NCT04771520

Avapritinib for the Treatment of CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors

Phase 2 Study of Avapritinib in Patients With CKIT or PDGFRA Mutation-Positive Malignant Solid Tumors

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies the effect of avapritinib in treating malignant solid tumors that have a genetic change (mutation) in CKIT or PDGFRA and have spread to nearby tissue or lymph nodes (locally advanced) or other places in the body (metastatic). Avapritinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Avapritinib may help to control the growth of malignant solid tumors.

Detailed description

PRIMARY OBJECTIVE: I. To determine the objective response rate (ORR) of avapritinib in patients with pathogenic CKIT or PDGFRA activating mutation-positive malignant solid tumors, as assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 or RANO criteria (as appropriate). SECONDARY OBJECTIVES: I. To evaluate the duration of response (DoR) to avapritinib in patients with pathogenic CKIT or PDGFRA activating mutation-positive malignant solid tumors. II. To evaluate the disease control rate (DCR) of avapritinib in patients with pathogenic CKIT or PDGFRA activating mutation-positive malignant solid tumors. III. To determine the safety and tolerability of avapritinib in patients with pathogenic CKIT or PDGFRA activating mutation-positive malignant solid tumors, as assessed by the National Cancer institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0. EXPLORATORY OBJECTIVES: I. To evaluate the overall survival (OS) of patients with pathogenic CKIT or PDGFRA activating mutation-positive malignant solid tumors receiving avapritinib. II. To evaluate the ORR and DoR of avapritinib in patients with measurable brain or central nervous system (CNS) metastases at baseline. III. To evaluate the progression-free survival (PFS) of patients with pathogenic CKIT or PDGFRA activating mutation-positive malignant solid tumors receiving avapritinib. IV. To evaluate the correlation between genomic mutations and clinical outcome. V. To evaluate time on treatment (including patients treated beyond progression). VI. To evaluate baseline genomics and circulating cell-free deoxyribonucleic acid (cfDNA) and functional analyses of variants. OUTLINE: Patients receive avapritinib orally (PO) once daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days and then every 8 weeks.

Conditions

• Anatomic Stage IV Breast Cancer AJCC v8
• Clinical Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8
• Clinical Stage IVA Gastroesophageal Junction Adenocarcinoma AJCC v8
• Clinical Stage IVB Gastroesophageal Junction Adenocarcinoma AJCC v8
• Locally Advanced Malignant Solid Neoplasm
• Locally Advanced Melanoma
• Locally Advanced Primary Malignant Central Nervous System Neoplasm
• Locally Advanced Sarcoma
• Metastatic Malignant Solid Neoplasm
• Metastatic Melanoma
• Metastatic Primary Malignant Central Nervous System Neoplasm
• Metastatic Sarcoma
• Pathologic Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8
• Pathologic Stage IVA Gastroesophageal Junction Adenocarcinoma AJCC v8
• Pathologic Stage IVB Gastroesophageal Junction Adenocarcinoma AJCC v8
• Postneoadjuvant Therapy Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8
• Postneoadjuvant Therapy Stage IVA Gastroesophageal Junction Adenocarcinoma AJCC v8
• Postneoadjuvant Therapy Stage IVB Gastroesophageal Junction Adenocarcinoma AJCC v8
• Prognostic Stage IIIC Breast Cancer AJCC v8
• Prognostic Stage IV Breast Cancer AJCC v8
• Stage IIIC Colorectal Cancer AJCC v8
• Stage IIIC Lung Cancer AJCC v8
• Stage IV Colorectal Cancer AJCC v8
• Stage IV Lung Cancer AJCC v8
• Stage IVA Colorectal Cancer AJCC v8
• Stage IVA Lung Cancer AJCC v8
• Stage IVB Colorectal Cancer AJCC v8
• Stage IVB Lung Cancer AJCC v8
• Stage IVC Colorectal Cancer AJCC v8

Interventions

Type	Name	Description
DRUG	Avapritinib	Given PO

Timeline

• Start date: 2021-01-20
• Primary completion: 2026-12-31
• Completion: 2026-12-31
• First posted: 2021-02-25
• Last updated: 2026-03-02

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study