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

Phase 1

• Conditions: Adult patients with low risk MDS; MedDRA version: 20.0Level: LLTClassification code 10028532Term: MyelodysplasiaSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2018-003542-17-IT
• Lead Sponsor: FONDAZIONE ITALIANA SINDROMI MIELODISPLASTICHE ETS

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-05-20
• Last Update Posted: 9 January 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

1) Diagnosis: Adult Myelodysplastic Syndrome (=18 years)
2) Revised IPSS: very low. low – intermediate
3) Having received 5-20 packed red blood cell units
4) Serum ferritin =300 ng/ml
5) Transferrin saturation =60%
6) Chelation naïve
7) Capability to provide informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

Exclusion Criteria

1) Patients aged <18 years old
2) Higher risk (revised IPSS) MDS (Intermediate 2, high)
3) Cumulative transfusion story of > 20 packed red cell units
4) 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
5) 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
6) 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)
7) ECOG performance status >2.
8) Left ventricular ejection fraction < 50% by echocardiograph
9) A history of repeated hospitalization for congestive heart failure.
10) Systemic diseases that would prevent study treatment (e.g. uncontrolled hypertension, cardiovascular, renal, hepatic, metabolic, etc.)
11) Clinical or laboratory evidence of chronic Hepatitis B or Hepatitis C (definition of chronic hepatitis follows EASL 2017 criteria)
12) History of HIV positive test result (ELISA or Western blot)
13) Treatment with systemic investigational drug within 4 weeks or topical investigational drug within 7 days of study start.
14) ALT or AST over 3 times superior to ULN at screening
15) ANC < 500/ microL
16) Platelets transfusion dependency
17) Total bilirubin over 1.5 times superior to ULN at screening (patients with Gilbert syndrome are allowed to enter the study)
18) Diagnosis of Child score C liver cirrhosis
19) Patients participating in another clinical trial other than an observational registry study
20) 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
21) History of non-compliance to medical regimens, or patients who are considered potentially unreliable and/or not cooperative
22) Presence of a surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
23) Pregnant, intending-to-become pregnant, or breast-feeding patients
24) History of drug or alcohol abuse within the 12 months prior to enrollment
25) Inability to provide a valid informed consent

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified