Gm1 Gangliosidosis Type 2 — Clinical Trials

MONDO:00092612 active·1 recruitingLast updated: Mar 16, 2026, 1:48 AM

Trial Landscape

Gm1 Gangliosidosis Type 2 has an emerging clinical trial program, driven by biological therapy and drug treatment.

Recruiting

Active Trials

By Approach

Biological Therapy2

Drug Treatment1

Procedure1

1 additional trials involve behavioral, dietary, diagnostic, or observational approaches.

By Phase

Phase 1

Phase 2

Sponsor Types

NIH1Industry1

Top Sponsors

Gemma Biotherapeutics

Trial Locations

United States2 trials

Brazil1 trial

Turkey (Türkiye)1 trial

United Kingdom1 trial

Trials often recruit across multiple countries, so totals exceed the trial count.

2 Trials

Phase 1RecruitingGene Therapy

NCT03952637

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

Sponsor:National Human Genome Research Institute (NHGRI)

United StatesStarted: Aug 2019Est. completion: Jan 2028Updated: Dec 2025

Phase 1Active, Not Recruiting

NCT04713475

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

Sponsor:Gemma Biotherapeutics

United StatesStarted: Mar 2021Est. completion: Feb 2029Updated: May 2025

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Data sourced from ClinicalTrials.gov via Kisho's automated pipeline. Trials refreshed every 4 hours for monitored diseases.

Last sync: March 16, 2026 at 1:48 AM