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Study of Vidaza (Azacitidine) Versus Support Treatment in Patients With Low Risk Myelodysplastic Syndrome (Low and Intermediate-1 IPSS) Without the 5q Deletion and Transfusion Dependent Anaemia (ABRAZA)

Primary Purpose

Myelodysplastic Syndrome (MDS)

Status
Completed
Phase
Phase 2
Locations
Spain
Study Type
Interventional
Intervention
Azacitidine
Sponsored by
Asociación Andaluza de Hematología y Hemoterapia
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myelodysplastic Syndrome (MDS) focused on measuring Low risk International Prognostic Scoring System (IPSS) (0 or Int-1), Patients who have not responded to previous treatment with erythropoietin (EPO), Eastern Cooperative Oncology Group (ECOG) score ≤ 3

Eligibility Criteria

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

Inclusion Criteria:

  1. Patients over 18 years of age.
  2. Patients who agree to take part in the study must understand the informed consent and sign it voluntarily.
  3. Patients must be able to comply with all the programmed visits and other study requirements.
  4. Patients with low risk International Prognostic Scoring System (IPSS 0 or Int-1) myelodysplastic syndrome (MDS) without chromosome 5 (5q) deletion and anaemia with transfusion needs. Transfusion dependence is defined as at least 2 units of erythrocyte concentrate (EC) during the 8 weeks prior to inclusion in the study, and symptomatic anaemia, defined by a haemoglobin value ≤9.0 gr/dl.
  5. Patients who have not responded to previous treatment with erythropoietin (EPO): With a response profile based on basal erythropoietin (EPO) levels of > 250 u/L, with no response alter 12 weeks of treatment at maximum doses (60.000 U or 250 µg darbepoetin (DAB), in combination with Granulocyte colony-stimulating factor (G-CSF) in cases of refractory anaemia with ringed sideroblasts (RARS)) , or with loss of the response obtained after an initial optimum response.
  6. Patients who are not candidates for intensive chemotherapy and transplant modalities.
  7. Patients with an Eastern Cooperative Oncology Group (ECOG) score ≤ 3
  8. Women of child-bearing age and heterosexual men whose partner is of child-bearing age, must undertake to use an effective contraceptive method for the duration of the treatment and for at least 3 months alter is has finalised.

Exclusion Criteria:

  1. The presence of a psychiatric or medical disease which prevents the patient from signing of the informed consent.
  2. Human immunodeficiency virus (HIV) Seropositive, hepatitis B antigen (AgVHB) positive or hepatitis C virus (HCV) polymerase chain reaction (PCR) positive.
  3. Pregnant or nursing women.
  4. Uncontrolled intercurrent disease: Active infection requiring parenteral antibiotics, Symptomatic chronic heart failure (New York Heart Association (NYHA) class III or IV), Instable angina pectoris, or Another neoplasia apart from his myelodysplastic syndromes (MDS).
  5. Have been treated with demethylating drugs at any moment prior to inclusion in the study.

Sites / Locations

  • Hospital General de Jerez
  • Hospital Costa del Sol
  • Hospital Reina Sofía, Servicio de Hematología
  • Hospital Universitario San Cecilio
  • Hospital Virgen de las Nieves
  • Hospital Juan Ramón Jiménez
  • Hospital Carlos Haya
  • Hospital Universitario Virgen del Rocío
  • Hospital Universitario Virgen de Valme

Arms of the Study

Arm 1

Arm 2

Arm Type

No Intervention

Experimental

Arm Label

Support treatment

Azacitidine

Arm Description

Transfusional support will be applied for symptomatic anaemia using clinical discretion and chelation therapy when ferritin is ≥ 1000 μgr/ml with the chelating agents allowed.

Azacitidine 75 mg/m2, for 5 days of each 20 day cycle. Transfusional support will be applied for symptomatic anaemia using clinical discretion and chelation therapy when ferritin is ≥ 1000 μgr/ml with the chelating agents allowed

Outcomes

Primary Outcome Measures

Erythroid haematologic response
To evaluate the efficacy of treatment with Azacitidine in patients with transfusion-dependent, low risk International Prognostic Scoring System (IPSS) 0 or int-1, Myelodysplastic Syndrome (MDS) without chromosome 5 (5q) deletion. The main objective will be based on the erythroid haematologic response according to International Working Group (IWG) 2006 criteria.

