Who is sponsoring NCT03725670?

NCT03725670 is sponsored by Shenzhen Geno-Immune Medical Institute.

What drugs are being tested in NCT03725670?

Interventions in NCT03725670: Intrathecal and intravenous LV gene therapy.

What condition does NCT03725670 study?

NCT03725670 studies Metachromatic Leukodystrophy (MLD).

Is NCT03725670 still recruiting?

NCT03725670 is currently recruiting now. Last updated 26 June 2025.

Last reviewed 2025-06-26 · How we verify

NCT03725670

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Direct Lentiviral Injection Gene Therapy for MLD

Recruiting now NA Last updated 26 June 2025

What this trial tests

NA trial testing Intrathecal and intravenous LV gene therapy in Metachromatic Leukodystrophy (MLD) in 10 participants. Currently enrolling.

Timeline

31 May 2025

Primary endpoint
31 May 2025

31 December 2030

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	31 May 2025
Primary completion	31 May 2025
Estimated completion	31 December 2030
Sites	1 location across China

Drugs / interventions tested

Intrathecal and intravenous LV gene therapy

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 functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical protocol.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Viral vector-based gene therapies in the clinic.
Zhao Z, Anselmo AC, Mitragotri S. · · 2022 · cited 171× · PMID 35079633 · DOI 10.1002/btm2.10258
Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord.
Jensen TL, Gøtzsche CR, Woldbye DPD. · · 2021 · cited 53× · PMID 34690692 · DOI 10.3389/fnmol.2021.695937
Therapeutic Approaches in Lysosomal Storage Diseases.
Fernández-Pereira C, San Millán-Tejado B, Gallardo-Gómez M, Pérez-Márquez T, et al · · 2021 · cited 43× · PMID 34944420 · DOI 10.3390/biom11121775
Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease.
Marquez Loza LI, Yuen EC, McCray PB. · · 2019 · cited 43× · PMID 30875857 · DOI 10.3390/genes10030218
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.
Massaro G, Geard AF, Liu W, Coombe-Tennant O, et al · · 2021 · cited 42× · PMID 33924076 · DOI 10.3390/biom11040611
Non-Integrating Lentiviral Vectors in Clinical Applications: A Glance Through.
Gurumoorthy N, Nordin F, Tye GJ, Wan Kamarul Zaman WS, et al · · 2022 · cited 41× · PMID 35052787 · DOI 10.3390/biomedicines10010107
Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement.
Sevin C, Deiva K. · · 2021 · cited 30× · PMID 34604300 · DOI 10.3389/fmolb.2021.624988
An overview of development in gene therapeutics in China.
Wang D, Wang K, Cai Y. · · 2020 · cited 20× · PMID 32528163 · DOI 10.1038/s41434-020-0163-7

Verify or expand the search:

Other trials of Intrathecal and intravenous LV gene therapy

Trials testing the same drug.

NCT03727555 — IT and IV Lentiviral Gene Therapy for X-ALD · NA · recruiting

Other recruiting trials for Metachromatic Leukodystrophy (MLD)

Currently open trials in the same condition.

NCT07046338 — Lentiviral Hematopoietic Stem Cell 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)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03725670 (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 Shenzhen Geno-Immune Medical Institute
Last refreshed: 26 June 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/NCT03725670.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing