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Sponsored by: |
Assistance Publique - Hôpitaux de Paris |
Information provided by: | Assistance Publique - Hôpitaux de Paris |
ClinicalTrials.gov Identifier: | NCT00426686 |
Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy defined by the spontaneous formation of platelet thrombi in the microvessels. These platelet microthrombi are responsible for a mechanical hemolytic anemia, a thrombocytopenia and a multivisceral ischemia. TTP is a rare but life-threatening disease in the absence of appropriate treatment (plasmatherapy). The onset of the disease usually occurs in adulthood (Moschcovitz syndrome) and rarely in childhood (Upshaw-Schulman syndrome). TTP is either sporadic or recurrent with multiple unpredictable relapses. TTP pathophysiology has remained obscure until a new metalloprotease, ADAMTS13, has been demonstrated to be involved in about 90% of all cases. Physiologically, ADAMTS13 function consists in limiting the size of von Willebrand factor (VWF) multimers and consequently, their hemostatic capacity. A large majority of TTP is associated with a severe deficiency of ADAMTS13. In most cases, ADAMTS13 severe deficiency is acquired via auto-antibodies to ADAMTS13; more rarely, ADAMTS13 deficiency is hereditary via ADAMTS13 gene mutations. ADAMTS13 auto-antibodies are either inhibitory of the catalytic activity or non inhibitory. ADAMTS13 mutations are spread all over the gene.
TTP prognosis is quite heterogeneous. Indeed, in about one third of the patients, TTP is refractory to plasmatherapy and/or chronic relapsing. Until now, TTP prognosis factors are not known. Their identification is however crucial both to adapt the curative treatment of an acute episode (addition of first intention immunosuppressive agents to plasmatherapy) and to prevent relapses.
In this context, the aim of the current project is to identify some ADAMTS13 related prognosis factors in TTP. A national prospective multicenter study including both adult and pediatric patients with TTP related to a severe ADAMTS13 deficiency will be designed over a three-year period. This study will involve our group as the French reference center for ADAMTS13 and 10 clinical departments from various French hospitals. Patients will be tested for ADAMTS13 activity and antigen, ADAMTS13 antibodies and ADAMTS13 gene sequencing. Our main hypothesis is that the inactivation of the ADAMTS13 domains crucial for its catalytic activity, either by inhibitory auto-antibodies (acquired TTP) or by genetic mutations (hereditary TTP) is a major bad prognosis factor.
Condition |
Thrombotic Thrombocytopenic Purpura |
Genetics Home Reference related topics: | factor V Leiden thrombophilia hemophilia thrombotic thrombocytopenic purpura |
Study Type: | Observational |
Study Design: | Case-Only, Other |
Official Title: | ADAMTS13-Related Prognostic Factors in Adult and Pediatric Thrombotic Thrombocytopenic Purpura |
March 2010
Estimated Enrollment: | 150 |
Study Start Date: | November 2006 |
Estimated Study Completion Date: | March 2010 |
Estimated Primary Completion Date: | March 2010 (Final data collection date for primary outcome measure) |
Show Detailed Description |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | Yes |
Sampling Method: | Non-Probability Sample |
Adult and pediatric patients with TTP related to a severe ADAMTS13 deficiency.
Inclusion Criteria:
Exclusion Criteria:
Contact: Agnes B VEYRADIER, MD, PhD | 33(0)145374305 | agnes.veyradier@abc.aphp.fr |
France | |||||
Hôpital Antoine Béclère | Recruiting | ||||
Clamart, France, 92140 | |||||
Contact: Agnès B VEYRADIER, MD, PhD 33 (0)145374305 agnes.veyradier@abc.aphp.fr |
Assistance Publique - Hôpitaux de Paris |
Principal Investigator: | paul COPPO, MD, PhD | Hôpital Saint Antoire, PARIS |
Study Director: | Elie AZOULAY, MD, PhD | Hôpital Saint Louis, PARIS |
Study Director: | Benoît SCHLEMMER, MD | Hôpital Saint Louis, PARIS |
Study Director: | Eric OKSENHENDLER, MD | Hôpital Saint Louis, PARIS |
Study Director: | Fadi FAKHOURI, MD | Hôpital Necker, PARIS |
Study Director: | Jean-Paul MIRA, MD, PhD | Hôpital Cochin, PARIS |
Study Director: | Eric RONDEAU, MD | Hôpital Tenon, PARIS |
Study Director: | Jean-paul VERNANT, MD | Hôpital la Pitié Salpétrière, PARIS |
Study Director: | Nicolas SCHLEINITZ, MD | CHU Conception, MARSEILLE |
Study Director: | Gilles KAPLANSKI, MD, PhD | CHU Conception, MARSEILLE |
Study Director: | Albert BENSMAN, MD | Hôpital Trousseau, PARIS |
Study Director: | Chantal LOIRAT, MD | Hôpital Robert Debré, PARIS |
Study Director: | Brigitte BADER-MEUNIER, MD | Hôpital Robert Debré, PARIS |
Study Director: | Christophe PIGUET, MD | Hôpital Dupuytren, LIMOGES |
Study Director: | Guy PUTET, MD | Hospices civils de LYON, LYON |
Study Director: | Béatrice DUCOT, MD | INSERM U569 Kremlin Bicêtre, Paris |
Study Director: | Agnes VEYRADIER, MD, PhD | Hôpital Antoine Béclère, CLAMART |
Study Director: | Thierry LEBLANC, MD | Hôpital Saint-Louis, PARIS |
Study Director: | Patrick NIAUDET, MD, PhD | Hôpital Necker, PARIS |
Study Director: | Christophe RIDEL, MD | Hôpital Tenon, PARIS |
Study Director: | Pascale POULLIN, MD | CHU de la Conception, MARSEILLE |
Study Director: | arlos FRANGIE, MD | Hôpital Bicêtre, LE KREMLIN BICETRE |
Study Director: | Hélène FRANCOIS, MD | Hôpital Bicêtre, LE KREMLIN BICETRE |
Study Director: | Olivier LAMBOTTE, MD | Hôpital Bicêtre, LE KREMLIN BICETRE |
Study Director: | Dominique BORDESSOULE, MD, PhD | Hôpital Dupytren, LIMOGES |
Study Director: | Stéphane GIRAULT, MD | Hôpital Dupytren, LIMOGES |
Study Director: | Hervé CHAMBOST, MD | Hôpital de la Timone Enfants, Marseilles |
Study Director: | Pierre BORDOGONI, MD, PhD | Hôpital de Brabois-Hôpital d'Enfants, Vandoeuvre-lès-Nancy |
Study Director: | Alexandra SALMON, MD | Hôpital de Brabois- hôpital d'enfants, Vandoeuvre-lès-Nancy |
Study Director: | Laurence CLEMENT, MD | Hôpital de Brabois-Hôpital d'enfants, Vandoeuvre-lès-Nancy |
Study Director: | Christian COMBE, MD, PhD | Hôpital Pellegrin, Bordeaux |
Study Director: | Sandrine MEUNIER, MD | Hôpital Edouard Heriot, Lyon |
Study Director: | Gwenaêlle ROUSSEY, MD | Hôpital Hotel Dieu, Nantes |
Study Director: | Mohammed HAMIDOU, MD, PhD | Hôpital Hotel Dieu, Nantes |
Study Director: | Bernard BONNOTTE, MD, PhD | Hôpital du Bocage, Dijon |
Study Director: | Yves TANTER, MD | Hôpital du Bocage, Dijon |
Study Director: | Jacques POURRAT, MD, PhD | Hôpital de Rangueil, Toulouse |
Study Director: | Marie-Christine THOURET, MD | CHU de l'Archet 2, Nice |
Study Director: | Philippe VANHILLE, MD | CH de Valenciennes, Valenciennes |
Study Director: | Nicolas LIMAL, MD | Hôpital Henri Mondor, Créteil |
Study Director: | Philippe REMY, MD | Hôpital Henri Mondor, Créteil |
Study Director: | Jean-Michel KORACH, MD | CHG Châlons-en-Champagne, Châlons-en-Champagne |
Study Director: | Carine GREIB, MD | Hôpital Haut-Lévêque, Pesac |
Study Director: | Jean-Louis PALLOT, MD | CHI André Grégoire, Montreuil |
Study Director: | Alain WYNCKEL, MD | CHU de Reims, Reims |
Study Director: | Claire CAZALETS, MD | Hôpital Sud, Rennes |
Study Director: | Bertrand DE CAGNY, MD | CHU d'Amiens, Amiens |
Study Director: | Claire PRESNE, MD | CHU D'Amiens, Amiens |
Study Director: | Cécile FOHRER, MD | Hôpital de Haute-Pierre, Strasbourg |
Study Director: | Karin BILGER, MD | Hôpital de Haute-Pierre, Strasbourg |
Study Director: | Bruno LIOURE, MD | Hôpital de Haute-Pierre, Strasbourg |
Study Director: | Raoul HERBRECHT, MD, PhD | Hôpital de Haute-Pierre, Strasbourg |
Study Director: | Dominique PLANTAZ, MD, PhD | Hôpital Nord, Grenoble |
Study Director: | Hubert NIVET, MD, PhD | Hôpital Gatien de Clovheville, Tours |
Study Director: | Emmanuel FLECK, MD | Hôpital Saint Louis, La Rochelle |
Responsible Party: | Clinical Research Delegation ( Zakia IDIR ) |
Study ID Numbers: | PO51064, AOM-05012, 31-06 (Ethical Committee) |
First Received: | January 24, 2007 |
Last Updated: | April 28, 2008 |
ClinicalTrials.gov Identifier: | NCT00426686 |
Health Authority: | France: Ministry of Health |
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