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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 below

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My brain or spinal cord disease is at a manageable stage.

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I agree not to breastfeed while participating in this study.

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I (or my guardian) can understand and sign the consent form.

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My condition did not improve after two initial treatments.

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My leukemia has returned or is not responding to treatment.

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My cancer has returned after treatment more than once.

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I am between 1 and 21 years old.

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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:

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I do not plan to receive any non-study cancer treatments during the trial.

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I am eligible for a stem cell transplant from a donor.

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I cannot take or tolerate medications by mouth.

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My leukemia has spread to my brain.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 years for reporting.

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 • NCT01607892

71%

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

Hypertension

Insomnia

Leukocytosis

Hyperuricaemia

Fall

Petechiae

Chills

Anxiety

Asthenia

International normalised ratio increased

Lymphopenia

Hyperhidrosis

Productive cough

Dry mouth

Haemorrhoids

Haematuria

Myalgia

Oropharyngeal pain

Pain in extremity

Hyperamylasaemia

Contusion

Rhinorrhoea

Tinnitus

Dysphagia

Gingival bleeding

Pollakiuria

Night sweats

Bone pain

Hyperlipasaemia

Urinary tract infection

Orthostatic hypotension

Oedema

Dyspepsia

Malignant neoplasm progression

Lung infection

Depression

Hyperbilirubinaemia

Upper respiratory tract infection

Bacteraemia

Non-cardiac chest pain

Dysuria

Muscle spasms

Tachycardia

Death

Respiratory failure

Staphylococcal infection

Sinus tachycardia

Neuropathy peripheral

Syncope

Amnesia

Cataract

Hypoacusis

Proteinuria

Skin lesion

Disease progression

Multiple organ dysfunction syndrome

Hypoxia

Haemoptysis

Gait disturbance

Malaise

Nasal congestion

Peripheral sensory neuropathy

Dry eye

Urinary incontinence

Alopecia

Neck pain

Embolism

Central nervous system haemorrhage

Agitation

Hypermagnesaemia

Pain

Performance status decreased

Sudden death

Post procedural haemorrhage

Transfusion reaction

Wrist fracture

Ejection fraction decreased

Pleural effusion

Respiratory arrest

Leukostasis syndrome

Encephalopathy

Seizure

Guillain-Barre syndrome

Haemorrhage intracranial

Somnolence

Gastrointestinal haemorrhage

Cholecystitis acute

Flank pain

Epiglottitis

Gastroenteritis

Localised infection

Mastoiditis

Mucormycosis

Dysarthria

Visual impairment

Hypercalcaemia

Acute respiratory distress syndrome

Cerebral haemorrhage

Nervous system disorder

Haematochezia

Pancreatitis

Upper gastrointestinal haemorrhage

Acute kidney injury

Joint effusion

Appendicitis

Cellulitis

Pharyngitis

Pneumonia fungal

Respiratory syncytial virus infection

Sinusitis

Skin infection

Spinal fracture

Atrial fibrillation

Ascites

Pseudomonal bacteraemia

100%

80%

60%

40%

20%

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

0 / 13Eligibility criteria met