A Study of Replagal in Children and Adults With Fabry Disease in India

Официальное название

A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Replagal (Agalsidase Alfa [r-DNA Origin]) in Indian Children and Adults With Fabry Disease

Обзор

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.

Вмешательства

• Биопрепарат Replagal
  Participants will receive Replagal 0.2 mg/kg, intravenous infusion at Day 1 and every 2 weeks.

Первичные конечные точки

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Срок оценки: 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) [Срок оценки: From the study drug administration up to Week 53]
• Number of Participants With Adverse Drug Reactions (ADRs) Related to Replagal [Срок оценки: From the study drug administration up to Week 53]
• Number of Participants With Infusion-related Reactions of Replagal [Срок оценки: From the study drug administration up to Week 53]

Вторичные конечные точки (8)

• Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) [Срок оценки: 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 [Срок оценки: Baseline up to Week 53]
• Change From Baseline in Urine Concentration of Globotriaosylceramide (Gb3) [Срок оценки: Baseline and at Weeks 13, 27, 39, and 53]
• Change From Baseline in Urine Protein Creatinine Ratio [Срок оценки: Baseline and at Weeks 13, 27, 39, and 53]
• Percent Change From Baseline in Left Ventricular Mass Index (LVMI) [Срок оценки: Baseline and at Weeks 27 and 53]
• Percent Change From Baseline in Left Ventricular Wall Thickness [Срок оценки: Baseline and at Weeks 27 and 53]
• Percent Change From Baseline in Ejection Fraction [Срок оценки: 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) [Срок оценки: Baseline and at Weeks 27 and 53]

Критерии участия

Критерии включения

• 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.
• Female participants of childbearing potential (example, nonsterilised, premenopausal female participants) must have a documented negative pregnancy test prior to administration of the first dose of Replagal in this study. In addition, all female participants of childbearing potential must use a two medically accepted forms of contraception throughout the study, that is, either a barrier method or hormonal contraceptive with norethindrone and ethinyl estradiol or similar active components.
• Male participant who is nonsterilised and sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (example, condom with or without spermicide) from signing of informed consent throughout the duration of the study.

Note: Female participants not of childbearing potential defined as those who have been surgically sterilized (hysterectomy, bilateral oophorectomy, or tubal ligation) or who are postmenopausal (example, defined as at least 1 year since last regular menses with an appropriate clinical profile \[that is, age appropriate, history of vasomotor symptoms\]).

Критерии исключения

• 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 (m\^2) 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.
• If female, the participant 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.
• Participant/LAR/guardian is unable to understand the nature, scope, and possible consequences of the study.
• Participant is unable to comply with the protocol, example, 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.
• If male, the participant intends to donate sperm during the course of this study.
• Participants who had participated in any other investigational drug study within the past 4 weeks prior to screening.
• Any participant deemed as unfit for this trial, as per investigator's clinical judgment.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Индия · 3 центра

• Institute of Child Health — Kolkata
• All India Institute of Medical Sciences (AIIMS) — New Delhi
• Sir Gangaram Hospital — New Delhi

Идентификаторы

NCT: NCT05067868 · TAK-675-4008 · 2022-004233-40

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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