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Selective Inhibitor of Nuclear Export
KPT-330 for Relapsed Childhood 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.
Must not have
Plan to administer non-protocol chemotherapy, radiation therapy, or immunotherapy during the study period.
Individuals eligible for allogeneic hematopoietic stem cell transplantation (HSCT).
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights
No Placebo-Only Group
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.
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.
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 belowSelect...
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.
Select...
I am mostly active and can do most of my daily activities.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I do not plan to receive any non-study cancer treatments during the trial.
Select...
I am eligible for a stem cell transplant from a donor.
Select...
I cannot take or tolerate medications by mouth.
Select...
My leukemia has spread to my brain.
Select...
My body does not respond to blood or platelet transfusions.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
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 study objectives
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 • NCT0160789271%
Fatigue
65%
Decreased appetite
58%
Nausea
49%
Thrombocytopenia
48%
Diarrhoea
48%
Anaemia
41%
Vomiting
36%
Hyponatraemia
34%
Dyspnoea
33%
Weight decreased
31%
Constipation
24%
Epistaxis
24%
Neutropenia
23%
Dysgeusia
23%
Hypokalaemia
22%
Vision blurred
22%
Oedema peripheral
21%
Pyrexia
21%
Dizziness
20%
Hypomagnesaemia
20%
Hypotension
19%
Hypercreatininaemia
19%
Hyperglycaemia
19%
Hypocalcaemia
18%
Stomatitis
17%
Headache
16%
Confusional state
16%
Cough
16%
Muscular weakness
16%
Dehydration
16%
Leukopenia
15%
Sepsis
15%
Arthralgia
14%
Back pain
14%
Hypophosphataemia
14%
Febrile neutropenia
12%
Hyperkalaemia
11%
Pneumonia
11%
Aspartate aminotransferase increased
11%
Electrocardiogram QT prolonged
11%
Hypoalbuminaemia
11%
Abdominal pain
11%
Alanine aminotransferase increased
9%
Hypertension
9%
Insomnia
9%
Leukocytosis
9%
Hyperuricaemia
8%
Fall
8%
Petechiae
7%
Chills
7%
Anxiety
7%
Asthenia
7%
International normalised ratio increased
7%
Lymphopenia
6%
Hyperhidrosis
6%
Productive cough
6%
Dry mouth
6%
Haemorrhoids
6%
Haematuria
6%
Myalgia
6%
Oropharyngeal pain
6%
Pain in extremity
6%
Hyperamylasaemia
5%
Contusion
5%
Rhinorrhoea
5%
Tinnitus
5%
Dysphagia
5%
Gingival bleeding
5%
Pollakiuria
5%
Night sweats
5%
Bone pain
5%
Hyperlipasaemia
5%
Urinary tract infection
5%
Orthostatic hypotension
5%
Oedema
5%
Dyspepsia
4%
Malignant neoplasm progression
4%
Lung infection
4%
Depression
4%
Hyperbilirubinaemia
4%
Upper respiratory tract infection
4%
Bacteraemia
4%
Non-cardiac chest pain
4%
Dysuria
4%
Muscle spasms
3%
Tachycardia
3%
Death
3%
Respiratory failure
3%
Staphylococcal infection
3%
Sinus tachycardia
3%
Neuropathy peripheral
3%
Syncope
3%
Amnesia
3%
Cataract
3%
Hypoacusis
3%
Proteinuria
3%
Skin lesion
2%
Disease progression
2%
Multiple organ dysfunction syndrome
2%
Hypoxia
2%
Haemoptysis
2%
Gait disturbance
2%
Malaise
2%
Nasal congestion
2%
Peripheral sensory neuropathy
2%
Dry eye
2%
Urinary incontinence
2%
Alopecia
2%
Neck pain
2%
Embolism
2%
Central nervous system haemorrhage
2%
Agitation
2%
Hypermagnesaemia
1%
Pain
1%
Performance status decreased
1%
Sudden death
1%
Post procedural haemorrhage
1%
Transfusion reaction
1%
Wrist fracture
1%
Ejection fraction decreased
1%
Pleural effusion
1%
Respiratory arrest
1%
Leukostasis syndrome
1%
Encephalopathy
1%
Seizure
1%
Guillain-Barre syndrome
1%
Haemorrhage intracranial
1%
Somnolence
1%
Gastrointestinal haemorrhage
1%
Cholecystitis acute
1%
Flank pain
1%
Epiglottitis
1%
Gastroenteritis
1%
Localised infection
1%
Mastoiditis
1%
Mucormycosis
1%
Dysarthria
1%
Visual impairment
1%
Hypercalcaemia
1%
Acute respiratory distress syndrome
1%
Cerebral haemorrhage
1%
Nervous system disorder
1%
Haematochezia
1%
Pancreatitis
1%
Upper gastrointestinal haemorrhage
1%
Acute kidney injury
1%
Joint effusion
1%
Appendicitis
1%
Cellulitis
1%
Pharyngitis
1%
Pneumonia fungal
1%
Respiratory syncytial virus infection
1%
Sinusitis
1%
Skin infection
1%
Spinal fracture
1%
Atrial fibrillation
1%
Ascites
1%
Pseudomonal bacteraemia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Acute Myeloid Leukemia (AML)
Other Hematological Malignancies (ALL, CML and CLL)
Diffuse Large B-cell Lymphoma (DLBCL)
Non-Hodgkin Lymphoma (NHL) Excluding DLBCL
Multiple Myeloma (MM)
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
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
1,113 Previous Clinical Trials
358,865 Total Patients Enrolled
William Lawrence and Blanche Hughes FoundationOTHER
Karyopharm Therapeutics IncIndustry Sponsor
88 Previous Clinical Trials
7,557 Total Patients Enrolled
Andrew E Place, MD,PhDPrincipal InvestigatorDana-Farber Cancer Institute
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My brain or spinal cord disease is at a manageable stage.I do not plan to receive any non-study cancer treatments during the trial.I have not taken certain medications recently.I need more chemotherapy to help treat my condition.I have recovered from side effects of my previous cancer treatments.I agree not to breastfeed while participating in this study.I (or my guardian) can understand and sign the consent form.I cannot take or tolerate medications by mouth.My condition did not improve after two initial treatments.My doctor thinks more chemotherapy won't help me.I do not have any major health, mental, or social issues affecting my daily life.I have had cancer before, but not acute leukemia or under certain conditions.My leukemia has returned or is not responding to treatment.My cancer has returned after treatment more than once.I am between 1 and 21 years old.My organs are functioning well.I am a woman who can have children and have a confirmed negative pregnancy test.I am eligible for a stem cell transplant from a donor.My leukemia has spread to my brain.My body does not respond to blood or platelet transfusions.I am mostly active and can do most of my daily activities.
Research Study Groups:
This trial has the following groups:- Group 1: KPT-330
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.