5-Azacitidine in Low-risk Myelodysplastic Syndromes (MDSs)

NCT00897130 | Completed Phase 2 DMC

• 1 other identifier: 2007-003943-55
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 7

Brief Summary

Azacitidine will be given at a dose of 75 mg/sqm (s.c) daily for 5 consecutive days every 28 days (every month) for a total of 8 courses to low risk MDSs according to IPSS scoring system. In fact, several studies produced high rates of trilineage responses, reduces the risk of progression to acute myeloid leukemia (AML) in high-risk MDS and improves the quality of life (QoL). The use of 5-Aza in the earlier phases of MDS could reduce the proliferative advantage of MDS clone and favour the regrowth of normal hematopoiesis.

Conditions

Myelodysplastic Syndrome

Keywords

low-risk (IPSS 0-INT1) MDS according to WHO classification

Outcome Measures

Primary Outcomes (2)

• To evaluate the efficacy (hematologic response) of five days monthly 5-Aza treatment schedule in patients with low-risk MDS (IPSS 0-1).

  36 months

• To evaluate the toxicity of five days monthly 5-Aza treatment schedule in patients with low-risk MDS (IPSS 0-1).

  36 months

Secondary Outcomes (1)

• To evaluate the QoL by the FACT-An questionnaire

  36 months

Study Arms (1)

Azacytidine

EXPERIMENTAL

Azacitidine will be given at a dose of 75mg/sqm subcutaneous daily for 5 consecutive days every 28 days (every month) for a total of 8 courses. 5-Aza dosages will be adjusted.

Drug: Azacytidine

Interventions

Azacytidine DRUG

Azacitidine will be given at a dose of 75mg/sqm subcutaneous daily for 5 consecutive days every 28 days (every month) for a total of 8 courses. 5-Aza dosages will be adjusted

Azacytidine

Eligibility Criteria

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

You may qualify if:

• Patients with low-risk (IPSS 0-INT1) MDS according to WHO classification, presenting one or more of the followings:
• Symptomatic anemia requiring RBC transfusion supportive therapy previously unresponsive to EPO or not expected to respond to EPO
• Thrombocytopenia requiring platelet transfusion with or without muco-cutaneous haemorrhagic syndrome
• Persistent (\> 3 months) absolute neutrophil count less then 1,5 x 109/L, with or without infections, requiring or not myeloid growth factor therapy
• ≥ 18 years old.
• Life expectancy ≥ 3 months.
• ECOG performance Status Grade 0-2.
• Serum bilirubin levels ≤ 1.5 upper limit of the normal (ULN)
• Serum GOT and GPT levels ≤ 2x UNL.
• Creatinine levels ≤ 1.5x UNL.
• Negative serum β-human chorionic gonadotropin (β-HCG) pregnancy test 24 hours prior to beginning of therapy with 5-AZA, for fertile women.
• Written informed consent.

You may not qualify if:

• Patients with MDS according to WHO classification with INT-2 or high IPSS risk.
• Life expectancy \< 3 months.
• ECOG performance Status Grade \> 2.
• Serum bilirubin levels \>1.5 upper limit of the normal (ULN).
• Serum GOT and GPT levels \> 2 x UNL.
• Creatinine levels \>1.5 x UNL.
• Pregnancy or breast feeding.
• Insulin-dependent diabetes and uncontrolled non insulin-dependent diabetes.
• Severe cardiac or pulmonary disease incompatible with the conduction of the protocol.
• Patient with a clear indication to receive long-term anticoagulant therapy.
• Other active hematologic or solid tumors.
• Severe CNS disease.
• Malignant hepatic tumors.
• Hypersensitivity to mannitol or azacitidine.
• No written informed consent.

Sponsors & Collaborators

• Università degli Studi di Brescia: lead
• University of Udine: collaborator
• University of Bologna: collaborator
• University of Genova: collaborator
• University of Siena: collaborator
• Cremona: collaborator
• Mantova: collaborator

Study Sites (7)

University of Bologna

Bologna, Italy

Location

Chair of Haematology, Bone Marrow Transplant Unit

Brescia, 25123, Italy

Location

Cremona

Cremona, Italy

Location

University of Genova

Genova, Italy

Location

Mantova

Mantova, Italy

Location

University of Siena

Siena, Italy

Location

University of Udine

Udine, Italy

Location

Related Publications (1)

• Abdulhaq H, Rossetti JM. The role of azacitidine in the treatment of myelodysplastic syndromes. Expert Opin Investig Drugs. 2007 Dec;16(12):1967-75. doi: 10.1517/13543784.16.12.1967.

  PMID: 18042004 RESULT

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 Officials

• Prof Domenico Russo, MD

  Chair of Haematology, Brescia University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Full Professor of Hematology

Study Record Dates

• First Submitted: May 8, 2009
• First Posted: May 12, 2009
• Study Start: August 1, 2008
• Primary Completion: December 1, 2009
• Study Completion: December 1, 2014
• Last Updated: December 3, 2014

Record last verified: 2014-12

Locations

IT (7)