Secondary Outcome Measures

Haematologic response
Haematologic response, bases on the following parameters: platelets, and neutrophils according to International Working Group (IWG) Criteria.
Medullary and cytogenetic response
Medullary and cytogenetic response according to International Working Group (IWG) 2006 criteria.
Quality of life
The effect of treatment response on quality of life, through the Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire.
Overall survival
Overall survival
Event-Free Survival
Event-Free Survival
Acute Leukaemia Transformation Rate
Acute Leukaemia Transformation Rate

Full Information

First Posted
April 13, 2011
Last Updated
January 25, 2016
Sponsor
Asociación Andaluza de Hematología y Hemoterapia
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1. Study Identification

Unique Protocol Identification Number
NCT01338337
Brief Title
Study of Vidaza (Azacitidine) Versus Support Treatment in Patients With Low Risk Myelodysplastic Syndrome (Low and Intermediate-1 IPSS) Without the 5q Deletion and Transfusion Dependent Anaemia
Acronym
ABRAZA
Official Title
Multicentre, Open-label, Randomized Phase II Study of Vidaza (Azacitidine) Versus Support Treatment in Patients With Low Risk Myelodysplastic Syndrome (Low and Intermediate-1 International Prognostic Scoring System(IPSS )) Without the 5q Deletion and Transfusion Dependent Anaemia
Study Type
Interventional

2. Study Status

Record Verification Date
January 2016
Overall Recruitment Status
Completed
Study Start Date
November 2010 (undefined)
Primary Completion Date
December 2015 (Actual)
Study Completion Date
December 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Asociación Andaluza de Hematología y Hemoterapia

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
Primary Outcome Measures: • To evaluate the efficacy of treatment with Azacitidine in patients with transfusion-dependent, low risk International Prognostic Scoring System (IPSS) 0 int-1, Myelodysplastic Syndrome (MDS) without chromosome 5 (5q) deletion. The main objective will be based on the erythroid haematologic response according to International Working Group (IWG) 2006 criteria. Secondary Outcome Measures: Haematologic response, bases on the following parameters: platelets, and neutrophils according to International Working Group (IWG) Criteria. Medullary and cytogenetic response according to International Working Group (IWG) 2006 criteria. The effect of treatment response on quality of life, through the Functional Assessment of Cancer Therapy-Anemia (FACT-an) questionnaire. Overall survival, Event-Free Survival and the Acute Leukaemia Transformation Rate.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndrome (MDS)
Keywords
Low risk International Prognostic Scoring System (IPSS) (0 or Int-1), Patients who have not responded to previous treatment with erythropoietin (EPO), Eastern Cooperative Oncology Group (ECOG) score ≤ 3

