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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.


Phase 2
18 Years
N/A
Open (Enrolling)
Both
Myelodysplastic Syndrome

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Trial Information

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.


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.


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.

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Evaluate the efficacy of the treatment in response rate terms

Outcome Time Frame:

6 months

Safety Issue:

No

Principal Investigator

Sanz Guillermo, Dr

Investigator Role:

Study Director

Investigator Affiliation:

Hospital La Fe

Authority:

Spain: Ministry of Health

Study ID:

2007-000972-18

NCT ID:

NCT00495547

Start Date:

February 2009

Completion Date:

December 2013

Related Keywords:

  • Myelodysplastic Syndrome
  • Myelodysplastic Syndrome
  • Red Cell transfusion dependent
  • Myelodysplastic Syndromes
  • Preleukemia

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