Trials / Active Not Recruiting
Active Not RecruitingNCT04480567
AAV Gene Therapy Study for Subjects with PKU
A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria
- Status
- Active Not Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 100 (estimated)
- Sponsor
- BioMarin Pharmaceutical · Industry
- Sex
- All
- Age
- 15 Years
- Healthy volunteers
- Not accepted
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 administration of BMN 307 and will be followed for safety and efficacy.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | BMN 307 | AAV Gene Therapy Infusion |
Timeline
- Start date
- 2020-09-24
- Primary completion
- 2027-12-01
- Completion
- 2027-12-01
- First posted
- 2020-07-21
- Last updated
- 2024-12-12
Locations
3 sites across 2 countries: United States, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04480567. Inclusion in this directory is not an endorsement.