External Control, Observational, Retrospective Study Comparing Pralsetinib to Best Available Therapy in Patients With RET-Fusion Positive NSCLC

NCT04697446 | Unknown

• 1 other identifier: BLU-667-2404
• Study Type: observational
• Target: 279
• Locations: 3 countries
• Sites: 3

Brief Summary

This is an external control, observational, retrospective study designed to compare clinical outcomes for pralsetinib compared with best available therapy for patients with RET-fusion positive advanced NSCLC.

Conditions

RET-fusion Non Small Cell Lung Cancer; Lung Neoplasm; Carcinoma, Non-Small-Cell Lung; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplasms by Site; Neoplasms; Lung Diseases; Respiratory Tract Diseases; Carcinoma, Bronchogenic; Bronchial Diseases; Head and Neck Neoplasms; Carcinoma; Neoplasms by Histologic Type; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Metastatic Non Small Cell Lung Cancer

Outcome Measures

Primary Outcomes (1)

• Comparative evaluation of real-world response rate (rwORR) between patients receiving best available therapy versus pralsetinib

  rwORR, defined as the proportion of patients with clinician-assess complete response (CR) or partial response (PR)

  Up to 12 years

Secondary Outcomes (8)

• Comparative evaluation between patients receiving best available therapy versus pralsetinib of Overall survival (OS)

  Up to 12 years

• Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world duration of response (rwDOR)

  Up to 12 years

• Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world disease control rate (rwDCR)

  Up to 12 years

• Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world clinical benefit rate (rwCBR)

  Up to 12 years

• Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world progression-free survival (rwPFS)

  Up to 12 years

Study Arms (2)

Patients from the BLU-667-1101 (ARROW) study

Patients with Non-Small Cell Lung Cancer (NSCLC) who received treatment with pralsetinib as part of the BLU-667-1101 (ARROW) study

External Control Group

Patients with Non-Small Cell Lung Cancer (NSCLC) that received best available therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

This study will include patients with locally advanced or metastatic RET-fusion positive non-small cell lung cancer (NSCLC)

You may qualify if:

• Must have a diagnosis of locally advanced (non-resectable) or metastatic RET-fusion positive NSCLC
• Must have received at least one line of systemic therapy for locally advanced (non-resectable) or metastatic RET-fusion positive NSCLC, which may include regimens containing:
• Chemotherapy, e.g., regimens containing platinum doublet-based therapy (carboplatin, cisplatin)
• Chemotherapy in combination with other drugs will be assessed, e.g., in combination with pemetrexed, immune checkpoint inhibitors (pembrolizumab), bevacizumab
• Ramucirumab in combination with docetaxel
• Immune checkpoint inhibitors, e.g., pembrolizumab, nivolumab, and atezolizumab
• MKIs, e.g., cabozantinib, alectinib, vandetanib, sunitinib, and nintedanib
• Must be aged ≥18 years of age at the initiation of first systemic line of therapy
• Must have availabile of performance status (e.g., Eastern Cooperative Oncology Group \[ECOG\] score or Karnofsky score)
• Must have an index date at least 3 months prior to the start of data collection (in order to include patients with at least 3 months of follow-up after index date), unless date of death occurred less than three months from index date
• Must have an approved waiver of informed consent or signed informed consent for participation in the retrospective chart review study, as applicable

You may not qualify if:

• Known primary driver alteration other than RET (e.g., targetable mutation in EGFR, ALK, ROS1, or BRAF)
• History of other malignancy, other than non-melanoma skin cancer, within 1 year prior to initiation of first systemic therapy
• Received pralsetinib as the first line of systemic therapy for RET-fusion positive NSCLC, or prior to initiation of first systemic therapy

Sponsors & Collaborators

• Blueprint Medicines Corporation: lead
• Analysis Group, Inc.: collaborator

Study Sites (3)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

University Hospital Center of Toulouse - Larrey Hospital

Toulouse, 31300, France

Location

Lucerne Cantonal Hospital

Lucerne, 6000, Switzerland

Location

MeSH Terms

Conditions

Lung Neoplasms; Carcinoma, Non-Small-Cell Lung; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplasms by Site; Neoplasms; Lung Diseases; Respiratory Tract Diseases; Carcinoma, Bronchogenic; Bronchial Diseases; Head and Neck Neoplasms; Carcinoma; Neoplasms by Histologic Type; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue

Condition Hierarchy (Ancestors)

Bronchial Neoplasms; Neoplasms, Glandular and Epithelial

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 16, 2020
• First Posted: January 6, 2021
• Study Start: December 1, 2020
• Primary Completion: October 31, 2021
• Study Completion: October 31, 2021
• Last Updated: August 10, 2021

Record last verified: 2021-08

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1); US (1); FR (1)