A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease

Takeda

Status and phase: Completed; Phase 3

Conditions: Fabry Disease

Treatments: Biological: REPLAGAL

Study type: Interventional

Funder types: Industry

Identifiers

NCT04974749

TAK-675-3001

2022-004246-35 (EudraCT Number)

Details and patient eligibility

About

The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks. Participants will visit their study clinic many times throughout the study.

Enrollment: 20 patients

Sex: All

Ages: 7 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participant and/or legally authorized representative must voluntarily sign an Institutional Review Board/Independent Ethics Committee approved written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the participant. For the participants less than (<) 18 years old, participants will give assent AND their parent(s)/legally authorized representative should sign the ICF accordingly.

• The participant has confirmed diagnosis of Fabry disease as determined by the investigator, according to medical record including:

  • For male participant, Fabry disease is confirmed by a deficiency of α-galactosidase A (GLA) activity and a mutation in the GLA gene
  • For female participant, Fabry disease is confirmed by a mutation in the GLA gene.

• The participant is 7 to 65 years of age, inclusive, at screening.

• Female participants of childbearing potential must have a negative pregnancy test at screening.

• Female participants of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study and for at least 14 days after the final investigational product infusion.

• The participant is deemed, as determined by the investigator, to have adequate general health to undergo the specified protocol-related procedures and to have no safety or medical contraindications for participation.

• The participant has not received any treatment (approved or investigational) specific to Fabry disease, such as ERT, chaperone therapy, or substrate reduction therapy.

• The adult participant (greater than or equal to [>=] 18 years old) must have an estimated glomerular filtration rate (eGFR) of 45 to 120 milliliter per minute per 1.73 meter square (mL/min/1.73 m^2) (inclusive). Serum creatinine is tested and the eGFR is calculated by central laboratory using the Chronic Kidney Disease Epidemiology (CKD-EPI) equation.

Exclusion criteria

• In the opinion of the investigator, the participant's life expectancy is less than or equal to (<=) 5 years.

• The participant has undergone or is scheduled to undergo kidney transplantation or is currently on dialysis or has any signs or symptoms of end stage renal disease.

• The participant has a urine protein/creatinine ratio of greater than (>) 500 milligram per gram (mg/g).

• The participant has a clinically relevant history of allergy or signs or symptoms of severe hypersensitivity, which in the investigator's judgment, will substantially increase the participant's risk if he or she participates in the study.

• In the opinion of the investigator, the participant has non-Fabry disease-related cause of end organ (renal, cardiovascular, central nervous system) dysfunction/failure or is receiving medications that may affect the rate of disease progression, as assessed by renal measures.

• The participant has a positive test result at screening for hepatitis B surface antigen with detectable hepatitis B viral deoxyribonucleic acid (DNA) load, hepatitis C virus (HCV) antibody with confirmation by HCV ribonucleic acid polymerase chain reaction testing, or human immunodeficiency virus antibody.

• The participant has received prior treatment with any of the following medications, with the exception of non-systemic use:

  • Chloroquine
  • Amiodarone
  • Monobenzone
  • Gentamicin

• The participant is pregnant or lactating.

• The participant has a body mass index >35 kilogram per square meter (kg/m^2).

• The participant is treated or has been treated with any investigational drug for indication other than Fabry disease within 30 days of study start.

• The participant and/or the participant's parent(s)/legal guardian is unable to understand the nature, scope, and possible consequences of the study.

• The participant is unable to comply with the protocol, example, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or is otherwise unlikely to complete the study, as determined by the investigator.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

REPLAGAL

Experimental group

Description:

Participants received REPLAGAL 0.2 milligrams per kilogram (mg/kg) body weight, intravenous (IV) infusion, every other week (EOW) from Day 1 (Week 0) up to Week 52.

Treatment:

Biological: REPLAGAL