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Prednisone or Dexamethasone in Treating Patients With Newly Diagnosed, Previously Untreated Primary Immune Thrombocytopenic Purpura (ITP0207)

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2014 by Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor:
Information provided by (Responsible Party):
Gruppo Italiano Malattie EMatologiche dell'Adulto
ClinicalTrials.gov Identifier:
NCT00657410
First received: April 11, 2008
Last updated: November 20, 2014
Last verified: November 2014
  Purpose

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.


Condition Intervention Phase
Nonneoplastic Condition
Drug: dexamethasone
Drug: prednisone
Procedure: quality-of-life assessment
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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.

Resource links provided by NLM:


Further study details as provided by Gruppo Italiano Malattie EMatologiche dell'Adulto:

Primary Outcome Measures:
  • Final response (complete, partial, and minimal response) rate from evaluation of initial response [ Time Frame: At day +180 from evaluation of initial response ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Initial response rate [ Time Frame: At day 42 (arm I), at day 46 (arm II) ] [ Designated as safety issue: No ]
  • Quality of response per arm [ Time Frame: At initial evaluation and at final evaluation ] [ Designated as safety issue: No ]
  • Final response rate [ Time Frame: At day 180 from the statement of initial response ] [ Designated as safety issue: No ]
  • Rate of bleeding events [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: Yes ]
  • 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 Frame: At day 42 or before day 180 from the first evaluation ] [ Designated as safety issue: No ]
  • Time to platelet number increase until a hemostatically effective level is reached and/or disappearance of bleeding symptoms [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: No ]
  • Rate of persistent response [ Time Frame: At 12 months from the statement of initial response ] [ Designated as safety issue: No ]
  • Association of type of initial response with final and persistent response (in patients with final and persistent response) [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: No ]
  • Rate of rescue interventions [ Time Frame: After day 180 from evaluation of initial response ] [ Designated as safety issue: No ]
  • Rate of splenectomy eligible patients [ Time Frame: At 12 months from enrollment ] [ Designated as safety issue: No ]
  • Rate of patients who have undergone splenectomy during follow-up [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: No ]
  • Rate of patients who develop connective tissue diseases or underlying hematological diseases (myelodysplastic syndromes, chronic lymphoproliferative diseases, others) during follow-up [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: No ]

Estimated Enrollment: 150
Study Start Date: April 2008
Estimated Study Completion Date: March 2015
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ARM A - PDN
PDN is administered orally at the daily dose of 1 mg/Kg for 4 consecutive weeks (from day 0 to day 28), then, therapy is tapered within 14 days. The patients considered NOT RESPONDER at day 42 or WHO HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be crossed to ARM B.
Drug: prednisone Procedure: quality-of-life assessment
Experimental: ARM B - DXM

DXM is administered orally at single fixed daily doses of 40 mg for 4 consecutive days, every 14 days, for 3 consecutive courses. If platelet count is £ 20x109/L or bleeding symptoms related to thrombocytopenia are present, lowdose DXM (0.035 mg/Kg/day) between courses is given. The patients (either from ARM A+B or from ARM B) considered NOT RESPONDER at day 46 or who HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be considered OFF TREATMENT.

For these patients a second line therapy will be considered, according to the medical practice of the Centre (splenectomy or other).

