Clinical trial NCT04566393: Expanded Access to Ulixertinib (BVD-523) in Patients with Advanced MAPK Pathway-Altered Malignancies
Official title: Expanded Access to Ulixertinib (BVD-523) in Patients with Advanced MAPK Pathway-Altered Malignancies
Study type: EXPANDED_ACCESS
Overall status: AVAILABLE
Conditions: Pancreatic Cancer
Small Bowel Cancer
Colorectal Cancer
Melanoma
Non Small Cell Lung Cancer
Thyroid Cancer
Bladder Cancer
Head and Neck Cancer
Gastric Cancer
Esophageal Cancer
Cholangiocarcinoma
Ovarian Cancer
Hepatocellular Carcinoma
Glioblastoma
MAPK Gene Mutation
KRAS Activating Mutation
BRAF Gene Mutation
NRAS Gene Mutation
HRAS Gene Mutation
MEK Mutation
ERK Mutation
Lead sponsor: xCures
Sponsor class: INDUSTRY
Collaborators: name: Cancer Commons
First posted: 2020-09-28
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT

Brief summary: The objective of this expanded access program is to provide ulixertinib (BVD-523) for compassionate use in advanced cancer patients with MAPK pathway-altered solid tumor(s), including but not limited to KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations who have incomplete response to or have exhausted available therapies.

Ulixertinib is available for treatment as monotherapy or in combination with other clinically tolerable agent(s), conditionally approved by the drug manufacturer.


Eligibility criteria: Inclusion Criteria:

* Main Inclusion Criterion:

  1. Patient has a MAPK pathway-altered solid tumor(s), including but not limited to KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations.
* Other Inclusion Criteria:

  1. In the opinion of the treating physician, the patient has exhausted or has inadequate response to available anti-cancer treatments.
  2. In the opinion of the treating physician, the patient has adequate organ function to tolerate ulixertinib as defined in section 6.1
  3. Male or female patients aged ≥ 12 years.
  4. Patient must be able to swallow and retain orally administered medication.

     Note: Ulixertinib is primarily absorbed in the duodenum and therefore patients with any prior stomach or duodenal resection should be evaluated with that understanding.
  5. For females, evidence of post-menopausal status or negative urinary or serum pregnancy test for pre-menopausal patients.
  6. Highly effective contraception for both male and female patients throughout the treatment and for at least 4 months after last treatment administration. In patients under the age of 18, who are not sexually active, abstinence is an acceptable form.
  7. Toxicities related to any prior treatments are either stable, stable on supportive therapy, resolved, or in the opinion of the treating physician, clinically non-significant
  8. Ability to understand a written informed consent document, and the willingness to sign it. Assent will be obtained when appropriate based on the patient's age.

Exclusion Criteria:

1. Patient is already participating in or qualifies for and is able to enroll in a clinical trial of ulixertinib (BVD-523).
2. Patient has received systemic therapy with an investigational agent within 5 half-lives or 14 days prior to starting ulixertinib treatment, whichever is shorter.
3. Patient has received radiotherapy within 14 days prior to the first dose of ulixertinib treatment other than for the allowable treatment of symptomatic bone metastasis.
4. A history of current evidence/risk of retinal vein occlusion (RVO) or central serous retinopathy (CSR)
5. Current evidence of uncontrolled, significant intercurrent illness that would, in the treating physician's judgment, contraindicate the patient's treatment with ulixertinib due to safety concerns.
6. Patients who, in the opinion of the treating physician, have not fully recovered from recent major surgery to a sufficient extent to tolerate treatment with ulixertinib.
7. Known hypersensitivity to ulixertinib or any component in its formulation.
8. Patients taking prohibited medications as described in current Investigator's Brochure.

   Note: Patients who require treatment with Drugs that are strong inhibitors or inducers of CYP1A2, CYP2D6, and CYP3A4 (see Appendix 3) were excluded from the FIH study of ulixertinib and should be discussed with xCures to review if any potential benefits outweigh the potential risks.
9. Patient is actively breastfeeding.
10. Prior stomach or duodenal resection that in the opinion of the treating physician would affect the breakdown and absorption of ulixertinib.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Huntsville
country: United States
facility: Clearview Cancer Institute
state: Alabama
city: Mobile
country: United States
facility: Infirmary Cancer Care
state: Alabama
city: Arroyo Grande
country: United States
facility: PCR Oncology
state: California
city: Newport Beach
country: United States
facility: Hoag Memorial Hospital Presbyterian
state: California
city: San Francisco
country: United States
facility: xCures Inc.
state: California
city: Santa Monica
country: United States
facility: Providence Saint John's Health Center
state: California
city: Washington
country: United States
facility: MedStar Georgetown University Hospital
state: District of Columbia
city: Orlando
country: United States
facility: Orlando Health
state: Florida
city: Cedar Rapids
country: United States
facility: Unity Point Health - St. Lukes Hospital
state: Iowa
city: Des Moines
country: United States
facility: Iowa Oncology Research Association
state: Iowa
city: Baton Rouge
country: United States
facility: Mary Bird Perkins Cancer Center
state: Louisiana
city: Sterling Heights
country: United States
facility: Oakland Macomb Cancer Specialists
state: Michigan
city: Lincoln
country: United States
facility: Cancer Partners of Nebraska
state: Nebraska
city: Flemington
country: United States
facility: Hunterdon Hematology Oncology
state: New Jersey
city: Long Branch
country: United States
facility: Monmouth Medical Center
state: New Jersey
city: Mickleton
country: United States
facility: The Minniti Center for Medical Oncology and Hematology
state: New Jersey
city: Summit
country: United States
facility: Atlantic Health System/Overlook Medical Center
state: New Jersey
city: Brooklyn
country: United States
facility: Hirschfeld Oncology
state: New York
city: Stony Brook
country: United States
facility: Stony Brook Cancer Center
state: New York
city: Cincinnati
country: United States
facility: The Christ Hospital
state: Ohio
city: Allentown
country: United States
facility: Lehigh Valley Health Network
state: Pennsylvania
city: Tacoma
country: United States
facility: Northwest Medical Specialties
state: Washington
Study officials:
References: