Clinical trial NCT03691207: A Study Of AL101In Patients With Adenoid Cystic Carcinoma (ACC) Bearing Activating Notch Mutations
Official title: A Phase 2, Open-Label, Multi-center Study of AL101 in Patients With Adenoid Cystic Carcinoma (ACC) Bearing Activating Notch Mutations
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Adenoid Cystic Carcinoma
Lead sponsor: Ayala Pharmaceuticals, Inc,
Sponsor class: INDUSTRY
First posted: 2018-10-01
Enrollment: 87
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label
Primary purpose: TREATMENT

Brief summary: This is a Phase 2, non comparative, open label, multicenter study of AL101 in patients with recurrent or metastatic ACC who harbor NOTCH 1,2,3,4 activating mutations.

Detailed description: This is a Phase 2, non-comparative, open-label, multicenter study of AL101 in patients with recurrent or metastatic ACC who harbor NOTCH 1,2,3,4 activating mutations.

The study includes 2 cohorts, ran in a sequential fashion:

Cohort 1 - AL101 4 mg once weekly (QW) intravenously (IV) Cohort 2 - AL101 6 mg QW IV

Eligibility criteria: 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 other than ACC in the past 2 years.
2. Uncontrolled, Active 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 ≥2.
7. Abnormal organ and marrow function

Primary outcomes:
measure: Overall Response Rate (ORR)
timeFrame: 3 years and 7 months
Secondary outcomes:
measure: Clinical Benefit Response Rate (CBR)
timeFrame: 3 years and 7 months
measure: Overall Survival
timeFrame: 3 years and 5 months
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: USC Norris Comprehensive Cancer center
state: California
city: Aurora
country: United States
facility: University of Colorado Cancer Center
state: Colorado
city: Miami
country: United States
facility: Sylvester Comprehensive Cancer Center
state: Florida
city: Tampa
country: United States
facility: H. Lee Moffitt Cancer Center & Research Institute
state: Florida
city: Baltimore
country: United States
facility: University of Maryland School of Medicine
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Rochester
country: United States
facility: Mayo Clinic Hospital
state: Minnesota
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Research Center
state: Washington
city: Calgary
country: Canada
facility: Tom Baker Cancer Centre
state: Alberta
city: Hamilton
country: Canada
facility: Hamilton Health Sciences Juravinski Cancer Centre
state: Ontario
city: London
country: Canada
facility: London Health Sciences Center
state: Ontario
city: Villejuif
country: France
facility: Institut Gustave Roussy
city: Petah Tikva
country: Israel
facility: Rabin Medical center
city: Nijmegen
country: Netherlands
facility: Radboud University
city: Manchester
country: United Kingdom
facility: The Christie NHS Foundation Trust
Study officials:
References: