A Replagal study in Indian Children and Adults With Fabry Disease
- Conditions
- Health Condition 1: E752- Other sphingolipidosis
- Registration Number
- CTRI/2021/12/038690
- Lead Sponsor
- Shire Biotech India Pvt Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Closed to Recruitment of Participants
- Sex
- Not specified
- Target Recruitment
- 0
1. Male and female Replagal naïve subjects (and who are not part of any other program at the time of study enrollment and during the study period) of any age with confirmed diagnosis of Fabry disease.
2. Subjects 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 ( <4.0 nmol/mL/hour in plasma or serum or <8% of average mean normal in leukocytes and sequencing of GLA gene for females).
3. Subject must have any clinical manifestations of Fabry disease based on investigatorâ??s discretion.
4. Subject/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.
5. Female subjects of childbearing potential (eg, nonsterilised, premenopausal female subjects) must have a documented negative pregnancy test prior to administration of the first dose of Replagal in this study. In addition, all female subjects of childbearing potential must use a medically accepted form of contraception throughout the study, ie, either a barrier method or hormonal contraceptive with norethindrone and ethinyl estradiol or similar active components.
6. Male subject who is nonsterilised and sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (eg, condom with or without spermicide) from signing of informed consent throughout the duration of the study.
Note: Female subjects not of childbearing potential defined as those who have been surgically sterilized (hysterectomy, bilateral oophorectomy, or tubal ligation) or who are postmenopausal (eg, defined as at least 1 year since last regular menses with an appropriate clinical profile [ie, age appropriate, history of vasomotor symptoms]
1. Subject who have received Replagal.
2. Subjects with poorly controlled hypertension as per investigatorâ??s discretion.
3. Subjects with chronic kidney disease (CKD) with estimated Glomerular Filtration rate less than 15 mL/min/1.73 m2 and who had/will have kidney transplantation or are currently on dialysis.
4. Subjects with any serious hepatic disorder who had abnormal hepatic function test values at screening (when either ALT or 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 investigstor for hematology and biochemistry. These abnormal laboratory values could be discussed with medical monitor before excluding the subject.
5. If female, the subject is pregnant or lactating or intending to become pregnant before participating in this study, during the study; or intending to donate ova during such time period.
6. Subject/LAR/guardian is unable to understand the nature, scope, and possible consequences of the study.
7. Subject is unable to comply with the protocol, eg, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the investigator.
8. If male, the subject intends to donate sperm during the course of this study.
9. Subjects who had participated in any other investigational drug study within the past 4 weeks prior to screening.
10. Any subject deemed as unfit for this trial, as per investigatorâ??s clinical judgment.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence of adverse events (AEs), serious AEs(SAEs), treatment emergent AEs, treatment emergent SAEs, adverse drug reactions(ADRs), infusion-related reactions, and deathTimepoint: 53 weeks
- Secondary Outcome Measures
Name Time Method Changes from baseline to Weeks 27 and 53 in the quality of life based on the questionnaire (36-item short form survey [SF-36])Timepoint: Baseline to Weeks 27 and 53;Changes in estimated glomerular filtration rate (eGFR)Timepoint: Baseline to Weeks 13, 27, 39, and 53;Changes in urine Gb3 (globotriaosylceramide)Timepoint: Baseline to Weeks 13,27,39,and 53;Changes in urine protein/creatinine ratioTimepoint: Baseline to Weeks 13, 27, 39, and 53;Frequency and changes in regimen of analgesic use for neuropathic painTimepoint: Baseline to 53 weeks;Percentage changes in ejection fractionTimepoint: Baseline to Weeks 27 and 53;Percentage changes in left ventricular mass index (LVMI) (g/m2)Timepoint: Baseline to Weeks 27 and 53;Percentage changes in left ventricular wall thicknessTimepoint: Baseline to Weeks 27 and 53