Fabry Disease Clinical Trial
— SWITCHOfficial 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
NCT number | NCT01268241 |
Other study ID # | SW02/2010 |
Secondary ID | |
Status | Completed |
Phase | |
First received | |
Last updated | |
Start date | December 2010 |
Est. completion date | April 2016 |
Verified date | April 2020 |
Source | CENTOGENE GmbH Rostock |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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®.
Status | Completed |
Enrollment | 200 |
Est. completion date | April 2016 |
Est. primary completion date | April 2016 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
Argentina | Juan Fernandez Hospital, Department of Neurology | Buenos Aires | |
Argentina | Unidad Renal Corrientes SRL, Medicina Interna Nefrólogo | Corrientes | |
Belgium | UZA - University Ziekenhuis Antwerpen) | Edegem | |
Croatia | University Hospital "Sestre Milosrdnice" Department of neuroimmunology and neurogenetic | Zagreb | |
Czechia | Miroslava Hajkova, 2nd Dept of Cardiology&Angiology, Fakultni poliklinika | Prague 2 | |
Denmark | National University Hosoital Rigshospitalet, Endokrinologisk ward | Copenhagen | |
France | Université de Versailles - Saint Quentin en YvelinesService de Génétique Médicale | Paris | |
Germany | Kinderklinik München-Schwabing Städt. Klinikum GmbH | Munich | |
United Kingdom | Royal Free Hospital, Dep. of Academic Haematology, Lysosomal Storage Disorders Unit | London |
Lead Sponsor | Collaborator |
---|---|
CENTOGENE GmbH Rostock |
Argentina, Belgium, Croatia, Czechia, Denmark, France, Germany, United Kingdom,
Banikazemi M, Bultas J, Waldek S, Wilcox WR, Whitley CB, McDonald M, Finkel R, Packman S, Bichet DG, Warnock DG, Desnick RJ; Fabry Disease Clinical Trial Study Group. Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Ann Intern Med. 2007 Jan 16;146(2):77-86. Epub 2006 Dec 18. — View Citation
Banikazemi M, Ullman T, Desnick RJ. Gastrointestinal manifestations of Fabry disease: clinical response to enzyme replacement therapy. Mol Genet Metab. 2005 Aug;85(4):255-9. — View Citation
Beck M, Ricci R, Widmer U, Dehout F, de Lorenzo AG, Kampmann C, Linhart A, Sunder-Plassmann G, Houge G, Ramaswami U, Gal A, Mehta A. Fabry disease: overall effects of agalsidase alfa treatment. Eur J Clin Invest. 2004 Dec;34(12):838-44. — View Citation
Eng CM, Banikazemi M, Gordon RE, Goldman M, Phelps R, Kim L, Gass A, Winston J, Dikman S, Fallon JT, Brodie S, Stacy CB, Mehta D, Parsons R, Norton K, O'Callaghan M, Desnick RJ. A phase 1/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studies. Am J Hum Genet. 2001 Mar;68(3):711-22. Epub 2001 Feb 1. — View Citation
Hajioff D, Goodwin S, Quiney R, Zuckerman J, MacDermot KD, Mehta A. Hearing improvement in patients with Fabry disease treated with agalsidase alfa. Acta Paediatr Suppl. 2003 Dec;92(443):28-30; discussion 27. — View Citation
Hoffmann B, Garcia de Lorenzo A, Mehta A, Beck M, Widmer U, Ricci R; FOS European Investigators. Effects of enzyme replacement therapy on pain and health related quality of life in patients with Fabry disease: data from FOS (Fabry Outcome Survey). J Med Genet. 2005 Mar;42(3):247-52. — View Citation
Hoffmann B, Reinhardt D, Koletzko B. Effect of enzyme-replacement therapy on gastrointestinal symptoms in Fabry disease. Eur J Gastroenterol Hepatol. 2004 Oct;16(10):1067-9. — View Citation
Kint JA. Fabry's disease: alpha-galactosidase deficiency. Science. 1970 Feb 27;167(3922):1268-9. — View Citation
MacDermot KD, Holmes A, Miners AH. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males. J Med Genet. 2001 Nov;38(11):750-60. — View Citation
Mehta A, Ricci R, Widmer U, Dehout F, Garcia de Lorenzo A, Kampmann C, Linhart A, Sunder-Plassmann G, Ries M, Beck M. Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey. Eur J Clin Invest. 2004 Mar;34(3):236-42. — View Citation
Moore DF, Altarescu G, Herscovitch P, Schiffmann R. Enzyme replacement reverses abnormal cerebrovascular responses in Fabry disease. BMC Neurol. 2002 Jun 18;2:4. — View Citation
Ramaswami U, Wendt S, Pintos-Morell G, Parini R, Whybra C, Leon Leal JA, Santus F, Beck M. Enzyme replacement therapy with agalsidase alfa in children with Fabry disease. Acta Paediatr. 2007 Jan;96(1):122-7. — View Citation
Ries M, Clarke JT, Whybra C, Timmons M, Robinson C, Schlaggar BL, Pastores G, Lien YH, Kampmann C, Brady RO, Beck M, Schiffmann R. Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease. Pediatrics. 2006 Sep;118(3):924-32. — View Citation
Rolfs A, Böttcher T, Zschiesche M, Morris P, Winchester B, Bauer P, Walter U, Mix E, Löhr M, Harzer K, Strauss U, Pahnke J, Grossmann A, Benecke R. Prevalence of Fabry disease in patients with cryptogenic stroke: a prospective study. Lancet. 2005 Nov 19;366(9499):1794-6. Erratum in: Lancet. 2006 Dec 23;368(9554):2210. — View Citation
Rolfs A, Martus P, Heuschmann PU, Grittner U, Holzhausen M, Tatlisumak T, Böttcher T, Fazekas F, Enzinger C, Ropele S, Schmidt R, Riess O, Norrving B; sifap1 Investigators. Protocol and methodology of the Stroke in Young Fabry Patients (sifap1) study: a prospective multicenter European study of 5,024 young stroke patients aged 18-55 years. Cerebrovasc Dis. 2011;31(3):253-62. doi: 10.1159/000322153. Epub 2010 Dec 21. — View Citation
Schiffmann R, Floeter MK, Dambrosia JM, Gupta S, Moore DF, Sharabi Y, Khurana RK, Brady RO. Enzyme replacement therapy improves peripheral nerve and sweat function in Fabry disease. Muscle Nerve. 2003 Dec;28(6):703-10. — View Citation
Schiffmann R, Kopp JB, Austin HA 3rd, Sabnis S, Moore DF, Weibel T, Balow JE, Brady RO. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA. 2001 Jun 6;285(21):2743-9. — View Citation
* Note: There are 17 references in all — Click here to view all references
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---|---|---|---|---|
Primary | Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease | 24 month |
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