Krabbe Disease — Clinical Trials

MONDO:00094998 active·3 recruitingLast updated: Mar 19, 2026, 7:49 AM

Trial Landscape

Krabbe Disease has a growing clinical research program, driven by biological therapy and drug treatment.

Recruiting

Active Trials

By Approach

Biological Therapy4

Drug Treatment1

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

By Phase

Phase 1

Phase 2

5 trials are observational or have no phase designation.

Sponsor Types

Academic/Other6Industry2

Top Sponsors

Forge Biologics, Inc

Trial Locations

United States8 trials

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

3 of 8 Trials

Phase 2Active, Not Recruiting

NCT02171104

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Sponsor:Masonic Cancer Center, University of Minnesota

United StatesStarted: Jul 2014Est. completion: Jul 2029Updated: Jan 2026

Phase 1Active, Not RecruitingGene Therapy

NCT05739643

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

Sponsor:Forge Biologics, Inc

United StatesStarted: Feb 2023Est. completion: Nov 2026Updated: Nov 2025

Phase 1Active, Not RecruitingGene Therapy

NCT04693598

Gene Transfer Clinical Trial for Krabbe Disease

Sponsor:Forge Biologics, Inc

United StatesStarted: Nov 2021Est. completion: Nov 2026Updated: Jan 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 19, 2026 at 7:49 AM