Fabry DisesasePhase 1/2NCT06858397
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
A Phase 1/2 study testing HM15421/GC1134A in people with Fabry Disesase, run by GC Biopharma Corp at 10 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disesase
- What is being tested?
- HM15421/GC1134A
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 10 sitesin 3 countries
- Study dates
- 14 May 2025to 30 Aug 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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:
- Female participants are eligible to participate if they are not pregnant or breastfeeding, and at least 1 of the following conditions applies:
- Is not a WOCBP, OR
- Is a WOCBP and using a contraceptive method that is highly effective (with a failure rate of < 1% per year), preferably with low user dependency, starting at least one menstrual cycle before first study drug administration and continuing for at least 30 days after the end of systemic exposure of the study drug and agrees not to donate eggs (ova, oocytes) for the purpose of reproduction during this period. The Investigator should evaluate the effectiveness of the contraceptive method in relationship to the first dose of study drug.
- A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within 24 hours before the first dose of study drug.
- If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.
- Women whose postmenopausal status is recent, may perform additional follicle stimulating hormone (FSH) testing.
- Informed Consent
- Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Who cannot join
- 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.
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- 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.
- Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator and/or Medical Monitor, would interfere with the participant's compliance with the requirements of the study.
- Participants who may have history of deliberate self-harm or suicidal ideation.
Trial locations and contacts
10 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
10 sites in 3 countries
United States6 sites, 6 recruiting
- Recruiting
Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3039
Robert J. Hopkin, MD
- Recruiting
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030
Ozlem Goker-Alpan, MD
- Recruiting
University of Kansas School of Medicine
Kansas City, Kansas, 66160-8500
Ahmad M. Tuffaha, MD
- Recruiting
David Geffen School of Medicine UCLA, UCLA Health
Los Angeles, California, 90095
Anjay Rastogi, MD
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
Chester B. Whitley, MD
- Recruiting
University of Pittsburgh Medical Center Children's Hoispital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Damara Ortiz, MD
Argentina2 sites, 2 recruiting
- Recruiting
Hospital Italiano de Buenos Aires
Buenos Aires, C1199ABB
Marcelo Rugiero, MD
- Recruiting
Centro Medico IPAM
Rosario, Santa Fe Province, S2000
Sebastian Jaurretche, MD
South Korea2 sites, 2 recruiting
- Recruiting
Yonsei University, College of Medicine
Seoul, 03722
Geu-Ru Hong, MD
- Recruiting
Pusan National University Children's Hospital in Yangsan
Yangsan, Gyeongsangnam-do, 50612
Chong Kun Cheon, MD
About this study
Official title: An Open Label, Dose Range, Proof-of-Concept Study to Assess the Safety and Efficacy of HM15421/GC1134A in Patients With Fabry Disease
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
- Being tested
- HM15421/GC1134A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HM15421/GC1134ADrug
SC
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Low dose Receives: Drug: HM15421/GC1134A |
| Cohort 2 | Experimental | Mid dose Receives: Drug: HM15421/GC1134A |
| Cohort 3 | Experimental | High dose Receives: Drug: HM15421/GC1134A |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidences and characteristics of adverse events
Measured: Up to 48 weeks
Secondary outcomes (17)
Maximum serum concentration (Cmax)
PK parameter
Measured: Up to 48 weeks
Time to reach maximum serum concentration (Tmax)
PK parameter
Measured: Up to 48 weeks
Trough serum concentration (Ctrough)
PK parameter
Measured: Up to 48 weeks
Area under the concentration-time curve in one dosing interval (AUC0-tau)
PK parameter
Measured: Up to 48 weeks
Terminal elimination half-life (t1/2)
PK parameter
Measured: Up to 48 weeks
Apparent clearance at steady state (CLss/F)
PK parameter
Measured: Up to 48 weeks
Apparant volume of distribution at steady state during the terminal phase (Vss/F)
PK parameter
Measured: Up to 48 weeks
Plasma Lyso-Gb3 level
PD parameter
Measured: Up to 48 weeks
Plasma Gb3 level
PD parameter
Measured: Up to 48 weeks
Urine Lyso-Gb3 level
PD Parameter
Measured: Up to 48 weeks
Urine Creatinine level
PD parameter
Measured: Up to 48 weeks
Urine Albumin level
PD parameter
Measured: Up to 48 weeks
Total Urine Protein Level
PD parameter
Measured: Up to 48 weeks
Urine Protein to Creatinine Ratio
PD parameter
Measured: Up to 48 weeks
Urine Albumin to Creatinine Ratio
PD parameter
Measured: Up to 48 weeks
Change in eGFR
PD parameter
Measured: Up to 48 weeks
Change in kidney Gb3 accumulation using the quantative Barisoni Lipid Inclusion Scoring System
PD parameter
Measured: Up to 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2025
- Study start14 May 2025
- Primary completion30 Aug 2028 (estimated)
- Study completion30 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DisesaseStudies of the same condition, open and under way.
