Compassionate Use of Brontictuzumab for Adenoid Cystic Carcinoma (ACC)

Study Overview
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Brief Summary
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The goal of this study is to start treatment with the NOTCH1 inhibitor brontictuzumab in an attempt to control tumors and prolong survival. The therapy involves participant being given the drug brontictuzumab to treat adenoid cystic carcinoma (ACC) with NOTCH 1 mutations. This is considered an investigational treatment.

Detailed Description
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Treatment: Participant will receive brontictuzumab by vein over 30 minutes one time every 3 weeks. Clinic Visits: On Day 1 of Cycle 1 and then every 3 weeks from then on: Participant will have a physical exam Blood (about 3 teaspoons) will be drawn for routine tests. Every 6 weeks, participant will have an MRI and CT scan to check the status of the disease. Length of Treatment: Participant may continue taking brontictuzumab for as long as the doctor thinks it is in their best interest. Participant will no longer be able to take the drug if the disease gets worse, they develop another illness that prevents receiving more treatment, or if intolerable side effects occur. Brontictuzumab is not FDA approved or commercially available. It is currently being used for research purposes only.

Official Title
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NOTCH 1 Inhibitor Brontictuzumab for Adenoid Cystic Carcinoma Bearing Two Activating NOTCH1 Mutations

Conditions
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Adenoid Cystic Carcinoma

Intervention / Treatment
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* Drug: Brontictuzumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Eastern Cooperative Oncology Group (ECOG) performance status 0 to 1 Organ and marrow function as follows: absolute neutrophil count (ANC) ≥1000/mm3, platelets ≥50,000/dL, hemoglobin ≥8 g/dL, bilirubin ≤ 1.5 times the upper limit of normal, serum creatinine ≤1.5 mg/dL or creatinine clearance ≥50 mL/min, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 times the upper limit of normal. Central Nervous System (CNS) metastases allowed if subject does not require steroids, brain metastases are clinically stable without symptomatic progression Capability to understand and comply with the protocol and signed informed consent document. Exclusion Criteria: Major medical conditions that might affect study participation (e.g. uncontrolled pulmonary, renal, or hepatic dysfunction, uncontrolled infection, cardiac disease) Inability or unwillingness to abide by the study protocol or cooperate fully with the investigator.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Brontictuzumab<br>1.5 mg/Kg of Brontictuzumab single agent intravenously every three weeks. | Drug: Brontictuzumab<br>* 1.5 mg/Kg intravenously every three weeks.<br>* Other names: OMP-52M51;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Disease Progression | Tumor response for measurable disease based on Response Evaluation Criteria In Solid Tumors (RECIST) to establish disease progression. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions | 6 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Brontictuzumab