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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Karyopharm Therapeutics, Inc Identifier:
First received: May 16, 2012
Last updated: February 22, 2016
Last verified: February 2016
The purpose of this research study are to find out more information such as: the highest dose of KPT-330 that can be given safely, the side effects it may cause, to examine how the body affects the study drug concentrations in the blood (pharmacokinetics or PK), to examine the effects of this study drug on the body (pharmacodynamics or PDn) and to gain some information on its effectiveness in treating cancer.

Condition Intervention Phase
Hematological Malignancies
Drug: KPT-330
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Karyopharm Therapeutics, Inc:

Primary Outcome Measures:
  • Number of participants with Adverse Events [ Time Frame: 2 and 12 months ] [ Designated as safety issue: Yes ]
    Severity of Adverse Events

Secondary Outcome Measures:
  • Area under the plasma concentration versus time curve (AUC) of KPT-330 [ Time Frame: 2 and 12 months ] [ Designated as safety issue: No ]
    Changes in AUC over time

Enrollment: 284
Study Start Date: June 2012
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: July 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: selinexor Drug: KPT-330
Other Name: Selinexor


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria

  1. Malignancies that are refractory to or who are intolerant of established therapy known to provide clinical benefit for their condition. Patients must not be candidates for anti-tumor regimens known to provide clinical benefit
  2. All patients on this study 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 all inclusion/exclusion criteria are met.

Exclusion Criteria

  1. 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.
  2. 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;
  3. Known active hepatitis A, B, or C infection; or known to be positive for HCV RNA or HBsAg (HBV surface antigen);
  4. 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.
  5. Significantly diseased or obstructed gastrointestinal tract or uncontrolled vomiting or diarrhea.
  6. Grade ≥2 peripheral neuropathy at baseline (within 14 days prior to cycle 1 day 1).
  7. Macular degeneration, uncontrolled glaucoma, or markedly decreased visual acuity.
  8. In the opinion of the investigator, patients who are significantly below their ideal body weight.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01607892

United States, Florida
Moffitt Cancer Center
Tampa, Florida, United States, 33612
United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
United States, Missouri
Washington University School of Medicine
St Louis, Missouri, United States, 63110
United States, New Jersey
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
United States, New York
Weill Cornell Medical Center
New York City, New York, United States, 10065
United States, Ohio
Gabrail Cancer Center Research
Canton, Ohio, United States, 44718
The Ohio State University
Columbus, Ohio, United States, 43210
United States, Tennessee
Sarah Cannon Research Institute
Nashville, Tennessee, United States, 37203
United States, Texas
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Canada, Alberta
Tom Baker Cancer Centre
Calgary, Alberta, Canada, T2W 4N2
Canada, Ontario
Princess Margaret Hospital
Toronto, Ontario, Canada, M5T 2M9
Copenhagen, Denmark, 2100
Sponsors and Collaborators
Karyopharm Therapeutics, Inc
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Karyopharm Therapeutics, Inc Identifier: NCT01607892     History of Changes
Other Study ID Numbers: KCP-330-001 
Study First Received: May 16, 2012
Last Updated: February 22, 2016
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Denmark: Danish Health and Medicines Authority

Keywords provided by Karyopharm Therapeutics, Inc:
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

Additional relevant MeSH terms:
Neoplasms processed this record on May 25, 2016