Status:
ACTIVE_NOT_RECRUITING
AAV Gene Therapy Study for Subjects with PKU
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Phenylketonuria (PKU)
Eligibility:
All Genders
15+ years
Phase:
PHASE1
PHASE2
Brief Summary
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single admini...
Eligibility Criteria
Inclusion
- Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency
- Ability and willingness to maintain dietary protein intake consistent with baseline intake
- Willingness to abstain from hepatotoxic substances post-BMN 307 administration
- Willingness and capable per investigator opinion to comply with study procedures and requirements
- Willingness to use effective methods of contraception
- Plasma Phe levels \> 600 µmol/L
Exclusion
- Subjects with primary BH4 deficiency or other forms of BH4 metabolism deficiency
- Clinically significant liver dysfunction or disease
- Prior treatment with gene therapy
- Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study
- History of malignancy
Key Trial Info
Start Date :
September 24 2020
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2027
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT04480567
Start Date
September 24 2020
End Date
December 1 2027
Last Update
December 12 2024
Active Locations (3)
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1
University of South Florida
Tampa, Florida, United States, 33606
2
Morristown Medical Center
Morristown, New Jersey, United States, 07960
3
University Hospital Birmingham NHS Foundation Trust
Birmingham, United Kingdom, B15 2TH