PRAN-16-52 Pracinostat in combination with Azacitidine in Adult AML
Research type
Research Study
Full title
PHASE III, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER, RANDOMIZED STUDY OF PRACINOSTAT IN COMBINATION WITH AZACITIDINE IN PATIENTS ≥18 YEARS WITH NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA UNFIT FOR STANDARD INDUCTION CHEMOTHERAPY
IRAS ID
225797
Contact name
Amit Patel
Contact email
Sponsor organisation
Helsinn Healthcare SA
Eudract number
2016-004724-34
Clinicaltrials.gov Identifier
79,597, IND Number
Duration of Study in the UK
3 years, 9 months, 25 days
Research summary
Acute Myeloid Leukemia is a type of blood cancer caused by a maturation arrest of bone marrow cells that does not allow them to become healthy cells also called blasts. The standard therapy approaches or hematopoietic stem cell transplant are often not appropriate in old patients or patients with poor healthy conditions. In US, no drugs are currently approved for this population and in EU hypomethylating agents (as Azacitidine) are available, even if the efficacy of this drug alone is modest and needs to be improved. The aim of this Phase III study is to show how effective and safe is Pracinostat used in combination with Azacitidine compared to Azacitidine alone, in patients 18 years of age or older with newly diagnosed AML and unfit to receive chemotherapy. Patients with acute promyelocytic leukemia (APL) and cytogenetic low-risk AML will be excluded. Up to 500 patients will participate in the study across approximately 140 medical centres worldwide.
Treatments will be given based on 28-day cycles, with pracinostat/placebo taken orally once every other day, 3 times a week for 3 weeks, followed by 1 week of rest. AZA will be given for 7 days at the beginning of each cycle. Study treatment will be stopped if the disease has advanced or if the patient has shown the disappearance of all signs of cancer in response to treatment (known as complete remission), but then relapses or if the patient has non-manageable toxicities. Once study treatment has permanently stopped, patients will enter a Follow-up phase of the study and will be followed up for assessment of the disease every 3 months until death.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
17/NW/0369
Date of REC Opinion
7 Aug 2017
REC opinion
Further Information Favourable Opinion