A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease

Part of paid clinical trials in Los Angeles, California.

Sponsor
GC Biopharma Corp
Study ID
NCT06858397
Phase
PHASE1/PHASE2
Status
Recruiting

Conditions

  • Fabry Disesase

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • HM15421/GC1134A — DRUG
    SC

Study Details

This Phase 1/2 first-in-human (FIH) study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of HM15421 in patients with FD.

Key Dates

Start date
May 14, 2025
Status verified
Nov 2025
Primary completion
Aug 30, 2028
Completion
Aug 30, 2028

Study Design

Enrollment
18 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
    Low dose
  • Experimental: Cohort 2
    Mid dose
  • Experimental: Cohort 3
    High dose

Primary Outcome Measure

Incidences and characteristics of adverse events [ Time Frame: Up to 48 weeks ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
David Geffen School of Medicine UCLA, UCLA HealthLos AngelesCalifornia90095
Anjay Rastogi, MD (PRINCIPAL_INVESTIGATOR)
University of Kansas School of MedicineKansas CityKansas66160-8500
Ahmad M. Tuffaha, MD (PRINCIPAL_INVESTIGATOR)
University of MinnesotaMinneapolisMinnesota55455
Chester B. Whitley, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital Medical CenterCincinnatiOhio45229-3039
Robert J. Hopkin, MD (PRINCIPAL_INVESTIGATOR)
University of Pittsburgh Medical Center Children's Hoispital of PittsburghPittsburghPennsylvania15224
Damara Ortiz, MD (PRINCIPAL_INVESTIGATOR)
Lysosomal and Rare Disorders Research and Treatment CenterFairfaxVirginia22030
Ozlem Goker-Alpan, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA