Fabry DiseasePhase 2NCT00837824
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
A Phase 2 study testing Fabrazyme (agalsidase beta) in people with Fabry Disease and Chronic Kidney Disease, Stage IV (Severe), run by Genzyme, a Sanofi Company at 9 sites in United States.
Trial summary
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
- What is being tested?
- Fabrazyme (agalsidase beta)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 9 sitesUnited States
- Study dates
- Dec 2002to Aug 2003
- Phase
- Phase 2
- 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
- provided written informed consent prior to any study-related procedures being performed.
- be ≥16 years old.
- have a current diagnosis of Fabry disease (defined as abnormal α-galactosidase (α GAL) enzyme levels or Fabry genotype).
- have one of the following clinical conditions present at enrollment: serum creatinine level greater than 3.0 mg/dL (an average of two values at least one week apart), or be currently on dialysis, or be status post kidney transplant by greater than 3 months.
- have the ability to comply with the requirements of the protocol
- have a negative pregnancy test, if a female patient of childbearing potential. In addition, all female patients of childbearing potential must use a medically accepted method of contraception throughout the study.
Who cannot join
- if they did not meet the specific inclusion criteria.
- if they had participated in a study employing an investigational drug within 30 days of the start of their participation in this trial.
- had previously received enzyme replacement therapy (ERT) for their Fabry disease.
- had diabetic nephropathy.
- were pregnant or lactating.
- were unwilling to comply with the requirements of the protocol.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Study site
Boston, Massachusetts
Study site
Coral Springs, Florida
Study site
Farmington, Connecticut
Study site
Houston, Texas
Study site
Kansas City, Kansas
Study site
Milwaukee, Wisconsin
Study site
New York, New York
Study site
Pittsburgh, Pennsylvania
Study site
Rochester, New York
About this study
Official title: A Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal Disease
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Being tested
- Fabrazyme (agalsidase beta)
- Keywords
- Fabry Disease patients with Severe Renal Disease
- Medical subject headings
- Fabry Disease, Renal Insufficiency, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fabrazyme (agalsidase beta)Biological
Also called r-hαGAL
1.0 mg/kg every 2 weeks
Fabrazyme (agalsidase beta)Biological
Also called r-hαGAL
3.0 mg/kg every 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| Fabrazyme 1mg/kg every 2 weeks | Experimental | Fabrazyme 1.0 mg/kg every 2 weeks Receives: Biological: Fabrazyme (agalsidase beta) |
| Fabrazyme 3mg/kg every 2 weeks | Experimental | Fabrazyme 3.0 mg/kg every 2 weeks Receives: Biological: Fabrazyme (agalsidase beta) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease
The trial was terminated early due to inadequate study design. During the study period of 7 months, only 1 patient had a clinical event, a stroke, in the Fabrazyme 1 mg/kg treatment arm. The time to event was determined from first dose of Fabrazyme to the date of event.
Measured: 7 months
Secondary outcomes (1)
Plasma Globotriaosylceramide (GL-3)
This outcome measure evaluated the mean plasma GL-3 values for all patients to see if it decreased while on Fabrazyme. Normal plasma GL-3 level is defined as ≤ 7.03 µg/mL.
Measured: Evaluated at Baseline, Month 3, and Final Visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2009 (estimated)
- Study startDec 2002
- Primary completionAug 2003
- Study completionAug 2003
Why it stopped: The trial was terminated early due to inadequate study design.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- CRL/Medinet
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00837824
- Sponsor's protocol ID
- AGAL-022-02
- Status last verified
- Mar 2015
- First submitted
- 23 Oct 2008
- Last updated
- 7 Apr 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal Disease
- ClinicalTrials.gov ID
- NCT00837824
- Acronym / sponsor ID
- AGAL-022-02
- Keywords
- Fabry Disease patients with Severe Renal Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The trial was terminated early due to inadequate study design.
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Collaborators
- CRL/Medinet
- Study start
- Dec 2002
- Primary completion
- Aug 2003 (actual)
- Study completion
- Aug 2003 (actual)
- First posted
- 5 Feb 2009 (estimated)
- Results
- Posted 5 Feb 2009
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Fabrazyme 1mg/kg every 2 weeks | Experimental | Biological: Fabrazyme (agalsidase beta)Fabrazyme 1.0 mg/kg every 2 weeks |
| Fabrazyme 3mg/kg every 2 weeks | Experimental | Biological: Fabrazyme (agalsidase beta)Fabrazyme 3.0 mg/kg every 2 weeks |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- provided written informed consent prior to any study-related procedures being performed.
- be ≥16 years old.
- have a current diagnosis of Fabry disease (defined as abnormal α-galactosidase (α GAL) enzyme levels or Fabry genotype).
- have one of the following clinical conditions present at enrollment: serum creatinine level greater than 3.0 mg/dL (an average of two values at least one week apart), or be currently on dialysis, or be status post kidney transplant by greater than 3 months.
- have the ability to comply with the requirements of the protocol
Key exclusion criteria (6 listed)
- if they did not meet the specific inclusion criteria.
- if they had participated in a study employing an investigational drug within 30 days of the start of their participation in this trial.
- had previously received enzyme replacement therapy (ERT) for their Fabry disease.
- had diabetic nephropathy.
- were pregnant or lactating.
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)Also: r-hαGAL1.0 mg/kg every 2 weeks | Biological | Fabrazyme 1mg/kg every 2 weeks |
| Fabrazyme (agalsidase beta)Also: r-hαGAL3.0 mg/kg every 2 weeks | Biological | Fabrazyme 3mg/kg every 2 weeks |
Primary outcome
Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease
Time frame: 7 months
The trial was terminated early due to inadequate study design. During the study period of 7 months, only 1 patient had a clinical event, a stroke, in the Fabrazyme 1 mg/kg treatment arm. The time to event was determined from first dose of Fabrazyme to the date of event.
1 secondary outcome
Plasma Globotriaosylceramide (GL-3)
Time frame: Evaluated at Baseline, Month 3, and Final Visit
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Apr 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Fabry Disease
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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
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fabrazyme (agalsidase beta) in people with Fabry Disease and Chronic Kidney Disease, Stage IV (Severe), run by Genzyme, a Sanofi Company at 9 sites in United States.
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 6 inclusion and 6 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: stopped early, as of the sponsor's last update on 7 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 9 sites in United States. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 5 Feb 2009). 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?
Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease (time frame: 7 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 7 Apr 2015.