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

Primary Purpose

Nonneoplastic Condition

Status
Completed
Phase
Phase 3
Locations
Italy
Study Type
Interventional
Intervention
dexamethasone
prednisone
quality-of-life assessment
Sponsored by
Gruppo Italiano Malattie EMatologiche dell'Adulto
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Nonneoplastic Condition focused on measuring idiopathic thrombocytopenic purpura

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • U.O. di Ematologia - Azienda Ospedaliera - Pia Fondazione di Culto e di Religione Card. G.Panico
  • 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
  • University of Bologna Medical School
  • Sezione di Ematologia e Trapianti Spedali Civili
  • Struttura Complessa di Oncologia Medica - Azienda Ospedaliera - Ospedale di Circolo di Busto Arsizio
  • Marche U.O. di Medicina Interna - ASUR Marche 8 - Ospedale Civile
  • 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
  • ASL Le1 P.O. Vito Fazzi - U.O. di Ematologia
  • 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
  • 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"
  • Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova
  • Ospedale "Infermi"
  • 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"
  • Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore
  • Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
  • 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
  • Ospedale San Bortolo

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

ARM A - PDN

ARM B - DXM

Arm Description

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.

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).

Outcomes

Primary Outcome Measures

Final response (complete, partial, and minimal response) rate from evaluation of initial response

Secondary Outcome Measures

Initial response rate
Quality 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

Full Information

First Posted
April 11, 2008
Last Updated
February 17, 2017
Sponsor
Gruppo Italiano Malattie EMatologiche dell'Adulto
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1. Study Identification

Unique Protocol Identification Number
NCT00657410
Brief Title
Prednisone or Dexamethasone in Newly Diagnosed, Previously Untreated Primary Immune Thrombocytopenic Purpura
Acronym
ITP0207
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 Type
Interventional

2. Study Status

Record Verification Date
February 2017
Overall Recruitment Status
Completed
Study Start Date
April 2008 (undefined)
Primary Completion Date
February 2016 (Actual)
Study Completion Date
February 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Gruppo Italiano Malattie EMatologiche dell'Adulto

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Nonneoplastic Condition
Keywords
idiopathic thrombocytopenic purpura

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
150 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ARM A - PDN
Arm Type
Experimental
Arm Description
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.
Arm Title
ARM B - DXM
Arm Type
Experimental
Arm Description
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).
Intervention Type
Drug
Intervention Name(s)
dexamethasone
Intervention Type
Drug
Intervention Name(s)
prednisone
Intervention Type
Procedure
Intervention Name(s)
quality-of-life assessment
Primary Outcome Measure Information:
Title
Final response (complete, partial, and minimal response) rate from evaluation of initial response
Time Frame
At day +180 from evaluation of initial response
Secondary Outcome Measure Information:
Title
Initial response rate
Time Frame
At day 42 (arm I), at day 46 (arm II)
Title
Quality of response per arm
Time Frame
At initial evaluation and at final evaluation
Title
Final response rate
Time Frame
At day 180 from the statement of initial response
Title
Rate of bleeding events
Time Frame
At 3 years from study entry
Title
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
Title
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
Title
Rate of persistent response
Time Frame
At 12 months from the statement of initial response
Title
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
Title
Rate of rescue interventions
Time Frame
After day 180 from evaluation of initial response
Title
Rate of splenectomy eligible patients
Time Frame
At 12 months from enrollment
Title
Rate of patients who have undergone splenectomy during follow-up
Time Frame
At 3 years from study entry
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Maria Gabriella Mazzucconi, MD
Organizational Affiliation
Gruppo Italiano Malattie EMatologiche dell'Adulto
Official's Role
Principal Investigator
Facility Information:
Facility Name
U.O. di Ematologia - Azienda Ospedaliera - Pia Fondazione di Culto e di Religione Card. G.Panico
City
Tricase
State/Province
(le)
Country
Italy
Facility Name
S.O.C. di Ematologia - Azienda Ospedaliera - SS. Antonio e Biagio e Cesare Arrigo
City
Alessandria
Country
Italy
Facility Name
Azienda ospedaliera Nuovo Ospedale "Torrette"
City
Ancona
Country
Italy
Facility Name
USL 8 - Ospedale S.Donato
City
Arezzo
Country
Italy
Facility Name
Dipartimento Area Medica - Presidio Ospedaliero "C. e G.Mazzoni"
City
Ascoli
Country
Italy
Facility Name
UO Ematologia con trapianto- AOU Policlinico Consorziale di Bari
City
Bari
Country
Italy
Facility Name
Ospedali Riuniti di Bergamo
City
Bergamo
ZIP/Postal Code
24100
Country
Italy
Facility Name
University of Bologna Medical School
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Sezione di Ematologia e Trapianti Spedali Civili
City
Brescia
ZIP/Postal Code
21125
Country
Italy
Facility Name
Struttura Complessa di Oncologia Medica - Azienda Ospedaliera - Ospedale di Circolo di Busto Arsizio
City
Busto Arsizio
Country
Italy
Facility Name
Marche U.O. di Medicina Interna - ASUR Marche 8 - Ospedale Civile
City
Civitanova - Marche
Country
Italy
Facility Name
U.O. Ematologia - P.O. Annunziata - A.O. di Cosenza
City
Cosenza
Country
Italy
Facility Name
Ospedale Maggiore - Div.Medicina Crema
City
Crema
Country
Italy
Facility Name
Struttura Complessa di Ematologia Ospedali Riuniti Foggia - Azienda Ospedaliero-Universitaria
City
Foggia
Country
Italy
Facility Name
Ospedale Santa Maria Goretti
City
Latina
ZIP/Postal Code
04100
Country
Italy
Facility Name
ASL Le1 P.O. Vito Fazzi - U.O. di Ematologia
City
Lecce
ZIP/Postal Code
73100
Country
Italy
Facility Name
Istituto Scientifico Romagnoli per lo Studio e la Cura dei Tumori- IRST
City
Meldola
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria - Policlinico G. Martino Dipartimento di Medicina Interna - U.O. Messina
City
Messina
Country
Italy
Facility Name
Azienda Ospedaliera San Paolo - Unità di Ematologia e Trombosi
City
Milano
Country
Italy
Facility Name
S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
City
Novara
Country
Italy
Facility Name
Divisione di Ematologia con trapianto di midollo - A.U. Policlinico "Paolo Giaccone"
City
Palermo
Country
Italy
Facility Name
Cattedra di Ematologia CTMO Università degli Studi di Parma
City
Parma
Country
Italy
Facility Name
Med. Int. ed Oncologia Medica IRCCS Policlinico S. Matteo
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
U.O. Ematologia Clinica - Azienda USL di Pescara
City
Pescara
Country
Italy
Facility Name
Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale di Piacenza
City
Piacenza
Country
Italy
Facility Name
Pordenone Unità operativa Medicina II Az. Osp. S. M. degli Angeli
City
Pordenone
Country
Italy
Facility Name
Dipartimento Oncologico - Ospedale S.Maria delle Croci
City
Ravenna
Country
Italy
Facility Name
Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli"
City
Reggio Calabria
Country
Italy
Facility Name
Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova
City
Reggio Emilia
Country
Italy
Facility Name
Ospedale "Infermi"
City
Rimini
Country
Italy
Facility Name
Az. Ospedaliera "Sant' Andrea"-Università la Sapienza Seconda Facoltà di Medicina e Chirurgia
City
Roma
Country
Italy
Facility Name
Divisione di Ematologia - Ospedale S. Camillo
City
Roma
Country
Italy
Facility Name
Divisione Ematologia - Università Campus Bio-Medico
City
Roma
Country
Italy
Facility Name
UOC Pronto Soccorso e Accettazione Ematologica - Dipartimento Biotecnologie Cellulari ed Ematologia - Università degli Studi di Roma "Sapienza"
City
Rome
ZIP/Postal Code
00161
Country
Italy
Facility Name
Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore
City
Rome
ZIP/Postal Code
00168
Country
Italy
Facility Name
Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
City
San Giovanni Rotondo
Country
Italy
Facility Name
U.O.C. Ematologia e Trapianti - A.O. Senese - Policlinico " Le Scotte"
City
Siena
Country
Italy
Facility Name
Divisione di Ematologia dell' Università degli Studi di Torino - "Città della Salute e della Scienza di Torino"
City
Torino
Country
Italy
Facility Name
Struttura Complessa II Medicina - Ematologia - Centro di Riferimento Ematologico - Ospedale Maggiore
City
Trieste
Country
Italy
Facility Name
Clinica Ematologica - Policlinico Universitario
City
Udine
Country
Italy
Facility Name
Policlinico G. B. Rossi - Borgo Roma
City
Verona
ZIP/Postal Code
37134
Country
Italy
Facility Name
Ospedale San Bortolo
City
Vicenza
ZIP/Postal Code
36100
Country
Italy

12. IPD Sharing Statement

Learn more about this trial

Prednisone or Dexamethasone in Newly Diagnosed, Previously Untreated Primary Immune Thrombocytopenic Purpura

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