Innovative Trial for Understanding the Impact of Targeted Therapies in NF2 (INTUITT-NF2)
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|ClinicalTrials.gov Identifier: NCT04374305|
Recruitment Status : Recruiting
First Posted : May 5, 2020
Last Update Posted : April 27, 2021
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
|Condition or disease||Intervention/treatment||Phase|
|Neurofibromatosis Type 2 Vestibular Schwannoma Non-vestibular Schwannoma Meningioma Ependymoma||Drug: Brigatinib||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||80 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||The study is a platform trial that includes a Master Protocol with embedded Drug Sub-studies. Initially, there is 1 drug substudy of brigatinib. The overall number of arms is not fixed by design because the investigators include arm-dropping rules for futility and allow for the possibility of arm addition by amendment.|
|Masking:||None (Open Label)|
|Official Title:||Innovative Trial for Understanding the Impact of Targeted Therapies in NF2 (INTUITT-NF2)|
|Actual Study Start Date :||June 20, 2020|
|Estimated Primary Completion Date :||December 1, 2029|
|Estimated Study Completion Date :||December 1, 2030|
Experimental: Brigatinib Sub-Study
Subjects treated in this arm will receive brigatinib 90 mg by mouth daily for 7 days and then increased to 180 mg by mouth daily if the drug is tolerated.
Oral daily per predetermined dosage per protocol.
Other Name: Alunbrig
- Radiographic response rate (for each drug substudy) [ Time Frame: 2 years ]
Radiographic response rates in target tumors according to tumor-associated criteria:
- VS, non-VS, and meningiomas: Dombi criteria (2013)
- Ependymomas: RECIST 1.12
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability [ Time Frame: 2 years ]Number of Participants with Treatment Emergent Adverse Events as Assessed CTCAE v5.0
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04374305
|Contact: Scott Plotkin, MDemail@example.com|
|United States, California|
|UCLA Medical Center||Recruiting|
|Los Angeles, California, United States, 90095|
|Contact: Phioanh (Leia) Nghiemphu, MD PNghiemphu@mednet.ucla.edu|
|Principal Investigator: Phioanh (Leia) Nghiemphu, MD|
|United States, Maryland|
|Johns Hopkins Hospital||Recruiting|
|Baltimore, Maryland, United States, 21287|
|Contact: Jaishri Blakeley, MD firstname.lastname@example.org|
|Principal Investigator: Jaishri Blakeley, MD|
|United States, Massachusetts|
|Massachusetts General Hospital||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: Scott Plotkin, MD 617-724-8770 email@example.com|
|Principal Investigator: Scott Plokin, MD|
|United States, Minnesota|
|Mayo Clinic Hospital - Rochester||Recruiting|
|Rochester, Minnesota, United States, 55905|
|Contact: Dusica Babovic-Vuksanovic, MD firstname.lastname@example.org|
|Principal Investigator: Dusica Babovic-Vuksanovic, MD|
|United States, New York|
|New York University Langone Medical Center||Recruiting|
|New York, New York, United States, 10016|
|Contact: Kaleb Yohay, MD Kaleb.Yohay@nyulangone.org|
|Principal Investigator: Kaleb Yohay, MD|
|Principal Investigator:||Scott Plotkin, MD||Massachusetts General Hospital|