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Funding withdrawn
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| Name | Class |
|---|---|
| Karyopharm Therapeutics Inc | INDUSTRY |
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The purpose of this study is to test the safety and efficacy of Selinexor and Dexamethasone and see what effects it has on AL amyloidosis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| selinexor/ dexamethasone (Sd) | Experimental | Selinexor • 60mg PO once weekly on days 1, 8, 15, 22 until disease progression or toxicity Dexamethasone • 20 mg PO administered 30-60 minutes prior to selinexor on days 1, 2, 8, 9, 15, 16, 22, 23 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Selinexor | Drug | Selinexor will be given orally at a dose of 60mg once weekly on the first day of the week (day 1, 8, 15, and 22) for Cycle 1 and up to Cycle 12. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Compare number of dose limiting toxicity (DLT) occurence to measure safety and toxicity | Safety and toxicity will be determined by how many occurrence of dose limiting toxicity (DLT) occured during 12 months of treatment for each subject | approximately 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Compare Hematologic Overall Response Rate (ORR) | Comparing proportion of subjects with Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR), No Response (NR) and Progressive Disease (PD) | approximately 12 months |
| Hematologic Very Good Partial Response (VGPR) or better rate |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Cara Rosenbaum, MD | Weill Medical College of Cornell University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Weill Cornell Medicine - Multiple Myeloma Center | New York | New York | 10065 | United States |
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| ID | Term |
|---|---|
| D000544 | Alzheimer Disease |
| D000686 | Amyloidosis |
| D000075363 | Immunoglobulin Light-chain Amyloidosis |
| ID | Term |
|---|---|
| D003704 | Dementia |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| C585161 | selinexor |
| D003907 | Dexamethasone |
| ID | Term |
|---|---|
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
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| Dexamethasone | Drug | Dexamethasone will be given orally at a dose of 20mg, if tolerated, or at a reduced dose if required, 30 to 60 minutes prior to selinexor for first 2 days of each week only (days 1, 2, 8, 9, 15, 16, 22, and 23 of each cycle). |
|
Percentage of subjects that achieved Very Good Partial Response (VGPR) or better response |
| approximately 12 months |
| Hematologic Complete Response (CR) rate | Percentage of subjects that achieved Complete Response (CR) | approximately 12 months |
| Stringent dFLC response rate | Percentage of subjects that has stringent dFLC (dFLC is defined as the difference in involved amyloidogenic and uninvolved serum-free light chains). | approximately 12 months |
| Number of patients with peripheral blood mass spectrometry for monoclonal protein detection (MALDI-TOF) | For each subject, detection of monoclonal protein will be analyzed at screening, day 1 of each cycle (only when in ≥VGPR), at each end of treatment (EOT) visits, and day 1 of each post-treatment follow, up until PD or 24 months, whichever occurs first. | End of Study (approximately 3 years) |
| minimal residual disease (MRD) negative CR/VGPR rate | Percentage of subjects that is MRD negative when in complete response (CR) and Very Good Partial Response (VGPR) | approximately 12 months |
| Percentage of participants with organ response | Organ response for kidney and cardiac is defined as the proportion of baseline organ involved participants who achieve organ response in each corresponding organ. Organ response defined for cardiac: N-terminal brain pronatriuretic peptide (NT-proBNP) response (> 30% and > 300 nanogram per liter [ng/L] decrease in participants with baseline NT-proBNP >= 650 ng/L) or New York Heart Association (NYHA) class response (>= 2 class decrease in participants with baseline NYHA class 3 or 4); for kidney: decrease in proteinuria by >=30% or below 0.5 grams /24 hours without renal progression. | End of Study (approximately 3 years) |
| Median hematologic Progression Free Survival (PFS) | Median estimate of months that participants have progression free survival and median estimate is calculated using the Kaplan-Meier methodology | End of Study (approximately 3 years) |
| Time to first hematologic response | Measured in months between the date of enrollment and the first efficacy evaluation at which the participant has met the criteria for hematologic complete response. | approximately 12 months |
| Time to best hematologic response | Measured in months between the date of enrollment and the best hematologic response the subject achieved while on treatment | approximately 12 months |
| Time to hematologic progression | Measured in months between the date of enrollment and the first efficacy evaluation at which the participant has met the criteria for hematologic progression | End of Study (approximately 3 years) |
| Duration of hematologic response | Duration of hematologic response is defined as the time between the date of initial documentation of hematologic response (i.e. Complete Response, Very Good Partial Response, Partial Response, No Response and Progressive Disease) to the date of the next hematologic response | End of Study (approximately 3 years) |
| Time to next therapy | Measured in months from the date of enrollment to the start date of subsequent treatment for AL amyloidosis | End of Study (approximately 3 years) |
| Median organ Progression Free Survival (PFS) | Measured in months between the date of enrollment and the first efficacy evaluation at which the participant has met the criteria for hematologic complete response. | End of Study (approximately 3 years) |
| Time to organ response | Measured in months between the date of enrollment and the organ response | approximately 12 months |
| Time to organ progression | Measured in months between the date of organ response to organ progression | End of Study (approximately 3 years) |
| Duration of organ response | Measured in months between the date of initial documentation of organ response to organ progression | End of Study (approximately 3 years) |
| D024801 |
| Tauopathies |
| D019636 | Neurodegenerative Diseases |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D054219 | Neoplasms, Plasma Cell |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D010265 | Paraproteinemias |
| D000072473 |
| Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |