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NCT07046338
Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
NA trial testing Lentiviral TYF-ARSA correction of patient's autologous HSCs in Metachromatic Leukodystrophy (MLD) in 10 participants. Currently enrolling.
1 June 2025
Quick facts
| Lead sponsor | Shenzhen Geno-Immune Medical Institute |
|---|---|
| Phase | NA |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 10 |
| Start date | 1 June 2025 |
| Primary completion | 1 June 2025 |
| Estimated completion | 30 September 2030 |
| Sites | 1 location across China |
Drugs / interventions tested
- Lentiviral TYF-ARSA correction of patient's autologous HSCs
Conditions studied
- Metachromatic Leukodystrophy (MLD) — all drugs for Metachromatic Leukodystrophy (MLD) →
Sponsor
Shenzhen Geno-Immune Medical Institute
Who can join
Adults 1 Month to 50, any sex, with Metachromatic Leukodystrophy (MLD). Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to transduce patient-derived hematopoietic stem cells (HSCs), with the goal of achieving therapeutic gene correction through transplantation of genetically modified HSCs. The primary objectives are to evaluate the safety and efficacy of the gene therapy clinical protocol.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT07046338
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
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Related trials
Other recruiting trials for Metachromatic Leukodystrophy (MLD)
Currently open trials in the same condition.
- NCT03725670 — Direct Lentiviral Injection Gene Therapy for MLD · NA · recruiting
- NCT03771898 — A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy · Phase 2 · active not recruiting
- NCT01887938 — An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy · Phase 1, PHASE2 · active not recruiting
Other Shenzhen Geno-Immune Medical Institute trials
Trials by the same sponsor.
- NCT07501156 — H3K27M-specific Immune Effector Cells Targeting DMG/DIPG · Phase 1, PHASE2 · recruiting
- NCT07495956 — cfMSC Therapy for Diabetes · Phase 1, PHASE2 · recruiting
- NCT07477600 — cfMSC Stem Cell Therapy Targeting COPD · Phase 1, PHASE2 · recruiting
- NCT07180927 — DLL3 CAR-T Therapy Targeting Brain Tumors · Phase 1, PHASE2 · recruiting
- NCT07193966 — NG2 and DLL3 CAR-T Cells Targeting Melanoma · Phase 1, PHASE2 · 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 NCT07046338 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Shenzhen Geno-Immune Medical Institute
- Last refreshed: 1 July 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/NCT07046338.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing