Prednisone or Dexamethasone in Treating Patients With Newly Diagnosed, Previously Untreated Primary Immune Thrombocytopenic Purpura
Information source: Gruppo Italiano Malattie EMatologiche dell'Adulto
Information obtained from ClinicalTrials.gov on February 07, 2013 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Nonneoplastic Condition
Intervention: dexamethasone (Drug); prednisone (Drug); quality-of-life assessment (Procedure)
Phase: Phase 3
Status: Recruiting
Sponsored by: Gruppo Italiano Malattie EMatologiche dell'Adulto Official(s) and/or principal investigator(s): Maria Gabriella Mazzucconi, MD, Principal Investigator, Affiliation: Gruppo Italiano Malattie EMatologiche dell'Adulto
Summary
RATIONALE: Drugs, such as prednisone and dexamethasone, may change the immune system and be
an effective treatment for primary immune thrombocytopenic purpura. It is not yet known
which drug is more effective in treating primary immune thrombocytopenic purpura.
PURPOSE: This randomized phase III trial is studying high-dose dexamethasone to see how well
it works compared to standard-dose prednisone in treating patients with newly diagnosed,
previously untreated primary immune thrombocytopenic purpura.
Clinical Details
Official title: Randomized Study of the Treatment of Primary Immune Thrombocytopenic Purpura (ITP) in Newly Diagnosed Untreated Adult Patients. Comparison of Standard Dose Prednisone Versus High-dose Dexamethasone.
Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Final response (complete, partial, and minimal response) rate from evaluation of initial response
Secondary outcome: Initial response rateQuality of response per arm Final response rate Rate of bleeding events Resumed response rate in non-responder patients (at day 42) or patients who have lost response before day 180 from the first evaluation (arm I only) Time to platelet number increase until a hemostatically effective level is reached and/or disappearance of bleeding symptoms Rate of persistent response Association of type of initial response with final and persistent response (in patients with final and persistent response) Rate of rescue interventions Rate of splenectomy eligible patients Rate of patients who have undergone splenectomy during follow-up Rate of patients who develop connective tissue diseases or underlying hematological diseases (myelodysplastic syndromes, chronic lymphoproliferative diseases, others) during follow-up
Detailed description:
OBJECTIVES:
Primary
- To evaluate the role of therapy intensification in adult patients with newly diagnosed,
previously untreated primary immune thrombocytopenic purpura with high-dose
dexamethasone (HD-DXM), in terms of improvement of response at 6 months after initial
response, in comparison with standard-doses of prednisone.
Secondary
- Compare rate of initial response.
- Compare quality of response.
- Compare rate of final responses and rate of persistent response.
- Compare rate of bleeding events.
- Determine rate of resumed response with HD-DXM in non-responder patients or patients
who have lost response (arm I only).
- Compare time to platelet number increase until a hemostatically effective level is
reached and/or disappearance of bleeding symptoms.
- Compare rate of rescue interventions.
- Compare rate of eligible patients for splenectomy.
- Compare rate of patients who underwent splenectomy.
- Compare rate of patients who develop connective tissue diseases or underlying
hematological diseases (myelodysplastic syndromes, chronic lymphoproliferative
diseases, others).
- Compare patient's self reported quality of life.
OUTLINE: This is a multicenter study. Patients are stratified by treating center. Patients
are randomized to 1 of 2 treatment arms.
- Arm I (Standard-dose prednisone): Patients receive oral prednisone at a standard dose
(1 mg/Kg) once daily on days 1-28 followed by a 14-day taper.
Patients considered non-responders at day 42 or who have lost response before evaluation of
final response (day 180) are crossed to arm II.
- Arm II (High-dose dexamethasone): Patients receive oral dexamethasone at a high dose
(40 mg/day) once daily on days 1-4. Treatment repeats every 14 days for 3 courses.
Quality of life is assessed at baseline, on day 42 (arm I) or 46 (arm II) (initial response
evaluation day), 180 days after initial response evaluation, and at 3, 9, 12 months after
randomization.
After completion of study treatment, patients are followed monthly until 1 year after
randomization, every 2 months for 1 year, and then every 3 months for 1 year.
Eligibility
Minimum age: 18 Years.
Maximum age: 80 Years.
Gender(s): Both.
Criteria:
Inclusion criteria
- Signed written informed consent according to IGH/EU/GCP and national local laws
- Newly diagnosed untreated ITP adult patients
- Age > 18 < 80 years
- Platelet count <20x109/L
- Platelet count > 20 x109/L and <50x109/L plus bleeding with score > 8 (according to
grading scale at paragraph 7. 1)
- Baseline Quality of Life evaluation questionnaire filled in Newly diagnosed untreated
ITP adult patients
- Age > 18 < 80 years
- Platelet count <20x109/L
- Platelet count > 20 x109/L and <50x109/L plus bleeding with score > 8 (according to
grading scale at paragraph 7. 1)
- Baseline Quality of Life evaluation questionnaire filled in
Exclusion criteria
- Active malignancy at time of study entry
- Steroids administration (PDN <1mg/Kg/day) for more than 5 days before randomization
- Concomitant treatment with anti-platelet and or anti-coagulant drugs
- Concomitant severe psychiatric disorders
- Not confirmed diagnosis of ITP for
- *Positivity of autoimmunity markers: antinucleus (≥1: 80), anti-tireoglobulin,
anti-tireoperoxidase, anti-cardiolipin (≥ 40 GPL UmL), anti-b2glycoprotein (≥ 40
IgG U/mL) antibodies, Lupus Anticoagulant (KCT ratio, dRVVT ratios ≥1. 5 times
the upper normal limit ), direct antiglobulin test (DAT ).
- Presence of autoimmune hemolytic anemia
- Presence of connective tissue disease
- Women who are pregnant or breastfeeding
- Cardiovascular diseases requiring treatment
- Severe non-controlled, despite therapy, hypertension and diabetes
- Liver and kidney function impairment (creatinine, ALT, AST >2 times upper normal
limit)
- HCVAb, HIVAb, HBsAg, HBcAb seropositive status
- Chronic liver disease
- Documented viral illness by the positivity of IgM, or vaccination both occurred one
month before diagnosis
- Intake of drugs not previously taken within one week before diagnosis
- Bleeding score 15 due to ICH or to GI bleeding (according to grading scale at
paragraph 7. 1, Tab. 3)
- Active gastric ulcer.
Locations and Contacts
Azienda ospedaliera Nuovo Ospedale "Torrette", Ancona, Italy; Recruiting Pietro Leoni, Email: pleoni@univpm.it Pietro Leoni, Pr., Principal Investigator
USL 8 - Ospedale S.Donato, Arezzo, Italy; Recruiting Antonio Benci, Dr., Email: ematolar@virgilio.it Antonio Benci, Dr., Principal Investigator
UO Ematologia con trapianto- AOU Policlinico Consorziale di Bari, Bari, Italy; Recruiting Rita RIZZI, dr, Email: ritarizzi@ematba.uniba.it Rita RIZZI, Dr., Principal Investigator
Ospedali Riuniti di Bergamo, Bergamo 24100, Italy; Recruiting Alessandro Rambaldi, MD, Phone: 39-35-269491 Alessandro RAMBALDI, Pr., Principal Investigator
University of Bologna Medical School, Bologna 40138, Italy; Recruiting Nicola VIANELLI, Dr., Email: nicola.vianelli@unibo.it Nicola Vianelli, Dr., Principal Investigator Riccardo Ragionieri, Dr., Sub-Investigator
Sezione di Ematologia e Trapianti Spedali Civili, Brescia 21125, Italy; Recruiting Giuseppe ROSSI, Email: rossig@med.unibs.it Giuseppe ROSSI, Pr., Principal Investigator
Struttura Complessa di Oncologia Medica - Azienda Ospedaliera - Ospedale di Circolo di Busto Arsizio, Busto Arsizio, Italy; Recruiting Marco Bregni, Dr., Email: mbregni@aobusto.it Marco Bregni, Dr., Principal Investigator
Marche U.O. di Medicina Interna - ASUR Marche 8 - Ospedale Civile, Civitanova - Marche, Italy; Recruiting Riccardo CENTURIONI, Dr., Email: ricentu@tin.it Riccardo CENTURIONI, Dr., Principal Investigator Rosanna RE, Dr., Sub-Investigator
U.O. Ematologia - P.O. Annunziata - A.O. di Cosenza, Cosenza, Italy; Recruiting Fortunato MORABITO, Dr., Email: fortunato_morabito@tin.it Fortunato MORABITO, Dr., Principal Investigator Brigida GULINO, Dr., Sub-Investigator
Ospedale Santa Maria Goretti, Latina 04100, Italy; Recruiting Giuseppe CIMINO, Pr., Email: cimino@bce.uniroma1.it Giuseppe CIMINO, Pr., Principal Investigator
ASL Le1 P.O. Vito Fazzi - U.O. di Ematologia, Lecce 73100, Italy; Recruiting Nicola DI RENZO, Email: direnzo.ematolecce@libero.it Nicola DI RENZO, Dr., Principal Investigator Claudia QUINTAVALLE, Dr., Sub-Investigator
Azienda Ospedaliera Universitaria - Policlinico G. Martino Dipartimento di Medicina Interna - U.O. Messina, Messina, Italy; Recruiting Caterina Musolino, Pr., Email: cmusolino@unime.it Caterina Musolino, Pr., Principal Investigator Alessandro Allegra, Dr., Sub-Investigator
S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro, Novara, Italy; Recruiting Gianluca GAIDANO, Dr., Email: gaidano@med.unipmn.it Monia LUNGHI, Dr. Gianluca GAIDANO, Pr., Principal Investigator Monia LUNGHI, Dr., Sub-Investigator
Divisione di Ematologia con trapianto di midollo - A.U. Policlinico "Paolo Giaccone", Palermo, Italy; Recruiting Gerlando Quintini, Dr., Email: gerlando.quintini@libero.it Gerlando Quintini, Dr., Principal Investigator
Cattedra di Ematologia CTMO Università degli Studi di Parma, Parma, Italy; Recruiting Cecilia CARAMATTI, Dr., Email: cecilia.caramatti@unipr.it Cecilia CARAMATTI, Dr., Principal Investigator Elena ROSETTI, Dr., Sub-Investigator
Med. Int. ed Oncologia Medica IRCCS Policlinico S. Matteo, Pavia 27100, Italy; Recruiting Patrizia NORIS, Email: p.noris@smatteo.pv.it Patrizia NORIS, Dr., Principal Investigator
U.O. Ematologia Clinica - Azienda USL di Pescara, Pescara, Italy; Recruiting Giuseppe Fioritoni, Dr., Email: gfioritoni@tin.it Giuseppe Fioritoni, Dr., Principal Investigator
Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale di Piacenza, Piacenza, Italy; Recruiting Daniele VALLISA, Dr., Email: d.vallisa@ausl.pc.it Daniele VALLISA, Dr., Principal Investigator Elena TRABACCHI, Dr., Sub-Investigator
Pordenone Unità operativa Medicina II Az. Osp. S. M. degli Angeli, Pordenone, Italy; Recruiting Luigi VIRGOLINI, Pr., Email: luigi.virgolini@aopn.fvg.it Luigi VIRGOLINI, Dr., Principal Investigator Anna Ermacora, Dr., Sub-Investigator
Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli", Reggio Calabria, Italy; Recruiting Francesco NOBILE, Pr., Email: f.nobile@netonline.it NOBILE, Pr., Principal Investigator Donatella VINCELLI, Dr., Sub-Investigator
Ospedale "Infermi", Rimini, Italy; Recruiting Patrizia Tosi, Dr., Email: patrizia.tosi@auslrn.net Patrizia Tosi, Dr., Principal Investigator
Divisione di Ematologia - Ospedale S. Camillo, Roma, Italy; Recruiting Ignazio MAJOLINO, Pr., Email: imajolino@scamilloforlanini.rm.it Ignazio MAJOLINO, Pr., Principal Investigator
Az. Ospedaliera "Sant' Andrea"-Università la Sapienza Seconda Facoltà di Medicina e Chirurgia, Roma, Italy; Recruiting Bruno MONARCA, Pr., Email: b.monarca@libero.it Bruno MONARCA, Pr., Principal Investigator Antonella FERRARI, Dr., Sub-Investigator
UOC Pronto Soccorso e Accettazione Ematologica - Dipartimento Biotecnologie Cellulari ed Ematologia - Università degli Studi di Roma "Sapienza", Rome 00161, Italy; Recruiting Maria Gabriella MAZZUCCONI, Pr., Phone: 39-06-4424-1984, Email: mazzucconi@bce.uniroma.it Maria Gabriella MAZZUCCONI, Pr., Principal Investigator
Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore, Rome 00168, Italy; Recruiting Giuseppe Leone, Pr., Email: gleone@rm.unicatt.it Giuseppe Leone, Pr., Principal Investigator
Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy; Recruiting Nicola CASCAVILLA, Pr., Email: n.cascavilla@operapadrepio.it Nicola CASCAVILLA, Pr., Principal Investigator
U.O.C. Ematologia e Trapianti - A.O. Senese - Policlinico " Le Scotte", Siena, Italy; Recruiting Francesco LAURIA, Pr., Email: lauria@unisi.it Francesco LAURIA, Pr., Principal Investigator Marzia Defina, Dr., Sub-Investigator
Struttura Complessa II Medicina - Ematologia - Centro di Riferimento Ematologico - Ospedale Maggiore, Trieste, Italy; Recruiting Giorgio Paladini, Pr., Email: giorgio.paladini@aots.sanita.fvg.it Giorgio Paladini, Pr., Principal Investigator Stefano BONETTO, Dr., Sub-Investigator
Policlinico G. B. Rossi - Borgo Roma, Verona 37134, Italy; Recruiting Giovanni Pizzolo, MD, Email: giovanni.pizzolo@univr.it Giovanni PIZZOLO, Pr., Principal Investigator Dino DE VENERI, Dr., Sub-Investigator
Ospedale San Bortolo, Vicenza 36100, Italy; Recruiting Francesco RODEGHIERO, Pr., Email: rodeghiero@hemato.ven.it Cicenzo RODEGHIERO, Pr., Principal Investigator Stefania Fortuna, Dr., Sub-Investigator
U.O. di Ematologia - Azienda Ospedaliera - Pia Fondazione di Culto e di Religione Card. G.Panico, Tricase, (le), Italy; Recruiting Vincenzo PAVONE, Pr., Email: salentoematologia@piafondazionepanico.it Vincenzo PAVONE, Pr., Principal Investigator
Additional Information
Starting date: April 2008
Last updated: June 6, 2012
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