NCT06858397 · GC Biopharma Corp
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
What this study is about
This Phase 1/2 first-in-human (FIH) study is designed to evaluate the safety, tolerability, how the drug moves through the body (PK), and effectiveness of HM15421 in patients with FD.
View original scientific description
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.
Interventions
DRUG
HM15421/GC1134A
SC
Primary outcome measures
Incidences and characteristics of adverse events
Time frame: Up to 48 weeks
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Participants must be ≥ 18 years of age or age considered as adult in the respective country at the time of signing the informed consent. 2. Documented diagnosis of FD with clinical symptoms. 3. Females: historical genetic test results based on identification of pathogenic or likely pathogenic GLA variant of FD. 4. 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). 5. 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. 6. Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m2 by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. 7. Plasma lyso-Gb3 levels greater than 1.5 times the upper limit of normal (ULN). 8. Male participants:
- Male participants are eligible to part
Where
- Los Angeles, California
- Kansas City, Kansas
- Minneapolis, Minnesota
- Cincinnati, Ohio
- Pittsburgh, Pennsylvania
- Fairfax, Virginia
Collaborators
Hanmi Pharmaceutical Company Limited
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Nov 4, 2025 · Source of record for eligibility and locations