A Phase I/II study of Azacitidine (Vidaza®) in pediatric patients with newly diagnosed or relapsed high-grade pediatric MDS or JMM

Phase 1

• Conditions: D46; C93.3; Myelodysplastic syndromes; Juvenile myelomonocytic leukaemia

• Registration Number: DRKS00005375
• Lead Sponsor: Erasmus MC-Sophia

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-11-25
• Study Completion: 2020-06-22
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

•stratum 1: newly diagnosed patients with primary advanced MDS (RAEB or RAEB-t) in a ‘pre stem cell transplantation window’.
•stratum 2: relapsed patients with advanced primary MDS in a ‘re-transplantation window’. At relapse azacitidine may also be continued when a 2nd transplant is not feasible, as long as the patient benefits from treatment.
•stratum 3: newly diagnosed patients with JMML in a ‘pre-stem cell transplantation window’.
•stratum 4: relapsed patients with JMML in a ‘re-transplantation window’. Azacitidine may also be continued when a 2nd transplant is not feasible and as long as the patient benefits from treatment
•stratum 5: newly diagnosed or relapsed patients with secondary advanced MDS, occurring after chemotherapy, radiotherapy and or stem-cell transplantation, or secondary cases after prior treatment for aplastic anemia.

Exclusion Criteria

•Other serious illnesses or medical conditions
•Genetic abnormalities indicative of AML
• JMML patients in whom a diagnosis of Noonan syndrome is suspected based on clinical history and/or presenting symptoms
• Patients with secondary MDS with underlying bone-marrow failure syndromes or with familial MDS
•Isolated extramedullary disease
•Symptomatic CNS-involvement
•Current uncontrolled infection
•Cardiac toxicity (shortening fraction below 28%)
•Concurrent treatment with any other anti-cancer therapy is not allowed
•Pregnant or lactating patients
•Patients who cannot be regularly followed up for psychological, social, familial or geographic reasons
•Patient with expected non compliance to toxicity management guidelines
•Prior treatment with a demethylating agent
•Allergy to azicitidine or mannitol.

Study & Design

• Study Type: interventional
• Study Design: Not specified