ABRAZA: 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

• Sponsor: Asociación Andaluza de Hematología y Hemoterapia (Other)
• Overall Status: Completed
• CT.gov ID: NCT01338337
• Collaborator: (none)
• Enrollment: 40
• Locations: 9
• Arms: 2
• Duration (Months): 61
• Patients Per Site: 4.4
• Patients Per Site Per Month: 0.1

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndrome (MDS)	Drug: Azacitidine	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 40 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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 Start Date: Nov 1, 2010
• Actual Primary Completion Date: Dec 1, 2015
• Actual Study Completion Date: Dec 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
No Intervention: Support treatment 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.
Experimental: Azacitidine 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	Drug: Azacitidine; 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

Outcome Measures

Primary Outcome Measures

1. Erythroid haematologic response [Approximately the primary outcome is measured on days 0, 252 and 504 (basal, cycle 9 and 18 (each cicle has 28 days +/-3 days))]

   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

1. Haematologic response [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).]

   Haematologic response, bases on the following parameters: platelets, and neutrophils according to International Working Group (IWG) Criteria.

2. Medullary and cytogenetic response [Approximately this secondary outcome is measured on days 252 and 504 (basal, cycle 9 and 18 (each cicle has 28 days +/-3 days)).]

   Medullary and cytogenetic response according to International Working Group (IWG) 2006 criteria.

3. Quality of life [Approximately this secondary outcome is measured on days 252 and 504 (basal, cycle 9 and 18 (each cicle has 28 days +/-3 days)).]

   The effect of treatment response on quality of life, through the Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire.

4. Overall survival [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)]

   Overall survival

5. Event-Free Survival [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)]

   Event-Free Survival

6. Acute Leukaemia Transformation Rate [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)]

   Acute Leukaemia Transformation Rate

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Hospital General de Jerez	Jerez	Cádiz	Spain	11407
2	Hospital Costa del Sol	Marbella	Malaga	Spain	29603
3	Hospital Reina Sofía, Servicio de Hematología	Cordoba		Spain	14004
4	Hospital Universitario San Cecilio	Granada		Spain	18012
5	Hospital Virgen de las Nieves	Granada		Spain	18840
6	Hospital Juan Ramón Jiménez	Huelva		Spain	21005
7	Hospital Carlos Haya	Malaga		Spain	29800
8	Hospital Universitario Virgen del Rocío	Sevilla		Spain	41013
9	Hospital Universitario Virgen de Valme	Sevilla		Spain	41014

Sponsors and Collaborators

• Asociación Andaluza de Hematología y Hemoterapia

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Joaquin Sanchez, MD. Ph D Hematologist, Asociación Andaluza de Hematología y Hemoterapia
• ClinicalTrials.gov Identifier: NCT01338337
• Other Study ID Numbers: ABRAZA
• First Posted: Apr 19, 2011
• Last Update Posted: Jan 26, 2016
• Last Verified: Jan 1, 2016

• Keywords provided by Joaquin Sanchez, MD. Ph D Hematologist, Asociación Andaluza de Hematología y Hemoterapia: 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
• Additional relevant MeSH terms: Preleukemia; Myelodysplastic Syndromes; Syndrome; Azacitidine

Study Results

No Results Posted as of Jan 26, 2016