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

NCT00432822 · Status: TERMINATED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2007-09-20

No results posted yet for this study

Summary

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.

Conditions

  • Phenylalanine Hydroxylase Deficiencies

Interventions

DRUG

tetrahydrobiopterin (BH4)

Sponsors & Collaborators

  • Orphanetics Pharma Entwicklungs GmbH

    lead INDUSTRY

Principal Investigators

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

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00432822 on ClinicalTrials.gov