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Tests treatment safety and results for Fabry Disesase
Official title A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
ClinicalTrials.gov ID: NCT06858397
What this study is testing
What is HM15421/GC1134A?
HM15421/GC1134A is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for fabry disesase.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- 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...
You likely can't join if
- 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...
- Urine protein to creatinine ratio (UPCR) \> 0.5 g/g and not treated with an angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor...
- Known history of hypersensitivity to any ingredient in the investigational product and to Gadolinium contrast agent that is not managed by the use of...
See the full eligibility 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.
- 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.
- 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...
- 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.
- 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...
- 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.
- 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 treatment study or using compassionate access product for FD. Participants who have participated in treatment trials for conditions not related to FD should be enrolled after the adequate wash out...
- 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...
- Participants who may have history of deliberate self-harm or suicidal ideation.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Kansas City, Kansas, United States
- Minneapolis, Minnesota, United States
- Cincinnati, Ohio, United States
- Pittsburgh, Pennsylvania, United States
- Fairfax, Virginia, United States
- Rosario, Santa Fe Province, Argentina
- Buenos Aires, Argentina
- Yangsan, Gyeongsangnam-do, South Korea
- Seoul, South Korea
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Kansas City, Kansas, United States; Minneapolis, Minnesota, United States; Cincinnati, Ohio, United States; Pittsburgh, Pennsylvania, United States; Fairfax, Virginia, United States and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.