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
Qualifiers
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
Treatments tested in this trial
- Replagal
Treatment groups
5 Participants
are divided into 1 treatment groupSponsors and collaborators
Shire
Lead sponsor
Takeda
Collaborator
Source ClinicalTrials.gov