The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease (SWITCH)
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Purpose
The current approved treatment for Fabry disease is enzyme replacement therapy (ERT). There are actually 2 products in this therapeutic class available: Replagal® (agalsidase alfa) and Fabrazyme® (agalsidase beta). Both are indicated for long-term treatment in patients with a confirmed diagnosis of Fabry disease (alfa-galactosidase A deficiency). Both have been commercially available in Europe for almost 10 years, yet little information is available about the clinical and safety profile of patients who switch from one therapy to the other. An extended shortage of Fabrazyme® that began in June 2009 has necessitated that a large number of patients switch from Fabrazyme® to Replagal®. This offers the possibility to study the clinical status and adverse events in patients who switch from Fabrazyme® to Replagal® on a large-scale basis. In addition, as a result of the increasing Fabrazyme® shortage, many of these patients received a reduced dosage of Fabrazyme® for an extended period before transitioning to treatment with Replagal®.
| Condition |
|---|
|
Fabry Disease Fabry´s Disease Anderson-Fabry Disease |
| Study Type: | Observational |
| Study Design: | Observational Model: Cohort |
| Official Title: | International Observational Retrospective Case Review of Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Males and Females With Anderson-Fabry Disease |
Plasma and urine GB3 and lyso-GB3 and agalsidase antibodies will be analyzed in central laboratories, there is consent for extra blood samples to be taken (at the time of the switch and after 6, 12 and 24 months)
| Estimated Enrollment: | 200 |
| Study Start Date: | November 2010 |
| Estimated Study Completion Date: | June 2013 |
| Estimated Primary Completion Date: | May 2013 (Final data collection date for primary outcome measure) |
| Groups/Cohorts |
|---|
|
Observation
Hemizygous male or heterozygous female patients of any age with genetically confirmed diagnosis of Anderson-Fabry disease.
|
Detailed Description:
Aim:
Anderson-Fabry disease is an X-linked lysosomal storage disorder resulting from deficiency of the hydrolytic enzyme alfa galactosidase A. Trials of specific therapy by replacement of alfa galactosidase A were commenced in 1999 and subsequently two preparations of alfa galactosidase A received marketing approval by the EMEA in 2001. Clinical trials, observational studies and registry data have provided evidence for efficacy of enzyme replacement therapy (ERT) with alfa galactosidase A in improving symptoms of pain, gastrointestinal disturbance, hypohidrosis, left ventricular mass index, glomerular filtration rate and quality of life in men. There is currently no long-term data showing the impact of enzyme replacement therapy on overall survival. It has been suggested that earlier therapy, before the onset of end organ manifestations, would be more likely to prevent further damage and therefore have the biggest effect on overall survival. There is as yet little evidence to substantiate this hypothesis however clinical trials have recently demonstrated safety and therapeutic effects of enzyme replacement in children.
So far, there are only limited data available on the clinical course of the disease and adverse events in patients, switching from one therapeutic alternative to the other. West and Lemoine (16) report clinical effects of a switch from Agalsidase beta to agalsidase alfa in 5 patients with Fabry disease due to shortage of agalsidase beta. The patients were treated with Replagal® for 44 weeks at an average.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Probability Sample |
Hemizygous male or heterozygous female patients at 18 years with genetically confirmed diagnosis of Anderson-Fabry disease
Inclusion Criteria:
- Hemizygous male or heterozygous female patients at 18 years with genetically confirmed diagnosis of Anderson-Fabry disease.
- Written informed consent
- Patient had received Fabrazyme® for at least 12 months prior to starting treatment with Replagal® in full dose (i.e. 1.0 mg/kg eow) or any reduced dose prescribed by the treating physician due to the shortage of the medication
- Patient has received or is receiving treatment commercially available Replagal® (0.2 mg/kg eow) for intravenous (IV) infusion prescribed by their treatment physician and administered in accordance with the Replagal® prescribing information.
