Clinical Trial: Idelalisib for Immunoglobulin M (IgM)-Associated Primary (AL) Amyloidosis

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor, Idelalisib (GS-1101), in IgM-Associated AL Amyloid

Brief Summary: The investigators expect to enroll 15 participants with relapsed or refractory IgM-associated AL amyloidosis onto this Phase II clinical trial. Idelalisib will be self-administered orally at a dose of 100 mg twice daily (may be increased to 150 mg (one tablet) twice daily after 3 months at investigator discretion). Participants will be treated until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression.

Detailed Summary:

This study includes the use of Idelalisib to treat previously treated patients with IgM-associated AL Amyloidosis at Boston Medical Center. Boston Medical Center is internationally recognized as a leader in amyloidosis research and patient care through the activities of the multidisciplinary Amyloid Center at Boston University. The problematic cell in most forms of AL amyloidosis shares similarities with multiple myeloma. However, in the small subset of AL Amyloidosis patients with an IgM paraprotein, the cells are more typically related to lymphoplasmacytic lymphoma or Waldenstrom's macroglobulinemia. Because clonal cluster of differentiation antigen 20 (CD20)+ lymphoplasmacytic cells are usually responsible for IgM paraproteins, treatment paradigms based on Waldenstrom's macroglobulinemia (WM) may be more appropriate than myeloma-based strategies. Idelalisib has been shown to be active and well tolerated in patients with relapsed/refractory non-Hodgkin lymphoma including chronic lymphocytic lymphoma, and lymphoplasmacytic lymphoma with or without Waldenström's macroglobulinemia (WM). The side effect profile of idelalisib merges well with the known predisposition to toxicity of amyloidosis patient.

The investigators expect to enroll 15 participants with IgM-associated AL amyloidosis onto this Phase II clinical trial. Idelalisib will be self-administered orally at a dose of 100 mg (1 tablet) twice daily (may be escalated to 150 mg (one tablet) twice daily after 3 months at investigator discretion). Participants will be treated until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression.


Sponsor: John Mark Sloan

Current Primary Outcome: Overall Response [ Time Frame: 3 months ]

Evaluate hematologic response according to standard criteria


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Progression Free Survival [ Time Frame: 1 year ]
    Evaluate time to progression
  • Organ Response [ Time Frame: 3 months ]
    Number of patients with organ response using standard AL amyloidosis criteria (protocol section 8.4)
  • Evaluate Safety and Tolerability of Agent [ Time Frame: 3 months ]
    Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v4.0
  • Quality of Life [ Time Frame: 3 months ]
    Evaluate quality of life according to Functional Assessment of Cancer Therapy Lymphoma Subscale (FACT-Lym) assessment tool


Original Secondary Outcome:

  • Progression Free Survival [ Time Frame: 1 year ]
    Evaluate time to progression
  • Number of patients with organ response using standard AL amyloidosis criteria (protocol section 8.4) [ Time Frame: 3 months ]
    Evaluate organ response according to standard criteria
  • Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v4.0 [ Time Frame: 3 months ]
    Evaluate safety and tolerability of agent
  • Quality of Life [ Time Frame: 3 months ]
    Evaluate quality of life according to FACT-Lym assessment tool


Information By: Boston Medical Center

Dates:
Date Received: September 9, 2015
Date Started: January 2016
Date Completion:
Last Updated: March 30, 2017
Last Verified: March 2017