Study of the Safety, Tolerability and Efficacy of KPT-8602 in Patients With Relapsed/Refractory Cancer Indications
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ClinicalTrials.gov Identifier: NCT02649790 |
Recruitment Status :
Active, not recruiting
First Posted : January 7, 2016
Last Update Posted : June 11, 2020
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This is a first-in-human, multi-center, open-label clinical study with separate dose escalation (Phase 1) and expansion (Phase 2) stages to assess preliminary safety, tolerability, and efficacy of the second generation oral XPO1 inhibitor KPT-8602 in patients with relapsed/refractory multiple myeloma (MM), metastatic colorectal cancer (mCRC), metastatic castration resistant prostate cancer (mCRPC), and higher risk myelodysplastic syndrome (HR-MDS).
Dose escalation and dose expansion may be included for all parts of the study as determined by ongoing study results.
Condition or disease | Intervention/treatment | Phase |
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Relapsed/Refractory Multiple Myeloma (RRMM) Metastatic Colorectal Cancer (mCRC) Metastatic Castration Resistant Prostate Cancer (mCRPC) Higher Risk Myelodysplastic Syndrome (HR-MDS) | Drug: KPT-8602 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 119 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 1/2 Open-Label Study of the Safety, Tolerability and Efficacy of the Selective Inhibitor of Nuclear Export (SINE) Compound KPT-8602 in Patients With Relapsed/Refractory Cancer Indications |
Actual Study Start Date : | January 2016 |
Estimated Primary Completion Date : | March 2021 |
Estimated Study Completion Date : | March 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: KPT-8602
Relapsed/Refractory Multiple Myeloma (RRMM) - CLOSED TO ENROLLMENT Metastatic Colorectal Cancer (CRC) - CLOSED TO ENROLLMENT Relapsed/Refractory Metastatic Castration Resistant Prostate Cancer (mCRPC) - CLOSED TO ENROLLMENT Higher Risk Myelodysplastic Syndrome (MDS) - CLOSED TO ENROLLMENT Starting dose for CRC, mCRPC, MDS is 20 mg of KPT-8602 |
Drug: KPT-8602 |
- Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) [ Time Frame: Approximately 4 weeks ]MTD will be based on the assessment of dose limiting toxicities (DLTs) during the first cycle of therapy and will be defined as the highest dose at which 1 of 6 patients, at most, experiences DLTs within Cycle 1. Dose escalation will continue until an RP2D is selected based on DLT assessment.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
INCLUSION CRITERIA
- Written informed consent obtained prior to any screening procedures and in accordance with federal, local, and institutional guidelines.
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Age ≥ 18 years.
Higher Risk Myelodysplastic Syndrome (Part F):
- Documented diagnosis of MDS with 5-19% myeloblasts.
- Patients should be intermediate-2 or high-risk MDS by International Prognostic Scoring System (IPSS).
- Patients believed to be IPSS high risk, without clearly meeting IPSS categories above should be discussed with the medical monitor prior to enrolling.
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HMA refractory patients including:
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≥ 2 cycles of azacitidine and/or decitabine or experimental agents (such as SGI-110 or ASTX727 or similar) with clear progressive disease (PD) (no count recovery with ≥50% increase in bone marrow blasts)
OR
- ≥ 4 cycles of azacitidine and/or decitabine (or other hypomethylating therapy) with lack of improvement (no CR/CRi/PR/HI).
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- Patients receiving a stable dose of erythropoiesis-stimulating agent (ESA) for at least 1 month at the time of study entry may continue to receive ESA.
EXCLUSION CRITERIA
Patients in All Parts of the Study:
- Major surgery within 4 weeks before C1D1.
- Impaired cardiac function or clinically significant cardiac diseases.
- Uncontrolled active severe systemic infection requiring parenteral antibiotics, antivirals, or antifungals within one week prior to C1D1.
- Patients with known symptomatic brain metastasis.
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Prior malignancies:
- Patients in All Parts of the Study: Patients with adequately resected basal or squamous cell carcinoma of the skin, or adequately resected carcinoma in situ (i.e. cervix) may enroll irrespective of the time of diagnosis.
- Patients with Higher Risk MDS only: Concomitant malignancies or previous malignancies with less than a 1-year disease free interval at the time of enrollment.
INDICATION-SPECIFIC EXCLUSION CRITERIA
Higher risk Myelodysplastic Syndrome (Part F):
- IPSS low or intermediate-1 risk MDS.
- Evidence of transformation to AML by World Health Organization (WHO) (≥20% blasts in bone marrow or peripheral blood).
- Patients receiving granulocyte-colony stimulating factor (G-CSF) or granulocyte macrophage-colony stimulating factor (GM-CSF) within the 3 weeks prior to C1D1.

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): NCT02649790
United States, Florida | |
Moffitt Cancer Center | |
Tampa, Florida, United States, 33612 | |
United States, New Jersey | |
John Theurer Cancer Center at Hackensack UMC | |
Hackensack, New Jersey, United States, 07601 | |
United States, New York | |
Weill Cornell Medical College | |
New York, New York, United States, 10021 | |
United States, Ohio | |
Ohio State University, The James Cancer Hospital and Solove Research Institute | |
Columbus, Ohio, United States, 43210 | |
United States, Pennsylvania | |
University of Pennsylvania Abramson Cancer Center Clinical Research Unit | |
Philadelphia, Pennsylvania, United States, 19104 | |
United States, Tennessee | |
Vanderbilt University Medical Center | |
Nashville, Tennessee, United States, 37232 | |
Canada, Ontario | |
Princess Margaret Cancer Research | |
Toronto, Ontario, Canada | |
Canada, Quebec | |
MUHC GLEN Site Cedars - Cancer Centre | |
Montréal, Quebec, Canada |
Study Director: | Jatin Shah, MD | Karyopharm Therapeutics Inc |
Responsible Party: | Karyopharm Therapeutics Inc |
ClinicalTrials.gov Identifier: | NCT02649790 |
Other Study ID Numbers: |
KCP-8602-801 |
First Posted: | January 7, 2016 Key Record Dates |
Last Update Posted: | June 11, 2020 |
Last Verified: | May 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
KPT-8602 Multiple Myeloma Karyopharm MM Phase 1 Relapsed/ Refractory Multiple Myeloma |
Myelodysplastic Syndrome MDS Metastatic Castration Resistant Prostate Cancer mCRPC CRC Metastatic Colorectal Cancer |
Prostatic Neoplasms Colorectal Neoplasms Multiple Myeloma Neoplasms, Plasma Cell Preleukemia Myelodysplastic Syndromes Genital Neoplasms, Male Urogenital Neoplasms Neoplasms by Site Neoplasms Prostatic Diseases Intestinal Neoplasms Gastrointestinal Neoplasms Digestive System Neoplasms Digestive System Diseases |
Gastrointestinal Diseases Colonic Diseases Intestinal Diseases Rectal Diseases Neoplasms by Histologic Type Hemostatic Disorders Vascular Diseases Cardiovascular Diseases Paraproteinemias Blood Protein Disorders Hematologic Diseases Hemorrhagic Disorders Lymphoproliferative Disorders Immunoproliferative Disorders Immune System Diseases |