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

• Conditions: Higher-risk-myelodysplastic syndromes; MedDRA version: 21.1Level: PTClassification code 10028533Term: Myelodysplastic syndromeSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2019-003997-25-IT
• Lead Sponsor: FONDAZIONE GIMEMA (GRUPPO ITALIANO MALATTIE EMATOLOGICHE DELL' ADULTO) FRANCO MANDELLI ONLUS

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-10-07
• Last Update Posted: 26 July 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 274

Inclusion Criteria

1. Patients with newly diagnosed higher-risk MDS, including IPSS Intermediate-2 and high, and IPSS-R intermediate to very-high
2. Age 18-70 years
3. Previously untreated for HR-MDS
4. HSCT – eligible
5. Life expectancy greater than or equal to 3 months;
6. Signed written informed consent according to ICH/EU/GCP and national local laws
7. Eastern Cooperative Oncology Group Performance Status Grade of 0-2
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 137
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 137

Exclusion Criteria

1. Acute myeloid leukaemia with >20% blasts in BM or peripheral blood (PB);
2. concurrent malignancy diagnosed in the past 12 months (with the exception of skin basalioma);
3. severe renal, cardiac, liver or lung impairment; 4. 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.
5. HIV infection; active, uncontrolled HCV or HBV infections or liver cirrhosis;
6. clinically relevant neurological or psychiatric diseases;
7. hypersensitivity (known or suspected) to AZA;
8. 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