Evaluating Fabagal for treating Fabry disease

A Phase 3 Randomized, Double-blinded, Active-controlled, Multicenter Trial to Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

This study is testing if a new treatment called Fabagal can help people with Fabry disease feel better and is comparing it to a similar existing treatment.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 8 Years and up
• Sex: All
• Sponsor: ISU Abxis Co., Ltd. (industry)
• Locations: 3 sites (Manila and 2 other locations)
• Trial ID: NCT06081062 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and efficacy of Fabagal® (Agalsidase beta) in patients diagnosed with Fabry disease. The primary objective is to compare the efficacy of Fabagal to an active comparator, Agalsidase beta, while secondary objectives will focus on evaluating safety, pharmacokinetics, and immunogenicity. Participants will be monitored for their response to the treatment and any adverse effects that may arise during the study. The trial will include patients aged 8 years and older who meet specific diagnostic criteria.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 8 years or older with a confirmed diagnosis of Fabry disease and specific enzyme activity levels.

Not a fit: Patients without a confirmed GLA mutation or those with normal alpha-galactosidase A activity may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for managing Fabry disease.

How similar studies have performed: Other studies have shown success with similar enzyme replacement therapies for Fabry disease, indicating a promising approach.

Eligibility criteria

Inclusion Criteria:

1. 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
2. Age: Those who are aged 8 years or older
3. Those who have at least one of the following symptoms and signs:

   * Glomerular filtration rate decreased (Inclusion criteria: 2 or more cases of 30 ≦ eGFR \< 90 mL/min/1.73 m2 \[adjusted for age \>40\] \[including results within 6 months of the screening visit, but including results within 12 months for patients with a 60 ≦ eGFR \< 90 mL/min/1.73 m2\])

     * Proteinuria that is equivalent to microalbuminuria or worse (Inclusion criteria: 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 albuminuria in 24-hour urine)

       * 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
4. Patients who have not previously received enzyme replacement therapy (ERT) or Chaperone therapy for treatment of Fabry disease
5. Patients who voluntarily consented and signed the informed consent form
6. 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 vasectomy) during the clinical study

Exclusion Criteria:

1. Patients who participated in other studies in which investigational products are administered within 30 days prior to the screening visit
2. Patients with chronic kidney disease stage 4 to 5 (CKD 4-5; see Section 16.1)
3. 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
4. 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
5. Patients who are pregnant, breastfeeding, or planning to become pregnant or breastfeed during the clinical study
6. Patients with a history of HIV, hepatitis B/C or HIV antibodies, hepatitis B surface antigens, or hepatitis C antibodies
7. 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

Where this trial is running

Manila and 2 other locations

• Philippine General Hospital — Manila, Philippines (RECRUITING)
• St.Luke's Medical Center — Manila, Philippines (RECRUITING)
• Seoul Asan Center — Seoul, Songpa-gu, South Korea (RECRUITING)

Study contacts

• Study coordinator: Beomhee Lee

Conditions: Fabry Disease