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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00225615
Other study ID # PKU-004
Secondary ID
Status Completed
Phase Phase 3
First received September 22, 2005
Last updated August 18, 2009
Start date November 2005
Est. completion date June 2006

Study information

Verified date August 2009
Source BioMarin Pharmaceutical
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Interventional

Clinical Trial Summary

The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.


Recruitment information / eligibility

Status Completed
Enrollment 100
Est. completion date June 2006
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group 8 Years and older
Eligibility Inclusion Criteria:

- 8 years of age and older

- Prior successful participation in Study PKU-003

- Willing and able to provide written informed consent or assent and written informed consent (if required) by a parent or legal guardian

- For females of child-bearing potential only: Negative urine pregnancy test within 24 hours prior to enrollment. Women using acceptable birth control measures must agree to continue to use those measures while participating in the study

- Willing and able to comply with study procedures

- Willing to continue current diet unchanged while participating in the study

Exclusion Criteria:

- Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable

- Withdrew from, or otherwise did not successfully complete, study PKU-003, except for subjects who were removed from the study because their blood Phe exceeded the alert level

- Expected to require any investigational agent or vaccine prior to completion of all scheduled study assessments

- Pregnant or breastfeeding, or planning pregnancy

- 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)

- Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)

- Concurrent use of levodopa

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
sapropterin dihydrochloride


Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Primary objective:
Primary - To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
Secondary Secondary objectives:
Secondary To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU.
Secondary To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels.
Secondary To evaluate the population pharmacokinetics of Phenoptin.
Secondary To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period.
Secondary To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels.
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