Clinical trial: Early and Low Dose Deferasirox (3.5 mg/kg FCT) to Suppress NTBI and LPI as Early Intervention to Prevent Tissue Iron Overload in Lower Risk MDS (NCT03920657)

Eligibility criteria:
Inclusion Criteria:

          -  Diagnosis: Adult Myelodysplastic Syndrome (≥18 years).

          -  Revised IPSS: very low. low - intermediate

          -  Having received 5-20 packed red blood cell units

          -  Serum ferritin ≥300 ng/ml

          -  Transferrin saturation ≥ 60%

          -  Chelation naïve

          -  Capability to provide informed consent

        Exclusion Criteria:

          -  Patients aged <18 years old

          -  Higher risk (revised IPSS) MDS (Intermediate 2, high)

          -  Cumulative transfusion story of > 20 packed red cell units

          -  Creatinine Clearance (CrCL): <60 ml/min. Patients with CrCl of 40-60ml/min will be
             included only individually if no other renal risk factors are present.

          -  Serum creatinine >2 x ULN at screening. If borderline serum creatinine will be
             measured within 7-10 days and the mean value will be used for eligibility criteria.

          -  Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg
             in a non-first void urine sample (or alternatively in two of three samples obtained
             for screening).

          -  ECOG performance status >2.

          -  Left ventricular ejection fraction < 50% by echocardiography

          -  A history of repeated hospitalization for congestive heart failure.

          -  Systemic diseases that would prevent study treatment (e.g. uncontrolled hypertension,
             cardiovascular, renal, hepatic, metabolic, etc.)

          -  Clinical or laboratory evidence of chronic Hepatitis B or Hepatitis C (definition of
             chronic hepatitis follows EASL 2017 criteria).

          -  History of HIV positive test result (ELISA or Western blot).

          -  Treatment with systemic investigational drug within 4 weeks or topical investigational
             drug within 7 days of study start.

          -  ALT or AST over 3 times superior to ULN at screening.

          -  ANC < 500/ microL

          -  Platelets transfusion dependency

          -  Total bilirubin over 1.5 times superior to ULN at screening (patients with Gilbert
             syndrome are allowed to enter the study)

          -  Diagnosis of Child score C liver cirrhosis.

          -  Patients participating in another clinical trial other than an observational registry
             study.

          -  Patients with a history of another malignancy within the past 3 years, with the
             exception of basal skin carcinoma or cervical carcinoma in situ or completely resected
             colonic polyps carcinoma in situ.

          -  History of non-compliance to medical regimens, or patients who are considered
             potentially unreliable and/or not cooperative.

          -  Presence of a surgical or medical condition which might significantly alter the
             absorption, distribution, metabolism or excretion of study drug.

          -  Pregnant, intending-to-become pregnant, or breast-feeding patients.

          -  Women of potential maternity age who do not agree to practice effective contraceptive
             methods fo the entire study duration.

          -  History of drug or alcohol abuse within the 12 months prior to enrollment.

          -  Hypersensitivity to the active substance or to any of the excipients.

          -  Inability to provide a valid informed consent
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.


Patient description (medical language): A 51-year-old man comes to the office complaining of fatigue and some sexual problems including lack of libido. The patient doesn't smoke or use any illicit drug.  Blood pressure is 120/80 mm Hg and pulse is 70/min.  Oxygen saturation is 99% on room air.  BMI is 24 kg/m2.  Skin examination shows increased pigmentation. Genotype testing is consistent with homozygosity for the C282Y mutation. Laboratory study shows transferrin saturation of 55% and serum ferritin of 550 μg/L. He is diagnosed as a case of hemochromatosis.

Patient description (patient's own words): I am 51 years old and I just came back from the doctor's office. I'm sick and tired of being that exhausted, reaching the point where me and my lovely wife are not touching each other anymore! I'm not smoking or doing drugs! My blood pressure was 120/80 mm Hg, and pulse was 70/min and my oxygen saturation 99%. My BMI is 24. My skin also turned a bit darker lately. He tested my genes and told me that I have a mutation of the C282Y gene. I also did lab tests where my transferrin saturation was 55% and serum ferritin was 550  μg/L. The doctor diagnosed me with iron overload.

Does the patient match the trial criteria? 0