Non-Small Cell Lung CancerNCT04204928
Pre-Approval Access Program (PAAP) for Pralsetinib (BLU-667) in Patients With Unresectable or Metastatic NSCLC or MTC
A study testing pralsetinib (BLU-667) in people with Non-Small Cell Lung Cancer and Medullary Thyroid Cancer, run by Blueprint Medicines Corporation.
Trial summary
This is a global, multicenter, open-label pre-approval access program to provide access to pralsetinib (BLU-667) until such time that pralsetinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Non-Small Cell Lung Cancer, Medullary Thyroid Cancer
- What is being tested?
- pralsetinib (BLU-667)
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 12 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 1a. Pathologically documented and definitively diagnosed non-resectable or metastatic NSCLC with a RET fusion for patients who are either treatment naïve, or who have been previously treated with systemic therapy. In the presence of a primary driver mutation, such as EGFR, ALK, ROS1, NTRK, or BRAF, the patient must be treated with the appropriate targeted therapy first. Patient is eligible if RET fusion is confirmed AND patient has undergone initial therapy for his/her driver mutation, or
- 1b. Pathologically documented and definitively diagnosed RET mutation in advanced MTC patients who are treatment naïve or who have been previously treated with MKI therapy, or
- 1c. Pathologically documented and definitively diagnosed advanced solid tumor with an oncogenic RET fusion previously treated with standard of care appropriate for the tumor type.
- 2. If previously treated with a selective RET inhibitor (e.g., RETEVMO), confirm patient has not progressed but has discontinued due to adverse event(s).
- 3. Patient is not eligible for an ongoing study of pralsetinib or cannot access an ongoing study of pralsetinib.
- 4. Patient is ≥ 12 years of age. 5. Patient has adequate vital organ function, including heart, lungs, liver, kidneys, bone marrow and endocrine, and is expected to tolerate therapy with a tyrosine kinase inhibitor.
Show all 14Show fewer
- 6. No presence of clinically symptomatic interstitial lung disease or interstitial pneumonitis, including radiation pneumonitis (i.e., affecting activities of daily living or requiring therapeutic intervention).
- 7. Patient or patient's legal guardian, if permitted by local regulatory authorities, intends to provide informed consent prior to the start of treatment with pralsetinib.
- 8. Patient does not require therapy with a concomitant medication that is a strong inhibitor or strong inducer of cytochrome P450 (CYP) 3A4.
- 9. Patient has not received treatment with any systemic anticancer therapy (except for immunotherapy or other antibody therapies) and all forms of radiotherapy within 14 days or 5 half-lives prior to the first dose of pralsetinib. Pralsetinib may be started within these washout periods if considered by the healthcare provider to be safe and within the best interest of the patient, with prior Sponsor approval.
- 10. Patient has not received treatment with any immunotherapy or other antibody therapy within 28 days prior to the first dose of pralsetinib (immune related toxicities must have resolved to < Grade 2 prior to starting pralsetinib).
- 11. Patient has not had a major surgical procedure (minor surgical procedures such as central venous catheter placement, tumor needle biopsy, and feeding tube placement are not considered major surgical procedures) within 14 days prior to the first dose of pralsetinib.
- 12. Women must be willing, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ highly effective contraception during pralsetinib administration period and for at least 30 days after the last dose of pralsetinib. Men, if not surgically sterile, must be willing to abstain from sexual intercourse or employ highly effective contraception during pralsetinib administration period and for at least 90 days after the last dose of pralsetinib.
- 13. Women must not be pregnant or breastfeeding.
Who cannot join
None listed.
About this study
This is a global, multicenter, open-label pre-approval access program to provide access to pralsetinib (BLU-667) until such time that pralsetinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.
- Being tested
- pralsetinib (BLU-667)
- Medical subject headings
- Carcinoma, Non-Small-Cell Lung, Carcinoma, Medullary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pralsetinib (BLU-667)Drug
Pralsetinib will be administered orally (PO) at a dose of 400 mg once daily (QD) in continuous 28 day cycles
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04204928
- Sponsor's protocol ID
- BLU-667-PAAP-01
- Status last verified
- Aug 2021
- First submitted
- 17 Dec 2019
- Last updated
- 13 Aug 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pre-Approval Access Program (PAAP) for Pralsetinib (BLU-667) in Patients With Unresectable or Metastatic NSCLC or MTC
- ClinicalTrials.gov ID
- NCT04204928
- Acronym / sponsor ID
- BLU-667-PAAP-01
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- First posted
- 19 Dec 2019 (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 a global, multicenter, open-label pre-approval access program to provide access to pralsetinib (BLU-667) until such time that pralsetinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.
Study design
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (14 listed)
- 1a. Pathologically documented and definitively diagnosed non-resectable or metastatic NSCLC with a RET fusion for patients who are either treatment naïve, or who have been previously treated with systemic therapy. In the presence of a primary driver mutation, such as EGFR, ALK, ROS1, NTRK, or BRAF, the patient must be treated with the appropriate targeted therapy first. Patient is eligible if RET fusion is confirmed AND patient has undergone initial therapy for his/her driver mutation, or
- 1b. Pathologically documented and definitively diagnosed RET mutation in advanced MTC patients who are treatment naïve or who have been previously treated with MKI therapy, or
- 1c. Pathologically documented and definitively diagnosed advanced solid tumor with an oncogenic RET fusion previously treated with standard of care appropriate for the tumor type.
- 2. If previously treated with a selective RET inhibitor (e.g., RETEVMO), confirm patient has not progressed but has discontinued due to adverse event(s).
- 3. Patient is not eligible for an ongoing study of pralsetinib or cannot access an ongoing study of pralsetinib.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| pralsetinib (BLU-667)Pralsetinib will be administered orally (PO) at a dose of 400 mg once daily (QD) in continuous 28 day cycles | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2021.
Related trials for Non-Small Cell Lung Cancer
Active, not recruitingPhase 3
A Study of Combination Amivantamab and Carboplatin-Pemetrexed Therapy, Compared With Carboplatin-Pemetrexed, in Participants With Advanced or Metastatic Non-Small Cell Lung Cancer Characterized by Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertions
Carcinoma, Non-Small-Cell Lung
RecruitingPhase 1
A Study to Find a Suitable Dose of ASP5834 in Adults With Solid Tumors
Solid Tumor, Non-Small-Cell Lung Cancer
RecruitingPhase 1/2
KEYMAKER-U01 Substudy 01A: Efficacy and Safety Study of Pembrolizumab (MK-3475) With or Without Chemotherapy When Used With Investigational Agents in Treatment-naïve Participants With Stage IV Non-small Cell Lung Cancer (NSCLC) (MK-3475-01A/KEYMAKER-U01A)
Carcinoma, Non-Small-Cell Lung
RecruitingPhase 3
A Global Phase III Study of Rilvegostomig or Pembrolizumab Plus Chemotherapy for First-Line Treatment of Locally Advanced or Metastatic Squamous Non-small Cell Lung Cancer (NSCLC)
Non-small Cell Lung Cancer
Active, not recruitingPhase 3
A Study of DZD9008 Versus Platinum-Based Doublet Chemotherapy in Local Advanced or Metastatic Non-small Cell Lung Cancer (WU-KONG28)
Non-small Cell Lung 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.
Questions about this trial
What is this clinical trial studying?
A study testing pralsetinib (BLU-667) in people with Non-Small Cell Lung Cancer and Medullary Thyroid Cancer, run by Blueprint Medicines Corporation.
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 14 inclusion and 0 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 13 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
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.
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 13 Aug 2021.