Innovative Trial for Understanding the Impact of Targeted Therapies in NF2
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- STATUS
- Recruiting
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- End date
- Dec 1, 2030
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- participants needed
- 80
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- sponsor
- Scott R. Plotkin, MD, PhD
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, Non-vestibular Schwannoma |
|---|---|
| Age | 12years - 100years |
| Treatment | Brigatinib |
| Clinical Study Identifier | NCT04374305 |
| Sponsor | Scott R. Plotkin, MD, PhD |
| Last Modified on | 19 February 2024 |
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
Learn moreIf 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 moreComplete 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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