Drug: dexamethasone Procedure: quality-of-life assessment

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

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00657410

Locations
Italy
U.O. di Ematologia - Azienda Ospedaliera - Pia Fondazione di Culto e di Religione Card. G.Panico Recruiting
Tricase, (le), Italy
Contact: Vincenzo PAVONE, Pr.       salentoematologia@piafondazionepanico.it   
Principal Investigator: Vincenzo PAVONE, Pr.         
S.O.C. di Ematologia - Azienda Ospedaliera - SS. Antonio e Biagio e Cesare Arrigo Recruiting
Alessandria, Italy
Contact: Roberto Santi       rsanti@ospedale.al.it   
Principal Investigator: Roberto Santi         
Azienda ospedaliera Nuovo Ospedale "Torrette" Recruiting
Ancona, Italy
Contact: Pietro Leoni       pleoni@univpm.it   
Principal Investigator: Pietro Leoni, Pr.         
USL 8 - Ospedale S.Donato Recruiting
Arezzo, Italy
Contact: Antonio Benci, Dr.       ematolar@virgilio.it   
Principal Investigator: Antonio Benci, Dr.         
Dipartimento Area Medica - Presidio Ospedaliero "C. e G.Mazzoni" Recruiting
Ascoli, Italy
Contact: Pero Galieni, Dr.       piero.galieni@sanita.marche.it   
Principal Investigator: Piero Galieni, Dr.         
UO Ematologia con trapianto- AOU Policlinico Consorziale di Bari Recruiting
Bari, Italy
Contact: Rita RIZZI, dr       ritarizzi@ematba.uniba.it   
Principal Investigator: Rita RIZZI, Dr.         
Ospedali Riuniti di Bergamo Recruiting
Bergamo, Italy, 24100
Contact: Alessandro Rambaldi, MD    39-35-269491      
Principal Investigator: Alessandro RAMBALDI, Pr.         
University of Bologna Medical School Recruiting
Bologna, Italy, 40138
Contact: Nicola VIANELLI, Dr.       nicola.vianelli@unibo.it   
Principal Investigator: Nicola Vianelli, Dr.         
Sub-Investigator: Riccardo Ragionieri, Dr.         
Sezione di Ematologia e Trapianti Spedali Civili Recruiting
Brescia, Italy, 21125
Contact: Domenico Russo       russo@med.unibs.it   
Principal Investigator: Domenico Russo, Pr.         
Sub-Investigator: Cecilia Carbone         
Struttura Complessa di Oncologia Medica - Azienda Ospedaliera - Ospedale di Circolo di Busto Arsizio Recruiting
Busto Arsizio, Italy
Contact: Marco Bregni, Dr.       mbregni@aobusto.it   
Principal Investigator: Marco Bregni, Dr.         
Marche U.O. di Medicina Interna - ASUR Marche 8 - Ospedale Civile Recruiting
Civitanova - Marche, Italy
Contact: Riccardo CENTURIONI, Dr.       ricentu@tin.it   
Principal Investigator: Riccardo CENTURIONI, Dr.         
Sub-Investigator: Rosanna RE, Dr.         
U.O. Ematologia - P.O. Annunziata - A.O. di Cosenza Recruiting
Cosenza, Italy
Contact: Fortunato MORABITO, Dr.       fortunato_morabito@tin.it   
Principal Investigator: Fortunato MORABITO, Dr.         
Sub-Investigator: Brigida GULINO, Dr.         
Ospedale Maggiore - Div.Medicina Crema Recruiting
Crema, Italy
Contact: Maria Cristina Pasquini, Dr.       vincri@hotmail.com   
Principal Investigator: Maria Cristina Pasquini, Dr.         
Struttura Complessa di Ematologia Ospedali Riuniti Foggia - Azienda Ospedaliero-Universitaria Recruiting
Foggia, Italy
Contact: Silvana Franca Capalbo, Dr.       scapalbo@ospedaliriunitifoggia.it   
Principal Investigator: Silvana Franca Capalbo, Dr.         
Ospedale Santa Maria Goretti Recruiting
Latina, Italy, 04100
Contact: Giuseppe CIMINO, Pr.       cimino@bce.uniroma1.it   
Principal Investigator: Giuseppe CIMINO, Pr.         
ASL Le1 P.O. Vito Fazzi - U.O. di Ematologia Recruiting
Lecce, Italy, 73100
Contact: Nicola DI RENZO       direnzo.ematolecce@libero.it   
Principal Investigator: Nicola DI RENZO, Dr.         
Sub-Investigator: Claudia QUINTAVALLE, Dr.         
Istituto Scientifico Romagnoli per lo Studio e la Cura dei Tumori- IRST Recruiting
