Phenylketonuria (PKU) MedDRA version: 6.1 Classification code 10034872
Conditions
Interventions
Product Name: Phenoptin
Product Code: T1401
Pharmaceutical Form: Tablet
INN or Proposed INN: Sapropterin, sapropterin dihydrochloride
CA
Sponsors
BioMarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Eight years of age and older; Received =80% of the scheduled doses in Study PKU-003, unless removed from the study after exceeding the Phe alert level value; Willing and able to provide written informed consent or, in the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian; Negative urine pregnancy test within 24 hours prior to enrollment (females of child-bearing potential only); Currently using acceptable birth control measures, as determined by the investigator, and willing 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. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.; Secondary Objective: To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU. To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels. To evaluate the population pharmacokinetics of Phenoptin. To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period. To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels. ; Primary end point(s): The primary measure of efficacy will be the blood Phe levels at the end of each dosing period. The efficacy analyses will include all subjects enrolled in this study. Each subject will have data from three two-week periods at each of three pre-established doses, followed by a 10 mg/kg/day dose for 4 weeks, followed by a subject-specific dose for 12 weeks. Analysis of variance for crossover designs will be used to estimate average within-person changes in Phe level for the three dose levels. | — |
Countries
Germany, Ireland, Italy, United Kingdom
Outcome results
None listed