Clinical trial NCT04697446: External Control, Observational, Retrospective Study Comparing Pralsetinib to Best Available Therapy in Patients With RET-Fusion Positive NSCLC
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
Study type: OBSERVATIONAL
Overall status: UNKNOWN
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
Lead sponsor: Blueprint Medicines Corporation
Sponsor class: INDUSTRY
Collaborators: name: Analysis Group, Inc.
First posted: 2021-01-06
Enrollment: 279
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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)

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.


Eligibility criteria: 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

Primary outcomes:
measure: Comparative evaluation of real-world response rate (rwORR) between patients receiving best available therapy versus pralsetinib
timeFrame: Up to 12 years
Secondary outcomes:
measure: Comparative evaluation between patients receiving best available therapy versus pralsetinib of Overall survival (OS)
timeFrame: Up to 12 years
measure: Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world duration of response (rwDOR)
timeFrame: Up to 12 years
measure: Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world disease control rate (rwDCR)
timeFrame: Up to 12 years
measure: Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world clinical benefit rate (rwCBR)
timeFrame: Up to 12 years
measure: Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world progression-free survival (rwPFS)
timeFrame: Up to 12 years
measure: Comparative evaluation between patients receiving best available therapy versus pralsetinib of Duration of treatment (DOT)
timeFrame: Up to 12 years
measure: Comparative evaluation between patients receiving best available therapy versus pralsetinib of Time to next treatment line (TtNTL)
timeFrame: Up to 12 years
measure: To characterize the safety profile and conduct comparative evaluation of safety between patients receiving best available care vs. pralsetinib
timeFrame: Up to 12 years
Other outcomes:
Locations:
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Toulouse
country: France
facility: University Hospital Center of Toulouse - Larrey Hospital
city: Lucerne
country: Switzerland
facility: Lucerne Cantonal Hospital
Study officials:
References: