Clinical trial NCT00495547: SIMIDIS: Azacitidine and Beta Erythropoietin Treatment in Patients With Myelodysplastic Syndrome
Official title: A Multicenter, Non-Randomized, Open-Label Study to Evaluate Efficacy and Safety of Azacitidine and Beta Erythropoietin Treatment in Patients With Myelodysplastic Syndrome Red Cell Transfusion Dependent With Low or Intermediate -1 Risk.
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Myelodysplastic Syndrome
Keywords: Myelodysplastic Syndrome
Red Cell transfusion dependent
Lead sponsor: PETHEMA Foundation
Sponsor class: OTHER
Collaborators: name: Roche Pharma AG
name: Celgene Corporation
First posted: 2007-07-03
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary objective is to evaluate the efficacy of the treatment in response rate terms. Otherwise this study wants to evaluate the safety of the treatment.

Detailed description: A total of up to 30 patients diagnosed of myelodysplastic syndrome red cell transfusion dependent with low or intermediate -1 risk will be included.

The patients will be evaluated at scheduled visits in up to three study periods: Pre-treatment, Treatment and Follow up.

The Pre-treatment includes Screening and baseline visits. After providing informed consent, patients will be evaluated for study eligibility.

During Treatment Period patients will be evaluated once a month although biochemistry and haematology parameters will be evaluated every 2 weeks.

If an erythroid response after 24 weeks is determined, a extension treatment will be carry out without disease progression.

Eligibility criteria: Inclusion Criteria:

1. Must voluntary sign the informed consent.
2. Age ≥ 18 years.
3. Must be able to comply with the protocol requirements
4. Patient recently diagnosed with Myelodysplastic syndrome red cell transfusion dependent with low or intermediate -1 risk according IPSS criteria.
5. Red cell transfusion dependent anemia.
6. El patient has to be able to complain with the protocol visits.
7. Women and man must accept to use high efficacy anticonceptive methods

Exclusion Criteria:

1. Pregnancy or breast-feed women.
2. Patients previously received treatment with azacytidine .
3. Patients previously received treatment with erythropoietin agents.
4. Proliferative Chronic myelomonocytic Leukaemia (leukocytes ≥ 12000/mL).
5. Patient with a previous clinical history of another cancer (except for basocellular carcinoma or spinocellular carcinoma in situ of cervix or breast) except if the patient is free of symptoms during ≥ 3 years.
6. Cytotoxic chemotherapy or experimental agents usage for myelodysplastic syndrome treatment during 28 days.
7. Previous haematopoietic transplant.
8. Mielosupresion and antitumoral treatment during the previous 28 days.
9. The following laboratory data:

   Absolute neutrophil count \< 1000 cel/ml (0.5x 109L) Platelet count \< 50000/μL (25 x 109/L) Creatinine \> 2.0 mg/dL (177 mmol/L) Aspartate transaminase (AST) or Alanine transaminase (ALT ) \> 5 x the upper limit of normal. Total bilirubin: \> 2 mg/dL
10. Patients with B12 vitamin, folic acid and ferrum deficiency.
11. Patient with positive VIH-1.
12. Any other organic or mental illness that could make impossible to sign the Inform consent or involve risk to the patient.
13. Patient has hypersensitivity previous to beta ,azacytidine, erythropoietin and/or mannitol.

Primary outcomes:
measure: Evaluate the efficacy of the treatment in response rate terms
timeFrame: 6 months
Secondary outcomes:
measure: Evaluate the safety of the treatment
timeFrame: 2 months
Other outcomes:
Locations:
city: Badalona
country: Spain
facility: Hospital Germans Trias i Pujol
city: Barcelona
country: Spain
facility: Hospital Clínic Universitari
city: Barcelona
country: Spain
facility: Hospital del Mar
city: Córdoba
country: Spain
facility: Hospital Reina Sofía
city: Leon
country: Spain
facility: Hospital Virgen Blanca
city: Madrid
country: Spain
facility: Hospital la Paz
city: Oviedo
country: Spain
facility: Hospital Central de Asturias
city: Palma de mallorca
country: Spain
facility: Hospital Son Llatzer
city: Salamanca
country: Spain
facility: Hospital Clinico Universitario
city: Valencia
country: Spain
facility: Hospital La Fe de Valencia
Study officials:
affiliation: Hospital La Fe
name: Sanz Guillermo, Dr
role: STUDY_DIRECTOR
affiliation: Hospital Clinico de Salamanca
name: Del Cañizo Consuelo, DR
role: STUDY_DIRECTOR
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