Idelalisib for Immunoglobulin M (IgM)-Associated Primary (AL) Amyloidosis
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|ClinicalTrials.gov Identifier: NCT02590588|
Recruitment Status : Terminated (Poor accrual)
First Posted : October 29, 2015
Results First Posted : May 10, 2017
Last Update Posted : September 21, 2017
|Condition or disease||Intervention/treatment||Phase|
|Amyloidosis||Drug: Idelalisib||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||1 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor, Idelalisib (GS-1101), in IgM-Associated AL Amyloid|
|Actual Study Start Date :||January 2016|
|Actual Primary Completion Date :||March 27, 2017|
|Actual Study Completion Date :||March 27, 2017|
Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.
Idelalisib daily until unacceptable toxicity or disease progression.
Other Name: GS-1101
- Overall Response [ Time Frame: 3 months ]Evaluate hematologic response according to standard criteria
- 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.
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02590588
|United States, Massachusetts|
|Boston Medical Center|
|Boston, Massachusetts, United States, 02118|
|Principal Investigator:||John "Mark" Sloan, MD||Boston Medical Center|