PhenylketonuriaPhase 3NCT00332189
Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
A Phase 3 study testing sapropterin dihydrochloride in people with Phenylketonuria, run by BioMarin Pharmaceutical at 14 sites in United States.
Trial summary
The objective of this study is to evaluate the safety of long-term treatment with Phenoptin in subjects with phenylketonuria (PKU) who participated in Phase 3 clinical studies with Phenoptin. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Phenylketonuria
- What is being tested?
- sapropterin dihydrochloride
- Type of treatment
- Pill or oral medicine
- How many people
- 111 participants
- Where
- 14 sitesUnited States
- Study dates
- Jul 2006to Aug 2009
- Phase
- Phase 3
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 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participation in study PKU-004 or PKU-006
- Willing and able to provide written, signed informed consent or, in the case of subjects under the age of 18 years, provide written assent (if required) and written informed consent by a parent or legal guardian, after the nature of the study has been explained, and prior to any research-related procedures
- Negative urine pregnancy test at screening (females of child-bearing potential)
- Willing and able to comply with all study procedures
Who cannot join
- Non-responsive to prior treatment with Phenoptin based on participation in PKU-004 or PKU-006
- Perceived to be unreliable or unavailable for study participation or, if under the age of 18 years, have parents or legal guardians who are perceived to be unreliable or unavailable
- Terminated early from PKU-004 or PKU-006, except for subjects in PKU-004 that rolled into PKU-008 at Week 22, subjects in PKU-006 that rolled into PKU-008 at Week 10, or subjects in PKU-006 that terminated due to elevated Phe levels following dietary Phe increases
- Use of any investigational product other than Phenoptin within 30 days prior to screening, or anticipated requirement for any investigational agent prior to completion of all scheduled study assessments
- Positive urine pregnancy test at screening (non-sterile females of child-bearing potential only), already known to be pregnant or breastfeeding or planning a pregnancy in self or partner during the study
- Female subjects of childbearing potential must be using an effective method of birth control, as determined by the PI, and willing to continue to use acceptable birth control measures
Show all 14Show fewer
- Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid-dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes)
- Any condition that, in the view of the PI, renders the subject at high risk from treatment compliance and/or completing the study
- ALT > 2 times the upper limit of normal (i.e., Grade 1 or higher based on World Health Organization Toxicity Criteria) at screening (see Appendix 2)
- Serious neuropsychiatric illness (e.g., major depression) not currently under medical control
- Prior history of organ transplantation
- Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)
- Concurrent use of levodopa
- Clinical diagnosis of primary BH4 deficiency
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
Study site
Atlanta, Georgia
Study site
Chicago, Illinois
Study site
Dallas, Texas
Study site
Los Angeles, California
Study site
Madison, Wisconsin
Study site
Minneapolis, Minnesota
Study site
New Haven, Connecticut
Study site
New York, New York
Study site
Portland, Oregon
Study site
Sacramento, California
Study site
Salt Lake City, Utah
Study site
San Jose, California
Study site
Seattle, Washington
Study site
St Louis, Missouri
About this study
Official title: A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
The objective of this study is to evaluate the safety of long-term treatment with Phenoptin in subjects with phenylketonuria (PKU) who participated in Phase 3 clinical studies with Phenoptin.
- Conditions
- Phenylketonuria
- Being tested
- sapropterin dihydrochloride
- Keywords
- PKU
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sapropterin dihydrochlorideDrug
Also called Kuvan
5-20mg/kg/day orally, dose may be adjusted up or down as needed at the discretion of the investigator in increments of 5mg/kg/day.
What is being measured
The primary outcome is the main question the study is designed to answer.
Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study.
Safety was assessed with regards to the rate and type of AEs, clinically significant changes to vital signs and/or physical examination findings, and clinically significant changes in laboratory test results.
Measured: Baseline through Final Visit (a Maximum of 30 months) with AEs collected at month 3 and 6 then at 6 month intervals
Secondary outcomes (1)
Following Blood Phe Levels.
There were no pre-specified efficacy analysis, blood Phe samples were taken at each visit. Blood Phe concentrations remained within levels consistent with local clinical site recommendations for blood Phe control.
Measured: Baseline through Final Visit (a Maximum of 30 months) with blood phe samples taken at months 3 and 6 then at 6 month intervals
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2006 (estimated)
- Study startJul 2006
- Primary completionAug 2009
- Study completionAug 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for PhenylketonuriaStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 111 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00332189
- Sponsor's protocol ID
- PKU-008
- Status last verified
- Oct 2012
- First submitted
- 30 May 2006
- Last updated
- 16 Nov 2012
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Burton BK, Nowacka M, Hennermann JB, Lipson M, Grange DK, Chakrapani A, Trefz F, Dorenbaum A, Imperiale M, Kim SS, Fernhoff PM. Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: results of a phase 3b study. Mol Genet Metab. 2011 Aug;103(4):315-22. doi: 10.1016/j.ymgme.2011.03.020. Epub 2011 Mar 31.
PubMed 21646032