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NCT06687733
Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
Phase 1, PHASE2 trial testing NGGT002 in Phenylketonurias in 18 participants. Currently enrolling.
30 July 2026
Quick facts
| Lead sponsor | NGGT (Suzhou) Biotechnology Co., Ltd. |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 18 |
| Start date | 25 July 2024 |
| Primary completion | 30 July 2026 |
| Estimated completion | 30 July 2031 |
| Sites | 2 locations across China |
Drugs / interventions tested
- NGGT002
Conditions studied
- Phenylketonurias — all drugs for Phenylketonurias →
Sponsor
NGGT (Suzhou) Biotechnology Co., Ltd. — full company profile →
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 a 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
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
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 NCT06687733
- 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.
- NCT06332807 — AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom) · Phase 1, PHASE2 · recruiting
Other recruiting trials for Phenylketonurias
Currently open trials in the same condition.
- NCT06332807 — AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom) · Phase 1, PHASE2 · recruiting
- 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
- 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
Other NGGT (Suzhou) Biotechnology Co., Ltd. trials
Trials by the same sponsor.
- NCT06706427 — Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects · Phase 1, PHASE2 · active not 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 NCT06687733 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 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 (Suzhou) Biotechnology Co., Ltd.
- Last refreshed: 14 November 2024
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/NCT06687733.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing