A Study Of AL101 In Patients With Adenoid Cystic Carcinoma (ACC) Bearing Activating Notch Mutations

Sponsor:

Collaborators:

Information provided by (Responsible Party):

，

Tracking Information

First Submitted Date ^ICMJE

September 22, 2018

First Posted Date ^ICMJE

October 1, 2018

Last Update Posted Date

October 1, 2018

Actual Study Start Date ^ICMJE

October 2018

Estimated Primary Completion Date

April 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Objective response rate (ORR; complete response [CR] and partial response [PR]) by RECIST v1.1[ Time Frame: Up to 36 month ]

Original Primary Outcome Measures ^ICMJE

Same as current

Current Secondary Outcome Measures ^ICMJE

Frequency, duration and severity of adverse events (AEs) and serious adverse events (SAEs);[ Time Frame: Up to 36 month ]

Descriptive Information

Brief Title ^ICMJE

A Study Of AL101 In Patients With Adenoid Cystic Carcinoma (ACC) Bearing Activating Notch Mutations

Official Title ^ICMJE

A Phase 2, Open-Label, Single-Arm, Multi-Center Study Of AL101 (BMS-906024) In Patients With Adenoid Cystic Carcinoma (ACC) Bearing Activating Notch Mutations

Brief Summary

This is a Phase 2, Simon 2-Stage optimal design, non comparative, open label, single-arm, multicenter study of AL101 in patients with recurrent or metastatic ACC who harbor NOTCH 1,2,3,4 activating mutations.

Detailed Description

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Allocation:
Intervention Model: Single Group Assignment
Intervention Model Description: Open label
Masking: Interventional
Masking Description:
Primary Purpose: Treatment

Condition ^ICMJE

Intervention ^ICMJE

Drug: AL101
AL101 is a small-molecule that inhibits gamma secretase, an enzyme which plays a key role in the activation of the Notch signaling pathway by releasing the Notch intracellular domain (NICD) of all four Notch receptors from the membrane. In patients with aberrant Notch signaling, AL101 may inhibit Notch signaling and potentially impede tumor growth.The drug is administered intravenous.

Study Arms

Experimental: SINGLE-ARM
AL101 is an inhibitor of gamma secretase-mediated Notch signaling.

Recruitment Information

Recruitment Status ^ICMJE

Not yet recruiting

Estimated Enrollment ^ICMJE

Original Estimated Enrollment ^ICMJE

Same as current

Estimated Study Completion Date

July 2021

Estimated Primary Completion Date

April 2021 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria: 1. Confirmed Adenoid Cystic Carcinoma with known NOTCH 1/2/3/4 activating mutation that is recurrent or metastatic, not amenable to potentially curative surgery or radiotherapy. 2. Evidence of radiographic or clinical disease progression within 6-months of signing informed consent; newly diagnosed metastatic patients will be allowed. 3. Patients must have Formalin-fixed, Paraffin-embedded tissue available . 4. Must have at least 1 target lesion that is measurable for patients with nodal or visceral metastasis. Exclusion Criteria: 1. Diagnosed with a malignancy in the past 2 years. 2. Infection 3. Gastrointestinal (GI) disease with increased risk of diarrhea [e.g. inflammatory bowel disease (IBD)] 4. Symptomatic central nervous system (CNS) metastases. 5. Unstable or severe uncontrolled medical condition 6. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1. 7. Abnormal organ and marrow function

Sex/Gender

Sexes Eligible for Study:

All

Ages

18 Years and older (Adult, Older Adult)

Accepts Healthy Volunteers

Listed Location Countries ^ICMJE

Removed Location Countries

Administrative Information

Has Data Monitoring Committee

Yes

U.S. FDA-regulated Product

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

Plan to Share IPD:

Responsible Party

，

Study Sponsor ^ICMJE

Ayala Pharmaceuticals, Inc,

Collaborators ^ICMJE

Investigators ^ICMJE

PRS Account

Verification Date

September 2018

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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