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

Part of paid clinical trials in Orange, California.

Sponsor
NGGT INC.
Study ID
NCT06332807
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Phenylketonurias

Eligibility Criteria

Sex
ALL
Age
18 Years - 55 Years
Healthy Volunteers
Not accepted

Interventions

  • NGGT002 — GENETIC
    adeno-associated viral vector with human phenylalanine hydroxylase gene

Study Details

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.

Key Dates

Start date
Jan 10, 2025
Status verified
Nov 2025
Primary completion
Dec 30, 2030
Completion
Dec 30, 2030

Study Design

Enrollment
12 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: NGGT002
    Low dose and high dose group: Six to twelve patients will be enrolled into two cohorts at two dose levels. The safety of this study can be ensured by selecting the highest dose under the No Observed Adverse Effect Level (NOAEL) doses observed in preclinical toxicology studies.

Primary Outcome Measure

Incidence and severity of Adverse Events (AEs) [ Time Frame: Baseline to Week 52 and during Year 1 to 5 ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Children's Hospital of Orange County HospitalOrangeCalifornia92868
Cheryl Willis
[email protected]
714-509-4225
Richard Chang, MD (PRINCIPAL_INVESTIGATOR)
University of MinnesotaMinneapolisMinnesota55454
Carrie Gibson
[email protected]
651-274-6024
Chester Whitley, MD (PRINCIPAL_INVESTIGATOR)
Atlantic Health SystemMorristownNew Jersey07960
Claudia Rohan
[email protected]
973-829-4581
Darius Adams, MD (PRINCIPAL_INVESTIGATOR)
University of Pittsburgh Medical CenterPittsburghPennsylvania15224
Angela Riemenschneider
[email protected]
412-692-5232
Gerard Vockley, MD,PhD (PRINCIPAL_INVESTIGATOR)
University or Texas, Southwestern medical CenterDallasTexas75390
Juana Luevano
[email protected]
214.645-7411
Markey McNutt, MD, PhD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Orange, CA

By research site
Children's Hospital of Orange County Hospital· Orange, CAUniversity of Minnesota· Minneapolis, MNAtlantic Health System· Morristown, NJUniversity of Pittsburgh Medical Center· Pittsburgh, PAUniversity or Texas, Southwestern medical Center· Dallas, TX

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