Active, not recruiting
Phase 1/2
A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001)
Condition(s) studied
Non-Small Cell Lung CancerMedullary Thyroid CancerColon CancerAny Solid Tumor
Investigational drug(s) / intervention(s)
LOXO-292: Oral LOXO-292
Study summary
This is an open-label, first-in-human study designed to evaluate the safety, tolerability, pharmacokinetics (PK) and preliminary anti-tumor activity of selpercatinib (also known as LOXO-292) administered orally to participants with advanced solid tumors, including rearranged during transfection (RET)-fusion-positive solid tumors, medullary thyroid cancer (MTC) and other tumors with RET activation.
Eligibility
Key Inclusion Criteria:
For Phase 1:
* Participants with a locally advanced or metastatic solid tumor that:
* Has progressed on or is intolerant to standard therapy, or
* For which no standard therapy exists, or in the opinion of the Investigator, are not candidates for or would be unlikely to tolerate or derive significant clinical benefit from standard therapy, or
* Decline standard therapy
* Prior multikinase inhibitors (MKIs) with anti-RET activity are allowed
* A RET gene alteration is not required initially. Once adequate PK exposure is achieved, evidence of RET gene alteration in tumor and/or blood is required as identified through molecular assays, as performed for clinical evaluation
* Measurable or non-measurable disease as determined by RECIST 1.1 or RANO as appropriate to tumor type
* Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2 or Lansky Performance Score (LPS) greater than or equal to (≥) 40 percent (%) (age less than \[\<\] 16 years) with no sudden deterioration 2 weeks prior to the first dose of study treatment
* Adequate hematologic, hepatic and renal function
* Life expectancy of at least 3 months
For Phase 2: As for phase 1 with the following modifications:
* For Cohort 1: Participants must have received prior standard therapy appropriate for their tumor type and stage of disease, or in the opinion of the Investigator, would be unlikely to tolerate or derive clinical benefit from appropriate standard of care therapy
* Cohorts 1 and 2:
* Enrollment will be restricted to participants with evidence of a RET gene alteration in tumor
* At least one measurable lesion as defined by RECIST 1.1 or RANO, as appropriate to tumor type and not previously irradiated
* Cohorts 3 and 4: Enrollment closed
* Cohort 5:
* Cohorts 1-4 without measurable disease
* MCT not meeting the requirements for Cohorts 3 or 4
* MTC syndrome spectrum cancers (e.g., MTC, pheochromocytoma), cancers with neuroendocrine features/differentiation, or poorly differentiated thyroid cancers with other RET alteration/activation may be allowed with prior Sponsor approval
* cfDNA positive for a RET gene alteration not known to be present in a tumor sample
* Cohort 6: Participants who otherwise are eligible for Cohorts 1, 2 or 5 who discontinued another RET inhibitor may be eligible with prior Sponsor approval
* Cohort 7: Participants with a histologically confirmed stage IB-IIIA NSCLC and a RET fusion; determined to be medically operable and tumor deemed resectable by a thoracic surgical oncologist, without prior systemic treatment for NSCLC
Key Exclusion Criteria (Phase 1 and Phase 2):
* Phase 2 Cohorts 1 and 2: an additional known oncogenic driver
* Cohorts 3 and 4: Enrollment closed
* Cohorts 1, 2 and 5: prior treatment with a selective RET inhibitor Notes: Participants otherwise eligible for Cohorts 1, 2, and 5 who discontinued another selective RET inhibitor may be eligible for Phase 2 Cohort 6 with prior Sponsor approval
* Investigational agent or anticancer therapy (including chemotherapy, biologic therapy, immunotherapy, anticancer Chinese medicine or other anticancer herbal remedy) within 5 half-lives or 2 weeks (whichever is shorter) prior to planned start of LOXO-292 (selpercatinib). In addition, no concurrent investigational anti-cancer therapy is permitted Note: Potential exception for this exclusion criterion will require a valid scientific justification and approval from the Sponsor
* Major surgery (excluding placement of vascular access) within 2 weeks prior to planned start of LOXO-292 (selpercatinib)
* Radiotherapy with a limited field of radiation for palliation within 1 week of planned start of LOXO-292 (selpercatinib), with the exception of participants receiving radiation to more than 30% of the bone marrow or with a wide field of radiation, which must be completed at least 4 weeks prior to the first dose of study treatment
* Any unresolved toxicities from prior therapy greater than Common Terminology Criteria for Adverse Events (CTCAE) Grade 1 at the time of starting study treatment with the exception of alopecia and Grade 2, prior platinum-therapy related neuropathy
* Symptomatic primary CNS tumor, metastases, leptomeningeal carcinomatosis, or untreated spinal cord compression. Participants are eligible if neurological symptoms and CNS imaging are stable and steroid dose is stable for 14 days prior to the first dose of LOXO-292 (selpercatinib) and no CNS surgery or radiation has been performed for 28 days, 14 days if stereotactic radiosurgery (SRS)
* Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months prior to planned start of LOXO-292 (selpercatinib) or prolongation of the QT interval corrected (QTcF) greater than (\>) 470 milliseconds (msec)
* Participants with implanted pacemakers may enter the study without meeting QTc criteria due to nonevaluable measurement if it is possible to monitor for QT changes.
