Completed

A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

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What is being tested

Fabrazyme (agalsidase beta)

+ Placebo
Biological
Who is being recruted

Fabry Disease

Over 16 Years
+16 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Placebo-Controlled
Phase 4
Interventional
Study Start: February 2001

See protocol details

Summary

Principal SponsorGenzyme, a Sanofi Company
Last updated: December 27, 2013
Sourced from a government-validated database.Claim as a partner
Study start date: February 1, 2001Actual date on which the first participant was enrolled.

People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid ("globotriaosylceramide" or "GL-3") levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.

Official TitleMulti-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients With Fabry Disease 
Principal SponsorGenzyme, a Sanofi Company
Last updated: December 27, 2013
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details
82 patients to be enrolledTotal number of participants that the clinical trial aims to recruit.
Treatment Study
These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

How participants are assigned to different groups/arms
In this clinical study, participants are placed into groups randomly, like flipping a coin. This ensures that the study is fair and unbiased, making the results more reliable. By assigning participants by chance, researchers can better compare treatments without external influences.

Other Ways to Assign Participants
Non-randomized allocation: Participants are assigned based on specific factors, such as their medical condition or a doctor's decision.
None (Single-arm trial): If the study has only one group, all participants receive the same treatment, and no allocation is needed.

How treatments are given to participants
Participants are divided into different groups, each receiving a specific treatment at the same time. This helps researchers compare how well different treatments work against each other.

Other Ways to Assign Treatments
Single-group assignment: Everyone gets the same treatment.
Cross-over assignment: Participants switch between treatments during the study.
Factorial assignment: Participants receive different combinations of treatments.
Sequential assignment: Participants receive treatments one after another in a specific order, possibly based on individual responses.
Other assignment: Treatment assignment does not follow a standard or predefined design.

How the effectiveness of the treatment is controlled
In a placebo-controlled study, some participants receive the experimental treatment, while others receive an inert substance (placebo) to compare outcomes. This method helps to isolate the effect of the treatment from the psychological effects of receiving any treatment at all.

Other Options
Non-placebo-controlled: No placebo is used. All participants receive the actual treatment or alternative interventions (often the Standard of Care), and comparisons are made between these treatments.

How the interventions assigned to participants is kept confidential
Participants, researchers, outcome assessors, and care providers do not know which treatment is being given. This is the most complete way to prevent bias and keep the study as neutral as possible.

Other Ways to Mask Information
Open-label: Everyone knows which treatment is being given.
Single-blind: Participants do not know which treatment they are receiving, but researchers do.
Double-blind: Neither participants nor researchers know which treatment is given.
Triple-blind: Participants, researchers, and outcome assessors do not know which treatment is given.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria
Any sexBiological sex of participants that are eligible to enroll.
Over 16 YearsRange of ages for which participants are eligible to join.
Healthy volunteers not allowedIf individuals who are healthy and do not have the condition being studied can participate.
Conditions
Pathology
Fabry Disease
Criteria
7 inclusion criteria required to participate
Patients must provide written informed consent
Patients must be at least 16 years old
Patients must have a current diagnosis of Fabry disease and have a clinical presentation consistent of Fabry disease (decreased sweating, Fabry pain, angiokeratoma, etc.)
Patients may not have received enzyme replacement therapy as a treatment for Fabry disease

Show More Criteria

9 exclusion criteria prevent from participating
Patient has undergone or is currently scheduled for kidney transplantation or is currently on dialysis
Patient has acute renal failure
Patient has participated in a study employing an investigational drug within 30 days of study entry
Patient has diabetes mellitus or presence of confounding renal disease

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives
2 intervention groups 

are designated in this study

50% chance 

of being blinded to the placebo group

Treatment Groups
Group I
Placebo
Patients randomized to placebo
Group II
Active Comparator
Patients randomized to Fabrazyme (agalsidase beta).
Study Objectives
Primary Objectives

The primary efficacy endpoint was the time to the first occurrence of a clinically significant renal (33% increase in serum creatinine, dialysis or transplant), cardiac (myocardial infarction, significant change in cardiac status, i.e., angina, congestive heart failure or symptomatic arrhythmia requiring medication or surgery) or cerebrovascular (stroke or transient ischemic attack) event and/or death (due to any cause) in Fabrazyme (agalsidase beta) patients as compared to placebo patients.
Secondary Objectives

Time to a clinically significant renal event (33% increase in serum creatinine, dialysis or transplant) in Fabrazyme (agalsidase beta) patients as compared to placebo patients.

Summary of slopes of eGFR by baseline eGFR subgroups (\>60 and \<=60 mL/min/1.73m\^2/year) comparing Placebo vs Fabrazyme (agalsidase beta) Patients.

Summary of slopes of inverse serum creatinine by baseline serum creatinine subgroups (\> or \<= 1.5 mg/dL) comparing Placebo vs Fabrazyme (agalsidase beta) patients.

Neuropathic pain was assessed by Question 12 of the Brief Pain Inventory (BPI) Questionnaire on a scale of 0 (no pain) to 10 (pain as bad as you can imagine)

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.
This study has 26 locations
Suspended
University of Alabama at BirminghamBirmingham, United StatesSee the location
Suspended
Cedars-Sinai Medical CenterLos Angeles, United States
Suspended
University of San FranciscoSan Francisco, United States
Suspended
University of Connecticut Health PartnersFarmington, United States
Completed26 Study Centers