Innovative Trial for Understanding the Impact of Targeted Therapies in NF2

  • STATUS
    Recruiting
  • End date
    Dec 1, 2030
  • participants needed
    80
  • sponsor
    Scott R. Plotkin, MD, PhD
Updated on 6 May 2021
schwannoma
neurofibromatosis type 2
measurable disease
cataract
meningioma
brigatinib
ependymoma
multiple meningiomas
investigational drug
experimental drug
bilateral tubal ligation
acoustic neuroma
nf2 gene

Summary

This is a multi-arm phase II platform-basket screening study designed to test multiple experimental therapies simultaneously in patients with neurofibromatosis type 2 (NF2) with associated progressive tumors of vestibular schwannomas (VS), non-vestibular schwannomas (non-VS), meningiomas, and ependymomas.

This Master Study is being conducted as a "basket" study that may allow people with multiple tumor types associated with NF2 to receive new drugs throughout this study. Embedded within the Master Study are individual drug substudies.

  • Investigational Drug Sub-study A: Brigatinib

Description

This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of investigational drugs to learn whether the drug(s) works in treating a specific disease.

The Master study is intended to enroll participants who will be placed into different treatment arms (sub-studies) which will each have an additional consent and enrollment processes.

-- The research study procedures include screening for eligibility, randomization to an experimental treatment sub-study, if qualified, and observation for up to 10 years.

  • Participants who have tumors grow during a treatment sub-study will be permitted to enroll in a different experimental treatment sub-study if they are eligible.
  • Participants who are not eligible for enrollment in a different treatment sub-study will be permitted to remain under observation on the Master Study to understand the growth pattern of these tumors (natural history)
  • Participants will be eligible to remain on this Master study for up to 10 years.
  • It is expected that about 80 people will take part in the Master Study
  • The study will randomize a maximum of 40 patients to each of the experimental arms. The overall size of the trial is not fixed by design because it include arm-dropping rules for futility and allow for the possibility of arm addition by amendment.

Drug Sub-study A will test the activity of brigatinib for treatment of NF2-related tumors.

  • Brigatinib is approved for the treatment of people with anaplastic lymphoma kinase (ALK)- positive metastatic non-small cell lung cancer (NSCLC) who have progressed or are intolerant to crizotinib. In preclinical models, brigatinib has shown evidence of activity against models of NF2-deficient tumors.
  • It is expected that 40 people will take part in the Brigatinib Sub-study.
  • The sub-study with brigatinib will include two stages.
  • In Stage 1, 20 subjects with any allowable tumor type will be accrued to each arm. A minimum of 2 subjects per tumor type (vestibular schwannoma, non-vestibular schwannoma, meningioma, and ependymoma) must be accrued in Stage 1. Interim analysis will be performed after Stage 1 to determine the radiographic response rate (RR) for each tumor types. Subsequently, in stage 2, another 20 subjects will be accrued into the 2 baskets with the most promising early results. If the results are equally promising for more than 2 baskets, subjects will be allocated to the appropriate number of baskets.

Details
Condition Acoustic Neuroma, Ependymoma, Meningioma, Glioma, Bilateral acoustic neurofibromatosis, Gliomas, Meningiomas, Non-vestibular Schwannoma, Non-vestibular Schwannoma, neurofibromatosis type 2, neurofibromatosis type ii, type 2 neurofibromatosis, vestibular schwannoma, Non-vestibular Schwannoma, Non-vestibular Schwannoma, Non-vestibular Schwannoma, Non-vestibular Schwannoma, Non-vestibular Schwannoma
Treatment brigatinib
Clinical Study IdentifierNCT04374305
SponsorScott R. Plotkin, MD, PhD
Last Modified on6 May 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Participants must meet all eligibility criteria outlined in the Master Study
Participants must be willing and able to provide written informed consent/assent for the brigatinib arm of the INTUITT-NF2 trial
Participant is 12 years of age and has body weight at least 40 kg on Day 1 of treatment
Patient must be able to swallow pills
Clinical laboratory values as specified below within 28 days before the first dose of study drug, as described in the protocol document
Female patients participating in this study should avoid becoming pregnant, and male patients should avoid impregnating a female partner. Non-sterilized female patients of reproductive age group and male patients should use effective methods of contraception through defined periods during and after study treatment as specified below
Female patients must meet 1 of the following
Postmenopausal for at least 1 year before the screening visit, or
Surgically sterile, or
If they are of childbearing potential, agree to practice 2 effective methods of contraception from the time of signing of the informed consent form through 4 months after the last dose of study drug, or agree to completely abstain from heterosexual intercourse. Brigatinib may decrease effectiveness of hormonal contraceptives, therefore, women are recommended to use non-hormonal methods of contraception. Highly effective non-hormonal birth control for women of child bearing potential with male partners includes
Sexual abstinence (no sexual intercourse)
Intrauterine device (IUD) or intrauterine system (IUS)
Bilateral tubal ligation (both tubes tied)
Vasectomized partner
Male patients, even if surgically sterilized (i.e., status post-vasectomy)
must agree to 1 of the following
Practice effective barrier contraception during the entire study treatment period and through 4 months after the last dose of study drug, or completely abstain from heterosexual intercourse

Exclusion Criteria

Female patients who are both lactating and breastfeeding or have a positive serum pregnancy test during the screening period or a positive urine pregnancy test on Day 1 before first dose of study drug (if applicable)
Any serious medical or psychiatric illness that could, in the investigator's opinion, potentially interfere with the completion of treatment according to this protocol
Treatment with any investigational products within 1 month or 5 half-lives (whichever is longer) before the first dose of study drug
Had major surgery within 30 days of the first dose of brigatinib. Minor surgical procedures such as catheter placement or minimally invasive biopsies are allowed
Have significant, uncontrolled, or active cardiovascular disease (as outlined in the protocol)
Have uncontrolled hypertension (defined as an average systolic blood pressure >160 or an average diastolic blood pressure >100 for adults; for children: please refer to table in protocol
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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