BMN 673 in Patients with Advanced Hematological Malignancies

• Research type

  Research Study

• Full title

  Phase 1, Two-arm, Open-label Study of Once Daily, Oral BMN 673 in Patients With Advanced Hematological Malignancies

• IRAS ID

  76986

• Contact name

  Ghulam Mufti

• Sponsor organisation

  BioMarin Pharmaceutical Inc.

• Eudract number

  2010-023964-42

• ISRCTN Number

  Unknown

• Research summary

  The purpose of this study is to test the safety of a new experimental oral study drug called BMN 673 and see what effects it has on patients and their cancer.patients will be enrolled into one of two areas depending on their disease. patients with Acute Myeloid Leukaemia (AML) or Myelodysplastic Syndrome (MDS) will be enrolled into Arm 1 and patients with Chronic Lymphocytic Leukaemia (CLL) or Mantle Cell Lymphoma (MCL) will be enrolled into Arm 2. The study is designed in two parts; Part 1 Dose Escalation and Part 2 Dose Expansion. Part 1 Dose Escalation: Part 1 of the study is designed to determine the safest dose to be given. Dose Escalation will begin with a 21 day cycle starting in the morning of Day 1 Cycle 1 when the patient will receive the initial dose of BMN 673. The patient will be observed for 10 hrs in which time they will have blood tests and vital signs taken to determine drug safety. The first patient at each dose level must complete 7 days of continuous dosing prior to 2 additional patients to be treated at that dose level. Once Cycle 1 is complete the patient will go on to Cycle 2 at the same dose level, which will consist of 21 days on a daily dose of BMN 673. During these 21 day cycles patients are monitored and tested formsafety. After 3 patients have completed through Cycle 1 Day 21, safety data will be reviewed by the sponsor and investigators to assess the next dose level of BMN 673 in patients These patient cycles will continue in cohorts of 3 patients receiving increasing doses of BMN 673 until the maximum tolerated dose (MTD) is determined.Part 2 Dose Expansion: will start once the MTD has been identified. This will consist of approximately 40 patients receiving the same dose level of BMN 673 in 21 day cycles starting with Cycle 1 Day 1. The purpose of Dose Expansion is to further assess safety at a single dose level.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  11/LO/1130

• Date of REC Opinion

  6 Oct 2011

• REC opinion

  Further Information Favourable Opinion