Multiple MyelomaPhase 1/2NCT02780609
Selinexor Plus High-Dose Melphalan (HDM) Before Autologous Hematopoietic Cell Transplantation for Multiple Myeloma
A Phase 1/2 study testing Selinexor, Melphalan and Dexamethasone in people with Multiple Myeloma, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- Selinexor, Melphalan and Dexamethasone
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Jul 2017to 23 Feb 2021
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Show all 10Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612
About this study
Official title: Phase 1/2 Investigator Sponsored Study of Selinexor in Combination With High-Dose Melphalan Before Autologous Hematopoietic Cell Transplantation for Multiple Myeloma
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.
- Conditions
- Multiple Myeloma
- Being tested
- Selinexor, Melphalan, Dexamethasone, Autologous Hematopoietic Cell Transplantation (HCT), Fosaprepitant
- Keywords
- autologous hematopoietic cell transplantation (HCT), high-dose melphalan, selinexor
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelinexorDrug
Also called KPT-330
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.
MelphalanDrug
Also called Alkeran
Melphalan 100 mg/m^2 IV over 30-45 minutes.
DexamethasoneDrug
Also called Decadron
Dexamethasone 20 mg PO (or IV) daily (on days -3, -2 and -1).
Autologous Hematopoietic Cell Transplantation (HCT)Procedure or surgery
Participant's own stem cells are collected from their blood, frozen, then given back to them after chemotherapy.
FosaprepitantDrug
Also called antiemetic agent, Standare of Care
Fosaprepitant at 150 mg IV on days -3 and -2.
| Study group | Role | What they receive |
|---|---|---|
| Selinexor Plus HDM HCT | Experimental | 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. Receives: Drug: Selinexor; Drug: Melphalan; Drug: Dexamethasone; Procedure: Autologous Hematopoietic Cell Transplantation (HCT); Drug: Fosaprepitant |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Up to 3 months
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.
Measured: 3 months post HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2016 (estimated)
- Study start20 Jul 2017
- Primary completion20 Feb 2021
- Study completion23 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Karyopharm Therapeutics Inc
- Responsible party
- Sponsor
- Investigators
- Taiga Nishihori, M.D., Principal investigator, H. Lee Moffitt Cancer Center and Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02780609
- Sponsor's protocol ID
- MCC-18630
- Status last verified
- Nov 2022
- First submitted
- 20 May 2016
- Last updated
- 4 Nov 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 33023948
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2 Investigator Sponsored Study of Selinexor in Combination With High-Dose Melphalan Before Autologous Hematopoietic Cell Transplantation for Multiple Myeloma
- ClinicalTrials.gov ID
- NCT02780609
- Acronym / sponsor ID
- MCC-18630
- Conditions
- Multiple Myeloma
- Keywords
- autologous hematopoietic cell transplantation (HCT), high-dose melphalan, selinexor
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Karyopharm Therapeutics Inc
- Study start
- 20 Jul 2017 (actual)
- Primary completion
- 20 Feb 2021 (actual)
- Study completion
- 23 Feb 2021 (actual)
- First posted
- 23 May 2016 (estimated)
- Results
- Posted 3 May 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Selinexor Plus HDM HCT | Experimental | Drug: Selinexor, Drug: Melphalan, Drug: Dexamethasone, Procedure: Autologous Hematopoietic Cell Transplantation (HCT), Drug: FosaprepitantThe 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SelinexorAlso: KPT-330Selinexor 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. | Drug | Selinexor Plus HDM HCT |
| MelphalanAlso: AlkeranMelphalan 100 mg/m^2 IV over 30-45 minutes. | Drug | Selinexor Plus HDM HCT |
| DexamethasoneAlso: DecadronDexamethasone 20 mg PO (or IV) daily (on days -3, -2 and -1). | Drug | Selinexor Plus HDM HCT |
| Autologous Hematopoietic Cell Transplantation (HCT)Participant's own stem cells are collected from their blood, frozen, then given back to them after chemotherapy. | Procedure or surgery | Selinexor Plus HDM HCT |
| FosaprepitantAlso: antiemetic agent, Standare of CareFosaprepitant at 150 mg IV on days -3 and -2. | Drug | Selinexor Plus HDM HCT |
Primary outcome
Phase I: Recommended Phase II Dose (RPh2D)
Time frame: Up to 3 months
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)
Time frame: 3 months post HCT
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33023948Turner 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.
- Moffitt Cancer Center Clinical Trials websiteLink provided by the sponsor.
Related trials for Multiple Myeloma
Not yet recruitingPhase 2
Study of YKST02 in Adults With Relapsed or Refractory Multiple Myeloma
Relapsed or Refractory Multiple Myeloma (RRMM)
RecruitingPhase 2
Engaging T-cells to Eliminate MRD in Newly Diagnosed Myeloma Optimizing Response With Talquetmab and Teclistamab (ROTATE)
Multiple Myeloma
Active, not recruitingPhase 3
A Phase III Study of Eque-cel in Subjects With Len-refractory RRMM (FUMANBA-03)
Multiple Myeloma
Active, not recruitingPhase 1/2
A Window of Opportunity Trial to Learn if Linvoseltamab is Safe and Well Tolerated, and How Well it Works in Adult Participants With Recently Diagnosed Multiple Myeloma Who Have Not Already Received Treatment
Multiple Myeloma
Active, not recruitingPhase 2
Testing AZD5363 as a Potential Targeted Treatment in Cancers With AKT Genetic Changes (MATCH-Subprotocol Y)
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Taiga Nishihori, M.D. principal investigator
H. Lee Moffitt Cancer Center and Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Selinexor, Melphalan and Dexamethasone in people with Multiple Myeloma, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 May 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase I: Recommended Phase II Dose (RPh2D) (time frame: Up to 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02780609, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2022.