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
40 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Support treatment
Arm Type
No Intervention
Arm Description
Transfusional support will be applied for symptomatic anaemia using clinical discretion and chelation therapy when ferritin is ≥ 1000 μgr/ml with the chelating agents allowed.
Arm Title
Azacitidine
Arm Type
Experimental
Arm Description
Azacitidine 75 mg/m2, for 5 days of each 20 day cycle. Transfusional support will be applied for symptomatic anaemia using clinical discretion and chelation therapy when ferritin is ≥ 1000 μgr/ml with the chelating agents allowed
Intervention Type
Drug
Intervention Name(s)
Azacitidine
Intervention Description
Azacitidine 75 mg/m2, for 5 days of each 20 day cycle. Transfusional support will be applied for symptomatic anaemia using clinical discretion and chelation therapy when ferritin is ≥ 1000 μgr/ml with the chelating agents allowed
Primary Outcome Measure Information:
Title
Erythroid haematologic response
Description
To evaluate the efficacy of treatment with Azacitidine in patients with transfusion-dependent, low risk International Prognostic Scoring System (IPSS) 0 or int-1, Myelodysplastic Syndrome (MDS) without chromosome 5 (5q) deletion. The main objective will be based on the erythroid haematologic response according to International Working Group (IWG) 2006 criteria.
Time Frame
Approximately the primary outcome is measured on days 0, 252 and 504 (basal, cycle 9 and 18 (each cicle has 28 days +/-3 days))
Secondary Outcome Measure Information:
Title
Haematologic response
Description
Haematologic response, bases on the following parameters: platelets, and neutrophils according to International Working Group (IWG) Criteria.
Time Frame
The secondary outcome is measured on days 0, 28, 56, 84, 112, 140, 168, 196, 224, 252, 280, 308, 336, 364, 392, 420, 448, 476 and 504 (the measurement is performed at basal line and in every cycle (every 28 days +/-3 days) up to a maximum of 18 cycles).
Title
Medullary and cytogenetic response
Description
Medullary and cytogenetic response according to International Working Group (IWG) 2006 criteria.
Time Frame
Approximately this secondary outcome is measured on days 252 and 504 (basal, cycle 9 and 18 (each cicle has 28 days +/-3 days)).
Title
Quality of life
Description
The effect of treatment response on quality of life, through the Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire.
Time Frame
Approximately this secondary outcome is measured on days 252 and 504 (basal, cycle 9 and 18 (each cicle has 28 days +/-3 days)).
Title
Overall survival
Description
Overall survival
Time Frame
The secondary outcome is measured from basal line until day 504 (average of days for 18 cycles), or until progresion, or until exitus... (from basal line until the end of the study)
Title
Event-Free Survival
Description
Event-Free Survival
Time Frame
The secondary outcome is measured from basal line until day 504 (average of days for 18 cycles), or until progresion, or until exitus... (from basal line until the end of the study)
Title
Acute Leukaemia Transformation Rate
Description
Acute Leukaemia Transformation Rate
Time Frame
The secondary outcome is measured from basal line until day 504 (average of days for 18 cycles), or until progresion, or until exitus... (from basal line until the end of the study)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients over 18 years of age. Patients who agree to take part in the study must understand the informed consent and sign it voluntarily. Patients must be able to comply with all the programmed visits and other study requirements. Patients with low risk International Prognostic Scoring System (IPSS 0 or Int-1) myelodysplastic syndrome (MDS) without chromosome 5 (5q) deletion and anaemia with transfusion needs. Transfusion dependence is defined as at least 2 units of erythrocyte concentrate (EC) during the 8 weeks prior to inclusion in the study, and symptomatic anaemia, defined by a haemoglobin value ≤9.0 gr/dl. Patients who have not responded to previous treatment with erythropoietin (EPO): With a response profile based on basal erythropoietin (EPO) levels of > 250 u/L, with no response alter 12 weeks of treatment at maximum doses (60.000 U or 250 µg darbepoetin (DAB), in combination with Granulocyte colony-stimulating factor (G-CSF) in cases of refractory anaemia with ringed sideroblasts (RARS)) , or with loss of the response obtained after an initial optimum response. Patients who are not candidates for intensive chemotherapy and transplant modalities. Patients with an Eastern Cooperative Oncology Group (ECOG) score ≤ 3 Women of child-bearing age and heterosexual men whose partner is of child-bearing age, must undertake to use an effective contraceptive method for the duration of the treatment and for at least 3 months alter is has finalised. Exclusion Criteria: The presence of a psychiatric or medical disease which prevents the patient from signing of the informed consent. Human immunodeficiency virus (HIV) Seropositive, hepatitis B antigen (AgVHB) positive or hepatitis C virus (HCV) polymerase chain reaction (PCR) positive. Pregnant or nursing women. Uncontrolled intercurrent disease: Active infection requiring parenteral antibiotics, Symptomatic chronic heart failure (New York Heart Association (NYHA) class III or IV), Instable angina pectoris, or Another neoplasia apart from his myelodysplastic syndromes (MDS). Have been treated with demethylating drugs at any moment prior to inclusion in the study.
Facility Information:
Facility Name
Hospital General de Jerez
City
Jerez
State/Province
Cádiz
ZIP/Postal Code
11407
Country
Spain
Facility Name
Hospital Costa del Sol
City
Marbella
State/Province
Malaga
ZIP/Postal Code
29603
Country
Spain
Facility Name
Hospital Reina Sofía, Servicio de Hematología
City
Cordoba
ZIP/Postal Code
14004
Country
Spain
Facility Name
Hospital Universitario San Cecilio
City
Granada
ZIP/Postal Code
18012
Country
Spain
Facility Name
Hospital Virgen de las Nieves
City
Granada
ZIP/Postal Code
18840
Country
Spain
Facility Name
Hospital Juan Ramón Jiménez
City
Huelva
ZIP/Postal Code
21005
Country
Spain
Facility Name
Hospital Carlos Haya
City
Malaga
ZIP/Postal Code
29800
Country
Spain
Facility Name
Hospital Universitario Virgen del Rocío
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
Hospital Universitario Virgen de Valme
City
Sevilla
ZIP/Postal Code
41014
Country
Spain

12. IPD Sharing Statement

Learn more about this trial

Study of Vidaza (Azacitidine) Versus Support Treatment in Patients With Low Risk Myelodysplastic Syndrome (Low and Intermediate-1 IPSS) Without the 5q Deletion and Transfusion Dependent Anaemia

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