Last reviewed · How we verify
NCT06332807
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
Phase 1, PHASE2 trial testing NGGT002 in Phenylketonurias in 12 participants. Currently enrolling.
30 December 2030
Quick facts
| Lead sponsor | NGGT INC. |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 12 |
| Start date | 10 January 2025 |
| Primary completion | 30 December 2030 |
| Estimated completion | 30 December 2030 |
| Sites | 5 locations across United States |
Drugs / interventions tested
- NGGT002
Conditions studied
- Phenylketonurias — all drugs for Phenylketonurias →
Sponsor
NGGT INC.
Who can join
Adults 18 to 55, any sex, with Phenylketonurias. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
Inborn Errors of Amino Acid Metabolism Revisited: Clinical Implications and Insights into Current Therapies.
Shakerdi AL, Nerney D, Molloy EJ, Knerr I. · · 2025 · PMID 41464651 · DOI 10.3390/jcm14248749 -
Advancing Gene Therapy for Phenylketonuria: From Precision Editing to Clinical Translation.
Yu I, Jeong J. · · 2025 · PMID 40943640 · DOI 10.3390/ijms26178722
Verify or expand the search:
- PubMed search for NCT06332807
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of NGGT002
Trials testing the same drug.
- NCT06687733 — Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects · Phase 1, PHASE2 · recruiting
Other recruiting trials for Phenylketonurias
Currently open trials in the same condition.
- NCT06560736 — Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria · NA · recruiting
- NCT06718842 — Walking Program in Fatty Liver Children With Phenylketonuria · NA · recruiting
- NCT06687733 — Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects · Phase 1, PHASE2 · recruiting
- NCT06061614 — Safety and Efficacy Study of NGGT002 in PKU Adult Subjects · EARLY_PHASE1 · recruiting
- NCT05128149 — Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference? · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06332807 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by NGGT INC.
- Last refreshed: 2 December 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06332807.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing