Conditions: Acute Myeloid Leukemia Leukemia Myelodysplastic Syndrome AML MDS
Keywords: RTK kinase inhibitor tyrosine acute FLT3 AC220 pharmacokinetic pharmacokinetics PK pharmacodynamic pharmacodynamics mutations PD receptor class III relapsed refractory t(8;21) q22;q22 AML1/ETO t(16;16 p13;q22 CBFbeta/MYH11 inv(16) p13q22 11q23 dysplasia myeloid myelomonocytic monoblastic monocytic erythroid erythroleukemia megakaryoblastic basophilic panmyelosis myelofibrosis
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 76
Healthy volunteers: no

Brief summary:
Patients received oral AC220 daily for 14 days to study the side effects, tolerability and best dose for treating relapsed or refractory acute myeloid leukemia, regardless of FLT3 status.

Detailed description:
This is a multi-center clinical study conducted in the USA and two international sites. This open-label, dose escalation study was designed to characterize the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of orally administered AC220 as a single agent given daily for 14 days. Cohorts of 3 patients received AC220 until dose limiting toxicity was noted (DLT). At that point cohorts expanded to 6 patients until MTD was determined. Patients not experiencing DLT or significant disease progression at Day 15 may have continued receiving AC220 at the discretion of the Investigator and Sponsor. FLT3 positive and negative patients were allowed to participate.

Arms:
arm 1: Determine safety, tolerability and pharmacokinetic (PK) parameters of AC220

Interventions: intervention 1: AC220
Intervention descriptions: intervention 1: Powder in bottle formulation supplied as 50mg or 350 mg in glass, crimped serum vials. Requires reconstitution by a pharmacist, and must be stored securely and protected from light.

Protocol document: