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

Neoplasms Clinical Trial

Official title:

An External Control, Observational, Retrospective Study Assessing the Effect of Pralsetinib Compared With Best Available Therapy for Patients With RET-Fusion Positive Advanced Non-Small Cell Lung Cancer

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04697446
• Other study ID #: BLU-667-2404
• Status: Enrolling by invitation
• Start date: December 1, 2020
• Est. completion date: October 31, 2021

Study information

• Verified date: August 2021
• Source: Blueprint Medicines Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 279
• Est. completion date: October 31, 2021
• Est. primary completion date: October 31, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

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

Locations

Country	Name	City	State
France	University Hospital Center of Toulouse - Larrey Hospital	Toulouse
Switzerland	Lucerne Cantonal Hospital	Lucerne
United States	Memorial Sloan Kettering Cancer Center	New York	New York

Sponsors (2)

Lead Sponsor	Collaborator
Blueprint Medicines Corporation	Analysis Group, Inc.

Countries where clinical trial is conducted

United States,  France,  Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Comparative evaluation between patients receiving best available therapy versus pralsetinib of Overall survival (OS)	OS, defined as time from initiation of a given line of therapy to death from any cause	Up to 12 years
Secondary	Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world duration of response (rwDOR)	rwDOR, defined as the duration of time from the first documented clinician-assessed response to the first documented clinician-assessed progressive disease or death due to any cause within 30 days of the last radiological exam, for each line of treatment	Up to 12 years
Secondary	Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world disease control rate (rwDCR)	rwDCR, defined as proportion of patients with clinician-assessed complete response, partial response, or stable disease	Up to 12 years
Secondary	Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world clinical benefit rate (rwCBR)	rwCBR, defined as proportion of patients who had documented clinician-assessed complete response or partial response, or stable disease lasting at least 16 weeks	Up to 12 years
Secondary	Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world progression-free survival (rwPFS)	rwPFS, defined as time from initiation of line of therapy to clinician-assessed disease progression or death from any cause, whichever occurs first	Up to 12 years
Secondary	Comparative evaluation between patients receiving best available therapy versus pralsetinib of Duration of treatment (DOT)	DOT, defined as time from initiation of line of systemic treatment to discontinuation of same line of treatment for any reason	Up to 12 years
Secondary	Comparative evaluation between patients receiving best available therapy versus pralsetinib of Time to next treatment line (TtNTL)	TtNTL, defined as the time from initiation of line of systemic treatment to the initiation of the next line of treatment	Up to 12 years
Secondary	To characterize the safety profile and conduct comparative evaluation of safety between patients receiving best available care vs. pralsetinib	Adverse events (AEs) that result in treatment modification or discontinuation, hospitalization, or death according to evaluation of responsible physician	Up to 12 years