Fabry DiseaseNCT00233870
A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.
An observational study of Fabry Disease, run by Genzyme, a Sanofi Company at 1 site in Japan.
Trial summary
The purpose of this survey is to identify any concerns regarding the following efficacy and safety-related issues in clinical practice with the new drugs "Fabrazyme for intravenous infusion 5mg" and "Fabrazyme for intravenous infusion 35mg" and to confirm the safety of these products in long-term use in the clinical… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Agalsidase beta (recombinant form)
- Type of treatment
- Injection or infusion
- How many people
- 405 participants
- Where
- 1 siteJapan
- Study dates
- Jun 2004to Mar 2011
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Japanese patients with Fabry Disease
Who may be able to join
- Patients in Japan with the indication of "Fabry Disease" and for whom the usual dosage and administration is 1mg of agalsidase beta (recombinant) per 1 kg body weight each time, administered by intravenous infusion every 2 weeks
- Because the efficacy evaluation of enzyme replacement therapy with Fabrazyme [agalsidase beta (recombinant form)] will require the comparison of findings before and after the start of enzyme replacement therapy, the efficacy evaluation set will be defined as including patients using Fabrazyme [agalsidase beta (recombinant form)] for the first time in the post-marketing setting and those for whom it is possible to obtain retrospective data for before the start of enzyme replacement therapy.
Who cannot join
- Patients registered in the post-marketing trials during the post-marketing clinical trial period.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Special Survey in Long-Term Use of Fabrazyme
The purpose of this survey is to identify any concerns regarding the following efficacy and safety-related issues in clinical practice with the new drugs "Fabrazyme for intravenous infusion 5mg" and "Fabrazyme for intravenous infusion 35mg" and to confirm the safety of these products in long-term use in the clinical setting.
- New adverse drug reactions (ADRs) that cannot be predicted from the Precautions (in particular, clinically significant ADRs)
- The incidence of ADRs under the actual conditions of use of the drug
- Causal factors that might potentially affect safety
- Efficacy evaluation in long-term use
Read more
This survey will be conducted in accordance with the approval condition established for Fabrazyme:
"To conduct a special surveillance of Efficacy and Safety in long term treatment and Pediatric with the drug."
Read the detailed description
Medical institutions or physicians will be asked to periodically complete the survey forms for all patients registered. Survey forms include baseline information available, and then data collected every 6 months, as available including: demographic information, concomitant medications/therapy, treatment record, ECG, Echocardiogram, computed tomography scan / magnetic resonance imaging (CT/MRI), Fabry symptoms, labs, functional disorder, blood concentration of GL-3, and anti-agalsidase beta antibody test (IgE testing) to survey whether the productions of antibodies to agalsidase beta is a causal factor of treatment-related reactions.
The survey period shall be approximately 7 years from June 1, 2004 during which survey shall be undertaken as follows:
- The observation period for each patient shall range from 1 to about 7 years after starting treatment
- Registration period: June 1, 2004 to March 31, 2010
- Survey period: June 1, 2004 to March 31, 2011
In institutions for which retrospective surveys are feasible, the survey period will trace back to the date of approval (January 29, 2004), as far as possible.
- Conditions
- Fabry Disease
- Being tested
- Agalsidase beta (recombinant form)
- 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 beta (recombinant form)Drug
Also called Fabrazyme
agalsidase beta 1.0 mg/kg body weight infused every 2 weeks as an intravenous infusion
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in blood GL-3 level
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Oct 2005 (estimated)
- Study startJun 2004
- Primary completionMar 2011
- Study completionMar 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 405 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
- NCT00233870
- Sponsor's protocol ID
- AGAL03004
- Status last verified
- May 2015
- First submitted
- 5 Oct 2005
- Last updated
- 8 May 2015
- Results posted
- No
Publications
Lee CS, Tsurumi M, Eto Y. Safety and tolerability of agalsidase beta infusions shorter than 90 min in patients with Fabry disease: post-hoc analysis of a Japanese post-marketing study. Orphanet J Rare Dis. 2023 Jul 24;18(1):209. doi: 10.1186/s13023-023-02803-5.
PubMed 37488580Tsurumi M, Suzuki S, Hokugo J, Ueda K. Long-term safety and efficacy of agalsidase beta in Japanese patients with Fabry disease: aggregate data from two post-authorization safety studies. Expert Opin Drug Saf. 2021 May;20(5):589-601. doi: 10.1080/14740338.2021.1891221. Epub 2021 Mar 10.
