Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

NCT06081062 | Recruiting Phase 3

• 1 other identifier: ISU303-003
• Study Type: interventional
• Target: 24
• Locations: 2 countries
• Sites: 3

Brief Summary

Evaluate the safety and efficacy of Fabagal® developed by ISU ABXIS Co., Ltd., which has similar efficacy to active comparator (Agalsidase beta).

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• Evaluate the efficacy of Fabagal compared with active comparator (Agalsidase beta)

  The proportion of patients achieving a GL-3 score (score 0) in renal cells after treatment with Fabagal or Active comparator (Agalsidase beta) in patients with Fabry disease The proportion of patients achieving a GL-3 score (score 0) in renal cells after treatment with Fabagal or Active comparator (agalsidase beta) in patients with Fabry disease The primary endpoint is a comparison of the proportion of subjects in each group who achieve a score of Zero on the renal capillary endothelium histology. The score is graded on a scale from 0-3 (normal, mild, moderate, and severe). * 0: signified no visible inclusions * 1: signified multiple discrete lipid granules * 2: signified single or multiple aggregates of lipid granules * 3: signified aggregates of lipid granules either large enough or numerous enough to cause clear distortion of the luminal surface

  Screening visit (Visit 0) and Visit 25 (Day 336)

Secondary Outcomes (5)

• Change from baseline in GL-3 levels in renal cells after administration of 24 doses of Fabagal compared to active comparator

  Screening visit (Visit 0) and Visit 25 (Day 336)

• Change from baseline in GL-3/lyso Gb-3 concentration in urine and blood after administration of 24 doses of Fabagal compared to active comparator

  Screening visit (Visit 0), Visits 3, 5, 7, 9, and 11 (Days 28, 56, 84, 112, and 140), Visit 13 (Day 168), Visits 15, 17, 19, 21, and 23 (Days 196, 224, 252, 280, and 308) and Visit 25 (Day 336)

• Change from baseline in renal function test values (estimated Glomerular Filtration Rate, eGFR) after administration

  Screening visit (Visit 0), Visit 13 (Day 168), and Visit 25 (Day 336)

• Change from baseline in pain score after administration of 24 doses of Fabagal compared to active comparator (Short Form McGill Pain Questionnaire-2)

  Visits 1, 13, and 25 (Days 0, 168, and 336)

• Change from baseline in quality of life after administration of 24 doses of Fabagal compared to active comparator (SF-36v2 questionnaire)

  Visits 1, 13, and 25 (Days 0, 168, and 336)

Study Arms (2)

Fabagal® (Agalsidase beta)

EXPERIMENTAL

1 mg/kg, administered every 2 weeks for 12 months

Biological: Fabagal® (Agalsidase beta)

Active Comparator (Agalsidase beta)

ACTIVE COMPARATOR

1 mg/kg, administered every 2 weeks for 12 months

Drug: Active comparator (Agalsidase beta)

Interventions

Fabagal® (Agalsidase beta) BIOLOGICAL

1 mg/kg every 2 weeks for 12 months

Fabagal® (Agalsidase beta)

Active comparator (Agalsidase beta) DRUG

1 mg/kg every 2 weeks for 12 months

Active Comparator (Agalsidase beta)

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify 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 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:
• 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 vasectomy) during the clinical study

You may not qualify 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 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

Sponsors & Collaborators

• ISU Abxis Co., Ltd.: lead

Study Sites (3)

Philippine General Hospital

Manila, Philippines

RECRUITING

St.Luke's Medical Center

Manila, Philippines

RECRUITING

Seoul Asan Center

Seoul, Songpa-gu, 05505, South Korea

RECRUITING

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase beta

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Central Study Contacts

Beomhee Lee

CONTACT

1688-7575; webmaster@amc.seoul.kr

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 8, 2023
• First Posted: October 12, 2023
• Study Start: April 14, 2023
• Primary Completion: June 1, 2025
• Study Completion: December 1, 2025
• Last Updated: July 29, 2024

Record last verified: 2024-07

Locations

PH (2); KR (1)