Feasibility Trial of Extracorporeal Iron Purification in Patients With Myelodysplastic Syndrome or Myelofibrosis

Official title

Feasibility, Tolerability and Efficacy of Extracorporeal Iron Purification in Patients With Myelodysplastic Syndrome or Myelofibrosis Intolerant of or Contraindicated to Oral or Subcutaneous Chelation Treatment.

Overview

In transfusion-dependent myelodysplastic syndromes patients, regular blood transfusions lead to iron overload, which can cause organ damage, hormonal imbalances, and increased infection risk, ultimately impacting patient survival. Standard oral iron chelation therapies can be intolerable for some patients due to adverse effects. The MEX-CD1 device (class III) could potentially offer an alternative for these patients by reducing serum iron levels through a novel, extracorporeal approach. MEXIRON clinical investigation focuses on the use of MEX-CD1, a medical device designed for extracorporeal chelation therapy to reduce iron overload in patients suffering from transfusion-dependent myelodysplastic syndromes (MDS) and myelofibrosis. MEXIRON aims to evaluate the device's use feasibility, safety, and effectiveness in reducing iron levels. Transfusions needs, patient experience and quality of life are also assessed. Each enrolled patients will undergo three low-volume continuous veno-venous haemodialysis cycles within one week. Following the three- haemodialysis cycles, patients will be monitored through on-site follow-up visits at 7 days, 28 days, and 90 days post-treatment to assess long-term effects.

Interventions

• Device Low-volume continuous veno-venous haemodialysis with MEX-CD1 use
  MEX-CD1 is a hyper-chelating colloidal solution that can be added to the dialysate to be used in low-volume continuous veno-venous hemodialysis. Intervention includes 3 low-volume continuous veno-venous hemodialysis for a duration of 3h20 each. For non-hospitalized patients, the treatment is performed on an outpatient basis.

Primary outcome measures

• Performance of MEX-CD1 on total iron chelation during a session epuration. [Time frame: Day 4]

Secondary outcome measures (7)

• Performance of MEX-CD1 on iron profile. [Time frame: Day 4]
• Performance of MEX-CD1 on iron profile. [Time frame: Day 4]
• Safety of MEX-CD1 use [Time frame: week 16]
• Impact on blood products patient's needs. [Time frame: week 16]
• Feasibility of MEX-CD1 use [Time frame: Day 4]
• Patient's reported tolerability of low-volume continuous veno-venous hemodialysis cycle. [Time frame: Day 4]
• Patient's reported quality of life. [Time frame: Week 16]

Eligibility criteria

Inclusion criteria

• Patient followed for myelodysplastic syndrome or myelofibrosis.
• Patient with platelet count ≥50 giga/L at inclusion.
• Patients with severe anemia and hemoglobin <70 g/L at baseline.
• Patient with intolerance or contraindication to oral or subcutaneous chelation therapy.
• Ferritinemia >1000 µg/L or hepatic iron concentration ≥7 mg/g or cardiac T2\* <20 ms at inclusion.
• Patient able to understand (French-speaking) and comply with protocol, having signed informed consent.

Exclusion criteria

• Patients with primary hemochromatosis (transferrin saturation coefficient CS-Tf > 45%).
• Patients with a contraindication to the use of MEX-CD1: weight < 30 kg, iron deficiency.
• Patients with a known allergy or contraindication to heparin or citrate.
• Patients undergoing azacitidine or other chemotherapy (or considered as such) for myelodysplastic syndrome or myelofibrosis.
• Patient who received treatment with luspatercept or erythropoietin (EPO) during the month prior to inclusion.
• Patients with indications for allogeneic bone marrow transplantation.
• Patients with a known allergy to shellfish (MEX-CD1 contains chitosan of animal origin) or to one of the other components of MEX-CD1.
• Patients with a peripheral vascular access that is difficult to access or that needs to be preserved.
• Patients participating in other interventional research that could interfere with the results of the study.
• Patients under legal protection or unable to express their consent.
• Patients under psychiatric care.
• Patient deprived of liberty by judicial or administrative decision.
• Pregnant or breast-feeding women.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

France · 2 centers

• Hôpital Lyon Sud — Oullins-Pierre-Bénite
• Hôpital Lyon Sud — Oullins-Pierre-Bénite

Identifiers

NCT: NCT06781099 · 69HCL23_0102 · 2024-A02473-44

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