A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

NCT00225615 | Completed Phase 3

• 1 other identifier: PKU-004
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 14

Brief Summary

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

Conditions

Phenylketonurias

Keywords

Phenylalanine Hydroxylase

Outcome Measures

Primary Outcomes (2)

• Primary objective:

• - To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.

Secondary Outcomes (6)

• Secondary objectives:

• 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.

Interventions

sapropterin dihydrochloride DRUG

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (14)

Unknown Facility

Los Angeles, California, United States

Location

Unknown Facility

Oakland, 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

Madison, Wisconsin, United States

Location

MeSH Terms

Conditions

Phenylketonurias

Interventions

sapropterin

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Alex Dorenbaum, MD

  BioMarin Pharmaceutical

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: September 22, 2005
• First Posted: September 26, 2005
• Study Start: November 1, 2005
• Study Completion: June 1, 2006
• Last Updated: August 20, 2009

Record last verified: 2009-08

Locations

US (14)