* Participants with bundle branch block may be considered for study entry if QTc is appropriate by a formula other than Fridericia's and if it is possible to monitor for QT changes.
* Required treatment with certain strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers and certain prohibited concomitant medications
* Phase 2 Cohort 7 (neoadjuvant treatment): Participant must not have received prior systemic therapy for NSCLC.
Primary outcome measure(s)
- Phase 1: Maximum Tolerated Dose (MTD) — Cycle 1 (cycle length = 28 days)
The MTD is defined as the highest dose level at which none of the first 3 treated patients, or not more than 1 of the first 6 treated patients, experiences a DLT. A DLT is any adverse events that starts on or after first administration of study drug, as defined by National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
* Any Grade(G) ≥3 nonhematologic toxicity, excluding
* G3 AST, ALT, and/or total bilirubin elevation for \<7 days.
* G3 neutropenia \<7 days
* G3 thrombocytopenia without clinically significant bleeding
* G3 or G4 lymphopenia.
* First occurrence of G3 or G4 electrolyte abnormalities
* G3 fatigue, weakness, nausea; other manageable constitutional symptom
* G3 or G4 vomiting or diarrhea that lasts for \<48hours with antiemetic/antidiarrheal medication in case of G3 and \<24 hours in case of G4
* G4 manageable constitutional symptom.
- Phase 1: Recommended Phase 2 Dose (RP2D) — Cycle 1 (cycle length = 28 days)
Phase 1: RP2D
- Phase 2: Objective Response Rate (ORR) Based on Independent Review Committee (IRC) Assessment — Approximately for up to 7 years 8 months
Objective Response Rate was defined as the percentage of participants with best overall response of confirmed response (CR), or Partial response (PR). Response was confirmed by a repeat assessment no less than 28 days.
* CR is defined as disappearance of all target lesions.
* PR is defined as at least a 30% decrease in the sum of diameter (LD for non-nodal lesions and short axis diameter \[SAD\] for nodal lesions) of target lesions, taking as reference the baseline sum LD.
ORR was assessed by independent review committee (IRC) using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. 95% confidence interval was calculated using Clopper-Pearson method.
Trial sites (85)
| Facility | City | Region | Status |
| Mayo Clinic of Scottsdale |
Scottsdale |
Arizona |
|
| City of Hope National Medical Center |
Duarte |
California |
|
| UCLA Medical Center |
Los Angeles |
California |
|
| Hoag Memorial Hospital Presbyterian |
Newport Beach |
California |
|
| Kaiser Permanente |
Oakland |
California |
|
| Irvine Medical Center |
Orange |
California |
|
| University of California - San Diego |
San Diego |
California |
|
| UCSF Medical Center at Mission Bay |
San Francisco |
California |
|
| Kaiser Permanente Medical Center |
Walnut Creek |
California |
|
| Sarah Cannon Research Institute at HealthOne |
Denver |
Colorado |
|
| Yale Cancer Center |
New Haven |
Connecticut |
|
| Mayo Clinic in Florida |
Jacksonville |
Florida |
|
| Memorial Hospital Pembroke |
Pembroke |
Florida |
|
| Emory University |
Atlanta |
Georgia |
|
| University of Chicago Medicine-Comprehensive Cancer Center |
Chicago |
Illinois |
|
| Ochsner Clinic Foundation |
New Orleans |
Louisiana |
|
| University of Maryland Medical Center |
Baltimore |
Maryland |
|
| Johns Hopkins University |
Baltimore |
Maryland |
|
| Massachusetts General Hospital |
Boston |
Massachusetts |
|
| Dana-Farber Cancer Institute |
Boston |
Massachusetts |
|
| University of Michigan |
Ann Arbor |
Michigan |
|
| START Midwest |
Grand Rapids |
Michigan |
|
| Mayo Clinic |
Rochester |
Minnesota |
|
| Washington University Medical School |
St Louis |
Missouri |
|
| Comprehensive Cancer Centers of Nevada |
Las Vegas |
Nevada |
|
| Roswell Park Cancer Institute |
Buffalo |
New York |
|
| NYU Langone |
New York |
New York |
|
| Memorial Sloan Kettering Cancer Center |
New York |
New York |
|
| University of North Carolina |
Chapel Hill |
North Carolina |
|
| Cleveland Clinic Foundation |
Cleveland |
Ohio |
|
| Ohio State University Hospital |
Columbus |
Ohio |
|
| Oregon Health and Science University |
Portland |
Oregon |
|
| University of Pennsylvania Hospital |
Philadelphia |
Pennsylvania |
|
| Thomas Jefferson University |
Philadelphia |
Pennsylvania |
|
| Sarah Cannon Research Institute SCRI |
Nashville |
Tennessee |
|
| Vanderbilt University Medical Center |
Nashville |
Tennessee |
|
| University of Texas Southwestern Medical Center at Dallas |
Dallas |
Texas |
|
| University of Texas MD Anderson Cancer Center |
Houston |
Texas |
|
| Huntsman Cancer Institute |
Salt Lake City |
Utah |
|
| USO-Virginia Cancer Specialists, PC |
Fairfax |
Virginia |
|
+ 45 more sites — see the full list on the official registry below.
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