Actively Recruiting
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
Led by GC Biopharma Corp · Updated on 2025-11-04
18
Participants Needed
10
Research Sites
172 weeks
Total Duration
On this page
Sponsors
G
GC Biopharma Corp
Lead Sponsor
H
Hanmi Pharmaceutical Company Limited
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be 18 years of age or older at the time of informed consent.
- Documented diagnosis of Fabry Disease with clinical symptoms.
- Females must have historical genetic test results showing a pathogenic or likely pathogenic GLA variant.
- Males must have plasma and/or leucocyte alpha galactosidase activity below the lower limit of normal.
- Patients must be naive to Fabry Disease therapy or have not received such therapy, including investigational treatments, in the past 6 months and have negative anti-drug antibody testing at screening.
- Estimated glomerular filtration rate (eGFR) of 60 mL/min/1.73 m2 or higher.
- Plasma lyso-Gb3 levels greater than 1.5 times the upper limit of normal.
- Male participants must agree to refrain from donating sperm and follow contraception guidelines including abstinence or condom use.
- Female participants must not be pregnant or breastfeeding and either not be women of childbearing potential (WOCBP) or use highly effective contraception, have a negative pregnancy test before dosing, and agree not to donate eggs during the study.
- Capable of giving signed informed consent and complying with study requirements.
You will not qualify if you...
- Women who are pregnant, planning pregnancy during the study, or breastfeeding.
- History of dialysis or renal transplantation.
- Chronic kidney disease stage 3 or higher.
- Acute kidney injury within 12 months before screening or specific kidney diseases.
- Urine protein to creatinine ratio above 0.5 g/g without treatment with ACE inhibitor or ARB.
- Known hypersensitivity to any study drug ingredient or Gadolinium contrast agent not managed by premedication.
- Cardiovascular event such as myocardial infarction or unstable angina within 6 months before screening.
- Congestive heart failure New York Heart Association Class IV.
- History of stroke.
- Pacemaker or other contraindications for MRI.
- Recent initiation or dose change of ACE inhibitor or ARB within 4 weeks before screening.
- Previous investigational gene therapy for Fabry Disease.
- Participation in other studies involving study drugs within 4 weeks before or during this study.
- Participation in interventional or compassionate use studies for Fabry Disease.
- Presence of HIV or active hepatitis B or C infections.
- Any medical, emotional, behavioral, or psychological condition that may interfere with study compliance.
- History of deliberate self-harm or suicidal ideation.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 10 locations
1
David Geffen School of Medicine UCLA, UCLA Health
Los Angeles, California, United States, 90095
Actively Recruiting
2
University of Kansas School of Medicine
Kansas City, Kansas, United States, 66160-8500
Actively Recruiting
3
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
4
Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
Actively Recruiting
5
University of Pittsburgh Medical Center Children's Hoispital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
6
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States, 22030
Actively Recruiting
7
Centro Medico IPAM
Rosario, Santa Fe Province, Argentina, S2000
Actively Recruiting
8
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina, C1199ABB
Actively Recruiting
9
Pusan National University Children's Hospital in Yangsan
Yangsan, Gyeongsangnam-do, South Korea, 50612
Actively Recruiting
10
Yonsei University, College of Medicine
Seoul, South Korea, 03722
Actively Recruiting
Research Team
G
GC Biopharma
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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