Fabry DiseasePhase 4NCT00081497
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
A Phase 4 study testing agalsidase beta in people with Fabry Disease, run by Genzyme, a Sanofi Company at 25 sites in 6 countries.
Trial summary
People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes certain types of fatty substances called "glycolipids". 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?
- agalsidase beta
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 67 participants
- Where
- 25 sitesin 6 countries
- Study dates
- Jan 2004to Sep 2005
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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-008-00 (NCT00074984)
- Patients must provide written informed consent prior to study participation
- Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception throughout the study
Who cannot join
- The patient was unable to complete AGAL-008-00 (NCT00074984)
- The patient has undergone kidney transplantation or is currently on dialysis
- The patient has diabetes mellitus or presence of confounding renal disease
- The patient has a clinically significant organic disease or an unstable condition that precludes participation
- The patient is unwilling to comply with the protocol requirements
Trial locations and contacts
25 sites in 6 countries.
25 sites in 6 countries
United States18 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
University of Alabama at Birmingham
Birmingham, Alabama, 35294-0006
Children's Hospital
Buffalo, New York, 14209
Children's Memorial Hospital
Chicago, Illinois, 60614
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Oncology Hematology Association
Coral Springs, Florida, 33065
Duke University Medical Center
Durham, North Carolina, 27710
Baylor College of Medicine
Houston, Texas, 77030
University of Kansas Medical Center
Kansas City, Kansas, 66160-7233
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics
Minneapolis, Minnesota, 55455
Mount Sinai School of Medicine
New York, New York, 10029
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, 15261
University of Rochester School of Medicine
Rochester, New York, 14642
University of San Francisco
San Francisco, California, 94143
University of Washington School of Medicine
Seattle, Washington, 98195
University of Connecticut Health Partners
West Hartford, Connecticut, 06119
Canada3 sites
Queen Elizabeth II Health Center
Halifax, Nova Scotia, B3H 1V8
Hopital du Sacre-Coeur de Montreal
Montreal, Quebec, H4J 1C5
North York General Hospital
Toronto, Ontario, M2K 1E1
About this study
Official title: Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study
People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes 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 analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.
- Conditions
- Fabry Disease
- Being tested
- agalsidase beta
- Keywords
- alpha-galactosidase A, a-GAL, 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.
agalsidase betaBiological
Also called Fabrazyme, r-hαGAL
1.0 mg/kg every 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| Fabrazyme 1.0 mg/kg every 2 weeks | Experimental | This is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment. Receives: Biological: agalsidase beta |
What is being measured
The primary outcome is the main question the study is designed to answer.
Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods
The primary efficacy analysis was the summary of change in slope of inverse serum creatinine for Placebo/Fabrazyme patients in the Intent to Treat (ITT) Population. It compared the placebo period slope with the Fabrazyme period slope.
Measured: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)
Secondary outcomes (4)
Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months
Pre-Fabrazyme=baseline visit of AGAL-008-00 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).
Measured: Pre-Fabrazyme, 6, 12, and 18 months
Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months
Pre-Fabrazyme=baseline visit of AGAL-00-800 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).
Measured: Pre-Fabrazyme, 6, 12, and 18 months
Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months
Pre-Fabrazyme=baseline visit of AGAL00800 for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL00800; assessment prior to first Fabrazyme infusion in AGAL02503 for placebo patients who did not transition to Fabrazyme in AGAL00800.
Measured: Pre-Fabrazyme and 6, 12, and 18 months
Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months
Pre-Fabrazyme=baseline visit of AGAL-008-00 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).
Measured: Pre-Fabrazyme and 6, 12, and 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2004 (estimated)
- Study startJan 2004
- Primary completionSep 2005
- Study completionSep 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00081497
- Sponsor's protocol ID
- AGAL02503
- Status last verified
- Mar 2015
- First submitted
- 14 Apr 2004
- Last updated
- 2 Apr 2015
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study
- ClinicalTrials.gov ID
- NCT00081497
- Acronym / sponsor ID
- AGAL02503
- Conditions
- Fabry Disease
- Keywords
- alpha-galactosidase A, a-GAL, r-haGAL, Fabry, GL-3, Fabrazyme
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 67 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Jan 2004
- Primary completion
- Sep 2005 (actual)
- Study completion
- Sep 2005 (actual)
- First posted
- 16 Apr 2004 (estimated)
- Results
- Posted 18 Aug 2010
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 alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes 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 analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fabrazyme 1.0 mg/kg every 2 weeks | Experimental | Biological: agalsidase betaThis is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment. |
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-008-00 (NCT00074984)
- Patients must provide written informed consent prior to study participation
- Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception throughout the study
Key exclusion criteria (5 listed)
- The patient was unable to complete AGAL-008-00 (NCT00074984)
- The patient has undergone kidney transplantation or is currently on dialysis
- The patient has diabetes mellitus or presence of confounding renal disease
- The patient has a clinically significant organic disease or an unstable condition that precludes participation
- The patient is unwilling to comply with the protocol requirements
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 |
|---|---|---|
| agalsidase betaAlso: Fabrazyme, r-hαGAL1.0 mg/kg every 2 weeks | Biological | Fabrazyme 1.0 mg/kg every 2 weeks |
Primary outcome
Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods
Time frame: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)
The primary efficacy analysis was the summary of change in slope of inverse serum creatinine for Placebo/Fabrazyme patients in the Intent to Treat (ITT) Population. It compared the placebo period slope with the Fabrazyme period slope.
4 secondary outcomes
Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months
Time frame: Pre-Fabrazyme, 6, 12, and 18 months
Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months
Time frame: Pre-Fabrazyme, 6, 12, and 18 months
Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months
Time frame: Pre-Fabrazyme and 6, 12, and 18 months
Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months
Time frame: Pre-Fabrazyme and 6, 12, and 18 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Apr 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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, a Sanofi Company
25 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing agalsidase beta in people with Fabry Disease, run by Genzyme, a Sanofi Company at 25 sites in 6 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 5 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 2 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, Canada, Czechia, Hungary, Poland and 1 more country. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Aug 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods (time frame: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2 Apr 2015.