Fabry DiseasePhase 4NCT00140621
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
A Phase 4 study testing Agalsidase beta in people with Fabry Disease, run by Genzyme, a Sanofi Company at 7 sites in Japan.
Trial summary
This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry disease. Read more about this study
- Who is it for?
- Ages 20 to 64 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Agalsidase beta
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 7 sitesJapan
- Study dates
- Jul 2005to Aug 2012
- 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 20 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Participants definitively diagnosed with cardiac Fabry disease (who fulfill all of the following criteria)
- In the case of male participants, documented plasma or leukocyte alpha-galactosidase A (α-GAL) activity was no more than 20 percent (%) of normal value (except for heterozygous female participants)
- Left ventricular hypertrophy was noted.
- Accumulation of globotriaosylceramide (GL-3) in the myocardium or a genetic deficiency associated with α-GAL was confirmed
- Or in the case of heterozygous female participants, when the family (father or son) was diagnosed with Fabry disease. (Father or son was related by birth.)
- Without symptoms or signs of Fabry, such as acroparesthesia, angiokeratomas, abnormal sweating, pain of distal extremities, chronic abdominal pain/diarrhea and corneal opacities were observed, except for proteinuria sign.
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- Participants with interventricular and posterior wall thickness of at least 13 millimeter (mm) on echocardiography within 3 months before signed date to informed consent
- Participants in whom cardiac function was rated as Class I or II according to the New York Heart Association (NYHA) classification when giving informed consent.
- Participants classification: inpatients and outpatients
- Participants who had given written informed consent before the study-related baseline tests.
Who cannot join
- Participants with severe hypertension (for example, blood pressure more than or equal to 180 millimeter of mercury [mmHg] and/or blood pressure more than or equal to 110 mmHg in spite of adequate medication)
- Participants whose serum creatinine level was higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent.
- Participants who had undergone kidney transplantation or were currently on dialysis.
- Participants with any serious hepatic disorder. Participants who had abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either alanine aminotransferase [ALT] or aspartate aminotransferase [AST] level exceeded the value five times as high as the upper normal limit).
- Permanent pacemaker or defibrillator implanted participants
- Pregnant or lactating women
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- Participants who had taken this drug for 6 months (26 weeks) or more before giving informed consent.
- Participants who had participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent.
- Enzyme replacement therapy history, except for agalsidase beta
- Participants who were unwilling to comply with the requirements of the protocol.
- Others judged by the investigator or sub-investigator to be ineligible for the study
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
Japan7 sites
Fujita Health University Hospital
Aichi, 470-1192
Sapporo Medical University Hospital
Hokkaido, 060-8543
Akune Citizen Hospital
Kagoshima, 899-1611
Tohoku University Hospital
Miyagi, 980-8574
Nihon University Itabashi Hospital
Tokyo, 173-8610
Nihon University Nerima Hikarigaoka Hospital
Tokyo, 179-0072
Yamanashi Prefectural Central Hospital
Yamanashi, 400-8506
About this study
Official title: A Multicenter Open-label Study of the Safety and Efficacy of α-galactosidase A (R-h α-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease
This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry disease.
Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.
- Conditions
- Fabry Disease
- Being tested
- Agalsidase beta
- Keywords
- cardiac fabry disease
- 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 betaDrug
Also called Fabrazyme®
| Study group | Role | What they receive |
|---|---|---|
| Agalsidase Beta | Experimental | Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks. Receives: Drug: Agalsidase beta |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Measured: Baseline to Week 156
Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Measured: Baseline to Week 156
Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156
Left ventricular mass was assessed by echocardiogram.
Measured: Baseline to Week 156
Change From Baseline in LVM at Week 156
Left ventricular mass was assessed by echocardiogram.