- More trials run by GC Biopharma CorpThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- GC Biopharma Corp
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hanmi Pharmaceutical Company Limited
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06858397
- Sponsor's protocol ID
- GC1134A_FD_P1201
- Status last verified
- Nov 2025
- First submitted
- 19 Feb 2025
- Last updated
- 4 Nov 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Dose Range, Proof-of-Concept Study to Assess the Safety and Efficacy of HM15421/GC1134A in Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT06858397
- Acronym / sponsor ID
- GC1134A_FD_P1201
- Conditions
- Fabry Disesase
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- GC Biopharma CorpIndustry (pharma, biotech, devices)
- Collaborators
- Hanmi Pharmaceutical Company Limited
- Study start
- 14 May 2025 (actual)
- Primary completion
- 30 Aug 2028 (estimated)
- Study completion
- 30 Aug 2028 (estimated)
- First posted
- 5 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: HM15421/GC1134ALow dose |
| Cohort 2 | Experimental | Drug: HM15421/GC1134AMid dose |
| Cohort 3 | Experimental | Drug: HM15421/GC1134AHigh dose |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- 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.
Key exclusion criteria (17 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HM15421/GC1134ASC | Drug | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
Incidences and characteristics of adverse events
Time frame: Up to 48 weeks
17 secondary outcomes
Maximum serum concentration (Cmax)
Time frame: Up to 48 weeks
Time to reach maximum serum concentration (Tmax)
Time frame: Up to 48 weeks
Trough serum concentration (Ctrough)
Time frame: Up to 48 weeks
Area under the concentration-time curve in one dosing interval (AUC0-tau)
Time frame: Up to 48 weeks
Terminal elimination half-life (t1/2)
Time frame: Up to 48 weeks
Apparent clearance at steady state (CLss/F)
Time frame: Up to 48 weeks
Apparant volume of distribution at steady state during the terminal phase (Vss/F)
Time frame: Up to 48 weeks
Plasma Lyso-Gb3 level
Time frame: Up to 48 weeks
Plasma Gb3 level
Time frame: Up to 48 weeks
Urine Lyso-Gb3 level
Time frame: Up to 48 weeks
Urine Creatinine level
Time frame: Up to 48 weeks
Urine Albumin level
Time frame: Up to 48 weeks
Total Urine Protein Level
Time frame: Up to 48 weeks
Urine Protein to Creatinine Ratio
Time frame: Up to 48 weeks
Urine Albumin to Creatinine Ratio
Time frame: Up to 48 weeks
Change in eGFR
Time frame: Up to 48 weeks
Change in kidney Gb3 accumulation using the quantative Barisoni Lipid Inclusion Scoring System
Time frame: Up to 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2025.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
10 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing HM15421/GC1134A in people with Fabry Disesase, run by GC Biopharma Corp at 10 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 23 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Argentina, South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact GC Biopharma (+82-031-260-9300, GC1134A@gccorp.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidences and characteristics of adverse events (time frame: Up to 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2025.