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

NCT01338337 | Completed Phase 2

• 1 other identifier: ABRAZA
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 9

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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.

  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 Outcomes (6)

• 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).

• 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)).

• 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)).

• 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)

• 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)

Study Arms (2)

Support treatment

NO INTERVENTION

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

EXPERIMENTAL

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

Interventions

Azacitidine DRUG

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

Azacitidine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Asociación Andaluza de Hematología y Hemoterapia: lead

Study Sites (9)

Hospital Reina Sofía, Servicio de Hematología

Córdoba, Cordoba, 14004, Spain

Location

Hospital General de Jerez

Jerez de la Frontera, Cádiz, 11407, Spain

Location

Hospital Universitario San Cecilio

Granada, Granada, 18012, Spain

Location

Hospital Virgen de las Nieves

Granada, Granada, 18840, Spain

Location

Hospital Juan Ramón Jiménez

Huelva, Huelva, 21005, Spain

Location

Hospital Costa del Sol

Marbella, Malaga, 29603, Spain

Location

Hospital Carlos Haya

Málaga, Malaga, 29800, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, Sevilla, 41013, Spain

Location

Hospital Universitario Virgen de Valme

Seville, Sevilla, 41014, Spain

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD. Ph D Hematologist

Study Record Dates

• First Submitted: April 13, 2011
• First Posted: April 19, 2011
• Study Start: November 1, 2010
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: January 26, 2016

Record last verified: 2016-01

Locations

ES (9)