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Azacitidine

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Combination of azacitidine at maximum tolerated dose and LDE255 at dose escalation, the starting dose will be 400 mg

Azacitidine at maximum tolerated dose. LDE255 at dose escalation (200, 400 or 800 mg)

Azacitidine and LDE255

Thomas PREBET, MD

Pierre FENAUX, MD, PHD

This study is a phase Ib add-on study of the combination of LDE255 to azacitidine in patients without marrow response after at least 6 cycles of azacitidine.

Dose escalation of LDE255 will be performed over time using a rolling 6 design to determine the safety of the combination with azacitidine. The starting dose will be 400 mg. Azacitidine will be continued at maximum tolerated dose (MTD). Evaluation will be based on cycle 1 toxicity profile. Once MTD will be determined, 20 additional patients will be treated at recommended dose.

A Study of LDE255 in Combination With Azacitidine for High Risk Myelodysplastic Syndrome Patients

A Single-arm Dose Finding Phase Ib Multicenter Study of the Oral Smoothened Antagonist LDE255 in Combination With Azacitidine for High Risk Myelodysplastic Syndrome Patients

Overall survival measured from the date of enrollment to death or the date of last contact

Novartis

Groupe Francophone des Myelodysplasies

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
A Study of LDE255 in Combination With Azacitidine for High Risk Myelodysplastic Syndrome Patients	NCT02323139	MDS	Azacitidine and...	18 Years -	Groupe Francophone des Myelodysplasies
A Study of LDE255 in Combination With Azacitidine for High Risk Myelodysplastic Syndrome Patients	NCT02323139	MDS	Azacitidine and...	18 Years -	Groupe Francophone des Myelodysplasies

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