A Study of Replagal in Children and Adults With Fabry Disease in India
ConditionFabry Disease
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorShire
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male and female Replagal naïve participants (and who are not part of any other program that allows participant to get access to free enzyme replacement therapy [ERT] at the time of study enrollment and during the study period) of any age with confirmed diagnosis of Fabry disease.
Participants who have documented confirmed diagnosis of Fabry disease based on proof of gene mutation: α-galactosidase A gene compatible with Fabry disease and/or a deficiency of α-galactosidase A (less than [<] 4.0 nanomole per milliliter per hour (nmol/mL/hour) in plasma or serum or <8 percent (%) of average mean normal in leukocytes and sequencing of GLA gene for females).
Participant must have any clinical manifestations of Fabry disease based on investigator's discretion.
Participant/legal authorized representative (LAR)/guardian is able to understand and willing to give written informed consent before performing any study specific procedures and willing to adhere to protocol requirements.
Disqualifiers
Participants who have received Replagal.
Participants with poorly controlled hypertension as per investigator's discretion.
Participants with chronic kidney disease (CKD) with estimated Glomerular Filtration rate less than 15 milliliter per minute (mL/min) /1.73 meter square (m²) and who had/will have kidney transplantation or are currently on dialysis.
Participants with any serious hepatic disorder who had abnormal hepatic function test values at screening (when either alanine aminotransferase [ALT] or aspartate aminotransferase [AST] level exceeded the value three times the upper limit of normal [ULN] and total bilirubin 1.5 times as high as the ULN); and deemed as clinically significant by investigator for hematology and biochemistry. These abnormal laboratory values could be discussed with medical monitor before excluding the participant.
Trial design
Design model
Single group
Treatments tested in this trial
Replagal
Biological/VaccineParticipants will receive Replagal 0.2 mg/kg, intravenous infusion at Day 1 and every 2 weeks.
Treatment groups
5 Participants
are divided into 1 treatment groupGroup A: ReplagalExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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 weeks2
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 533
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 534
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 53Secondary outcomes
1
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 532
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 533
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 534
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 53Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Shire
Lead sponsor
Takeda
Collaborator
Source ClinicalTrials.gov