NCT04273269

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

Terminated Phase 1, PHASE2 Last updated 9 June 2023

What this trial tests

Phase 1, PHASE2 trial testing LYS-GM101 in GM1 Gangliosidosis in 5 participants. Terminated before completion.

Timeline

11 May 2021

Primary endpoint
22 May 2023

22 May 2023

Quick facts

Lead sponsor	LYSOGENE
Phase	Phase 1, PHASE2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	5
Start date	11 May 2021
Primary completion	22 May 2023
Estimated completion	22 May 2023
Sites	3 locations across France, United Kingdom, United States

Drugs / interventions tested

• LYS-GM101

Conditions studied

• GM1 Gangliosidosis — all drugs for GM1 Gangliosidosis →

Sponsor

LYSOGENE — full company profile →

Who can join

Under 3, any sex, with GM1 Gangliosidosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

LYS-GM101 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the central nervous system. This study will assess, in a 2-stage adaptive-design, the safety and efficacy of treatment in subjects with infantile GM1 gangliosidosis.

Publications & conference data

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

1. Viral vector-based gene therapies in the clinic.
   Zhao Z, Anselmo AC, Mitragotri S. · · 2022 · cited 171× · PMID 35079633 · DOI 10.1002/btm2.10258
2. Novel vectors and approaches for gene therapy in liver diseases.
   Maestro S, Weber ND, Zabaleta N, Aldabe R, et al · · 2021 · cited 81× · PMID 34159305 · DOI 10.1016/j.jhepr.2021.100300
3. GM1 Gangliosidosis-A Mini-Review.
   Nicoli ER, Annunziata I, d'Azzo A, Platt FM, et al · · 2021 · cited 69× · PMID 34539759 · DOI 10.3389/fgene.2021.734878
4. GM1 Gangliosidosis: Mechanisms and Management.
   Rha AK, Maguire AS, Martin DR. · · 2021 · cited 56× · PMID 33859490 · DOI 10.2147/tacg.s206076
5. 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
6. Immune Responses and Immunosuppressive Strategies for Adeno-Associated Virus-Based Gene Therapy for Treatment of Central Nervous System Disorders: Current Knowledge and Approaches.
   Prasad S, Dimmock DP, Greenberg B, Walia JS, et al · · 2022 · cited 49× · PMID 35994385 · DOI 10.1089/hum.2022.138
7. Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.
   Bradbury AM, Bagel JH, Nguyen D, Lykken EA, et al · · 2020 · cited 49× · PMID 32773406 · DOI 10.1172/jci133953
8. Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders.
   Daci R, Flotte TR. · · 2024 · cited 39× · PMID 38256124 · DOI 10.3390/ijms25021050