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

NCT06858397 | Recruiting Phase 1 FDA Drug

• 1 other identifier: GC1134A_FD_P1201
• Study Type: interventional
• Target: 18
• Locations: 3 countries
• Sites: 10

Brief Summary

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.

Conditions

Fabry Disesase

Outcome Measures

Primary Outcomes (1)

• Incidences and characteristics of adverse events

  Up to 48 weeks

Secondary Outcomes (17)

• Maximum serum concentration (Cmax)

  Up to 48 weeks

• Time to reach maximum serum concentration (Tmax)

  Up to 48 weeks

• Trough serum concentration (Ctrough)

  Up to 48 weeks

• Area under the concentration-time curve in one dosing interval (AUC0-tau)

  Up to 48 weeks

• Terminal elimination half-life (t1/2)

  Up to 48 weeks

Study Arms (3)

Cohort 1

EXPERIMENTAL

Low dose

Drug: HM15421/GC1134A

Cohort 2

EXPERIMENTAL

Mid dose

Drug: HM15421/GC1134A

Cohort 3

EXPERIMENTAL

High dose

Drug: HM15421/GC1134A

Interventions

HM15421/GC1134A DRUG

SC

Cohort 1; Cohort 2; Cohort 3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must be ≥ 18 years of age or age considered as adult in the respective country at the time of signing the informed consent.
• Documented diagnosis of FD with clinical symptoms.
• Females: historical genetic test results based on identification of pathogenic or likely pathogenic GLA variant of FD.
• Males: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2 nmol/hr/mL, LLN in leucocytes=32 nmol/hr/mg/protein).
• Patients who are naive or have not received FD therapy including investigational therapy for FD within the past 6 months prior to screening and have negative ADA testing at screening.
• Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m2 by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation.
• Plasma lyso-Gb3 levels greater than 1.5 times the upper limit of normal (ULN).
• Male participants:
• Male participants are eligible to participate if they agree to the following during the study treatment period:
• Refrain from donating sperm,
• PLUS either:
• Be abstinent from heterosexual intercourse with a woman of childbearing potential (WOCBP) as their preferred and usual lifestyle (abstinent on a longterm and persistent basis) and agree to remain abstinent, OR
• Must agree to use a male condom when engaging in any activity that allows for passage of ejaculate to another person,
• In addition to male condom, use of highly effective method of contraception may be considered in WOCBP partners of male participants.
• Female participants:

You may not qualify if:

• Women who are pregnant, planning to become pregnant during the study, or are breast feeding.
• History of dialysis or renal transplantation.
• CKD stage ≥ 3.
• History of acute kidney injury within 12 months prior to screening, including specific kidney diseases (eg, acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (eg, ischemia, toxic injury); as well as extrarenal pathology (eg, prerenal azotemia, and acute postrenal obstructive nephropathy).
• Urine protein to creatinine ratio (UPCR) \> 0.5 g/g and not treated with an angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB).
• Known history of hypersensitivity to any ingredient in the investigational product and to Gadolinium contrast agent that is not managed by the use of premedication.
• Cardiovascular event (myocardial infarction, unstable angina) within 6 months before screening.
• Congestive heart failure New York Heart Association (NYHA) Class IV
• History of stroke.
• Pacemaker or other contraindication for magnetic resonance imaging (MRI) scanning.
• Angiotensin converting enzyme inhibitor or ARB therapy initiated or dose changed in the 4 weeks prior to screening.
• Patients who received investigational gene therapy for FD.
• Participation in other studies involving study drugs within 4 weeks prior to study entry and/or during study participation.
• Participating in interventional study or using compassionate access product for FD. Participants who have participated in interventional trials for conditions not related to FD should be enrolled after the adequate wash out period is over, which is 5 half-lives or 30 days whichever is longer.
• Presence of human immunodeficiency virus (HIV) and/or active (acute or chronic) hepatitis B and/or Hepatitis C infections.

Sponsors & Collaborators

• GC Biopharma Corp: lead
• Hanmi Pharmaceutical Company Limited: collaborator

Study Sites (10)

David Geffen School of Medicine UCLA, UCLA Health

Los Angeles, California, 90095, United States

RECRUITING

University of Kansas School of Medicine

Kansas City, Kansas, 66160-8500, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

RECRUITING

University of Pittsburgh Medical Center Children's Hoispital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Lysosomal and Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030, United States

RECRUITING

Centro Medico IPAM

Rosario, Santa Fe Province, S2000, Argentina

RECRUITING

Hospital Italiano de Buenos Aires

Buenos Aires, C1199ABB, Argentina

RECRUITING

Pusan National University Children's Hospital in Yangsan

Yangsan, Gyeongsangnam-do, 50612, South Korea

RECRUITING

Yonsei University, College of Medicine

Seoul, 03722, South Korea

RECRUITING

Central Study Contacts

GC Biopharma

CONTACT

+82-031-260-9300; GC1134A@gccorp.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2025
• First Posted: March 5, 2025
• Study Start: May 14, 2025
• Primary Completion (Estimated): August 30, 2028
• Study Completion (Estimated): August 30, 2028
• Last Updated: November 4, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (6); KR (2); AR (2)