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

Los Angeles, CA

Phase 1 & 2

Waitlist Available

Research Sponsored by GC Biopharma Corp

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

1. 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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 48 weeks

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.

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Eligible Conditions

Fabry disease

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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1. Participants must be ≥ 18 years of age or age considered as adult in the respective country at the time of signing the informed consent.

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2. Documented diagnosis of FD with clinical symptoms.

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3. Females: historical genetic test results based on identification of pathogenic or likely pathogenic GLA variant of FD.

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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).

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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.

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6. Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m2 by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation.

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7. Plasma lyso-Gb3 levels greater than 1.5 times the upper limit of normal (ULN).

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* Refrain from donating sperm

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* 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

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* In addition to male condom, use of highly effective method of contraception may be considered in WOCBP partners of male participants.