A Phase 4 interventional study of Replagal in Fabry Disease, sponsored by Shire. Recruiting at 3 sites in India. Per ClinicalTrials.gov, last updated 2026-09-03.
Sponsored by Shire (part of Takeda) · Phase 4, Interventional, and Treatment
The main aim of this study is to learn more about the safety profile of Replagal.
Participants will receive Replagal every 2 weeks at the clinic for about 1 year.
244 studies on the registry are indexed under Fabry Disease; 55 are open to participants now.
This study's planned enrollment of 5 is below the median of 22 across 108 interventional studies indexed under Fabry Disease.
Browse Fabry Disease studies →Shire is the lead sponsor of 351 studies on the registry; 2 are open to participants now.
Of its 48 completed or terminated interventional studies of FDA-regulated products, 48 (100%) have results posted.
First submitted after Shire became part of Takeda.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Note: Female participants not of childbearing potential defined as those who have been surgically sterilized (hysterectomy, bilateral oophorectomy, or tubal ligation) or who are (example, defined as at least 1 year since last regular menses with an appropriate clinical profile [that is, age appropriate, history of vasomotor symptoms]).
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants with fabry disease will receive Replagal 0.2 milligram per kilogram (mg/kg) intravenous infusion on Day 1 and every 2 weeks up to Week 51.
Biological: Replagal
Participants will receive Replagal 0.2 mg/kg, intravenous infusion at Day 1 and every 2 weeks.
Also known as: Agalsidase alfa
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. An SAE is any untoward medical occurrence (whether considered to be related to study product or not) that at any dose results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality or birth defect, an important medical event.
Time frame: From the start of study up to 53 weeks
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
AE is any unfavorable and unintended sign, symptom, or disease temporally associated with study or use of investigational drug product (IP), whether or not the AE is considered related to IP. TEAEs: AEs occurring or worsening at or after first dose of IP or ongoing at time of enrollment. SAE :untoward medical occurrence that at any dose met one, more of the following criteria: results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent, significant disability/incapacity, a congenital abnormality/birth defect, an important medical event. Severity: Mild: event that does not generally interfere with usual activities of daily living; Moderate: event that interferes with usual activities of daily living, causing discomfort, permanent risk of harm; Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention.
Time frame: From the study drug administration up to Week 53
Number of Participants With Adverse Drug Reactions (ADRs) Related to Replagal
An ADR is defined as a response to a drug which is noxious and unintended, and which occurs at doses normally used in humans for prophylaxis, diagnosis, or therapy of disease, or for the modification of physiological function. Number of participants with ADRs will be reported.
Time frame: From the study drug administration up to Week 53
Number of Participants With Infusion-related Reactions of Replagal
Number of participants with infusion-related reactions of Replagal will be reported.
Time frame: From the study drug administration up to Week 53
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
Change in eGRF levels will be assessed from baseline up to week 53.
Time frame: Baseline and at Weeks 13, 27, 39, and 53
Number of Participants With Change in Frequency and Regimen of Analgesic use of Replagal for Neuropathic Pain
Participants who are already taking analgesics, any change in frequency and regimen of analgesic use will be monitored throughout the study. Number of participants with change in frequency and regimen of analgesic use of Replagal for neuropathic pain will be reported.
Time frame: Baseline up to Week 53
Change From Baseline in Urine Concentration of Globotriaosylceramide (Gb3)
Change from baseline in urine concentration of Gb3 will be assessed.
Time frame: Baseline and at Weeks 13, 27, 39, and 53
Change From Baseline in Urine Protein Creatinine Ratio
Urine protein creatinine ratio will be assessed.
Time frame: Baseline and at Weeks 13, 27, 39, and 53
Percent Change From Baseline in Left Ventricular Mass Index (LVMI)
The 2-dimensional (2D) echocardiogram will be performed within one month of participant enrolment or at the time of screening, Week 27, and Week 53 and the percent change values from baseline in LVMI (gram per meter square \[g/m\^2\]) will be measured and reported.
Time frame: Baseline and at Weeks 27 and 53
Percent Change From Baseline in Left Ventricular Wall Thickness
The 2D echocardiogram will be performed within one month of participant enrolment or at the time of screening, Week 27, and Week 53 and the percent change values from baseline in left ventricular muscular thickness will be measured and reported.
Time frame: Baseline and at Weeks 27 and 53
Percent Change From Baseline in Ejection Fraction
The 2D echocardiogram will be performed within one month of participant enrolment or at the time of screening, Week 27, and Week 53 and the percent change values from baseline in ejection fraction will be measured and reported.
Time frame: Baseline and at Weeks 27 and 53
Percent Change From Baseline in Quality of Life Based on Questionnaire 36-itme Form Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores)
SF-36 is a generic quality-of-life instrument that has been widely used to assess health-related quality of life (HRQL) of participants. SF-36 consist of 36 items that are aggregated into 8 multi-item scales (physical functioning \[1=yes, limited a lot to 3=no, not limited at all\], role-physical \[1=all of the time to 5=none of the time\], bodily pain \[1=very severe to 6=none\], general health \[1=poor to 5=excellent\], vitality \[1=none of the time to 5=all of the time\], social functioning \[1=all of the time: to 5=none of the time\], role emotional \[1=all of the time to 5=none of the time\] and mental health \[1=all of the time to 5=none of the time\]). Four domains comprised physical component summary (PCS) score (physical functioning, role-physical, bodily pain, general health) and remaining 4 domains comprised mental component summary (MCS) score (vitality, social functioning, role-emotional, mental health). The scores ranges from 0 to 100. Higher scores indicating better HRQL.
Time frame: Baseline and at Weeks 27 and 53
Plan to share: Yes — Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report
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.
Data sharing details ↗ · vivli.org
Started Nov 2022 (actual). Primary completion is estimated for Oct 2027, 59 months after the start.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Agalsidase alfa