Prednisone or Dexamethasone in Newly Diagnosed, Previously Untreated Primary Immune Thrombocytopenic Purpura (ITP0207)
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|ClinicalTrials.gov Identifier: NCT00657410|
Recruitment Status : Completed
First Posted : April 14, 2008
Last Update Posted : February 20, 2017
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 or disease||Intervention/treatment||Phase|
|Nonneoplastic Condition||Drug: dexamethasone Drug: prednisone Procedure: quality-of-life assessment||Phase 3|
- 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.
- 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||150 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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 Start Date :||April 2008|
|Actual Primary Completion Date :||February 2016|
|Actual Study Completion Date :||February 2016|
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|
- Final response (complete, partial, and minimal response) rate from evaluation of initial response [ Time Frame: At day +180 from evaluation of initial response ]
- Initial response rate [ Time Frame: At day 42 (arm I), at day 46 (arm II) ]
- Quality of response per arm [ Time Frame: At initial evaluation and at final evaluation ]
- Final response rate [ Time Frame: At day 180 from the statement of initial response ]
- Rate of bleeding events [ Time Frame: At 3 years from study entry ]
- 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 ]
- 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 ]
- Rate of persistent response [ Time Frame: At 12 months from the statement of initial response ]
- 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 ]
- Rate of rescue interventions [ Time Frame: After day 180 from evaluation of initial response ]
- Rate of splenectomy eligible patients [ Time Frame: At 12 months from enrollment ]
- Rate of patients who have undergone splenectomy during follow-up [ Time Frame: At 3 years from study entry ]
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00657410
|U.O. di Ematologia - Azienda Ospedaliera - Pia Fondazione di Culto e di Religione Card. G.Panico|
|Tricase, (le), Italy|
|S.O.C. di Ematologia - Azienda Ospedaliera - SS. Antonio e Biagio e Cesare Arrigo|
|Azienda ospedaliera Nuovo Ospedale "Torrette"|
|USL 8 - Ospedale S.Donato|
|Dipartimento Area Medica - Presidio Ospedaliero "C. e G.Mazzoni"|
|UO Ematologia con trapianto- AOU Policlinico Consorziale di Bari|
|Ospedali Riuniti di Bergamo|
|Bergamo, Italy, 24100|
|University of Bologna Medical School|
|Bologna, Italy, 40138|
|Sezione di Ematologia e Trapianti Spedali Civili|
|Brescia, Italy, 21125|
|Struttura Complessa di Oncologia Medica - Azienda Ospedaliera - Ospedale di Circolo di Busto Arsizio|
|Busto Arsizio, Italy|
|Marche U.O. di Medicina Interna - ASUR Marche 8 - Ospedale Civile|
|Civitanova - Marche, Italy|
|U.O. Ematologia - P.O. Annunziata - A.O. di Cosenza|
|Ospedale Maggiore - Div.Medicina Crema|
|Struttura Complessa di Ematologia Ospedali Riuniti Foggia - Azienda Ospedaliero-Universitaria|
|Ospedale Santa Maria Goretti|
|Latina, Italy, 04100|
|ASL Le1 P.O. Vito Fazzi - U.O. di Ematologia|
|Lecce, Italy, 73100|
|Istituto Scientifico Romagnoli per lo Studio e la Cura dei Tumori- IRST|
|Azienda Ospedaliera Universitaria - Policlinico G. Martino Dipartimento di Medicina Interna - U.O. Messina|
|Azienda Ospedaliera San Paolo - Unità di Ematologia e Trombosi|
|S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro|
|Divisione di Ematologia con trapianto di midollo - A.U. Policlinico "Paolo Giaccone"|
|Cattedra di Ematologia CTMO Università degli Studi di Parma|
|Med. Int. ed Oncologia Medica IRCCS Policlinico S. Matteo|
|Pavia, Italy, 27100|
|U.O. Ematologia Clinica - Azienda USL di Pescara|
|Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale di Piacenza|
|Pordenone Unità operativa Medicina II Az. Osp. S. M. degli Angeli|
|Dipartimento Oncologico - Ospedale S.Maria delle Croci|
|Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli"|
|Reggio Calabria, Italy|
|Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova|
|Reggio Emilia, Italy|
|Az. Ospedaliera "Sant' Andrea"-Università la Sapienza Seconda Facoltà di Medicina e Chirurgia|
|Divisione di Ematologia - Ospedale S. Camillo|
|Divisione Ematologia - Università Campus Bio-Medico|
|UOC Pronto Soccorso e Accettazione Ematologica - Dipartimento Biotecnologie Cellulari ed Ematologia - Università degli Studi di Roma "Sapienza"|
|Rome, Italy, 00161|
|Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore|
|Rome, Italy, 00168|
|Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza|
|San Giovanni Rotondo, Italy|
|U.O.C. Ematologia e Trapianti - A.O. Senese - Policlinico " Le Scotte"|
|Divisione di Ematologia dell' Università degli Studi di Torino - "Città della Salute e della Scienza di Torino"|
|Struttura Complessa II Medicina - Ematologia - Centro di Riferimento Ematologico - Ospedale Maggiore|
|Clinica Ematologica - Policlinico Universitario|
|Policlinico G. B. Rossi - Borgo Roma|
|Verona, Italy, 37134|
|Ospedale San Bortolo|
|Vicenza, Italy, 36100|
|Principal Investigator:||Maria Gabriella Mazzucconi, MD||Gruppo Italiano Malattie EMatologiche dell'Adulto|