NCT00332189

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
111

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Jul 2006

Typical duration for phase_3

Geographic Reach
1 country

14 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 30, 2006

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 1, 2006

Completed
1 month until next milestone

Study Start

First participant enrolled

July 1, 2006

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2009

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2009

Completed
3.3 years until next milestone

Results Posted

Study results publicly available

November 16, 2012

Completed
Last Updated

November 16, 2012

Status Verified

October 1, 2012

Enrollment Period

3.1 years

First QC Date

May 30, 2006

Results QC Date

September 13, 2012

Last Update Submit

October 17, 2012

Conditions

Phenylketonuria

Keywords

PKU

Outcome Measures

Primary Outcomes (1)

  • 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.

    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.

    Baseline through Final Visit (a Maximum of 30 months) with blood phe samples taken at months 3 and 6 then at 6 month intervals

Interventions

sapropterin dihydrochlorideDRUG

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.

Also known as: Kuvan

Eligibility Criteria

Age4 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

Unknown Facility

Los Angeles, California, United States

Location

Unknown Facility

Sacramento, California, United States

Location

Unknown Facility

San Jose, California, United States

Location

Unknown Facility

New Haven, Connecticut, United States

Location

Unknown Facility

Atlanta, Georgia, United States

Location

Unknown Facility

Chicago, Illinois, United States

Location

Unknown Facility

Minneapolis, Minnesota, United States

Location

Unknown Facility

St Louis, Missouri, United States

Location

Unknown Facility

New York, New York, United States

Location

Unknown Facility

Portland, Oregon, United States

Location

Unknown Facility

Dallas, Texas, United States

Location

Unknown Facility

Salt Lake City, Utah, United States

Location

Unknown Facility

Seattle, Washington, United States

Location

Unknown Facility

Madison, Wisconsin, United States

Location

Related Publications (1)

  • 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.

    PMID: 21646032DERIVED

MeSH Terms

Conditions

Phenylketonurias

Interventions

sapropterin

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title
Medical Information Services
Organization
BioMarin Pharmaceutical, Inc.
medinfo@bmrn.com
1-800-983-4587

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 30, 2006

First Posted

June 1, 2006

Study Start

July 1, 2006

Primary Completion

August 1, 2009

Study Completion

August 1, 2009

Last Updated

November 16, 2012

Results First Posted

November 16, 2012

Record last verified: 2012-10

Locations

US(14)