Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety

September 19, 2007 updated by: Orphanetics Pharma Entwicklungs GmbH

Double-Blind, Placebo Controlled, Multicentre Study With an Open Label Extension to Evaluate the Efficacy and Safety of Tetrahydrobiopterin (BH4) in Children and Adolescents With Hyperphenylalaninemia Caused by Phenylalanine Hydroxylase Deficiency

The aim of the study is to confirm the efficacy and safety of BH4 in the treatment of hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency in patients responsive to BH4. The primary objective is to assess the effect of BH4 on phenylalanine tolerance compared to placebo under optimal blood phenylalanine control and to demonstrate safety in 12 months long-term treatment. Additionally population PK will be assessed.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

50

Phase

  • Phase 2
  • Phase 3

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Female and male patients, aged 0-18 years
  • Phenylalanine-4-hydroxylase (PAH) deficiency shown by mutation analysis
  • Blood phenylalanine concentration in the target range under dietary treatment
  • Written consent of a parent or legal representative
  • Assumed availability within the period of study participation
  • Patients/parents willing and able to follow the recommended diet
  • Use of an effective method of contraception in female patients of child bearing potential

Exclusion Criteria:

  • BH4-deficiency due to genetic disorders in biosynthesis or recycling of BH4
  • History or current evidence of poor diet compliance
  • History or current evidence of clinically relevant allergic or idiosyncratic reactions to drugs or food
  • History of allergic reactions to BH4 or its excipients
  • Positive pregnancy test (ß-HCG in serum) and lactating females
  • Participation in other drug trials within the last 30 days before start for the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

What is the study measuring?

Primary Outcome Measures

Outcome Measure
safety
dietary phenylalanine tolerance

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Orphanetics Pharma Entwicklungs GmbH

Investigators

  • Principal Investigator: Andrea Superti-Furga, Prof., Centre for Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

February 7, 2007

First Submitted That Met QC Criteria

February 7, 2007

First Posted (Estimate)

February 8, 2007

Study Record Updates

Last Update Posted (Estimate)

September 20, 2007

Last Update Submitted That Met QC Criteria

September 19, 2007

Last Verified

September 1, 2007

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BH4/III/05/001
  • EudraCT Number: 2006-000648-15

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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