NCT00272792

Brief 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.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
45

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Feb 2006

Shorter than P25 for phase_3

Geographic Reach
1 country

9 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

January 5, 2006

Completed
4 days until next milestone

First Posted

Study publicly available on registry

January 9, 2006

Completed
23 days until next milestone

Study Start

First participant enrolled

February 1, 2006

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2006

Completed
2.7 years until next milestone

Results Posted

Study results publicly available

July 9, 2009

Completed
Last Updated

August 13, 2015

Status Verified

July 1, 2015

First QC Date

January 5, 2006

Results QC Date

January 29, 2009

Last Update Submit

July 23, 2015

Conditions

Phenylketonurias

Outcome Measures

Primary Outcomes (1)

  • Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria

    at Week 10

Secondary Outcomes (1)

  • Change in Phenylalanine Levels From Baseline to Week 3

    Baseline to Week 3

Study Arms (2)

Sapropterin Dihydrochloride

EXPERIMENTAL

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.

Drug: Sapropterin Dihydrochloride

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

Sapropterin DihydrochlorideDRUG

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.

Sapropterin Dihydrochloride
PlaceboDRUG

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.

Placebo

Eligibility Criteria

Age4 Years - 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

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

Chicago, Illinois, United States

Location

Unknown Facility

Boston, Massachusetts, United States

Location

Unknown Facility

Minneapolis, Minnesota, United States

Location

Unknown Facility

St Louis, Missouri, United States

Location

Unknown Facility

Salt Lake City, Utah, United States

Location

Related Publications (1)

  • Trefz FK, Burton BK, Longo N, Casanova MM, Gruskin DJ, Dorenbaum A, Kakkis ED, Crombez EA, Grange DK, Harmatz P, Lipson MH, Milanowski A, Randolph LM, Vockley J, Whitley CB, Wolff JA, Bebchuk J, Christ-Schmidt H, Hennermann JB; Sapropterin Study Group. Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study. J Pediatr. 2009 May;154(5):700-7. doi: 10.1016/j.jpeds.2008.11.040. Epub 2009 Mar 4.

    PMID: 19261295DERIVED

Related Links

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
RANDOMIZED
Masking
DOUBLE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

January 5, 2006

First Posted

January 9, 2006

Study Start

February 1, 2006

Study Completion

November 1, 2006

Last Updated

August 13, 2015

Results First Posted

July 9, 2009

Record last verified: 2015-07

Locations

US(9)