Selinexor Plus High-Dose Melphalan (HDM) Before Autologous Hematopoietic Cell Transplantation for Multiple Myeloma
Primary Purpose
Multiple Myeloma
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Selinexor
Melphalan
Dexamethasone
Autologous Hematopoietic Cell Transplantation (HCT)
Fosaprepitant
Sponsored by
About this trial
This is an interventional treatment trial for Multiple Myeloma focused on measuring autologous hematopoietic cell transplantation (HCT), high-dose melphalan, selinexor
Eligibility Criteria
Inclusion Criteria:
- 18 years of age or older with histologically confirmed multiple myeloma
- Achieving partial response (PR) or very good partial response (VGPR) with systemic chemotherapy
- Received less than 4 lines of anti-myeloma therapy.
- Karnofsky performance status of >= 70%
- Adequate pulmonary, cardiac, hepatic and renal function as outlined in the protocol
- Signed informed consent form in accordance with institutional policies prior to the initiation of high-dose therapy
Exclusion Criteria:
- Non-secretory multiple myeloma
- Have achieved complete response (CR) prior to autologous hematopoietic cell transplantation (HCT)
- Central nervous system (CNS) involvement
- Uncontrolled bacterial, viral or fungal infections
- Myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.
- Prior malignancies within the last 5 years except resected basal cell carcinoma or treated cervical carcinoma in situ.
- Females who are pregnant or breastfeeding
- Have received other investigational drugs within 14 days prior to screening
- Prior autologous or allogeneic HCT
- Prior organ transplant or autoimmune disease requiring immunosuppressive therapy
Sites / Locations
- H. Lee Moffitt Cancer Center and Research Institute
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Selinexor Plus HDM HCT
Arm Description
The conditioning regimen begins 3 days prior to autologous transplant. Day 0 is the day of the autologous hematopoietic cell transplant. Melphalan will be given intravenously (IV) on Day -3 and Day -2; Dexamethasone will be given through via IV on Day -3, Day -2 and Day -1; fosaprepitant at 150 IV on days -3 and -2 will be given to patients an an antiemetic.Selinexor will be taken by mouth (PO) daily on the same day participants receive chemotherapy with melphalan.
Outcomes
Primary Outcome Measures
Phase I: Recommended Phase II Dose (RPh2D)
RPh2D/Maximum Tolerated Dose (MTD) of Selinexor when used in combination with high-dose melphalan as a conditioning regimen for hematopoietic cell transplant. MTD: the highest dose level at which 1 or less of 6 participants experience a dose limiting toxicity (DLT).
Complete Response (CR)
Complete response (CR) conversion rate. CR: Negative immunofixation of serum and urine, disappearance of any soft tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow.
tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow.
Complete Response conversion rate. CR: Negative immunofixation of serum and urine, disappearance of any soft tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow.
Secondary Outcome Measures
Full Information
NCT ID
NCT02780609
First Posted
May 20, 2016
Last Updated
November 2, 2022
Sponsor
H. Lee Moffitt Cancer Center and Research Institute
Collaborators
Karyopharm Therapeutics Inc
1. Study Identification
Unique Protocol Identification Number
NCT02780609
Brief Title
Selinexor Plus High-Dose Melphalan (HDM) Before Autologous Hematopoietic Cell Transplantation for Multiple Myeloma
Official Title
Phase 1/2 Investigator Sponsored Study of Selinexor in Combination With High-Dose Melphalan Before Autologous Hematopoietic Cell Transplantation for Multiple Myeloma
Study Type
Interventional
2. Study Status
Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
July 20, 2017 (Actual)
Primary Completion Date
February 20, 2021 (Actual)
Study Completion Date
February 23, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
H. Lee Moffitt Cancer Center and Research Institute
Collaborators
Karyopharm Therapeutics Inc
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Phase I: The primary purpose of this study phase is to determine the best dose also referred to as the maximum tolerated dose (MTD) of Selinexor when used in combination with high-dose melphalan as a conditioning regimen for hematopoietic cell transplant.
Phase II: The primary purpose of this study phase is to assess the complete response (CR) conversion rate.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Multiple Myeloma
Keywords
autologous hematopoietic cell transplantation (HCT), high-dose melphalan, selinexor
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
22 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Selinexor Plus HDM HCT
Arm Type
Experimental
Arm Description
The conditioning regimen begins 3 days prior to autologous transplant. Day 0 is the day of the autologous hematopoietic cell transplant. Melphalan will be given intravenously (IV) on Day -3 and Day -2; Dexamethasone will be given through via IV on Day -3, Day -2 and Day -1; fosaprepitant at 150 IV on days -3 and -2 will be given to patients an an antiemetic.Selinexor will be taken by mouth (PO) daily on the same day participants receive chemotherapy with melphalan.