Measured: Baseline to Week 156
Secondary outcomes (3)
Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test
Overall cardiac function assessment was assessed by tests (echocardiogram,cardiac catheterization (optional),electrocardiogram,B-type natriuretic peptide [BNP]), clinical symptoms (subjective symptoms) and the New York Heart Association (NYHA) cardiac functional classification.Overall assessment of cardiac function was assessed based on the evaluation items including interventricular septum thickness, left ventricular posterior wall thickness, left ventricular mass, clinical function tests and clinical symptoms. A subject was considered to be Improved: if Improved in 2 items or more, Unchanged: Improved in one item and unchanged in 2 items or unchanged in all 3 items, Aggravated: Aggravated in one item or more.
Measured: Baseline to Week 156
Percent Change From Baseline in GL-3 Plasma Levels at Week 156
Measured: Baseline to Week 156
Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156
The 36-Item Short-Form Health Survey (SF-36) is a standardized survey evaluating 8 aspects of functional health and well-being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. These 8 aspects can also be summarized as physical component score (PCS) and mental component score (MCS). The score for a section is an average of the individual question scores, which are scaled 0-100 (100=highest level of functioning).
Measured: Baseline to Week 156
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2005 (estimated)
- Study startJul 2005
- Primary completionAug 2012
- Study completionAug 2012
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
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00140621
- Sponsor's protocol ID
- AGAL03204
- Status last verified
- Apr 2015
- First submitted
- 30 Aug 2005
- Last updated
- 12 May 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Open-label Study of the Safety and Efficacy of α-galactosidase A (R-h α-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease
- ClinicalTrials.gov ID
- NCT00140621
- Acronym / sponsor ID
- AGAL03204
- Conditions
- Fabry Disease
- Keywords
- cardiac fabry disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Jul 2005
- Primary completion
- Aug 2012 (actual)
- Study completion
- Aug 2012 (actual)
- First posted
- 1 Sep 2005 (estimated)
- Results
- Posted 15 Apr 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry disease.
Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Agalsidase Beta | Experimental | Drug: Agalsidase betaAgalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks. |
Who may qualify
- Ages
- 20 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Participants definitively diagnosed with cardiac Fabry disease (who fulfill all of the following criteria)
- Participants with interventricular and posterior wall thickness of at least 13 millimeter (mm) on echocardiography within 3 months before signed date to informed consent
- Participants in whom cardiac function was rated as Class I or II according to the New York Heart Association (NYHA) classification when giving informed consent.
- Participants classification: inpatients and outpatients
- Participants who had given written informed consent before the study-related baseline tests.
Key exclusion criteria (11 listed)
- Participants with severe hypertension (for example, blood pressure more than or equal to 180 millimeter of mercury [mmHg] and/or blood pressure more than or equal to 110 mmHg in spite of adequate medication)
- Participants whose serum creatinine level was higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent.
- Participants who had undergone kidney transplantation or were currently on dialysis.
- Participants with any serious hepatic disorder. Participants who had abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either alanine aminotransferase [ALT] or aspartate aminotransferase [AST] level exceeded the value five times as high as the upper normal limit).
- Permanent pacemaker or defibrillator implanted participants
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® | Drug | Agalsidase Beta |
Primary outcome
Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Time frame: Baseline to Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Time frame: Baseline to Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156
Time frame: Baseline to Week 156
Left ventricular mass was assessed by echocardiogram.
Change From Baseline in LVM at Week 156
Time frame: Baseline to Week 156
Left ventricular mass was assessed by echocardiogram.
3 secondary outcomes
Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test
Time frame: Baseline to Week 156
Percent Change From Baseline in GL-3 Plasma Levels at Week 156
Time frame: Baseline to Week 156
Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156
Time frame: Baseline to Week 156
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 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
7 sites in 1 country: 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 7 sites in Japan.
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 10 inclusion and 11 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 12 May 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Japan. 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 15 Apr 2015). 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?
Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 (time frame: Baseline to Week 156). The study lists 4 primary outcomes in all. 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 12 May 2015.