Hematological MalignanciesPhase 1NCT01607892

Safety Study of the Selective Inhibitor of Nuclear Export (SINE) KPT-330 in Patients With Advanced Hematological Cancer

A Phase 1 study testing KPT-330 in people with Hematological Malignancies, run by Karyopharm Therapeutics Inc at 12 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

The purpose of this research study is to find out more information relating to the highest dose of KCP-330 that can be given safely and side effects it may cause, to examine how the body affects KCP-330 concentrations in the blood (pharmacokinetics or PK), to examine the effects of KCP-330 on the body… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Hematological Malignancies
What is being tested?: KPT-330
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 286 participants
Where: 12 sitesin 3 countries
Study dates: 23 Jul 2012to 13 Oct 2015
Phase: Phase 1
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

Malignancies that are refractory to or intolerant of established therapy known to provide clinical benefit. Patients must not be candidates for anti-tumor regimes known to provide clinical benefit.
All patients must have evidence of progressive disease on study entry. Previously untreated patients who are not chemotherapy candidates on Arm 2 may have advanced disease (without clear progression). There is no upper limit on the number of prior treatments provided that all inclusion criteria are met, and exclusion criteria are not met.

Who cannot join

Radiation, chemotherapy, or immunotherapy or any other anticancer therapy ≤2 weeks prior to cycle 1 day 1 and mitomycin C and radioimmunotherapy 6 weeks prior to cycle 1 day 1.
Patients with active graft versus host disease after allogeneic stem cell transplantation. At least 3 months must have elapsed since completion of allogeneic stem cell transplantation except for patients with AML, where at least 2 months must have elapsed;
Known active hepatitis A, B, or C infection; or known to be positive for HCV RNA or HBsAg (HBV surface antigen);
Patients with active CNS malignancy. Asymptomatic small lesions are not considered active. Treated lesions may be considered inactive if they are stable for at least 3 months. Patient with malignant cells in their cerebrospinal fluid (CSF) without CNS symptom may be included.
Significantly diseased or obstructed gastrointestinal tract or uncontrolled vomiting or diarrhea.
Grade ≥2 peripheral neuropathy at baseline (within 14 days prior to cycle 1 day 1).
Macular degeneration, uncontrolled glaucoma, or markedly decreased visual acuity.
In the opinion of the investigator, patients who are significantly below their ideal body weight.

Trial locations and contacts

12 sites in 3 countries.

Country

City, postcode or hospital

12 sites in 3 countries

United States9 sites

Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Gabrail Cancer Center Research
Canton, Ohio, 44718
The Ohio State University
Columbus, Ohio, 43210
Hackensack University Medical Center
Hackensack, New Jersey, 07601
MD Anderson Cancer Center
Houston, Texas, 77030
Sarah Cannon Research Institute
Nashville, Tennessee, 37203
Weill Cornell Medical Center
New York, New York, 10065
Washington University School of Medicine
St Louis, Missouri, 63110
Moffitt Cancer Center
Tampa, Florida, 33612

Other trials in United States

Canada2 sites

Tom Baker Cancer Centre
Calgary, Alberta, T2W 4N2
Princess Margaret Hospital
Toronto, Ontario, M5T 2M9

Other trials in Canada

Denmark1 site

Rigshospitalet
Copenhagen, 2100

Other trials in Denmark

About this study

Official title: A Phase I Study of the Safety, Pharmacokinetics and Pharmacodynamics of Escalating Doses of the Selective Inhibitor of Nuclear Export/SINE™ Compound KPT-330 in Patients With Advanced Hematological Malignancies

Conditions: Hematological Malignancies
Being tested: KPT-330
Keywords: Selinexor, KPT-330, Multiple Myeloma, non-Hodgkin Lymphoma, Chronic Lymphocytic Leukemia, Waldenström's macroglobulinemia, Peripheral T-Cell Lymphoma, Cutaneous T-Cell Lymphoma, Chronic Myelocytic Leukemia, Acute Lymphoblastic Leukemia
Medical subject headings: Hematologic Neoplasms, Multiple Myeloma, Lymphoma, Non-Hodgkin, Leukemia, Lymphocytic, Chronic, B-Cell, Waldenstrom Macroglobulinemia, Lymphoma, T-Cell, Peripheral, Lymphoma, T-Cell, Cutaneous, Leukemia, Myelogenous, Chronic, BCR-ABL Positive

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

KPT-330Drug
Also called Selinexor

Study groups in NCT01607892
Study group	Role	What they receive
selinexor	Experimental	Not described Receives: Drug: KPT-330

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product during the course of a study and which does not necessarily have to have a causal relationship with this treatment. An Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. TEAEs are any untoward medical incidence in a participant during administered study treatment, whether or not these events were related to study treatment.
Measured: From first dose of study drug administration to end of treatment (up to 27 months)
Recommended Phase 2 Dose (RP2D) of Selinexor
Recommended Phase 2 dose was determined by maximum tolerated dose (MTD). MTD was defined as the next lower dose level below the one in which > 1 of 3 participants or ≥ 2 of 6 participants experienced dose-limiting toxicity (DLT), provided that that dose level is ≤25% lower than the highest dose tested. If the projected MTD was >25% lower than the highest dose tested, then an additional cohort of ≥3 participants were added at a dose that was intermediate between the intolerable dose and the next lower dose.
Measured: From first dose of study drug administration to end of treatment (up to 27 months)

Secondary outcomes (13)

Maximum Observed Plasma Concentration (Cmax) of Selinexor
Cmax was defined as the maximum observed concentration, taken directly from the plasma concentration.
Measured: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Time to Maximum Observed Concentration (Tmax) of Selinexor
Tmax was defined as time of first observation of Cmax, taken directly from the plasma concentration data.
Measured: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Average Concentration From Time 0 to 24 Hours (Cavg0-24h) of Selinexor
Cavg0-24h was defined as average concentration from time 0 to 24 hours.
Measured: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Area Under the Concentration-Time Curve From Time 0 to t (AUC0-t) of Selinexor
AUC0-t was defined as area under the concentration-time curve from time zero to the last non-zero concentration.
Measured: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Area Under the Concentration-Time Curve From Time 0 Extrapolated to Infinity (AUC0-inf) of Selinexor
AUC0-inf was defined as the area under the concentration-time curve from time zero to infinity (extrapolated).
Measured: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Apparent Volume of Distribution Uncorrected for Fraction Absorbed (Vd/F) of Selinexor
Apparent volume of distribution was calculated as Dose/ (kel *AUC0-inf), uncorrected for fraction absorbed, reported normalized by participant body weight (kilogram [kg]).
Measured: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Apparent Total Body Clearance, Uncorrected for Fraction Absorbed (Cl/F) of Selinexor
Cl/F was calculated as Dose/AUC0-inf, uncorrected for fraction absorbed, reported normalized by participant body weight (kg).
Measured: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Terminal Half-Life (t½) of Selinexor
t½ was, calculated as ln(2)/kel, where kel is elimination rate constant, calculated using linear regression on the terminal portion of the log-linear concentration versus time curve.
Measured: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Number of Participants With Overall Response of Selinexor
Objective response for each malignancy was defined using the disease response criteria by malignancy; For NHL (including DLBCL, PTCL, and CTCL), objective response included complete response (CR) and partial response (PR). For MM, objective response included stringent complete response (sCR), CR, very good partial response (VGPR), and PR. For WM, objective response included CR, VGPR, and PR. For CLL, ALL, and AML, objective response included complete remission and Partial remission. For CML, objective response includes complete cytogenic response, and complete hematologic response (CHR).
Measured: From first dose of study drug administration to end of treatment (up to 27 months)
Duration of Response
Duration of response was defined as the time from the first occurrence of objective response to first documented evidence of disease recurrence or progression. Participants without evidence of progression were censored at time of last evaluable disease assessment. Objective response was defined as any response of partial response/remission or better for all malignancies; for AML, a response of morphologic leukemia-free state is also included for ORR. Duration of response was calculated by Kaplan-Meier method.
Measured: From first dose of study drug administration to end of treatment (up to 27 months)
Progression-free Survival
Progression-free survival (PFS) was calculated from the date of first dose of study drug to first documented evidence of disease recurrence or progression or death due to any cause. Participants who were last known to be alive and without evidence of progression were censored at time of last evaluable disease assessment. If date of progression or death occurred after more than 1 missed disease assessment interval, participants were censored at the time of last evaluable disease assessment prior to the missed assessment.
Measured: Cycle 1 Day 1 to End of Treatment (up to 27 months)
Duration of at Least Stable Disease
Duration of at least stable disease was defined as the time from the date of first dose of study drug to first documented evidence of disease recurrence or progression. Participants without evidence of progression were censored at time of last evaluable disease assessment.
Measured: Cycle 1 Day 1 to End of Treatment (up to 27 months)
Overall Survival
Overall Survival was calculated from the date of first dose of study drug to date of death due to any cause. Participants who were last known to be alive were censored at time of last contact. Overall survival was calculated by Kaplan-Meier method.
Measured: Cycle 1 Day 1 to End of Treatment (up to 27 months)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

23 Jul 201213 Oct 2015

First posted30 May 2012 (estimated)
Study start23 Jul 2012
Primary completion13 Oct 2015
Study completion13 Oct 2015

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 (4)Papers the sponsor has linked to this study.
Other trials for Hematological MalignanciesStudies of the same condition, open and under way.
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
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 286 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01607892
Sponsor's protocol ID: KCP-330-001
Status last verified: Jan 2023
First submitted: 16 May 2012
Last updated: 26 Jan 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes

Publications

Chen C, Siegel D, Gutierrez M, Jacoby M, Hofmeister CC, Gabrail N, Baz R, Mau-Sorensen M, Berdeja JG, Savona M, Savoie L, Trudel S, Areethamsirikul N, Unger TJ, Rashal T, Hanke T, Kauffman M, Shacham S, Reece D. Safety and efficacy of selinexor in relapsed or refractory multiple myeloma and Waldenstrom macroglobulinemia. Blood. 2018 Feb 22;131(8):855-863. doi: 10.1182/blood-2017-08-797886. Epub 2017 Dec 4.
PubMed 29203585
Kuruvilla J, Savona M, Baz R, Mau-Sorensen PM, Gabrail N, Garzon R, Stone R, Wang M, Savoie L, Martin P, Flinn I, Jacoby M, Unger TJ, Saint-Martin JR, Rashal T, Friedlander S, Carlson R, Kauffman M, Shacham S, Gutierrez M. Selective inhibition of nuclear export with selinexor in patients with non-Hodgkin lymphoma. Blood. 2017 Jun 15;129(24):3175-3183. doi: 10.1182/blood-2016-11-750174. Epub 2017 May 3.
PubMed 28468797
Garzon R, Savona M, Baz R, Andreeff M, Gabrail N, Gutierrez M, Savoie L, Mau-Sorensen PM, Wagner-Johnston N, Yee K, Unger TJ, Saint-Martin JR, Carlson R, Rashal T, Kashyap T, Klebanov B, Shacham S, Kauffman M, Stone R. A phase 1 clinical trial of single-agent selinexor in acute myeloid leukemia. Blood. 2017 Jun 15;129(24):3165-3174. doi: 10.1182/blood-2016-11-750158. Epub 2017 Mar 23.
PubMed 28336527
Schmidt J, Braggio E, Kortuem KM, Egan JB, Zhu YX, Xin CS, Tiedemann RE, Palmer SE, Garbitt VM, McCauley D, Kauffman M, Shacham S, Chesi M, Bergsagel PL, Stewart AK. Genome-wide studies in multiple myeloma identify XPO1/CRM1 as a critical target validated using the selective nuclear export inhibitor KPT-276. Leukemia. 2013 Dec;27(12):2357-65. doi: 10.1038/leu.2013.172. Epub 2013 Jun 11.
PubMed 23752175

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Hematological Malignancies trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase I Study of the Safety, Pharmacokinetics and Pharmacodynamics of Escalating Doses of the Selective Inhibitor of Nuclear Export/SINE™ Compound KPT-330 in Patients With Advanced Hematological Malignancies
ClinicalTrials.gov ID: NCT01607892
Acronym / sponsor ID: KCP-330-001
Conditions: Hematological Malignancies
Keywords: Selinexor, KPT-330, Multiple Myeloma, non-Hodgkin Lymphoma, Chronic Lymphocytic Leukemia, Waldenström's macroglobulinemia, Peripheral T-Cell Lymphoma, Cutaneous T-Cell Lymphoma
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 286 participants (actual)
Lead sponsor: Karyopharm Therapeutics IncIndustry (pharma, biotech, devices)
Study start: 23 Jul 2012 (actual)
Primary completion: 13 Oct 2015 (actual)
Study completion: 13 Oct 2015 (actual)
First posted: 30 May 2012 (estimated)
Results: Posted 1 Apr 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
selinexor	Experimental	Drug: KPT-330

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (2 listed)

Malignancies that are refractory to or intolerant of established therapy known to provide clinical benefit. Patients must not be candidates for anti-tumor regimes known to provide clinical benefit.
All patients must have evidence of progressive disease on study entry. Previously untreated patients who are not chemotherapy candidates on Arm 2 may have advanced disease (without clear progression). There is no upper limit on the number of prior treatments provided that all inclusion criteria are met, and exclusion criteria are not met.

Key exclusion criteria (8 listed)

Radiation, chemotherapy, or immunotherapy or any other anticancer therapy ≤2 weeks prior to cycle 1 day 1 and mitomycin C and radioimmunotherapy 6 weeks prior to cycle 1 day 1.
Patients with active graft versus host disease after allogeneic stem cell transplantation. At least 3 months must have elapsed since completion of allogeneic stem cell transplantation except for patients with AML, where at least 2 months must have elapsed;
Known active hepatitis A, B, or C infection; or known to be positive for HCV RNA or HBsAg (HBV surface antigen);
Patients with active CNS malignancy. Asymptomatic small lesions are not considered active. Treated lesions may be considered inactive if they are stable for at least 3 months. Patient with malignant cells in their cerebrospinal fluid (CSF) without CNS symptom may be included.
Significantly diseased or obstructed gastrointestinal tract or uncontrolled vomiting or diarrhea.

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
KPT-330Also: Selinexor	Drug	selinexor

Primary outcome

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
Time frame: From first dose of study drug administration to end of treatment (up to 27 months)
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product during the course of a study and which does not necessarily have to have a causal relationship with this treatment. An Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. TEAEs are any untoward medical incidence in a participant during administered study treatment, whether or not these events were related to study treatment.
Recommended Phase 2 Dose (RP2D) of Selinexor
Time frame: From first dose of study drug administration to end of treatment (up to 27 months)
Recommended Phase 2 dose was determined by maximum tolerated dose (MTD). MTD was defined as the next lower dose level below the one in which > 1 of 3 participants or ≥ 2 of 6 participants experienced dose-limiting toxicity (DLT), provided that that dose level is ≤25% lower than the highest dose tested. If the projected MTD was >25% lower than the highest dose tested, then an additional cohort of ≥3 participants were added at a dose that was intermediate between the intolerable dose and the next lower dose.

13 secondary outcomes

Maximum Observed Plasma Concentration (Cmax) of Selinexor
Time frame: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Time to Maximum Observed Concentration (Tmax) of Selinexor
Time frame: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Average Concentration From Time 0 to 24 Hours (Cavg0-24h) of Selinexor
Time frame: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Area Under the Concentration-Time Curve From Time 0 to t (AUC0-t) of Selinexor
Time frame: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Area Under the Concentration-Time Curve From Time 0 Extrapolated to Infinity (AUC0-inf) of Selinexor
Time frame: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Apparent Volume of Distribution Uncorrected for Fraction Absorbed (Vd/F) of Selinexor
Time frame: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Apparent Total Body Clearance, Uncorrected for Fraction Absorbed (Cl/F) of Selinexor
Time frame: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Terminal Half-Life (t½) of Selinexor
Time frame: 0 (pre-dose), 0.5, 1, 2, 4, 8, 24 and 48 hours post-dose on Day 1, 8 of Cycle 1 and Days 15, 16 or 17 of Cycle 2
Number of Participants With Overall Response of Selinexor
Time frame: From first dose of study drug administration to end of treatment (up to 27 months)
Duration of Response
Time frame: From first dose of study drug administration to end of treatment (up to 27 months)
Progression-free Survival
Time frame: Cycle 1 Day 1 to End of Treatment (up to 27 months)
Duration of at Least Stable Disease
Time frame: Cycle 1 Day 1 to End of Treatment (up to 27 months)
Overall Survival
Time frame: Cycle 1 Day 1 to End of Treatment (up to 27 months)

Results and safety

Results posted to the registry on 1 Apr 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Diffuse Large B-cell Lymphoma (DLBCL)	58	0
Non-Hodgkin Lymphoma (NHL) Excluding DLBCL	27	0
Multiple Myeloma (MM)	81	0
Acute Myeloid Leukemia (AML)	95	0
Other Hematological Malignancies (ALL, CML and CLL)	24	0

Primary outcome: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

Time frame: From first dose of study drug administration to end of treatment (up to 27 months). Count of participants. Unit: Participants

Measure	Diffuse Large B-cell Lymphoma (DLBCL) n = 58	Non-Hodgkin Lymphoma (NHL) Excluding DLBCL n = 27	Multiple Myeloma (MM) n = 81	Acute Myeloid Leukemia (AML) n = 95	Other Hematological Malignancies (ALL, CML and CLL) n = 24
At Least One TEAE	58	27	81	95	24
At Least One Serious TEAE	33	7	52	73	13

Population analysed: Safety population consisted of all participants who received at least 1 dose of selinexor.

Primary outcome: Recommended Phase 2 Dose (RP2D) of Selinexor

Time frame: From first dose of study drug administration to end of treatment (up to 27 months). Number. Unit: milligram per square meter (mg/m^2)

Measure	Advanced Hematological Malignancies. n = 285
Value	35

Population analysed: Efficacy analysis set included all participants who had either completed 1 cycle of treatment or discontinued treatment prior to completing the first cycle due to documented progressive disease (PD), death related to disease, or treatment-related toxicity. Here, data was not planned and analyzed based on individual specific disease, hence overall data was analyzed for this outcome measure.

13 secondary outcomes are reported in the full results.

Adverse events

Time frame: From first dose of study drug administration to end of treatment (up to 27 months). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Diffuse Large B-cell Lymphoma (DLBCL)	33 of 58 (56.9%)	58 of 58 (100%)	Not reported
Non-Hodgkin Lymphoma (NHL) Excluding DLBCL	7 of 27 (25.9%)	27 of 27 (100%)	Not reported
Multiple Myeloma (MM)	52 of 81 (64.2%)	80 of 81 (98.8%)	Not reported
Acute Myeloid Leukemia (AML)	73 of 95 (76.8%)	94 of 95 (98.9%)	Not reported
Other Hematological Malignancies (ALL, CML and CLL)	13 of 24 (54.2%)	24 of 24 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 29203585Chen C, Siegel D, Gutierrez M, Jacoby M, Hofmeister CC, Gabrail N, Baz R, Mau-Sorensen M, Berdeja JG, Savona M, Savoie L, Trudel S, Areethamsirikul N, Unger TJ, Rashal T, Hanke T, Kauffman M, Shacham S, Reece D. Safety and efficacy of selinexor in relapsed or refractory multiple myeloma and Waldenstrom macroglobulinemia. Blood. 2018 Feb 22;131(8):855-863. doi: 10.1182/blood-2017-08-797886. Epub 2017 Dec 4.
Derived from this study: PubMed 28468797Kuruvilla J, Savona M, Baz R, Mau-Sorensen PM, Gabrail N, Garzon R, Stone R, Wang M, Savoie L, Martin P, Flinn I, Jacoby M, Unger TJ, Saint-Martin JR, Rashal T, Friedlander S, Carlson R, Kauffman M, Shacham S, Gutierrez M. Selective inhibition of nuclear export with selinexor in patients with non-Hodgkin lymphoma. Blood. 2017 Jun 15;129(24):3175-3183. doi: 10.1182/blood-2016-11-750174. Epub 2017 May 3.
Derived from this study: PubMed 28336527Garzon R, Savona M, Baz R, Andreeff M, Gabrail N, Gutierrez M, Savoie L, Mau-Sorensen PM, Wagner-Johnston N, Yee K, Unger TJ, Saint-Martin JR, Carlson R, Rashal T, Kashyap T, Klebanov B, Shacham S, Kauffman M, Stone R. A phase 1 clinical trial of single-agent selinexor in acute myeloid leukemia. Blood. 2017 Jun 15;129(24):3165-3174. doi: 10.1182/blood-2016-11-750158. Epub 2017 Mar 23.
Derived from this study: PubMed 23752175Schmidt J, Braggio E, Kortuem KM, Egan JB, Zhu YX, Xin CS, Tiedemann RE, Palmer SE, Garbitt VM, McCauley D, Kauffman M, Shacham S, Chesi M, Bergsagel PL, Stewart AK. Genome-wide studies in multiple myeloma identify XPO1/CRM1 as a critical target validated using the selective nuclear export inhibitor KPT-276. Leukemia. 2013 Dec;27(12):2357-65. doi: 10.1038/leu.2013.172. Epub 2013 Jun 11.
Individual participant dataSharing planned: Yes

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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.

The record lists no contact. Use the official record to reach the sponsor.

12 sites in 3 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing KPT-330 in people with Hematological Malignancies, run by Karyopharm Therapeutics Inc at 12 sites in 3 countries.

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 2 inclusion and 8 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 26 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United States, Canada, Denmark. 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 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.

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 1 Apr 2021). 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?

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) (time frame: From first dose of study drug administration to end of treatment (up to 27 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 NCT01607892, which also lists 4 linked publications.

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 26 Jan 2023.