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NCT06332807 · ClinicalTrials.gov registry record · Phase 1
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
A Phase 1 study of Phenylketonurias, sponsored by NGGT.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 5
- Study locations
NCT06332807: Recruiting Phase 1 study of Phenylketonurias, sponsored by NGGT.
NCT06332807 is a Phase 1 study of Phenylketonurias that is actively recruiting participants, run by NGGT. The registered enrollment target is 12 participants, below the 43-participant average among 5 other Phenylketonurias trials with a reported enrollment target (72% lower). The trial reports 5 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06332807, a Phase 1 study of Phenylketonurias, is actively recruiting participants, sponsored by NGGT.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 5
- Study locations
Study Summary
This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is an rAAV8 based vector carrying a functional copy of the human PAH gene. Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
Primary Outcome
Incidence and severity of AEs, including serious AEs (SAEs) as assessed by CTCAE v5.0 of a single administration of NGGT002.
Conditions Studied
Interventions
- GENETIC NGGT002
Study Locations (5)
California
- Children's Hospital of Orange County Hospital - Orange
Minnesota
- University of Minnesota - Minneapolis
New Jersey
- Atlantic Health System - Morristown
Pennsylvania
- University of Pittsburgh Medical Center - Pittsburgh
Texas
- University or Texas, Southwestern medical Center - Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2025-01-10 |
| Est. Completion | 2030-12-30 |
| Phase | Phase 1 |
What NCT06332807 shows while recruiting
NCT06332807 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 43-participant average among 5 other Phenylketonurias trials with a reported enrollment target (72% lower).
The record links to 1 condition, with Phenylketonurias appearing as the primary indexed condition, and to 1 intervention - of which NGGT002 is the first listed.
NCT06332807 lists 5 locations in 5 states (California, Minnesota, New Jersey).
Frequently Asked Questions
What is clinical trial NCT06332807 about?
NCT06332807 is a clinical study titled "AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)". This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is an rAAV8 based vector carrying a functional copy of the human PAH gene. Participants will rece...
What is the current status of trial NCT06332807?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2025-01-10. Estimated completion is 2030-12-30.
What conditions does trial NCT06332807 study?
This clinical trial studies the following conditions: Phenylketonurias.
What interventions are being tested in trial NCT06332807?
The interventions under investigation include: NGGT002 (GENETIC).
Who is sponsoring clinical trial NCT06332807?
This trial is sponsored by NGGT, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06332807 being conducted?
This trial has 5 study locations across California, Minnesota, New Jersey, Pennsylvania, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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