ClinicalTrials.gov record NCT05067868

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 4A study of a treatment that is already approved, to learn more about its long-term effects, safety or use in everyday practice.
SexAll
LocationIndia
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This open-label, single-arm Phase IV study gives IV Replagal to people with confirmed Fabry disease who have not previously received Replagal. Participants receive Replagal every two weeks for about one year and are followed for safety and measures of kidney, heart, urine biomarkers, pain medication use, and quality of life.

What it is studying. The study is testing the safety profile of Replagal (tracking adverse events, treatment-emergent events, drug-related adverse reactions, and infusion-related reactions) and measuring its effects on kidney function, urine Gb3 and protein levels, heart structure and function, neuropathic pain medication use, and health-related quality of life over about 53 weeks.

What the researchers hope to learn. To collect safety data on Replagal in Indian children and adults with Fabry disease and to gather information on kidney, cardiac, urine biomarker, pain medication use, and quality-of-life outcomes.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological
  • Replagal

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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]).

Exclusion criteria

  • 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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive Replagal 0.2 mg/kg by intravenous infusion on Day 1 and then every 2 weeks at the clinic for about one year (treatment through Week 51 with safety follow-up to Week 53). They will attend regular clinic visits for monitoring, which include heart ultrasound (echocardiogram) at specified weeks, urine and blood tests to track kidney and biochemical markers (including urine Gb3 and urine protein/creatinine), and quality-of-life questionnaires. Staff will monitor and record adverse events, including any infusion-related reactions; pregnancy testing before the first dose and contraception requirements apply. Participants must be able to return for visits and follow study procedures.

How the study is structured. Interventional treatment study with a single treatment group (no placebo or randomization). Open-label Phase IV trial. Participants receive intravenous Replagal 0.2 mg/kg on Day 1 and then every two weeks through Week 51, with safety and effectiveness assessments through Week 53.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in India. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Fabry disease

Clinical trials47 trials listed for Fabry diseaseEvery trial the registry lists for Fabry disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Fabry disease researchLatest monthly update: September 2026. Includes: SGLT2 outcome link. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Nov 1, 2022
Primary completion
Oct 31, 2027 (estimated)
Study completion
Oct 31, 2027 (estimated)
Target enrollment
5 participants (estimated)
Last updated on registry
Sep 3, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Shire
Collaborators
  • Takeda

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (3)

Terms used on this page · explanations by MedicalUpdates.ai

enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
LAR
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
written informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
estimated Glomerular Filtration rate
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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