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

Send

Updated on 19 February 2024

See if I qualify

investigational drug

measurable disease

vasectomy

tubal ligation

karnofsky performance status

experimental drug

brigatinib

cataract

investigational therapy

ependymoma

bilateral tubal ligation

schwannoma

acoustic neuroma

neurofibromatosis type 2

conventional imaging

meningioma

conventional mri

intrauterine device (iud)

experimental therapy

multiple meningiomas

nf2 gene

neurofibromatosis

glioma

neurofibroma

meningiomatosis

Summary

Show definitions

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, Non-vestibular Schwannoma
Age	12years - 100years
Treatment	Brigatinib
Clinical Study Identifier	NCT04374305
Sponsor	Scott R. Plotkin, MD, PhD
Last Modified on	19 February 2024

How understandable was the trial content above?

Hard to understand

Easy to understand

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

How to participate?

Step 1 Connect with a study center

Send a message

Enter your contact details to connect with study team

First name*

Last name*

Email*

Phone number*

Other language

I am at least 18 years old.

By sending a message, I agree to share my information with the study team for the selected site and consent to receive emails and text messages for trial updates, notifications, and related communication. TrialX values your privacy; your information will be handled in accordance with our Privacy Policy

Step 2 Get screened

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

Step 3 Enroll in the clinical study

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.

Learn more

Step 4 Get your study results

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

Learn more

Similar trials to consider

Browse trials for

karnofsky performance status

experimental drug

brigatinib

cataract

investigational therapy

ependymoma

bilateral tubal ligation

schwannoma

acoustic neuroma

neurofibromatosis type 2

conventional imaging

meningioma

conventional mri

intrauterine device (iud)

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Innovative Trial for Understanding the Impact of Targeted Therapies in NF2