Clinical Trial: Study of Oral IXAZOMIB in Adult Patients With Relapsed or Refractory Light Chain Amyloidosis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: An Open-Label, Dose-Escalation, Phase 1 Study of the Oral Formulation of IXAZOMIB (MLN9708) Administered Weekly in Adult Patients With Relapsed or Refractory Light-Chain Amyloido

Brief Summary: This study will include patients with previously treated systemic relapsed or refractory light-chain (AL) amyloidosis who require further therapy and will be aimed at determining the safety profile and the maximum tolerated dose/recommended phase 2 dose of IXAZOMIB administered orally.

Detailed Summary:
Sponsor: Millennium Pharmaceuticals, Inc.

Current Primary Outcome:

  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: From start of study through 30 days after last dose or until initiation of subsequent antineoplastic therapy ]
  • Maximum Tolerated Dose and Recommended phase 2 dose of MLN9708 [ Time Frame: Dose Limiting Toxcities determined in Cycle 1 and adverse events monitored throughout the study will inform the recommended phase 2 dose ]
    Based on toxicity and efficacy outcomes


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Whole blood 20S proteasome activity profile and inhibition parameters of multiple-dose oral MLN9708 [ Time Frame: Days 1,2,8,15,16,22 ]
    Plasma pharmacokinetic and whole blood pharmacodynamic effect of MLN9708
  • Number of patients with hematologic response rate during study period [ Time Frame: Duration of treatment and then every 6 weeks thereafter until disease progression or initiation of subsequent antineoplastic therapy ]
    Complete response, very good partial response, partial response
  • Time to and duration of organ response and organ improvement [ Time Frame: After cycles 3,6,9,12; every 6 months thereafter until disease progression or initiation of subsequent antineoplastic therapy ]
    According to standardized criteria
  • Time to hematologic and organ disease progression [ Time Frame: Until evidence of hematologic and/or disease progression ]
  • Time from the date of the first dose of MLN9708 to the date of confirmed organ or hematologic disease progression, respectively [ Time Frame: Every 6 weeks from end of treatment until disease progression or initiation of subsequent antineoplastic therapy ]
    Progression free survival
  • Patient survival probability at 1 year after the first dose of MLN9708 [ Time Frame: Every 12 weeks after progressive disease or initiation of subsequent antineoplastic therapy ]


Original Secondary Outcome: Same as current

Information By: Takeda

Dates:
Date Received: March 15, 2011
Date Started: May 2011
Date Completion: December 2017
Last Updated: March 9, 2017
Last Verified: March 2017