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Study of Imatinib in Children With Neurofibromatosis and Airway Tumors

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03688568
Recruitment Status : Recruiting
First Posted : September 28, 2018
Last Update Posted : February 17, 2020
Sponsor:
Information provided by (Responsible Party):
Kent A Robertson, Indiana University

Brief Summary:
The purpose of this study is to look at a subset of plexiform neurofibromas and determine if the airway tumors are more sensitive to imatinib therapy. Funding Source - FDA OOPD

Condition or disease Intervention/treatment Phase
Neurofibroma, Plexiform Drug: Imatinib Mesylate Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Study of Imatinib in Children With Neurofibromatosis and Airway Tumors
Actual Study Start Date : September 1, 2018
Estimated Primary Completion Date : September 1, 2020
Estimated Study Completion Date : September 1, 2021


Arm Intervention/treatment
Experimental: Imatinib Mesylate Arm
Imatinib Mesylate, given daily orally, 55 mg PO BID, if tolerated for 2 weeks increase to 110 mg/m2 BID, and further increase to 165 and final dosage to 220 mg/m2 bid if tolerated. Can continue for 12 months.
Drug: Imatinib Mesylate
Imatinib given orally as dose escalation treatment.




Primary Outcome Measures :
  1. Quantitative Functional Airway Response [ Time Frame: 12 months ]
    Sleep study or pulmonary function test


Secondary Outcome Measures :
  1. Radiologic response of tumor [ Time Frame: 12 months ]
    Volumetric MRI measurements

  2. Quality of Life Assessment [ Time Frame: 12 months ]
    Quality of Life Questionnaire

  3. Cytokine Biomarker [ Time Frame: 12 months ]
    Immunoassay

  4. Inflammatory Cell Biomarker [ Time Frame: 12 months ]
    Flow cytometry



Information from the National Library of Medicine

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, Learn About Clinical Studies.


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Ages Eligible for Study:   6 Months to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients aged: > 6 months and < 12 years of age.
  2. Diagnosis of neurofibromatosis type 1 (NF1).
  3. Presence of symptomatic airway plexiform neurofibromas ; defined by abnormal sleep study or pulmonary function testing.
  4. Patients must have measurable (> 1.5 cm in two dimensions or able to assess a minimum of 3 slices) disease by magnetic resonance imaging (MRI).
  5. Patients must have a Karnofsky of > 70% or Lansky of > 50% and a life expectancy of > 2 months.
  6. Adequate end organ function, defined as the following:

    total bilirubin < 1.5 x ULN, SGOT and SGPT < 2.5 x UNL, creatinine < 1.5 x ULN, ANC > 1.5 x 109/L, platelets > 100 x 109/L.

  7. Patients must be able to swallow whole pills or crushed pills in a soft food such as pudding or apple sauce; or have other GI access such as a G-tube.
  8. Written, voluntary informed consent/assent.

Exclusion Criteria:

  1. Patient has received any other investigational agents within 14 days of first day of study drug dosing.
  2. Patient is < 5 years free of another primary malignancy except: if the other primary malignancy is not currently clinically significant nor requiring active intervention, or if other primary malignancy is a basal cell skin cancer or a cervical carcinoma in situ. Existence of any other malignant disease is not allowed.
  3. Patient with Grade III/IV cardiac problems as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study)
  4. Patient has a severe and/or uncontrolled medical disease (i.e., uncontrolled diabetes, chronic renal disease, or active uncontrolled infection).
  5. Patient has a known brain metastasis. Non-specific CNS changes on MRI/CT characteristic of NF1 are allowed, but not known CNS malignancies requiring therapeutic intervention.
  6. Patient has known chronic liver disease (i.e., chronic active hepatitis, and cirrhosis).
  7. Patient has a known diagnosis of human immunodeficiency virus (HIV) infection.
  8. Patient received chemotherapy within 4 weeks (6 weeks for nitrosourea or mitomycin-C) prior to study entry.
  9. Patient previously received radiotherapy to > 25 % of the bone marrow
  10. Patient had a major surgery within 2 weeks prior to study entry.
  11. Patient/parent with any significant history of non-compliance to medical regimens or with inability to grant reliable informed consent.
  12. Patients who have or anticipate receiving permanent (or semi-permanent) metallic structures attached to their body. (e.g., braces on teeth, body piercings), which their physicians believe will interfere with the MRI.
  13. Patient has an unstable airway requiring more urgent intervention or deemed unable to travel due to unstable airway by referring MD.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03688568


Contacts
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Contact: Kent Robertson, MD 317-944-87874 krobert@iu.edu
Contact: Jen Pencek, RN 317-944-2832 jpencek@iu.edu

Locations
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United States, Indiana
Riley Hospital for Children - Indiana University Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Kent Robertson, MD    317-944-8784    krobert@iu.edu   
Contact: Jen Pencek, RN    317-944-2832    jpencek@iu.edu   
Sponsors and Collaborators
Indiana University
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Responsible Party: Kent A Robertson, Associate Professor, Indiana University
ClinicalTrials.gov Identifier: NCT03688568    
Other Study ID Numbers: 1505569560
1R01FD004830-01A2 ( U.S. FDA Grant/Contract )
First Posted: September 28, 2018    Key Record Dates
Last Update Posted: February 17, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Neurofibroma, Plexiform
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Imatinib Mesylate
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action