Fabry DiseasePhase 4NCT00312767
A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.
A Phase 4 study testing Fabrazyme (agalsidase beta) in people with Fabry Disease, run by Genzyme, a Sanofi Company at 1 site in United States.
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 18 to 65 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Fabrazyme (agalsidase beta)
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2006to not given
- Phase
- Phase 4
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient must provide signed, written informed consent prior to any study-related procedures being performed.
- Patient is between 18 and 65 years of age, inclusive.
- Patient has documented Fabry disease.
- Patient has received bi-weekly 1 mg/kg infusions of Fabrazyme for at least 6 months prior to enrollment into the study.
- Patient has not experienced moderate or severe infusion-associated reactions (IARs) from Fabrazyme infusions, which were also associated with a rate reduction, within 3 months prior to enrollment into the study.
- Patient has been receiving chronic hemodialysis for treatment of end-stage renal insufficiency for at least 3 months prior to enrollment into the study.
- Patient has good vascular access for hemodialysis.
- Patient has not and will not have any other (investigational) drug(s) infused during their hemodialysis, and is expected to have a stable concomitant medication regimen at all PK assessments.
Who cannot join
- Patient's hemoglobin is < 9 g/100 mL at Screening/Baseline.
- Patient has a clinically significant organic disease or an unstable condition that, in the opinion of the Investigator, would preclude participation in the study.
- Patient has a medical condition, serious intercurrent illness, or extenuating circumstance that would significantly decrease study compliance.
- Patient has participated in a study employing an investigational drug within 30 days prior to the start of their participation in this study.
- Patient is unwilling to comply with the requirements of the protocol.
Trial locations and contacts
1 site in 1 country.
United States1 site
Trident Nephrology Associates
North Charleston, South Carolina, 29405
About this study
Official title: A Multicenter, Open-Label, Cross-Over Trial to Evaluate the Pharmacokinetics of Fabrazyme During Simultaneous Fabrazyme Infusion and Chronic Hemodialysis 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 (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 is designed to verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis in patients currently receiving Fabrazyme at a dose of 1.0 mg/kg every 2 weeks.
- Conditions
- Fabry Disease
- Being tested
- Fabrazyme (agalsidase beta)
- Keywords
- Agalsidase beta, alpha Galactosidase A, aGAL, rh aGAL, Fabry, GL3, Fabrazyme, dialysis, hemodialysis
- 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.
Verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis with a low-flux membrane.
Secondary outcomes (1)
Verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis with a high-flux membrane.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2006 (estimated)
- Study startApr 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Crossover
- Masking
- None (open label)
- Enrolment
- 0 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
- NCT00312767
- Sponsor's protocol ID
- AGAL03505
- Status last verified
- Feb 2014
- First submitted
- 7 Apr 2006
- Last updated
- 5 Feb 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label, Cross-Over Trial to Evaluate the Pharmacokinetics of Fabrazyme During Simultaneous Fabrazyme Infusion and Chronic Hemodialysis in Patients With Fabry Disease.
- ClinicalTrials.gov ID
- NCT00312767
- Acronym / sponsor ID
- AGAL03505
- Conditions
- Fabry Disease
- Keywords
- Agalsidase beta, alpha Galactosidase A, aGAL, rh aGAL, Fabry, GL3, Fabrazyme, dialysis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Apr 2006
- First posted
- 11 Apr 2006 (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 (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 is designed to verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis in patients currently receiving Fabrazyme at a dose of 1.0 mg/kg every 2 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Patient must provide signed, written informed consent prior to any study-related procedures being performed.
- Patient is between 18 and 65 years of age, inclusive.
- Patient has documented Fabry disease.
- Patient has received bi-weekly 1 mg/kg infusions of Fabrazyme for at least 6 months prior to enrollment into the study.
- Patient has not experienced moderate or severe infusion-associated reactions (IARs) from Fabrazyme infusions, which were also associated with a rate reduction, within 3 months prior to enrollment into the study.
Key exclusion criteria (5 listed)
- Patient's hemoglobin is < 9 g/100 mL at Screening/Baseline.
- Patient has a clinically significant organic disease or an unstable condition that, in the opinion of the Investigator, would preclude participation in the study.
- Patient has a medical condition, serious intercurrent illness, or extenuating circumstance that would significantly decrease study compliance.
- Patient has participated in a study employing an investigational drug within 30 days prior to the start of their participation in this study.
- Patient is unwilling to comply with the requirements of the protocol.
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
Verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis with a low-flux membrane.
1 secondary outcome
Verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis with a high-flux membrane.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2014.
Related trials for Fabry Disease
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A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
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Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
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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
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Fabrazyme (agalsidase beta) in people with Fabry Disease, run by Genzyme, a Sanofi Company at 1 site 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 8 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: withdrawn, as of the sponsor's last update on 5 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis with a low-flux membrane.. 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 5 Feb 2014.