NCT05221320

Trial of Ulixertinib in Combination With Hydroxychloroquine in Patients With Advanced Gastrointestinal (GI) Malignancies

A Phase 2 Basket Trial of Ulixertinib (BVD-523) in Combination With Hydroxychloroquine in Patients With Advanced GI Malignancies Harboring Mitogen-activated Protein Kinase (MAPK) Pathway Mutations (BVD-523-HCQ)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 47 (actual)
• Sponsor: BioMed Valley Discoveries, Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, prospective phase two basket trial assessing the efficacy of ulixertinib in combination with hydroxychloroquine in patients with advanced gastrointestinal malignancies. All patients enrolled must have a mitogen-activated protein kinase (MAPK) activating mutation to be deemed eligible for trial participation. Each disease-based basket will open to enrollment in two-stages. The opening of stage two will be dependent on the observed responses in the patients enrolled in the first stage.

Detailed description

This is an open-label, multicenter, phase II basket study of ulixertinib in combination with hydroxychloroquine in patients with advanced gastrointestinal malignancies harboring rat sarcoma virus (RAS), a member of the rapidly accelerated fibrosarcoma (non-V600 BRAF), extracellular signal-regulated kinase (ERK), or mitogen-activated protein kinase (MEK) mutations. The trial will have five baskets based on disease primary as listed below. Basket 1: Cholangiocarcinoma including intrahepatic cholangiocarcinoma, perihilar cholangiocarcinoma, or extrahepatic cholangiocarcinoma; Basket 2: Pancreatic adenocarcinoma; Basket 3: Colorectal adenocarcinoma; Basket 4: Esophageal adenocarcinoma, esophageal squamous cell carcinoma, or gastroesophageal junction (GEJ) adenocarcinoma; Basket 5: Gastric adenocarcinoma. While the overall trial is a basket design, each basket will operate as a Simon two-stage design and therefore, will open to enrollment in two-stages. Total enrollment for Stage 1 is targeted at approximately 65 patients with 13 patients per group. Additional patients may be enrolled as appropriate. Total enrollment for Stage 2 is targeted to approximately 150 patients with up to 30 patients per group. Additional patients may be enrolled as appropriate.

Conditions

• Tumor, Solid
• Gastrointestinal Cancer

Interventions

Type	Name	Description
DRUG	Ulixertinib	small molecule ERK 1/2 inhibitor
DRUG	Hydroxychloroquine	Autophagy inhibitor

Timeline

• Start date: 2022-05-26
• Primary completion: 2024-05-08
• Completion: 2024-07-14
• First posted: 2022-02-02
• Last updated: 2025-09-25
• Results posted: 2025-09-25

Locations

9 sites across 1 country: United States

Regulatory

• FDA-regulated drug study