Neurofibroma, PlexiformPhase 2NCT03688568
Study of Imatinib in Children With Neurofibromatosis and Airway Tumors
A Phase 2 study testing Imatinib Mesylate in people with Neurofibroma, Plexiform, run by Indiana University at 1 site in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 6 months to 12 years
- Conditions the study is for
- Neurofibroma, Plexiform
- What is being tested?
- Imatinib Mesylate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Sep 2018to 1 Sep 2021
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients aged: > 6 months and < 12 years of age.
- Diagnosis of neurofibromatosis type 1 (NF1).
- Presence of symptomatic airway plexiform neurofibromas ; defined by abnormal sleep study or pulmonary function testing.
- 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).
- Patients must have a Karnofsky of > 70% or Lansky of > 50% and a life expectancy of > 2 months.
- Adequate end organ function, defined as the following:
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- 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.
- 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.
- Written, voluntary informed consent/assent.
Who cannot join
- Patient has received any other investigational agents within 14 days of first day of study drug dosing.
- 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.
- 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)
- Patient has a severe and/or uncontrolled medical disease (i.e., uncontrolled diabetes, chronic renal disease, or active uncontrolled infection).
- 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.
- Patient has known chronic liver disease (i.e., chronic active hepatitis, and cirrhosis).
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- Patient has a known diagnosis of human immunodeficiency virus (HIV) infection.
- Patient received chemotherapy within 4 weeks (6 weeks for nitrosourea or mitomycin-C) prior to study entry.
- Patient previously received radiotherapy to > 25 % of the bone marrow
- Patient had a major surgery within 2 weeks prior to study entry.
- Patient/parent with any significant history of non-compliance to medical regimens or with inability to grant reliable informed consent.
- 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.
- Patient has an unstable airway requiring more urgent intervention or deemed unable to travel due to unstable airway by referring MD.
Trial locations and contacts
1 site in 1 country.
United States1 site
Riley Hospital for Children - Indiana University
Indianapolis, Indiana, 46202
About this study
Official title: Phase 2 Study of Imatinib in Children With Neurofibromatosis and Airway Tumors
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
- Conditions
- Neurofibroma, Plexiform
- Being tested
- Imatinib Mesylate
- Medical subject headings
- Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Imatinib MesylateDrug
Imatinib given orally as dose escalation treatment.
| Study group | Role | What they receive |
|---|---|---|
| Imatinib Mesylate Arm | Experimental | 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. Receives: Drug: Imatinib Mesylate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantitative Functional Airway Response
Sleep study or pulmonary function test
Measured: 12 months
Secondary outcomes (4)
Radiologic response of tumor
Volumetric MRI measurements
Measured: 12 months
Quality of Life Assessment
Quality of Life Questionnaire
Measured: 12 months
Cytokine Biomarker
Immunoassay
Measured: 12 months
Inflammatory Cell Biomarker
Flow cytometry
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2018
- Study start1 Sep 2018
- Primary completion1 Sep 2020
- Study completion1 Sep 2021 (estimated)
Why it stopped: Enrollment not feasible
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibroma, PlexiformStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kent A Robertson, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03688568
- Sponsor's protocol ID
- 1505569560
- Other IDs
- 1R01FD004830-01A2
- Status last verified
- Feb 2021
- First submitted
- 11 Sep 2018
- Last updated
- 12 Feb 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of Imatinib in Children With Neurofibromatosis and Airway Tumors
- ClinicalTrials.gov ID
- NCT03688568
- Acronym / sponsor ID
- 1505569560
- Conditions
- Neurofibroma, Plexiform
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Enrollment not feasible
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Study start
- 1 Sep 2018 (actual)
- Primary completion
- 1 Sep 2020 (actual)
- Study completion
- 1 Sep 2021 (estimated)
- First posted
- 28 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Imatinib Mesylate Arm | Experimental | Drug: Imatinib MesylateImatinib 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. |
Who may qualify
- Ages
- 6 months to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Patients aged: > 6 months and < 12 years of age.
- Diagnosis of neurofibromatosis type 1 (NF1).
- Presence of symptomatic airway plexiform neurofibromas ; defined by abnormal sleep study or pulmonary function testing.
- 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).
- Patients must have a Karnofsky of > 70% or Lansky of > 50% and a life expectancy of > 2 months.
Key exclusion criteria (13 listed)
- Patient has received any other investigational agents within 14 days of first day of study drug dosing.
- 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.
- 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)
- Patient has a severe and/or uncontrolled medical disease (i.e., uncontrolled diabetes, chronic renal disease, or active uncontrolled infection).
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Imatinib MesylateImatinib given orally as dose escalation treatment. | Drug | Imatinib Mesylate Arm |
Primary outcome
Quantitative Functional Airway Response
Time frame: 12 months
Sleep study or pulmonary function test
4 secondary outcomes
Radiologic response of tumor
Time frame: 12 months
Quality of Life Assessment
Time frame: 12 months
Cytokine Biomarker
Time frame: 12 months
Inflammatory Cell Biomarker
Time frame: 12 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Imatinib Mesylate in people with Neurofibroma, Plexiform, run by Indiana University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 12 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quantitative Functional Airway Response (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Feb 2021.