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

NCT04974749 | Completed Phase 3 Results

• 2 other identifiers: TAK-675-30012022-004246-35
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 6

Brief Summary

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.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs)

  An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this investigational product (IP) or medicinal product. Serious AE was any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality/birth defect, and was an important medical event. A TEAE was defined as any event emerging at or after the initiation of treatment with an IP or any existing event that worsened in either intensity or frequency following exposure to the IP until the end of the safety follow-up period.

  From start of study drug administration up to 14 days after end of treatment (EOT) [up to Week 54]

Secondary Outcomes (28)

• Number of Participants With TEAEs

  From start of study drug administration up to 14 days after EOT (up to Week 54)

• Number of Participants With Infusion-related Reactions (IRRs)

  From start of study drug administration up to Week 52

• Number of Participants With Positive Anti-drug Antibodies (ADA) to REPLAGAL

  Baseline up to Week 52

• Number of Participants With Positive Neutralizing Antibodies (NAb) to REPLAGAL

  Baseline up to Week 52

• Number of Participants With Clinically Meaningful Changes in Laboratory Parameters

  From start of study drug administration up to Week 52

Study Arms (1)

REPLAGAL

EXPERIMENTAL

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.

Biological: REPLAGAL

Interventions

REPLAGAL BIOLOGICAL

REPLAGAL IV infusion.

Also known as: Agalsidase Alfa, TAK-675

REPLAGAL

Eligibility Criteria

• Age: 7 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (6)

Peking Union Medical College Hospital

Beijing, Dongcheng District, China

Location

Xiangya Hospital, Central South University

Changsha, Kaifu District, China

Location

West China Hospital, Sichuan University

Chengdu, Wuhou District, China

Location

The Children's Hospital of Zhejiang University School of Medicine

Hangzhou, Xiacheng District, China

Location

Shandong Provincial Hospital

Jinan, Huaiyin District, China

Location

Ruijin Hospital, Shanghai Jiaotong Uni. School of Med.

Shanghai, Huangpu District, China

Location

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase alfa

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

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Medical Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 21, 2021
• First Posted: July 23, 2021
• Study Start: May 1, 2022
• Primary Completion: January 3, 2024
• Study Completion: January 3, 2024
• Last Updated: October 8, 2024
• Results First Posted: October 8, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

CN (6)