AAV Gene Therapy Study for Subjects With PKU
Purpose
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 administration of BMN 307 and will be followed for safety and efficacy.
Condition
- Phenylketonuria (PKU)
Eligibility
- Eligible Ages
- Over 15 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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 Criteria
- 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
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental Dose 1 of BMN 307 |
|
|
Experimental Dose 2 of BMN 307 |
|
|
Experimental Dose 3 of BMN 307 |
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More Details
- Status
- Active, not recruiting
- Sponsor
- BioMarin Pharmaceutical