Meldola, Italy
Contact: Dino Amadori, Dr.       sergio.amadori@ptvonline.it   
Principal Investigator: Dino Amadori, Dr.         
Azienda Ospedaliera Universitaria - Policlinico G. Martino Dipartimento di Medicina Interna - U.O. Messina Recruiting
Messina, Italy
Contact: Caterina Musolino, Pr.       cmusolino@unime.it   
Principal Investigator: Caterina Musolino, Pr.         
Sub-Investigator: Alessandro Allegra, Dr.         
Azienda Ospedaliera San Paolo - Unità di Ematologia e Trombosi Recruiting
Milano, Italy
Contact: Marco Cattaneo, Dr.       marco.cattaneo@unimi.it   
Principal Investigator: Marco Cattaneo, Dr.         
S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro Recruiting
Novara, Italy
Contact: Gianluca GAIDANO, Dr.       gaidano@med.unipmn.it   
Contact: Monia LUNGHI, Dr.         
Principal Investigator: Gianluca GAIDANO, Pr.         
Sub-Investigator: Monia LUNGHI, Dr.         
Divisione di Ematologia con trapianto di midollo - A.U. Policlinico "Paolo Giaccone" Recruiting
Palermo, Italy
Contact: Gerlando Quintini, Dr.       gerlando.quintini@libero.it   
Principal Investigator: Gerlando Quintini, Dr.         
Cattedra di Ematologia CTMO Università degli Studi di Parma Recruiting
Parma, Italy
Contact: Cecilia CARAMATTI, Dr.       cecilia.caramatti@unipr.it   
Principal Investigator: Cecilia CARAMATTI, Dr.         
Sub-Investigator: Elena ROSETTI, Dr.         
Med. Int. ed Oncologia Medica IRCCS Policlinico S. Matteo Recruiting
Pavia, Italy, 27100
Contact: Patrizia NORIS       p.noris@smatteo.pv.it   
Principal Investigator: Patrizia NORIS, Dr.         
U.O. Ematologia Clinica - Azienda USL di Pescara Recruiting
Pescara, Italy
Contact: Paolo Di Bartolomeo, Dr.       pescaratmo@virgilio.it   
Principal Investigator: Paolo Di Bartolomeo, Dr.         
Sub-Investigator: Antonella Sau         
Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale di Piacenza Recruiting
Piacenza, Italy
Contact: Daniele VALLISA, Dr.       d.vallisa@ausl.pc.it   
Principal Investigator: Daniele VALLISA, Dr.         
Sub-Investigator: Elena TRABACCHI, Dr.         
Pordenone Unità operativa Medicina II Az. Osp. S. M. degli Angeli Recruiting
Pordenone, Italy
Contact: Luigi VIRGOLINI, Pr.       luigi.virgolini@aopn.fvg.it   
Principal Investigator: Luigi VIRGOLINI, Dr.         
Sub-Investigator: Anna Ermacora, Dr.         
Dipartimento Oncologico - Ospedale S.Maria delle Croci Recruiting
Ravenna, Italy
Contact: Alfonso Zaccaria, Pr.       a.zaccaria@ausl.ra.it   
Principal Investigator: Alfonso Zaccaria, Pr.         
Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli" Recruiting
Reggio Calabria, Italy
Contact: Francesco NOBILE, Pr.       f.nobile@netonline.it   
Principal Investigator: NOBILE, Pr.         
Sub-Investigator: Donatella VINCELLI, Dr.         
Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova Recruiting
Reggio Emilia, Italy
Contact: Barbara Gamberi       barbara.gamberi@asmn.re.it   
Principal Investigator: Barbara Gamberi         
Sub-Investigator: Katia Codeluppi         
Ospedale "Infermi" Recruiting
Rimini, Italy
Contact: Patrizia Tosi, Dr.       patrizia.tosi@auslrn.net   
Principal Investigator: Patrizia Tosi, Dr.         
Az. Ospedaliera "Sant' Andrea"-Università la Sapienza Seconda Facoltà di Medicina e Chirurgia Recruiting
Roma, Italy
Contact: Bruno MONARCA, Pr.       b.monarca@libero.it   
Principal Investigator: Bruno MONARCA, Pr.         
Sub-Investigator: Antonella FERRARI, Dr.         
Divisione di Ematologia - Ospedale S. Camillo Recruiting
Roma, Italy
Contact: Ignazio MAJOLINO, Pr.       imajolino@scamilloforlanini.rm.it   
Principal Investigator: Ignazio MAJOLINO, Pr.         
Divisione Ematologia - Università Campus Bio-Medico Recruiting
