Study on the Effect of Ibrutinib on High Risk Smoldering Multiple Myeloma Patients
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|ClinicalTrials.gov Identifier: NCT02943473|
Recruitment Status : Recruiting
First Posted : October 24, 2016
Last Update Posted : March 22, 2019
The purpose of this research study is to test whether the drug ibrutinib (trademark name: IMBRUVICA®) is effective at preventing the development of multiple myeloma in people who currently have smoldering myeloma. The researchers conducting this trial) have reason to believe that ibrutinib can delay the development of multiple myeloma, thus giving people who currently have smoldering myeloma a longer period of time when they feel healthy and well.
Smoldering myeloma is an abnormal condition that is considered to be an early phase of the disease multiple myeloma. In this disorder, there is an abnormal growth of plasma cells, which is a type of blood cell found in the bone marrow. This growth is not as severe in people with smoldering myeloma as it is in multiple myeloma, so people with smoldering myeloma do not have any symptoms and tend to feel well. However, they have a higher risk of developing multiple myeloma than people in the general population.
Some people with smoldering myeloma are at an especially high risk of developing myeloma - 50% of these people will develop multiple myeloma 2 years after they are diagnosed with smoldering myeloma. The investigators identify these people by looking at the amount of myeloma in the bone marrow (called "bone marrow plasma cell percentage") and the amount of myeloma protein (called "serum protein electrophoresis" and "serum free light chain assay") in the blood. To be considered high risk, individuals must have highly abnormal levels for these tests.
Based upon current guidelines, people with smoldering myeloma do not require any treatment. However, known is that many of these people will develop multiple myeloma in the near future. Currently there have been no proven and effective way of preventing these people from developing multiple myeloma, which remains an incurable disease.
|Condition or disease||Intervention/treatment||Phase|
|High Risk Smoldering Multiple Myeloma||Drug: Ibrutinib||Phase 2|
This is a phase 2, open-label, single center, prospective pilot study designed to assess the efficacy of ibrutinib in subjects with high risk smoldering multiple myeloma.
All enrolled subjects will be treated with ibrutinib 560 mg (4 capsules, each containing 140 mg) taken PO daily for 12 cycles (28 days each). If a subject demonstrates benefit from ibrutinib, therapy may be extended beyond 12 cycles to a maximum of 2 years. Subjects who progress and meet criteria for symptomatic multiple myeloma will be withdrawn from study.
An initial cohort of 15 subjects will be accrued. If 4 or more patients progress to symptomatic myeloma in one year, then the study will be reviewed with the FDA to determine whether to employ a higher dose of ibrutinib, or to stop for futility. Otherwise, 21 additional patients will be accrued for a total sample size of 36.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||36 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2 Study of the Effect of the Bruton's Tyrosine Kinase Inhibitor Ibrutinib on Disease Response in Patients With High Risk Smoldering Multiple Myeloma|
|Study Start Date :||November 2016|
|Estimated Primary Completion Date :||December 2023|
|Estimated Study Completion Date :||December 2023|
Ibrutinib (trademark name is IMBRUVICA®). 560 mg dose administered on a continuous basis
Ibrutinib is a type of drug called a "kinase inhibitor." Kinases are proteins inside cells that help cells live and grow. Ibrutinib blocks a specific kinase protein in our bodies. This protein is thought to be very important in helping blood cancer cells live and grow. By blocking this kinase protein, ibrutinib stops cancer cells from growing.
Other Name: IMBRUVICA
- Number of patients without symptomatic myeloma [ Time Frame: up to 1 year ]Disease response - the proportion of patients with high risk smoldering multiple myeloma who do not progress to symptomatic myeloma as defined by the IMWG.
- Overall Response Rate [ Time Frame: up to 1 year ]Overall response rate, defined as partial response or better per IMWG criteria. (IMWG response criteria are - Complete Response, Very good partial response, partial response, Minimal response, stable disease, and progressive disease)
- Bone Density Changes [ Time Frame: baseline and one year ]Changes in bone density, particularly in patients with osteopenia (defined as T-score on bone densitometry testing (DEXA) of -1 to -2.5).
- PET-MRI Changes [ Time Frame: baseline and one year ]Changes in PET-MRI, particularly in patients with osteopenia
- Change in Serum interleukin-6 (IL-6) [ Time Frame: baseline and up to one year ]Bone Related Biomarker Changes
- Change in Serum stromal cell-derived factor-1 (SDF-1) [ Time Frame: baseline and up to one year ]Bone Related Biomarker Changes
- Change in Serum receptor activator of nuclear-factor kappa B ligand (RANKL) [ Time Frame: baseline and up to one year ]Bone Related Biomarker Changes
- Change in Serum macrophage inflammatory protein-1α (MIP-1α) [ Time Frame: baseline and up to one year ]Bone Related Biomarker Changes
- Change in Serum Dickkopf-1 (DKK-1) [ Time Frame: baseline and up to one year ]Bone Related Biomarker Changes
- Change in Serum C-terminal telopeptide (CTX) [ Time Frame: baseline and up to one year ]Bone Related Biomarker Changes
- Change in Urine N-terminal telopeptide (NTx) [ Time Frame: baseline and up to one year ]Bone Related Biomarker Changes
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): NCT02943473
|Contact: Lisa La, MSemail@example.com|
|Contact: Ajai Chari, MDfirstname.lastname@example.org|
|United States, New York|
|Icahn School of Medicine at Mount Sinai||Recruiting|
|New York, New York, United States, 10029|
|Contact: Ajai Chari, MD 212-241-7873 email@example.com|
|Principal Investigator: Ajai Chari, MD|
|Principal Investigator:||Ajai Chari, MD||Icahn School of Medicine at Mount Sinai|