PubMed 33599146
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Special Survey in Long-Term Use of Fabrazyme
- ClinicalTrials.gov ID
- NCT00233870
- Acronym / sponsor ID
- AGAL03004
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 405 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Jun 2004
- Primary completion
- Mar 2011 (actual)
- Study completion
- Mar 2011 (actual)
- First posted
- 6 Oct 2005 (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.
The purpose of this survey is to identify any concerns regarding the following efficacy and safety-related issues in clinical practice with the new drugs "Fabrazyme for intravenous infusion 5mg" and "Fabrazyme for intravenous infusion 35mg" and to confirm the safety of these products in long-term use in the clinical setting.
- New adverse drug reactions (ADRs) that cannot be predicted from the Precautions (in particular, clinically significant ADRs)
- The incidence of ADRs under the actual conditions of use of the drug
- Causal factors that might potentially affect safety
- Efficacy evaluation in long-term use
This survey will be conducted in accordance with the approval condition established for Fabrazyme:
"To conduct a special surveillance of Efficacy and Safety in long term treatment and Pediatric with the drug."
Detailed description
Medical institutions or physicians will be asked to periodically complete the survey forms for all patients registered. Survey forms include baseline information available, and then data collected every 6 months, as available including: demographic information, concomitant medications/therapy, treatment record, ECG, Echocardiogram, computed tomography scan / magnetic resonance imaging (CT/MRI), Fabry symptoms, labs, functional disorder, blood concentration of GL-3, and anti-agalsidase beta antibody test (IgE testing) to survey whether the productions of antibodies to agalsidase beta is a causal factor of treatment-related reactions.
The survey period shall be approximately 7 years from June 1, 2004 during which survey shall be undertaken as follows:
- The observation period for each patient shall range from 1 to about 7 years after starting treatment
- Registration period: June 1, 2004 to March 31, 2010
- Survey period: June 1, 2004 to March 31, 2011
In institutions for which retrospective surveys are feasible, the survey period will trace back to the date of approval (January 29, 2004), as far as possible.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Japanese patients with Fabry Disease
Key inclusion criteria (2 listed)
- Patients in Japan with the indication of "Fabry Disease" and for whom the usual dosage and administration is 1mg of agalsidase beta (recombinant) per 1 kg body weight each time, administered by intravenous infusion every 2 weeks
- Because the efficacy evaluation of enzyme replacement therapy with Fabrazyme [agalsidase beta (recombinant form)] will require the comparison of findings before and after the start of enzyme replacement therapy, the efficacy evaluation set will be defined as including patients using Fabrazyme [agalsidase beta (recombinant form)] for the first time in the post-marketing setting and those for whom it is possible to obtain retrospective data for before the start of enzyme replacement therapy.
Key exclusion criteria (1 listed)
- Patients registered in the post-marketing trials during the post-marketing clinical trial period.
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 beta (recombinant form)Also: Fabrazymeagalsidase beta 1.0 mg/kg body weight infused every 2 weeks as an intravenous infusion | Drug | – |
Primary outcome
Change in blood GL-3 level
Time frame: 6 months
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 8 May 2015.
- Derived from this study: PubMed 37488580Lee CS, Tsurumi M, Eto Y. Safety and tolerability of agalsidase beta infusions shorter than 90 min in patients with Fabry disease: post-hoc analysis of a Japanese post-marketing study. Orphanet J Rare Dis. 2023 Jul 24;18(1):209. doi: 10.1186/s13023-023-02803-5.
- Derived from this study: PubMed 33599146Tsurumi M, Suzuki S, Hokugo J, Ueda K. Long-term safety and efficacy of agalsidase beta in Japanese patients with Fabry disease: aggregate data from two post-authorization safety studies. Expert Opin Drug Saf. 2021 May;20(5):589-601. doi: 10.1080/14740338.2021.1891221. Epub 2021 Mar 10.
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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?
An observational study of Fabry Disease, run by Genzyme, a Sanofi Company at 1 site 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 2 inclusion and 1 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 8 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 1 site 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.
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?
Change in blood GL-3 level (time frame: 6 months). 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 NCT00233870, which also lists 2 linked publications.
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 8 May 2015.