An Open-label, Phase II, Randomized, Pilot Study to Assess the Effect in Term of Erythroid Improvement of Deferasirox Combined With Erythropoietin Compared to Erythropoietin Alone in Patients With low-and Int-1-risk Myelodysplastic Syndrome.

CompletadoFase 2ClinicalTrials.gov

ID: NCT01868477Tipo: INTERVENTIONALInicio: 28 de ene de 2014Fin estimado: 5 de abr de 2017

Traducción no disponible, mostrando original

Resumen

The primary purpose of this trial was is to assess the effect of treatment with deferasirox combined with erythropoietin vs. erythropoietin alone on erythropoiesis in patients with low- and int-1-risk myelodysplastic syndrome. The addition of deferasirox to erythropoietin can lead to a potential synergism with the reduction of reactive oxygen species, through both the NF-kB pathway and the control of free toxic iron. This may create a better environment in the bone marrow for a better response with erythropoietin. This study was designed to test in a prospective way the combination of deferasirox with erythropoietin in terms of their effect on hematopoiesis.

Descripción detallada

This study did not meet the original enrollment objective of 60 patients and was terminated without extending enrollment past original planned LPFV of 31-Oct-2016.

Elegibilidad

Edad mínima: 18 YearsSexo: ALL

Criterios de inclusión

Patients who had low- and Int-1-risk myelodysplastic syndrome
Documented diagnosis of the following:
Myelodysplastic syndrome that lasted ≥ 3 months and \< 3 years Disease must not have been secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases
A hemoglobin \< 10 g/dL and ≥ 8 g/dL
History of transfusions \< 10 RBC units and must not have been RBC transfusion dependent
ng/mL \< serum ferritin \< 1,500 ng/mL (Values within 10% difference above 1500 ng/ml or 10% difference below 300 ng/ml could have been accepted at the investigator's discretion.
Endogenous erythropoietin levels \< 500 units/L
Serum creatinine ≤ 1.5 times upper limit of normal (ULN)
Creatinine clearance above the concentration limit in locally approved prescribing information (PI). Patients with creatinine clearance between 40 and less than 60 mL/min, who did not present with additional risk factors that might impair renal function, were eligible at the discretion of the investigator

Criterios de exclusión

Patients who had MDS with isolated del(5q)
Patients who had received prior EPO treatment or other recombinant growth factors regardless of the outcome (Patient who had received prior EPO treatment or other recombinant growth factors for less than 4 weeks and not within 3 months before screening without a documented response are allowed)
Patients who had received steroids or immunosuppressive therapy for the improvement of hematological parameters (stable steroid treatment for adrenal failure or chronic medical conditions, and intermittent dexamethasone as antiemetics were allowed).
B12 and folate deficient patients with and without clinical symptoms (patients were rescreened after successful therapy of B12 and folate deficiency)
Uncontrolled seizures or uncontrolled hypertension

Intervenciones

drug

Deferasirox DFX, DT

provided as dispersible tablets for oral use in 125 and 250, 500 mg

drug

Erythropoietin alpha

drug

Deferasirox DFX, FCT

provided as film-coated tablet for oral use in 90, 180, 360 mg strengths

Ubicaciones

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Novartis Investigative Site

La Plata, Buenos Aires, Argentina

Patrocinadores

PrincipalNovartis Pharmaceuticals

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