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Tests treatment safety and results for Fabry Disease
Official title Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
ClinicalTrials.gov ID: NCT06081062
What this study is testing
What is Fabagal® (Agalsidase beta)?
Fabagal® (Agalsidase beta) is an investigational medicine, being studied as a potential treatment for fabry disease.
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
- Evaluate the safety and efficacy of Fabagal® developed by ISU ABXIS Co., Ltd., which has similar efficacy to active comparator (Agalsidase beta).
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 8 and older
You may be able to join if
- Those who have been diagnosed with Fabry disease by genetic and alpha-galactosidase A enzyme tests and grouped by sex are as follows:
- Male: Those who have confirmed GLA mutation (variation of α-galactosidase A gene) by genetic testing, and whose activity of alpha-galactosidase A in...
- Female: Those who have confirmed GLA mutation by genetic testing, and whose alpha-galactosidase A is within the normal range or is deficient
- Age: Those who are aged 8 years or older
- Those who have at least one of the following symptoms and signs:
You likely can't join if
- Patients who participated in other studies in which investigational products are administered within 30 days prior to the screening visit
- Patients with chronic kidney disease stage 4 to 5 (CKD 4-5; see Section 16.1)
- Patients who are currently on dialysis or have a history of kidney transplantation, or patients scheduled for dialysis at the time of screening, or...
- Patients who have started angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) treatment within 4 weeks prior to the...
- Patients who are pregnant, breastfeeding, or planning to become pregnant or breastfeed during the clinical study
- Patients with a history of HIV, hepatitis B/C or HIV antibodies, hepatitis B surface antigens, or hepatitis C antibodies
See the full eligibility criteria
- Those who have been diagnosed with Fabry disease by genetic and alpha-galactosidase A enzyme tests and grouped by sex are as follows:
- Male: Those who have confirmed GLA mutation (variation of α-galactosidase A gene) by genetic testing, and whose activity of alpha-galactosidase A in leukocytes is 5% or less than the normal mean value
- Female: Those who have confirmed GLA mutation by genetic testing, and whose alpha-galactosidase A is within the normal range or is deficient
- Age: Those who are aged 8 years or older
- Those who have at least one of the following symptoms and signs:
- Glomerular filtration rate decreased ( 2 or more cases of 30 ≦ eGFR \ 40] [including results within 6 months of the screening visit, but including results within 12 months for patients with a 60 ≦ eGFR \< 90 mL/min/1.73...
- Proteinuria that is equivalent to microalbuminuria or worse ( 2 or more cases of creatinine 30 mg/g in random urine at least 24 hours apart [including results within 6 months of the screening visit] or ≥30 mg of...
- For 24 hr urinary protein extraction (\>4 mg/m2/hr) or for spot urinary protein/creatinine ratio (≥200 mg/g [Cr]) \ Pediatrics: Aged \<19 years
- Abnormal left ventricular function as evidenced by MRI or echocardiography
- Left ventricular mass index (LVMI)\ \>115 g/m2 (male), \>95 g/m2 (female) or
- Left ventricular wall thickness \>12 mm (However, in the case of patients with hypertension, patients must have blood pressure treatment for at least 6 months prior to administration of the same drug) etc.
- Clinically significant arrhythmias and conduction disturbances, etc.
- Stroke or transient ischemic attack, etc., as evidenced by objective testing
- Patients who have not previously received enzyme replacement therapy (ERT) or Chaperone therapy for treatment of Fabry disease
- Patients who voluntarily consented and signed the informed consent form
- Patients (female patients and partners of male patients who are of childbearing potential) who have agreed to use a medically appropriate method of contraception (intrauterine device, condoms, surgical methods such as...
- Patients who participated in other studies in which investigational products are administered within 30 days prior to the screening visit
- Patients with chronic kidney disease stage 4 to 5 (CKD 4-5; see Section 16.1)
- Patients who are currently on dialysis or have a history of kidney transplantation, or patients scheduled for dialysis at the time of screening, or waitlisted for kidney transplantation
- Patients who have started angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) treatment within 4 weeks prior to the screening visit or whose dose has been changed
- Patients who are pregnant, breastfeeding, or planning to become pregnant or breastfeed during the clinical study
- Patients with a history of HIV, hepatitis B/C or HIV antibodies, hepatitis B surface antigens, or hepatitis C antibodies
- Patient whose medical, emotional, behavioral, or psychological conditions appear to interfere with compliance with the requirements of the clinical study according to the investigator's judgment
The study team makes the final eligibility decision.
Where it's taking place
- Manila, Philippines
- Seoul, Songpa-gu, 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, 8 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Manila, Philippines; Seoul, Songpa-gu, South Korea. 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.