Status:
TERMINATED
Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety
Lead Sponsor:
Orphanetics Pharma Entwicklungs GmbH
Conditions:
Phenylalanine Hydroxylase Deficiencies
Eligibility:
All Genders
Up to 18 years
Phase:
PHASE2
PHASE3
Brief 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 ob...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT00432822
Last Update
September 20 2007
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