RET-fusion Non Small Cell Lung CancerNCT04697446
External Control, Observational, Retrospective Study Comparing Pralsetinib to Best Available Therapy in Patients With RET-Fusion Positive NSCLC
An observational study of RET-fusion Non Small Cell Lung Cancer and Lung Neoplasm, run by Blueprint Medicines Corporation at 3 sites in 3 countries.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- RET-fusion Non Small Cell Lung Cancer, Lung Neoplasm, Carcinoma, Non-Small-Cell Lung
- How many people
- 279 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 1 Dec 2020to 31 Oct 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: This study will include patients with locally advanced or metastatic RET-fusion positive non-small cell lung cancer (NSCLC)
Who may be able to join
- 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
Show all 11Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
United States1 site
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
France1 site
University Hospital Center of Toulouse - Larrey Hospital
Toulouse, 31300
About this study
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
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
- Medical subject headings
- Lung Neoplasms, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms, Thoracic Neoplasms, Neoplasms by Site, Neoplasms, Lung Diseases, Respiratory Tract Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: Up to 12 years
Secondary outcomes (8)
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
Measured: Up to 12 years
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
Measured: Up to 12 years
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
Measured: Up to 12 years
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
Measured: Up to 12 years
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
Measured: Up to 12 years
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
Measured: Up to 12 years
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
Measured: Up to 12 years
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
Measured: Up to 12 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jan 2021
- Study start1 Dec 2020
- Primary completion31 Oct 2021 (estimated)
- Study completion31 Oct 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RET-fusion Non Small Cell Lung CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 279 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Analysis Group, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04697446
- Sponsor's protocol ID
- BLU-667-2404
- Status last verified
- Aug 2021
- First submitted
- 16 Dec 2020
- Last updated
- 10 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04697446
- Acronym / sponsor ID
- BLU-667-2404
- Conditions
- RET-fusion Non Small Cell Lung Cancer, Lung Neoplasm, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms, Thoracic Neoplasms, Neoplasms by Site
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 279 participants (estimated)
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- Collaborators
- Analysis Group, Inc.
- Study start
- 1 Dec 2020 (actual)
- Primary completion
- 31 Oct 2021 (estimated)
- Study completion
- 31 Oct 2021 (estimated)
- First posted
- 6 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- This study will include patients with locally advanced or metastatic RET-fusion positive non-small cell lung cancer (NSCLC)
Key inclusion criteria (11 listed)
- 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:
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Comparative evaluation of real-world response rate (rwORR) between patients receiving best available therapy versus pralsetinib
Time frame: Up to 12 years
rwORR, defined as the proportion of patients with clinician-assess complete response (CR) or partial response (PR)
8 secondary outcomes
Comparative evaluation between patients receiving best available therapy versus pralsetinib of Overall survival (OS)
Time frame: Up to 12 years
Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world duration of response (rwDOR)
Time frame: Up to 12 years
Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world disease control rate (rwDCR)
Time frame: Up to 12 years
Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world clinical benefit rate (rwCBR)
Time frame: Up to 12 years
Comparative evaluation between patients receiving best available therapy versus pralsetinib of Real-world progression-free survival (rwPFS)
Time frame: Up to 12 years
Comparative evaluation between patients receiving best available therapy versus pralsetinib of Duration of treatment (DOT)
Time frame: Up to 12 years
Comparative evaluation between patients receiving best available therapy versus pralsetinib of Time to next treatment line (TtNTL)
Time frame: Up to 12 years
To characterize the safety profile and conduct comparative evaluation of safety between patients receiving best available care vs. pralsetinib
Time frame: Up to 12 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2021.
- Individual participant dataSharing planned: No
Related trials for RET-fusion Non Small Cell Lung Cancer
Active, not recruitingPhase 3
A Study of Selpercatinib (LY3527723) in Participants With Advanced or Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer
Non-Small Cell Lung Cancer
RecruitingPhase 2
Cabozantinib in Patients With RET Fusion-Positive Advanced Non-Small Cell Lung Cancer and Those With Other Genotypes: ROS1 or NTRK Fusions or Increased MET or AXL Activity
Non-Small Cell Lung Cancer
Active, not recruitingPhase 1/2
Amivantamab With Tyrosine Kinase Inhibitors (TKI) for Advanced NSCLC With ALK, ROS1, or RET Alterations
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RecruitingPhase 3
A Prospective, Single-center, Non-randomized, Interventional Study on the Effectiveness and Safety of Combined Immunotargeted Therapy in Patients With Metastatic Non-small Cell Cancer With a RET Gene Translocation
NSCLC (Non-small Cell Lung Cancer)R
RecruitingPhase 4
Pralsetinib DDI Study in Patients With Advanced or Metastatic Solid Tumors
NSCLC (Non-small Cell Lung Cancer), Thyroid Cancer
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of RET-fusion Non Small Cell Lung Cancer and Lung Neoplasm, run by Blueprint Medicines Corporation at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 10 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, France, Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Comparative evaluation of real-world response rate (rwORR) between patients receiving best available therapy versus pralsetinib (time frame: Up to 12 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Aug 2021.