NCT01718366

A Study of Combined Deferasirox, Vitamin D and Azacytidine in High Risk MDS

Phase I-II Study of Association of Deferasirox, Vitamin D and Azacytidine as Treatment of High Risk MDS (IPSS Int-2 and High)

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 50 (actual)
• Sponsor: Groupe Francophone des Myelodysplasies · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Determinate safety and response rate of the association Deferasirox -Vitamine D - Azacitidine in treatment of high risk MDS Deferasirox Exjade: The dose of Deferasirox will be assigned according to the ferritin level. Dose escalation is scheduled during the phase I, with 5 additional patients per group. The maximal tolerated dose of Deferasirox will be required for the phase II of the study. The first dose will be assigned according to the ferritin level of the patient at time of inclusion: 5 mg/kg/d if the ferritin is \>300ng/ml and \< 1000ng/ml in Group 1 10 mg/kg/d if the ferritin is ≥1000ng/ml) in Group 2 Group 1 : Ferritin 300 to 1000ng /ml: * cohort 1 : 5 mg/kg/d * cohort 2 : 10mg/kg/d * cohort 3 : 15 mg/kg/d Group 2 : Ferritin \> 1000ng /ml: * cohort 1 : 10 mg/kg/d * cohort 2 : 15mg/kg/d * cohort 3 : 20 mg/kg/d 5 patients will be treated by cohort. In absence of toxicity (extra-hematological toxicity grade 3 or 4 or hematological grade 4), 5 additional patients will be included in the next cohort. Deferasirox will be administrated once daily during all the study period. Uvedose will be administrated once weekly during all the study period (100.000 UI P.O). Azacitidine will be administrated sc at 75 mg/m²/d, during 7 days, J1 to J7 of each cycles(One cycle is 28 days) During phase I and II, Deferasirox will always be associated with Vitamin D and Azacitidine Patients will be received 6 cycles of treatment (except if progression, unacceptable toxicity or withdrawn of patients occured) After 3 and 6 cycles, an evaluation will be done to evaluate the efficacy of the treatment. No dose modification of deferasirox will be done after 3 cycles of treatment except in case of progression). After 6 cycles, patients with CR, PR, marrow CR or HI will be treated with the same dose of Deferasirox until progression .

Detailed description

Deferasirox will be administrated once daily in the morning on an empty stomach, 30 minutes before meal. Deferasirox will be stopped if the ferritin level is under 100 ng/ml,and could be restarted is the ferritin level increase to 200 ng/ml Uvedose dose could be adjusted according to the phosphocalcic metabolism parameters and the plasma Vitamin D3 level.

Conditions

• MDS

Interventions

Type	Name	Description
DRUG	Deferasirox, Vitamin D and Azacitidine	association of Deferasirox (group 1: 5-10-15/mg/kg/day according to dose level group), Vitamine D (100000U/week) and Azacitidine (75 mg/kg/day day1-day7)

Timeline

• Start date: 2013-02-01
• Primary completion: 2019-02-01
• Completion: 2019-02-01
• First posted: 2012-10-31
• Last updated: 2018-01-10

Locations

15 sites across 2 countries: Belgium, France