HM15421 for Fabry Disease

Recruiting at 9 trial locations

Overseen By Hayoung Lee, MPH

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: GC Biopharma Corp

Must not be taking: ACE inhibitors, ARBs

Disqualifiers: Pregnancy, Dialysis, Stroke, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 48 weeks

18 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have started or changed the dose of an ACE inhibitor or ARB in the 4 weeks before screening. If you are on FD therapy, you must not have received it in the past 6 months.

How is the drug HM15421/GC1134A different from other treatments for Fabry disease?

The drug HM15421/GC1134A is unique because it may offer a new approach compared to existing enzyme replacement therapies (ERT) and chaperone therapy, which are the current standard treatments for Fabry disease. While ERT requires lifelong intravenous administration and chaperone therapy is limited to specific genetic mutations, HM15421/GC1134A could potentially provide a novel mechanism or administration method, although specific details are not provided in the available research. ^{1 2 3 4 5}

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for adults with Fabry Disease who have specific levels of a disease marker in their blood, haven't had certain treatments for six months, and can follow the study rules. Women must not be pregnant or breastfeeding and meet genetic test criteria; men need to agree to contraception terms.

Inclusion Criteria

Plasma lyso-Gb3 levels greater than 1.5 times the ULN

I agree to follow the rules about birth control and not donating sperm.

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

Exclusion Criteria

Pacemaker or other contraindication for MRI scanning

Patients who received investigational gene therapy for FD

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive HM15421/GC1134A to evaluate safety, tolerability, pharmacokinetics, and efficacy

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• HM15421/GC1134A

Trial Overview The study tests HM15421/GC1134A's safety and how well it works in humans with Fabry Disease. It looks at how the body processes the drug (pharmacokinetics) and its effectiveness in treating symptoms of this genetic disorder.

Participant Groups

Group I: Cohort 3 Experimental Treatment 1 Intervention

Group II: Cohort 2 Experimental Treatment 1 Intervention

Group III: Cohort 1 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

GC Biopharma Corp

Lead Sponsor

Trials

10

Recruited

660+

Hanmi Pharmaceutical co., ltd.

Collaborator

Trials

8

Recruited

10,700+

Findings from Research

Enzyme replacement therapy (ERT) for adults with Fabry disease showed a statistically significant decrease in left ventricular mass index (LVMI) and a reduction in the risk of proteinuria, indicating potential benefits in heart health and kidney function over time.

In children with Fabry disease, the study found no significant evidence of effectiveness from ERT, suggesting that the treatment may not have the same benefits for younger patients as it does for adults.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term effectiveness of enzyme replacement therapy in Fabry disease: results from the NCS-LSD cohort study. Anderson, LJ., Wyatt, KM., Henley, W., et al.[2022]

References

1. England pubmed.ncbi.nlm.nih.gov

Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Long-term effectiveness of enzyme replacement therapy in Fabry disease: results from the NCS-LSD cohort study. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Baseline characteristics of patients enrolled in the Canadian Fabry Disease Initiative. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Fabry disease: baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Developments in the treatment of Fabry disease. [2021]