Long-Term Tetrahydrobiopterin Treatment in PKU Patients o... | NCT00432822

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

NCT00432822

Sponsor

Orphanetics Pharma Entwicklungs GmbH

Status

Terminated

Last Update Posted

Sep 20, 2007Estimated

Enrollment

50Estimated

Phase

Phase 2Phase 3

Conditions

Phenylalanine Hydroxylase Deficiencies

Interventions

tetrahydrobiopterin (BH4)

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
EudraCT Number: 2006-000648-15

Status Module

Record Verification Date

Sep 2007

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Feb 7, 2007

First Submission Date that Met QC Criteria

Feb 7, 2007

First Posted Date

Feb 8, 2007Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 19, 2007

Last Update Posted Date

Sep 20, 2007Estimated

Description Module

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 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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

50Estimated

Name	Type	Description	Arm Group Labels	Other Names
tetrahydrobiopterin (BH4)	Drug

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Andrea Superti-Furga, Prof.	Centre for Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany	Principal Investigator

Locations

Not provided

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
12501224	Background	Muntau AC, Roschinger W, Habich M, Demmelmair H, Hoffmann B, Sommerhoff CP, Roscher AA. Tetrahydrobiopterin as an alternative treatment for mild phenylketonuria. N Engl J Med. 2002 Dec 26;347(26):2122-32. doi: 10.1056/NEJMoa021654.

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Measure	Description	Time Frame
dietary phenylalanine tolerance
safety