The study is aimed at evaluating the feasibility of a hypomethylating treatment combined with hematopoietic stem cell transplantation in comparison to hematopoietic stem cell transplantation in patients with high-risk myelodysplastic syndrome.

Phase 1

Recruiting

• Conditions: Higher-risk-myelodysplastic syndromes; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]; MedDRA version: 21.1Level: PTClassification code: 10028533Term: Myelodysplastic syndrome Class: 100000004864

• Registration Number: CTIS2023-510515-19-00
• Lead Sponsor: Fondazione Gimema Franco Mandelli Onlus

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-06-04
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 274

Inclusion Criteria

Patients with newly diagnosed higher-risk MDS, including IPSS Intermediate-2 and high, and IPSS-R intermediate to very-high, Age 18-70 years, Previously untreated for HR-MDS, HSCT – eligible, Life expectancy greater than or equal to 3 months, Signed written informed consent according to ICH/EU/GCP and national local laws, Eastern Cooperative Oncology Group Performance Status Grade of 0-2

Exclusion Criteria

Acute myeloid leukaemia with >20% blasts in BM or peripheral blood (PB), concurrent malignancy diagnosed in the past 12 months (with the exception of skin basalioma), severe renal, cardiac, liver or lung impairment, pregnant or lactating or potentially fertile (both males and females), who have not agreed to avoid pregnancy during the trial period; Women of childbearing potential and men must agree to use effective contraception during and up to 3 months after treatment with azacitidine., HIV infection; active, uncontrolled HCV or HBV infections or liver cirrhosis, clinically relevant neurological or psychiatric diseases, hypersensitivity (known or suspected) to AZA, prior Treatments: a) prior investigational drugs (within 30 days); b) radiotherapy, chemotherapy, or cytotoxic therapy for non-MDS conditions within the previous 6 months; c) growth factors (EPO, G-CSF or GM-CSF) during the previous 21 days; d) androgenic hormones during the previous 14 days; e) prior transplantation or cytotoxic therapy, including azacitidine, AZA or chemotherapy, administered to treat MDS.

Study & Design

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