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AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

NCT06332807 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-12-02

No results posted yet for this 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.

Conditions

  • Phenylketonurias

Interventions

GENETIC

NGGT002

adeno-associated viral vector with human phenylalanine hydroxylase gene

Sponsors & Collaborators

  • NGGT INC.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-01-10
Primary Completion
2030-12-30
Completion
2030-12-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06332807 on ClinicalTrials.gov