A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001)

Active:	Yes
Cancer Type:	Brain & Spinal Cord Tumor Colon/Rectal Cancer Head and Neck Cancer Lung Cancer Neuroendocrine Tumor Solid Tumor Thyroid Cancer Unknown Primary	NCT ID:	NCT03157128
Trial Phases:	Phase I Phase II	Protocol IDs:	LOXO-RET-17001 (primary) NCI-2017-00976 2017-000800-59
Eligibility:	12 Years and older, Male and Female	Study Type:	Treatment
Study Sponsor:	Loxo Oncology, Inc.
NCI Full Details:	http://clinicaltrials.gov/show/NCT03157128

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.

Objectives

This is an open-label, multi-center Phase 1/2 study in participants with advanced solid
tumors, including RET fusion-positive solid tumors, MTC, and other tumors with RET
activation. The trial will be conducted in 2 parts: Phase 1 (dose escalation - completed)
and phase 2 (dose expansion). Participants with advanced cancer are eligible if they have
progressed on or are intolerant to available standard therapies, or no standard or
available curative therapy exists, or in the opinion of the Investigator, they would be
unlikely to tolerate or derive significant clinical benefit from appropriate standard of
care therapy, or they declined standard therapy. A dose of 160 milligrams (mg) twice a
day (BID) has been selected as the recommended phase 2 dose (RP2D). Approximately 875
participants with advanced solid tumors harboring a RET gene alteration in tumor and/or
blood will be enrolled to one of seven phase 2 cohorts:

- Cohort 1: Advanced RET fusion positive solid tumor other than NSCLC or thyroid
cancer for participants who progressed on or intolerant to first line therapy (open)

- Cohort 2: Advanced RET fusion positive solid tumor other than NSCLC or thyroid
cancer for treatment naïve participants (open)

- Cohort 3: Advanced RET-mutant MTC participants who progressed on or intolerant to
first line therapy (closed)

- Cohort 4: Advanced RET-mutant MTC participants who are treatment naïve (closed)

- Cohort 5: Advanced RET-altered solid tumor for participants other than NSCLC or
thyroid cancer and RET-mutant MEN2 spectrum tumors (e.g. pheochromocytoma) otherwise
ineligible for cohorts 1-4. See details in inclusion/exclusion criteria (open)

- Cohort 6: Participants otherwise eligible for Cohorts 1-5 who discontinued another
RET inhibitor due to intolerance may be eligible with prior Sponsor approval
(closed)

- Cohort 7: RET fusion positive early-stage non-small cell lung cancer (NSCLC)
participants who are candidates for definitive surgery. Participants will receive
selpercatinib in a neoadjuvant and adjuvant setting. Participants will be followed
for disease recurrence for up to 5 years from the date of surgery (closed)

Treatment Sites in Georgia

Emory University School of Medicine
1440 Clifton Road
Atlanta, GA 30322

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