AAV Gene Therapy Study for Subjects with PKU
← catalyst calendarNCT04480567 · readout ≤ 499 d
Sponsored by BioMarin Pharmaceutical (industry) · BMRN — their whole pipeline →.
Phase
Phase 1/2
Status
Active, not recruiting
Study type
Interventional
Design
Non randomized · None · Treatment
Enrollment
100estimated
Sites
3
Countries
United Kingdom, United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2020-09-24 | actual | When the study began enrolling |
| Primary completion | Dec 2027 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Dec 2027 | estimated | The whole study's end, after follow-up |
| First posted | 2020-07-21 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2024-12-12 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Phenylketonuria (PKU)
Intervention
- Drug: BMN 307
Primary outcome
what the primary-completion date above is the date OFChange from baseline in mean Plasma Phe levels
measured baseline, week 12
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