | through study completion, an average of 2 years |
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