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Selective Inhibitor of Nuclear Export

KPT-330 for Leukemia

Phase 1
Waitlist Available
Led By Andrew E Place, MD,PhD
Research Sponsored by Dana-Farber Cancer Institute
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients with CNS 1 or CNS 2 disease are eligible.
Female patients with infants must agree not to breastfeed their infants while on this study.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights

Study Summary

This trial is for leukemia patients who have relapsed or become resistant to standard therapies. They will be given the drug KPT-330 to see if it prevents leukemia cells from growing and leads to their destruction.

Who is the study for?
This trial is for children and adolescents aged between 1 and 21 years with relapsed or refractory acute lymphoblastic leukemia (ALL) or acute myelogenous leukemia (AML), including other specific types of leukemia. Participants must have recovered from previous treatments, not be pregnant, agree to use contraception, and have no severe concurrent diseases.Check my eligibility
What is being tested?
The study tests KPT-330's effectiveness on participants with certain types of leukemia that haven't responded to standard therapies. The focus is on understanding the side effects when given to young patients. It explores how KPT-330 might help in destroying cancer cells by activating cell death processes.See study design
What are the potential side effects?
While the trial aims to identify all side effects of KPT-330, potential risks may include typical chemotherapy-related reactions such as nausea, fatigue, blood count changes leading to increased infection risk, organ inflammation, and possible allergic responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My brain or spinal cord disease is at a manageable stage.
Select...
I agree not to breastfeed while participating in this study.
Select...
I (or my guardian) can understand and sign the consent form.
Select...
My condition did not improve after two initial treatments.
Select...
My leukemia has returned or is not responding to treatment.
Select...
My cancer has returned after treatment more than once.
Select...
I am between 1 and 21 years old.
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I am mostly active and can do most of my daily activities.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Maximum tolerated dose (MTD) of KPT-330 determined by incidence of dose limiting toxicities.
Toxicity profile of KPT-330 assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Secondary outcome measures
Assessment of anti-leukemic activity of KPT-330 measured by objective response rates.
Biomarker analysis including measurements of cytokine levels and expression of XPO1 in white blood cells.
Measurement of KPT-330 in the blood, urine and cerebrospinal fluid.

Side effects data

From 2015 Phase 1 trial • 286 Patients • NCT01607892
75%
Nausea
71%
Thrombocytopenia
67%
Anaemia
67%
Fatigue
54%
Decreased appetite
46%
Hyponatraemia
42%
Neutropenia
42%
Diarrhoea
38%
Vomiting
33%
Leukopenia
25%
Vision blurred
25%
Dyspnoea
25%
Lymphopenia
25%
Constipation
21%
Electrocardiogram QT prolonged
21%
Dizziness
21%
Headache
17%
Fall
17%
Contusion
17%
Peripheral sensory neuropathy
17%
Urinary tract infection
17%
Proteinuria
17%
Abdominal pain
17%
Arthralgia
13%
Weight decreased
13%
Hypercreatininaemia
13%
Hyperglycaemia
13%
Hypertension
13%
Epistaxis
13%
Cough
13%
Leukocytosis
13%
Haematuria
13%
Night sweats
13%
Muscular weakness
13%
Hypocalcaemia
8%
Pneumonia
8%
Photopsia
8%
Aspartate aminotransferase increased
8%
Alanine aminotransferase increased
8%
Confusional state
8%
Gait disturbance
8%
Insomnia
8%
Agitation
8%
Chills
8%
Lung infection
8%
Tachycardia
8%
Stomatitis
8%
Urinary incontinence
8%
Dysuria
8%
Hyperbilirubinaemia
8%
Hypercalcaemia
8%
Hypokalaemia
8%
Alopecia
8%
Hypotension
8%
Oedema peripheral
8%
Petechiae
4%
Hypomagnesaemia
4%
Hypophosphataemia
4%
Nasal congestion
4%
Overdose
4%
Pleural effusion
4%
Respiratory arrest
4%
Transient ischaemic attack
4%
Ejection fraction decreased
4%
Pyrexia
4%
Dysgeusia
4%
Palpitations
4%
Cataract
4%
Back pain
4%
Pain in extremity
4%
Musculoskeletal chest pain
4%
Dehydration
4%
Hyperuricaemia
4%
Hyperkalaemia
4%
Upper respiratory tract infection
4%
Myalgia
4%
Death
4%
Lung consolidation
4%
Bacteraemia
4%
Urosepsis
4%
Malaise
4%
Oedema
4%
Depression
4%
Febrile neutropenia
4%
Dyspepsia
4%
Hypoalbuminaemia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Other Hematological Malignancies (ALL, CML and CLL)
Diffuse Large B-cell Lymphoma (DLBCL)
Non-Hodgkin Lymphoma (NHL) Excluding DLBCL
Multiple Myeloma (MM)
Acute Myeloid Leukemia (AML)

Trial Design

1Treatment groups
Experimental Treatment
Group I: KPT-330Experimental Treatment1 Intervention
KPT-330 will be administered twice a week on Days 1 and 3 for four weeks. Starting dose 30 mg/m2.In the dose-escalation cohort, three patients will initially be enrolled at each dose level and will be monitored for a DLT during the 28-day treatment cycle before dose escalation may occur.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
KPT-330
2015
Completed Phase 1
~430

Find a Location

Who is running the clinical trial?

Dana-Farber Cancer InstituteLead Sponsor
1,079 Previous Clinical Trials
340,961 Total Patients Enrolled
77 Trials studying Leukemia
10,404 Patients Enrolled for Leukemia
William Lawrence and Blanche Hughes FoundationOTHER
Karyopharm Therapeutics IncIndustry Sponsor
87 Previous Clinical Trials
7,563 Total Patients Enrolled
12 Trials studying Leukemia
657 Patients Enrolled for Leukemia

Media Library

KPT-330 (Selective Inhibitor of Nuclear Export) Clinical Trial Eligibility Overview. Trial Name: NCT02091245 — Phase 1
Leukemia Research Study Groups: KPT-330
Leukemia Clinical Trial 2023: KPT-330 Highlights & Side Effects. Trial Name: NCT02091245 — Phase 1
KPT-330 (Selective Inhibitor of Nuclear Export) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02091245 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Have any similar trials been run before this one?

"Since its debut in 2014, KPT-330 has become the subject of 48 ongoing studies around the world. Initially sponsored by Karyopharm Therapeutics Inc., this medication achieved Phase 1 approval after a 16 patient trial completed in 2014. Currently, clinical trials for KPT-330 are being conducted across 28 countries and 259 cities worldwide."

Answered by AI

Is eligibility for this experiment restricted to participants above the age of 30?

"According to the established guidelines, patient enrolment will be accepted from as young as 12 months old up until 21 years of age."

Answered by AI

What prerequisites must be met to access this medical trial?

"This trial is seeking to enrol sixteen individuals aged between one year and twenty-one years old that have been diagnosed with leukemia. Furthermore, the patient must not be responding to induction cycles, would not benefit from more cytotoxic chemotherapy as determined by their physician, have a CNS 1 or 2 disease status (excluding isolated relapse of CNS 3). Please refer to Section 11.1.3 for definitions of neurological diseases and how traumatic lumbar punctures should be interpreted in this instance."

Answered by AI

To what extent is KPT-330 potentially perilous to individuals?

"Due to limited data on safety and efficacy, KPT-330 was assigned a rating of 1 when evaluated by our team at Power."

Answered by AI

Are any participants being accepted for this research endeavor at present?

"Unfortunately, this clinical trial is not taking on applicants at the moment. It was initially published on March 1st 2014 and last updated November 28th 2022. If you are still searching for trials that may be of interest to you, there are currently 1561 studies actively recruiting patients with leukemia and 48 other medical trials looking for participants treated with KPT-330."

Answered by AI

Has KPT-330 been investigated in any other investigations?

"In 2014, KPT-330 began to be studied at Children's Healthcare of Atlanta. Currently, there are 29 completed studies and 48 ongoing trials distributed around San Francisco in the Golden State of California."

Answered by AI

How many different sites are overseeing this research endeavor?

"At present, 10 different sites are accepting patients for this clinical trial. These locations include San Francisco, New york and Milwaukee as well as 7 other metropolitan centres. To avoid excessive travel expenses associated with the study, it is recommend that you select a site closest to your residence."

Answered by AI

How many subjects are participating in the research experiment?

"Unfortunately, this clinical trial is no longer enrolling patients. First posted on March 1st 2014 and last updated on November 28th 2022, it has now concluded its recruitment process. However, if you are still searching for studies related to cancer or KPT-330 specifically, there are currently 1561 trials recruiting participants with leukemia and 48 actively looking for volunteers taking part in the KPT-330 study respectively."

Answered by AI
~1 spots leftby Apr 2025