Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

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 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.

View on ClinicalTrials.gov ↗

View your shortlist →

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.

Conditions Studied

Phenylketonurias

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

FieldValue
Enrollment Target 12 participants
Start Date 2025-01-10
Est. Completion 2030-12-30
Phase Phase 1

Sponsor

NGGT

1 total trials

Interested in This Trial?

Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06332807

The ClinicalTrials.gov registry entry for NCT06332807 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 43-participant average among 5 other Phenylketonurias trials with a reported enrollment target (72% lower). The listed sponsor is NGGT, which has 1 total studies on file at ClinicalTrials.gov.

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 reports 5 study locations spanning 5 distinct geographic areas - top geographies include 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.

Learn More About Clinical Trials

Similar trials for Phenylketonurias

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Explore more on PlainTrial

Phenylketonurias

All registered trials for this condition

California

All trials with a study site in California

Recruiting now

Find trials open to new participants near you

Rankings

Top sponsors, conditions, and phases

Read our methodology - how this data is sourced, computed, and verified.

Related

Browse all → | Rankings →
Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.