Clinical Trial: Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (advSM) and Relapsed or Refractory Myeloid Malignancies
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 1 Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (advSM) and Relapsed or Refractory Myeloid Malignancies
Brief Summary: This is a Phase 1, open-label, dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antineoplastic activity of BLU-285, administered orally (PO), in adult patients with advanced systemic mastocytosis and other relapsed or refractory myeloid malignancies. The study consists of 2 parts, a dose-escalation part (Part 1) and an expansion part (Part 2).
Detailed Summary:
Sponsor: Blueprint Medicines Corporation
Current Primary Outcome:
- Maximum tolerated dose (MTD) of BLU-285 [ Time Frame: During cycle 1 (28 days) of treatment ]
- Number of patients with adverse and serious adverse events and changes in physical findings, vital signs, clinical laboratory results and ECG findings [ Time Frame: Approximately 24 months ]
- Recommended Phase 2 dose (RP2D) of BLU-285 [ Time Frame: Approximately 24 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Maximum plasma concentration of BLU-285 [ Time Frame: Every cycle (28 days) up to cycle 4 ]Blood samples may be taken at pre-dose, and 0.5, 1, 2, 4, 8 and 24 hrs post dose on Cycle 1 Day 1 and Cycle 1 Day 15, Pre-dose of Cycle 2 to 4, Day 1
- Time to maximum plasma concentration of BLU-285 [ Time Frame: Every cycle (28 days) up to cycle 4 ]Blood samples may be taken at pre-dose, and 0.5, 1, 2, 4, 8 and 24 hrs post dose on Cycle 1 Day 1 and Cycle 1 Day 15, Pre-dose of Cycle 2 to 4, Day 1
- Overall Response Rate [ Time Frame: 8, 24, 40, 68 and every 24 weeks until patient terminates from the study (approximately 24 months) ]Defined as either complete remission (CR) or partial remission (PR); Clinical improvement rate, Duration of response; and Progression Free Survival will be measured as per International Working Group Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European competence network on mastocytosis (ECNM) consensus response criteria
- Changes in levels of serum tryptase and levels of V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog (KIT) D816V allele burden in blood [ Time Frame: Cycle (C)1Day (D)1, C1D15, C2D1, C3D1, C5D1, C7D1, C11D1, C18D1 every 6 cycles thereafter and at disease progression. (approximately 24 months) ]
- Changes in patient reported symptoms and quality of life using the Patient Global Impression of Change (PGIC) scale [ Time Frame: Part 2 only - Day 1 of Cycles 2-12 ]Defined as change from Day 1 of Cycle 2 on a 7 point scale
- Change in patient reported quality of life using the European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C-30) [ Time Frame: Part 2 only - Day 1 of Cycles 1-12 ]Defined as change from baseline on a scale of 0-100
Original Secondary Outcome: Same as current
Information By: Blueprint Medicines Corporation
Dates:
Date Received: September 15, 2015
Date Started: December 2015
Date Completion: May 2019
Last Updated: January 3, 2017
Last Verified: January 2017