Fabry DiseasePhase 3NCT00074971
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
A Phase 3 study testing Fabrazyme (agalsidase beta) in people with Fabry Disease, run by Genzyme, a Sanofi Company at 20 sites in 5 countries.
Trial summary
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Fabrazyme (agalsidase beta)
- How many people
- 58 participants
- Where
- 20 sitesin 5 countries
- Study dates
- Oct 1999to Dec 2004
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients must have successfully completed the previous double-blind study (AGAL-1-002-98)
- Patients must provide written informed consent prior to study participation
- Female patients must have a negative pregnancy test prior to each dosing and use a medically accepted method of contraception throughout the study
Who cannot join
- Patient has undergone kidney transplant or is currently on dialysis
- Patient is pregnant or lactating
- Patient is unwilling to comply with the requirements of the protocol
- Patient has a clinically significant organic disease (with the exception of symptoms related to Fabry disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would preclude participation in the study
Trial locations and contacts
20 sites in 5 countries.
20 sites in 5 countries
United States14 sites
University of New Mexico
Albuquerque, New Mexico, 87131
Greater Baltimore Medical Center
Baltimore, Maryland, 21204
Fetal Diagnostic and Imaging Center
Billings, Montana, 59101
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Children's Memorial Hospital
Chicago, Illinois, 60614
Northwest Oncology & Hematology Associates
Coral Springs, Florida, 33065
Hart Family Practice
Hickory, North Carolina, 28601
University of Iowa Hospital and Clinics
Iowa City, Iowa, 52242
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Mount Sinai School of Medicine
New York, New York, 10029
University of Rochester School of Medicine
Rochester, New York, 14642
Hematology/Oncology Associates of South Texas
San Antonio, Texas, 78229
University of California San Fransisco
San Francisco, California, 94143
University of Washington School of Medicine
Seattle, Washington, 98195
France2 sites
Hospital Edouard Herriot
Lyon, Cedex 03
Hospital Europeen Georges Pompidou
Paris, Cedex 15
United Kingdom2 sites
National Hospital for Neurology and Neurosurgery
London, WC1N 3BG
Hope Hospital
Manchester, M6 8HD
About this study
Official title: A Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme 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 ("globatriaosylceramide" 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.
- Conditions
- Fabry Disease
- Being tested
- Fabrazyme (agalsidase beta)
- Keywords
- a-Galactosidase A, aGAL, r-haGAL, Fabry, GL-3, Fabrazyme
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fabrazyme (agalsidase beta)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and efficacy
Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
Secondary outcomes (6)
Changes in McGill Pain Questionnaire
Autonomic status
Glomerular filtration
Functional assessment of urinary protein excretion Ophthalmic changes
SF-36 Health Survey
Physician's assessment of Fabry Symptoms and pain medication
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Dec 2003 (estimated)
- Study startOct 1999
- Study completionDec 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 58 participants
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Medical Monitor, Study director, Genzyme Coorporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00074971
- Sponsor's protocol ID
- AGAL-005-99
- Status last verified
- Dec 2013
- First submitted
- 24 Dec 2003
- Last updated
- 4 Dec 2013
- Results posted
- No
Publications
Germain DP, Charrow J, Desnick RJ, Guffon N, Kempf J, Lachmann RH, Lemay R, Linthorst GE, Packman S, Scott CR, Waldek S, Warnock DG, Weinreb NJ, Wilcox WR. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. J Med Genet. 2015 May;52(5):353-8. doi: 10.1136/jmedgenet-2014-102797. Epub 2015 Mar 20.
PubMed 25795794
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT00074971
- Acronym / sponsor ID
- AGAL-005-99
- Conditions
- Fabry Disease
- Keywords
- a-Galactosidase A, aGAL, r-haGAL, Fabry, GL-3, Fabrazyme
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 58 participants
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Oct 1999
- Study completion
- Dec 2004
- First posted
- 25 Dec 2003 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme 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 ("globatriaosylceramide" 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients must have successfully completed the previous double-blind study (AGAL-1-002-98)
- Patients must provide written informed consent prior to study participation
- Female patients must have a negative pregnancy test prior to each dosing and use a medically accepted method of contraception throughout the study
Key exclusion criteria (4 listed)
- Patient has undergone kidney transplant or is currently on dialysis
- Patient is pregnant or lactating
- Patient is unwilling to comply with the requirements of the protocol
- Patient has a clinically significant organic disease (with the exception of symptoms related to Fabry disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would preclude participation in the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fabrazyme (agalsidase beta) | Drug | – |
Primary outcome
Safety and efficacy
Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
6 secondary outcomes
Changes in McGill Pain Questionnaire
Autonomic status
Glomerular filtration
Functional assessment of urinary protein excretion Ophthalmic changes
SF-36 Health Survey
Physician's assessment of Fabry Symptoms and pain medication
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2013.
- Derived from this study: PubMed 25795794Germain DP, Charrow J, Desnick RJ, Guffon N, Kempf J, Lachmann RH, Lemay R, Linthorst GE, Packman S, Scott CR, Waldek S, Warnock DG, Weinreb NJ, Wilcox WR. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. J Med Genet. 2015 May;52(5):353-8. doi: 10.1136/jmedgenet-2014-102797. Epub 2015 Mar 20.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme Coorporation
20 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Fabrazyme (agalsidase beta) in people with Fabry Disease, run by Genzyme, a Sanofi Company at 20 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, France, United Kingdom, Netherlands, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and efficacy. The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00074971, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2013.