Study Stopped
Poor accrual
Idelalisib for Immunoglobulin M (IgM)-Associated Primary (AL) Amyloidosis
Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor, Idelalisib (GS-1101), in IgM-Associated AL Amyloid
1 other identifier
interventional
1
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jan 2016
Shorter than P25 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 9, 2015
CompletedFirst Posted
Study publicly available on registry
October 29, 2015
CompletedStudy Start
First participant enrolled
January 1, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 27, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
March 27, 2017
CompletedResults Posted
Study results publicly available
May 10, 2017
CompletedSeptember 21, 2017
August 1, 2017
1.2 years
September 9, 2015
March 30, 2017
August 23, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Overall Response
Evaluate hematologic response according to standard criteria
3 months
Secondary Outcomes (4)
Progression Free Survival
1 year
Organ Response
3 months
Evaluate Safety and Tolerability of Agent
3 months
Quality of Life
3 months
Study Arms (1)
Idelalisib
EXPERIMENTALIdelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.
Interventions
Idelalisib daily until unacceptable toxicity or disease progression.
Eligibility Criteria
You may qualify if:
- IgM paraprotein identified on serum immunofixation electrophoresis OR light chain-restricted CD20+ lymphoplasmacytic population on biopsy of bone marrow or lymph node (identified by H\&E/immunohistochemistry or flow cytometry) OR positive myeloid differentiation primary response gene 88 (MYD88-L265P) OR CXCR4WHIM mutation (CXCR4 mutation - warts, hypogammaglobulinemia, infections, myelokathexis) on submitted samples
- Biopsy-proven relapsed or refractory AL amyloidosis
- Age ≥ 18 years
- Eastern Cooperative Oncology Group (ECOG) performance status \<2 (see Appendix A.)
- Difference between serum free light chains (FLC) of \>30 mg/L or quantifiable IgM paraprotein \>0.5 g/L
- Participants must have normal organ and marrow function as defined below:
- Absolute neutrophil count \> 1,000/mm3
- Platelets \> 50,000/mm3
- Ability to understand and the willingness to sign a written informed consent document.
You may not qualify if:
- Previous treatment with idelalisib
- Glomerular filtration rate (GFR) \<15 ml/min
- Cardiac biomarker Stage III disease as determined by B-type natriuretic peptide (BNP) \>100 pg/mL and Troponin-I \>0.1 ng/mL (Girnius 2014)
- alanine-aminotransferase (ALT)/aspartate aminotransferase (AST) values \>2.5x upper limit of normal, Bilirubin \>1.5 upper limit of normal (ULN)
- Central nervous system (CNS) malignancy or other active malignancy
- Lactating or pregnant women
- Exposure to another investigational drug within 4 weeks prior to start of study treatment
- Ongoing alcohol or drug addiction as determined by investigator
- Amyloid-directed therapy within the past 28 days
- History of Human Immunodeficiency Virus (HIV), active Hepatitis B Virus (HBV) (assessed by positive Hepatitis B polymerase chain reaction assay (PCR) or Hepatitis B Surface Antigen), and/or Hepatitis C Virus (HCV) infection
- t(11,14) translocation identified on bone marrow cytogenetics or by Fluorescence in situ hybridization (FISH)
- Known lytic bone lesions
- Positive cytomegaly virus (CMV) Polymerase chain reaction (PCR)
- Previously untreated AL amyloidosis (Newly diagnosed)
- Unwilling or unable to comply with the protocol
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- John Mark Sloanlead
- Gilead Sciencescollaborator
Study Sites (1)
Boston Medical Center
Boston, Massachusetts, 02118, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
Early termination leading to small numbers of subjects analyzed.
Results Point of Contact
- Title
- Mark Sloan MD
- Organization
- Boston Medical Center
Study Officials
- PRINCIPAL INVESTIGATOR
John "Mark" Sloan, MD
Boston Medical Center
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Associate Professor of Medicine
Study Record Dates
First Submitted
September 9, 2015
First Posted
October 29, 2015
Study Start
January 1, 2016
Primary Completion
March 27, 2017
Study Completion
March 27, 2017
Last Updated
September 21, 2017
Results First Posted
May 10, 2017
Record last verified: 2017-08
Data Sharing
- IPD Sharing
- Will not share