Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

Phenylketonurias Clinical Trial

Official title:

A Phase 3, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00272792
• Other study ID #: PKU-006
• Status: Completed
• Phase: Phase 3
• First received: January 5, 2006
• Last updated: July 23, 2015
• Start date: February 2006
• Est. completion date: November 2006

Study information

• Verified date: July 2015
• 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 ability of Phenoptin to increase phenylalanine (phe) tolerance in children with phenylketonuria who are following a phe-restricted diet.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 45
• Est. completion date: November 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 4 Years to 12 Years

Eligibility

Inclusion Criteria:

• Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by at least one blood Phe measurement >/=360 umol/L (6 mg/dL)

• Under dietary control with a Phe-restricted diet as evidenced by:· Estimated daily Phe tolerance </=1000 mg/day

• At least 6 months of blood Phe control (mean level of </=480 µmol/L) prior to enrolling in the study

• Aged 4 to 12 years inclusive at screening

• A blood Phe level </=480 µmol/L at screening

• Female subjects of childbearing potential (as determined by the principal investigator) must have a negative blood or urine pregnancy test at entry (prior to the first dose). Note: All female subjects of childbearing potential and sexually mature male subjects must be advised to use a medically accepted method of contraception throughout the study. Female subjects of childbearing potential must be willing to undergo periodic pregnancy tests during the course of the study

• Willing and able to comply with all study procedures

• Willing to provide written assent (if applicable) 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

Exclusion Criteria:

• Any condition that, in the view of the PI, renders the subject at high risk from treatment compliance and/or completing the study

• Prior history of organ transplantation

• Perceived to be unreliable or unavailable for study participation or have parents or legal guardians who are perceived to be unreliable or unavailable

• Use of any investigational agent within 30 days prior to screening, or requirement for any investigational agent or vaccine prior to completion of all scheduled study assessments

• ALT > 2 times the upper limit of normal (i.e., Grade 1 or higher based on World Health Organization Toxicity Criteria) at screening

• 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)

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Phenylketonurias

Intervention

Drug:
• Sapropterin Dihydrochloride
Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
• Placebo
Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.

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	Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria		at Week 10	No
Secondary	Change in Phenylalanine Levels From Baseline to Week 3		Baseline to Week 3	No

External Links

•   BioMarin Pharmaceutical Inc website
•   NDA Review Package