Intervention Type
Drug
Intervention Name(s)
Selinexor
Other Intervention Name(s)
KPT-330
Intervention Description
Selinexor will be given orally 2 to 3 hours prior to high dose-melphalan IV infusion. Phase I: Dose escalation beginning with 40 mg to determine the recommended Phase II dose (RPh2D). Phase II: Treatment at RPh2D.
Intervention Type
Drug
Intervention Name(s)
Melphalan
Other Intervention Name(s)
Alkeran
Intervention Description
Melphalan 100 mg/m^2 IV over 30-45 minutes.
Intervention Type
Drug
Intervention Name(s)
Dexamethasone
Other Intervention Name(s)
Decadron
Intervention Description
Dexamethasone 20 mg PO (or IV) daily (on days -3, -2 and -1).
Intervention Type
Procedure
Intervention Name(s)
Autologous Hematopoietic Cell Transplantation (HCT)
Intervention Description
Participant's own stem cells are collected from their blood, frozen, then given back to them after chemotherapy.
Intervention Type
Drug
Intervention Name(s)
Fosaprepitant
Other Intervention Name(s)
antiemetic agent, Standare of Care
Intervention Description
Fosaprepitant at 150 mg IV on days -3 and -2.
Primary Outcome Measure Information:
Title
Phase I: Recommended Phase II Dose (RPh2D)
Description
RPh2D/Maximum Tolerated Dose (MTD) of Selinexor when used in combination with high-dose melphalan as a conditioning regimen for hematopoietic cell transplant. MTD: the highest dose level at which 1 or less of 6 participants experience a dose limiting toxicity (DLT).
Time Frame
Up to 3 months
Title
Complete Response (CR)
Description
Complete response (CR) conversion rate. CR: Negative immunofixation of serum and urine, disappearance of any soft tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow.
tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow.
Complete Response conversion rate. CR: Negative immunofixation of serum and urine, disappearance of any soft tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow.
Time Frame
3 months post HCT
Other Pre-specified Outcome Measures:
Title
Phase 1 and Phase 2 Percentage of Participants Treated at Dose Level 3/RP2D With Progression Free Survival (PFS)
Description
Progression Free Survival defined as the time from start of treatment to the time of progression or death.
Time Frame
at 24 months
Title
Overall Survival (OS)
Description
Rate of participants' survival at time of evaluation.
Time Frame
at 24 months
Title
Rate of Minimal Residual Disease (MRD)
Description
Rate of participants who did not have Minimal Residual Disease (MRD) as assessed by flow cytometry.
Time Frame
3 months post HCT
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
18 years of age or older with histologically confirmed multiple myeloma
Achieving partial response (PR) or very good partial response (VGPR) with systemic chemotherapy
Received less than 4 lines of anti-myeloma therapy.
Karnofsky performance status of >= 70%
Adequate pulmonary, cardiac, hepatic and renal function as outlined in the protocol
Signed informed consent form in accordance with institutional policies prior to the initiation of high-dose therapy
Exclusion Criteria:
Non-secretory multiple myeloma
Have achieved complete response (CR) prior to autologous hematopoietic cell transplantation (HCT)
Central nervous system (CNS) involvement
Uncontrolled bacterial, viral or fungal infections
Myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.
Prior malignancies within the last 5 years except resected basal cell carcinoma or treated cervical carcinoma in situ.
Females who are pregnant or breastfeeding
Have received other investigational drugs within 14 days prior to screening
Prior autologous or allogeneic HCT
Prior organ transplant or autoimmune disease requiring immunosuppressive therapy
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Taiga Nishihori, M.D.
Organizational Affiliation
H. Lee Moffitt Cancer Center and Research Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
H. Lee Moffitt Cancer Center and Research Institute
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
33023948
Citation
Turner JG, Cui Y, Bauer AA, Dawson JL, Gomez JA, Kim J, Cubitt CL, Nishihori T, Dalton WS, Sullivan DM. Melphalan and Exportin 1 Inhibitors Exert Synergistic Antitumor Effects in Preclinical Models of Human Multiple Myeloma. Cancer Res. 2020 Dec 1;80(23):5344-5354. doi: 10.1158/0008-5472.CAN-19-0677. Epub 2020 Oct 6.
Results Reference
derived
Links:
URL
https://moffitt.org/clinical-trials-research/
Description
Moffitt Cancer Center Clinical Trials website
Learn more about this trial
Selinexor Plus High-Dose Melphalan (HDM) Before Autologous Hematopoietic Cell Transplantation for Multiple Myeloma
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