Expanded Access to Ulixertinib (BVD-523) in Patients With Advanced MAPK Pathway-Altered Malignancies

• ClinicalTrials.gov Identifier: NCT04566393
• Expanded Access Status: Available
• First Posted: September 28, 2020
• Last Update Posted: February 8, 2022
• Sponsor: xCures
• Collaborator: Cancer Commons
• Information provided by (Responsible Party): xCures

Study Description

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.

Condition or disease	Intervention/treatment
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	Drug: Ulixertinib (BVD-523)

Study Design

• Study Type: Expanded Access
• Expanded Access Type: Intermediate-size Population

See clinical trials of the intervention/treatment in this expanded access record.

• Official Title: Expanded Access to Ulixertinib (BVD-523) in Patients With Advanced MAPK Pathway-Altered Malignancies

Interventions

Intervention Details:

• Drug: Ulixertinib (BVD-523)
  Ulixertinib (BVD-523) is an oral, first-in-class ERK1/2 inhibitor

Eligibility Criteria

• Ages Eligible for Study: 12 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All

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.

Contacts and Locations

Contacts

Contact: xCures Clinical Operations; (707) 641-4475; expandedaccess@xcures.com

Locations

United States, Alabama

University of Alabama at Birmingham; Available

Birmingham, Alabama, United States, 35294

Principal Investigator: Louis B Nabors, MD

United States, California

Kaiser Permanente Los Angeles Medical Center; Available

Los Angeles, California, United States, 90027

Principal Investigator: Richard M Green, MD

Hoag Memorial Hospital Presbyterian; Available

Newport Beach, California, United States, 92663

Principal Investigator: Michael Demeure, MD

xCures Inc.; Available

San Francisco, California, United States, 94105

Providence Saint John's Health Center; Available

Santa Monica, California, United States, 90404

Principal Investigator: Naveed Wagle, MD

United States, Colorado

University of Colorado Denver Anschutz Medical Campus; Available

Aurora, Colorado, United States, 80045

Principal Investigator: Rene Gonzalez-Cotarelo, MD

United States, District of Columbia

MedStar Georgetown University Hospital; Available

Washington, District of Columbia, United States, 20007

Principal Investigator: Benjamin A Weinberg, MD

United States, Indiana

Franciscan Health; Available

Indianapolis, Indiana, United States, 46237

Principal Investigator: Meghana Raghavendra, MD

United States, Maine

Harold Alfond Center for Cancer Care; Available

Augusta, Maine, United States, 04330

Principal Investigator: Rachit Kumar, MD

United States, Michigan

Cancer and Leukemia Center; Available

Sterling Heights, Michigan, United States, 48314

Principal Investigator: Andrew Muskovitz, MD

United States, Nebraska

Nebraska Cancer Specialists; Available

Omaha, Nebraska, United States, 68130

Principal Investigator: Joel M Michalski, MD, PhD

United States, New Jersey

Monmouth Medical Center; Available

Long Branch, New Jersey, United States, 07740

Principal Investigator: Seth Cohen, MD

United States, North Carolina

Duke Health; Available

Durham, North Carolina, United States, 27710

Principal Investigator: Lars M Wagner, MD

United States, Pennsylvania

Lehigh Valley Health Network; Available

Allentown, Pennsylvania, United States, 18103

Principal Investigator: Suresh Nair, MD

United States, Texas

Texas Oncology - Denton; Available

Denton, Texas, United States, 76201

Principal Investigator: Sharad K Jain, MD

United States, Washington

Seattle Cancer Care Alliance; Available

Seattle, Washington, United States, 98109

Principal Investigator: Elena G Chiorean, MD

Sponsors and Collaborators

xCures

Cancer Commons

More Information

• Responsible Party: xCures
• ClinicalTrials.gov Identifier: NCT04566393
• Other Study ID Numbers: ULI-EAP-100
• First Posted: September 28, 2020
• Last Update Posted: February 8, 2022
• Last Verified: February 2022

Additional relevant MeSH terms:

Glioblastoma; Thyroid Neoplasms; Cholangiocarcinoma; Neoplasms by Site; Neoplasms; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Nerve Tissue; Endocrine Gland Neoplasms; Endocrine System Diseases; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Astrocytoma; Glioma; Neoplasms, Neuroepithelial; Head and Neck Neoplasms; Thyroid Diseases