Efficacy & Safety Study of KPT-330 in Erythropoietin-Refractory Lower-Risk Myelodysplastic Syndrome Patients

NCT02431351 | Withdrawn Phase 2

• 1 other identifier: KCP-330-014
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is an open-label study designed to evaluate the safety and efficacy of the selective inhibitor of nuclear export (SINE) compound, Selinexor given orally to patients with transfusion-dependent, EPO-refractory lower-risk MDS (Low risk and Intermediate-1 as defined by IPSS).

Conditions

Myelodysplastic Syndrome

Keywords

KPT-330; Karyopharm; Selinexor; SIER; Myelodysplastic Syndrome (MDS); Erythropoietin (EPO); Refractory

Outcome Measures

Primary Outcomes (1)

• Hematologic improvement (based on the 2006 International Working Group (IWG) response criteria)

  Hematologic improvement in erythroid, platelet, and neutrophil counts, evaluated based on the 2006 International Working Group (IWG) response criteria for MDS.

  6 months

Secondary Outcomes (2)

• Overall response rate

  6 months

• Quality of Life (QOL-E) MDS Questionnaire)

  6 months

Study Arms (1)

Selinexor

EXPERIMENTAL

60 mg once weekly

Drug: Selinexor

Interventions

Selinexor DRUG

60 mg on Day 1 of each week for a 4 week cycle, given for ≥6 cycles.

Also known as: KPT-330

Selinexor

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of Low risk or Intermediate-1 risk MDS with any cytogenetic abnormalities (according to the IPSS criteria); patients must have bone marrow biopsy/aspiration or tumor tissue available from within 1 month prior to first dose or collected during the Screening period.
• Patients \>18 years at Screening who are not candidates for hematopoietic cell transplantation.
• Received 1 prior line of treatment; typically erythroid-stimulating agents (ESAs).
• Red blood cell (RBC) transfusion-dependent anemia while treated with or after discontinuation of EPO. Transfusion dependence is defined as the requirement for at least 2 units of RBCs transfused during the 8 weeks prior to study initiation.

You may not qualify if:

• Use of recombinant EPO within 8 weeks prior to screening.
• Patient has a concurrent active malignancy or prior history of malignancy other than MDS (except basal cell or squamous cell carcinoma of the skin and in situ of the cervix) unless free of disease for at least 1 year.
• Unstable cardiovascular function:
• Symptomatic ischemia,
• Uncontrolled clinically significant conduction abnormalities (i.e., ventricular tachycardia on anti-arrhythmic agents are excluded; 1st degree atrioventricular (AV) block or asymptomatic Left anterior fascicular block/Right bundle branch block (LAFB/RBBB) will not be excluded), or
• Congestive heart failure (CHF) New York Heart Association (NYHA) Class ≥3, or myocardial infarction (MI) within 3 months.
• Uncontrolled infection requiring parenteral antibiotics, antivirals, or antifungals within 1 week prior to first dose. Infections controlled on concurrent anti-microbial agents are acceptable, and anti-microbial prophylaxis per institutional guidelines is acceptable.
• Active bleeding Grade 3-4, in the last 4 weeks prior to enrollment.

Sponsors & Collaborators

• Karyopharm Therapeutics Inc: lead

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

selinexor

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 27, 2015
• First Posted: May 1, 2015
• Study Start: November 1, 2015
• Primary Completion: December 1, 2017
• Study Completion: May 1, 2019
• Last Updated: January 26, 2023

Record last verified: 2023-01