Roma, Italy
Contact: Giuseppe Avvisati       g.avvisati@unicampus.it   
Principal Investigator: Giuseppe Avvisati         
Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore Recruiting
Rome, Italy, 00168
Contact: Giuseppe Leone, Pr.       gleone@rm.unicatt.it   
Principal Investigator: Giuseppe Leone, Pr.         
UOC Pronto Soccorso e Accettazione Ematologica - Dipartimento Biotecnologie Cellulari ed Ematologia - Università degli Studi di Roma "Sapienza" Recruiting
Rome, Italy, 00161
Contact: Maria Gabriella MAZZUCCONI, Pr.    39-06-4424-1984    mazzucconi@bce.uniroma.it   
Principal Investigator: Maria Gabriella MAZZUCCONI, Pr.         
Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza Recruiting
San Giovanni Rotondo, Italy
Contact: Nicola CASCAVILLA, Pr.       n.cascavilla@operapadrepio.it   
Principal Investigator: Nicola CASCAVILLA, Pr.         
U.O.C. Ematologia e Trapianti - A.O. Senese - Policlinico " Le Scotte" Recruiting
Siena, Italy
Contact: Monica Bocchia, Pr.       bocchia@unisi.it   
Principal Investigator: Monica Bocchia, Pr.         
Sub-Investigator: Marzia Defina, Dr.         
Divisione di Ematologia dell' Università degli Studi di Torino - "Città della Salute e della Scienza di Torino" Recruiting
Torino, Italy
Contact: Piercarla Schinco       pcschinco@hototmail.com   
Principal Investigator: Piercarla Schinco         
Struttura Complessa II Medicina - Ematologia - Centro di Riferimento Ematologico - Ospedale Maggiore Recruiting
Trieste, Italy
Contact: Giorgio Paladini, Pr.       giorgio.paladini@aots.sanita.fvg.it   
Principal Investigator: Giorgio Paladini, Pr.         
Sub-Investigator: Stefano BONETTO, Dr.         
Clinica Ematologica - Policlinico Universitario Recruiting
Udine, Italy
Contact: Francesco Zaja, Pr.       zaja.francesco@aoud.sanita.fvg.it   
Principal Investigator: Francesco Zaja, Pr.         
Policlinico G. B. Rossi - Borgo Roma Recruiting
Verona, Italy, 37134
Contact: Giovanni Pizzolo, MD       giovanni.pizzolo@univr.it   
Principal Investigator: Giovanni PIZZOLO, Pr.         
Sub-Investigator: Dino DE VENERI, Dr.         
Ospedale San Bortolo Recruiting
Vicenza, Italy, 36100
Contact: Francesco RODEGHIERO, Pr.       rodeghiero@hemato.ven.it   
Principal Investigator: Cicenzo RODEGHIERO, Pr.         
Sub-Investigator: Stefania Fortuna, Dr.         
Sponsors and Collaborators
Gruppo Italiano Malattie EMatologiche dell'Adulto
Investigators
Principal Investigator: Maria Gabriella Mazzucconi, MD Gruppo Italiano Malattie EMatologiche dell'Adulto
  More Information

No publications provided

Responsible Party: Gruppo Italiano Malattie EMatologiche dell'Adulto
ClinicalTrials.gov Identifier: NCT00657410     History of Changes
Other Study ID Numbers: ITP0207, GIMEMA-ITP-0207, Eudract 2008-000417-30, EU-20839
Study First Received: April 11, 2008
Last Updated: November 20, 2014
Health Authority: Italy: Ethics Committee

Keywords provided by Gruppo Italiano Malattie EMatologiche dell'Adulto:
idiopathic thrombocytopenic purpura

Additional relevant MeSH terms:
Blood Platelet Disorders
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Autoimmune Diseases
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Hemorrhagic Disorders
Immune System Diseases
Pathologic Processes
Signs and Symptoms
Skin Manifestations
Thrombocytopenia
Thrombotic Microangiopathies
BB 1101
Dexamethasone
Dexamethasone 21-phosphate
Dexamethasone acetate
Prednisone
Anti-Inflammatory Agents
Antiemetics
Antineoplastic Agents
Antineoplastic Agents, Hormonal
Autonomic Agents
Central Nervous System Agents
Enzyme Inhibitors
Gastrointestinal Agents
Glucocorticoids
Hormones

ClinicalTrials.gov processed this record on November 27, 2014