- The switch of the medication from Fabrazyme® to Replagal® had to be taken place from September 2009 onwards at the earliest
- Patient data includes disease history, measures of Fabry related disease and safety measures
Exclusion Criteria:
- Concomitant use of Fabrazyme®
- Any switch of medication from Fabrazyme® to Replagal® before September 2009
- Any switch from Fabrazyme® to Replagal® for other reasons than Fabrazyme® shortage
- Patient has received treatment with any investigational drug or device within the 30 days prior to study entry
- No written informed consent
Contacts and Locations| Contact: Arndt Rolfs, MD | 49-381-494 ext 9540 | arndt.rolfs@med.uni-rostock.de |
| Contact: Kristin Bruederlein | 49-381-494 ext 4737 | kristin.bruederlein@med.uni-rostock.de |
| Argentina | |
| Juan Fernandez Hospital, Department of Neurology | Recruiting |
| Buenos Aires, Argentina, Cerviño 3356 | |
| Contact: Juan Politei, MD +54 11 480 82600 jpolitei@hotmail.com | |
| Sub-Investigator: Juan Politei, MD | |
| Unidad Renal Corrientes SRL, Medicina Interna Nefrólogo | Recruiting |
| Corrientes, Argentina, CP N°3400 | |
| Contact: Luis Rolando Urtiaga, MD 54-3783-15603369 urtiaga55@hotmail.com | |
| Sub-Investigator: Carlos Martin Cedrolla, MD | |
| Belgium | |
| UZA - University Ziekenhuis Antwerpen) | Recruiting |
| Edegem, Belgium, 2650 | |
| Contact: François Eyskens, MD +32 3821 ext 5745 francois.eyskens@pcma.provant.be | |
| Principal Investigator: François Eyskens, Prof. MD | |
| Croatia | |
| University Hospital "Sestre Milosrdnice" Department of neuroimmunology and neurogenetic | Recruiting |
| Zagreb, Croatia, 10000 | |
| Contact: Vanja Basic Kes, Prof. MD +385 1 3768282 vanjakes@net.hr | |
| Sub-Investigator: Iris Zavoreo, MD | |
| Czech Republic | |
| Miroslava Hajkova, 2nd Dept of Cardiology&Angiology, Fakultni poliklinika | Recruiting |
| Prague 2, Czech Republic, 12800 | |
| Contact: Lubor Golan, MD +42 0224966711 lubor.golan@seznam.cz | |
| Principal Investigator: Lubor Golan, MD | |
| Denmark | |
| National University Hosoital Rigshospitalet, Endokrinologisk ward | Recruiting |
| Copenhagen, Denmark, 2100 | |
| Contact: Ulla Feldt Rasmussen, Prof MD +45 35451023 ufeldt@rh.dk | |
| Principal Investigator: Ulla Feldt Rasmussen, Prof. MD | |
| France | |
| Université de Versailles - Saint Quentin en YvelinesService de Génétique Médicale | Recruiting |
| Paris - Garches, France, 92380 | |
| Contact: Dominique Germain, Prof. MD + 33 1 47 10 44 38 dominique.germain@rpc.aphp.fr | |
| Sub-Investigator: Karelle Benistan, MD | |
| Sub-Investigator: Nadege Laovar, MD | |
| Germany | |
| Kinderklinik München-Schwabing Städt. Klinikum GmbH | Recruiting |
| Munich, Germany, 80804 | |
| Contact: Jochen Baumkoetter, MD +49 89 3068 3378 jochen.baumkoetter@lrz.tum.de | |
| Sub-Investigator: Jochen Baumkoetter, MD | |
| United Kingdom | |
| Royal Free Hospital, Dep. of Academic Haematology, Lysosomal Storage Disorders Unit | Recruiting |
| London, United Kingdom, NW3 2QG | |
| Contact: Atul Mehta,, Prof. MD +44 207 8302814 atul.mehta1@nhs.net | |
| Principal Investigator: Derralynn Hughes, MD | |
| Principal Investigator: | Arndt Rolfs, MD | University of Rostock, Albrecht-Kossel-Institute for Neuroregeneration |
More Information
Publications:
| Responsible Party: | Prof. Dr. Arndt Rolfs, Prof. Dr. med., University of Rostock |
| ClinicalTrials.gov Identifier: | NCT01268241 History of Changes |
| Other Study ID Numbers: | SW02/2010 |
| Study First Received: | December 28, 2010 |
| Last Updated: | March 15, 2013 |
| Health Authority: | Germany: Ethics Commission |
Keywords provided by University of Rostock:
|
Fabry Disease Fabry´s Disease Anderson-Fabry Disease |
Additional relevant MeSH terms:
|
Fabry Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |
ClinicalTrials.gov processed this record on June 18, 2013