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Plexiform NeurofibromasPhase 2NCT02177825

Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas

A Phase 2 study testing Imatinib Mesylate in people with Plexiform Neurofibromas, run by St. Justine's Hospital at 1 site in Canada.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This phase II trial will test the hypothesis that inhibition of c-kit signalling pathways in pediatric patients with Neurofibromatosis Type I(NF-1) and progressing plexiform neurofibroma will result in objective reduction and/or inhibition of plexiform neurofibromas progression. Read more about this study

Who is it for?
Ages 2 to 21 years
Conditions the study is for
Plexiform Neurofibromas
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
5 participants
Where
1 siteCanada
Study dates
Jun 2014to 1 Mar 2019
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 2 to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age Greater than or equal to 2 years and up to 21 years of age at time of study enrolment
  • Diagnosis Patients with NF1 and an inoperable plexiform NFs that has the potential to cause significant morbidity.
  • Patients must have measurable disease by magnetic resonance imaging (MRI) and progressive plexiform neurofibroma(s) with or without clinical symptoms.
  • Patients must have a recent FDG-PET scan imaging study done in the last 3 months before being offered participation to the study
  • Surgery/Residual disease: Patients are only eligible if complete tumor resection is not feasible, or if a patient with a surgical option refuses surgery. Evidence of recurrent or progressive disease is NOT necessary. Patients must be at least 21 days from surgery, if performed, prior to receiving their first dose of study drug
  • Performance level Patients must have a Karnofsky of > 70% or Lansky of >50% and a life expectancy of > 6 months.
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  • Previous use of imatinib is permitted if there was no progressive disease during treatment.
  • Prior therapy Patients must be at least 28 days without any treatment before enrolment in this study.
  • Patient is free of another primary malignancy except if the other primary malignancy neither currently clinically significant nor requiring active intervention.
  • Organ function requirement
  • Creatinine < 1.5 x upper limit of normal (ULN)
  • Total bilirubin < 1.5 x ULN and SGOT and SGPT < 2.5 x ULN
  • ANC > 1.5 x 109/L and Platelets > 100 x 109/L
  • Reproductive potential Female patients of childbearing potential must have negative pregnancy test within 7 days before initiation of study drug dosing. Male and female patients of reproductive potential must agree to employ an effective barrier method of birth control throughout the study and for up to 3 months following discontinuation of study drug.

Who cannot join

  • Patient has received any other investigational agents within 28 days of first day of study drug dosing.
  • Patient with rapidly progressing disease may be enrolled before the 28 days period. In these cases, only the study chair can take this decision.
  • Patient with Grade III/IV cardiac problems as defined by the New York Heart Association Criteria.
  • Female patients who are pregnant or breast-feeding.
  • 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 central nervous system (CNS) changes on MRI/CT characteristic of NF1 are allowed, but not known CNS malignancies.
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  • Patient has known chronic liver disease (i.e., chronic active hepatitis, and cirrhosis).
  • Patient has a known diagnosis of human immunodeficiency virus (HIV) infection.
  • Patient received chemotherapy within 4 weeks prior to study entry.
  • Patient previously received radiotherapy to greater than or equal to 25% of the bone marrow within 24 months.
  • Patient had a major surgery within 2 weeks prior to study entry.
  • Patient with any significant history of non-compliance to medical regimens.
  • 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.

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • CHU Sainte-Justine

    Montreal, Quebec, H3T 1C5

Other trials in Canada

About this study

Official title: Phase II Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas

This phase II trial will test the hypothesis that inhibition of c-kit signalling pathways in pediatric patients with Neurofibromatosis Type I(NF-1) and progressing plexiform neurofibroma will result in objective reduction and/or inhibition of plexiform neurofibromas progression.

This will be a Phase II study of imatinib mesylate given orally. Patients with stable or responding disease may receive the drug for a period not exceeding one year.

Read the detailed description

Clinical objectives

  1. Demonstrate the clinical benefit of imatinib in a pediatric patient population with progressing and metabolically active plexiform neurofibromas (NF)
  2. Demonstrate the need or not to pursue treatment for more than a year in responders to imatinib

Biological studies objectives

  1. Identify biological markers of plexiform neurofibroma progression and response to treatment
  2. Identify biological markers of mast cell responses to imatinib, given that mast cells are required for tumorigenesis and are a target for imatinib

Imaging studies objectives

Using 18-Fluorodeoxyglucose-positron Emission Tomography (FDG PET/CT):

  1. Identify imaging characteristics of progressing plexiform neurofibromas
  2. Assess the role of F18-FDG PET/CT in comparison with CT/MRI to evaluate response to imatinib ¸

Pharmacological study

  1. Evaluate trough plasma levels of imatinib and its active metabolite (NDMIL N-desmethyl imatinib) achieved in this pediatric population
  2. Identify potential correlation between imatinib (and NDMI) trough levels achieved and clinical response
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

    Also called Imatinib

    oral administration

Study groups in NCT02177825
Study groupRoleWhat they receive
Imatinib MesylateExperimentalImatinib Mesylate at 110 mg/m2 up to 440mg/m2 PO per day for twelve months taken in one morning dose (if dose is less than 200 mg/day) or two doses (morning and evening)
Receives: Drug: Imatinib Mesylate

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Demonstrate the clinical benefit of imatinib in a pediatric patient population with progressing and metabolically active plexiform NFs

    Time to tumor progression as assessed by volumetric MRI and FDG-PETScan analysis at baseline, after 3, 6, 9, and 12 months on therapy.

    Measured: 12 months

Secondary outcomes (1)

  1. Changes in NF1 biomarkers after treatment with imatinib

    Patients on imatinib will have blood samples and urine taken at baseline and every 3 months until treatment completion or until disease progression. Mastocyte activation biomarkers will be monitored and evaluated as potential disease state indicators.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Jun 2014 (estimated)
  2. Study startJun 2014
  3. Primary completion1 Mar 2019
  4. Study completion1 Mar 2019

Why it stopped: Slow accrual 5 patients out of 25 expected. Primary objectives could not be met without recruiting more patients.

Resources

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
5 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02177825
Sponsor's protocol ID
PLNEU SJ 01
Status last verified
Apr 2019
First submitted
25 Jun 2014
Last updated
8 Apr 2019
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Imatinib Mesylate in people with Plexiform Neurofibromas, run by St. Justine's Hospital at 1 site in Canada.

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 14 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: stopped early, as of the sponsor's last update on 8 Apr 2019. 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 Canada. 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 stopped early (reason given: Slow accrual 5 patients out of 25 expected. Primary objectives could not be met without recruiting more patients.). Results are not available in the source record.

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?

Demonstrate the clinical benefit of imatinib in a pediatric patient population with progressing and metabolically active plexiform NFs (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02177825.

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 8 Apr 2019.

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