Selpercatinib for Cancer in Children
(LIBRETTO-121 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET) alteration and an advanced solid or primary CNS tumor.
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you have uncontrolled thyroid issues that required a change in medication in the last 7 days, you may not be eligible.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, if you have uncontrolled thyroid issues that required a change in medication recently, you may not be eligible.
What safety data is available for Selpercatinib in treating cancer?
Selpercatinib, also known as Retevmo, has been evaluated in several clinical trials, including the phase I/II LIBRETTO-001 trial. It has shown a manageable safety profile with common adverse events such as hypertension, elevated liver enzymes, edema, diarrhea, fatigue, and dry mouth. Most adverse events were manageable with dose adjustments, and only a small proportion of patients discontinued due to treatment-related adverse events. The drug has been approved for various RET fusion-positive cancers, indicating its safety and efficacy in these conditions.12345
What safety data exists for Selpercatinib in humans?
Selpercatinib has been shown to have a manageable safety profile in clinical trials for various cancers, with common side effects including high blood pressure, liver enzyme changes, swelling, diarrhea, fatigue, dry mouth, abdominal pain, constipation, rash, nausea, and headache. Most side effects were manageable with dose adjustments, and only a small number of patients stopped treatment due to side effects.12345
Is the drug Selpercatinib a promising treatment for cancer in children?
Selpercatinib is a promising drug because it has been approved for treating certain types of cancer in adults, showing strong and lasting effects. It targets specific cancer changes, helping to stop cancer growth. This success in adults suggests it could be effective for children with similar cancer types.12678
How is the drug Selpercatinib unique for treating cancer in children?
Selpercatinib is unique because it is the first selective inhibitor specifically targeting RET (rearranged during transfection) alterations, which are genetic changes found in some cancers. It is taken orally and has shown effectiveness in treating cancers with these RET alterations, offering a targeted approach compared to traditional chemotherapy.12678
What data supports the idea that Selpercatinib for Cancer in Children is an effective drug?
The available research shows that Selpercatinib has been effective in treating certain types of cancer, specifically RET fusion-positive non-small-cell lung cancer and thyroid cancer. In clinical trials, patients treated with Selpercatinib experienced strong and lasting responses, even those who had previously undergone other treatments. The drug was generally well-tolerated, with manageable side effects. Although the studies focus on adults, these results suggest that Selpercatinib could be a promising option for treating similar cancers in children.12579
What data supports the effectiveness of the drug Selpercatinib for cancer in children?
Who Is on the Research Team?
Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST)
Principal Investigator
Eli Lilly and Company
Are You a Good Fit for This Trial?
This trial is for children with advanced solid tumors or brain tumors that have a specific genetic change called RET alteration. They must be stable, able to take oral medication, and not pregnant. Participants need functioning major organs and haven't had recent surgery or uncontrolled diseases.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Phase 1: Dose Escalation
Participants are enrolled using a rolling 6 dose escalation scheme to determine the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of LOXO-292
Phase 2: Dose Expansion
Participants are enrolled into one of four dose expansion cohorts based on tumor histology and genotype, receiving the MTD/recommended dose from Phase 1
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- LOXO-292
LOXO-292 is already approved in United States for the following indications:
- Non-small cell lung cancer (NSCLC) with RET fusion
- Medullary thyroid cancer (MTC) with RET mutation
- Thyroid cancer with RET fusion
Find a Clinic Near You
Who Is Running the Clinical Trial?
Eli Lilly and Company
Lead Sponsor
Dr. Daniel Skovronsky
Eli Lilly and Company
Chief Medical Officer since 2018
MD from Harvard Medical School
David A. Ricks
Eli Lilly and Company
Chief Executive Officer since 2017
BSc from Purdue University, MBA from Indiana University
Loxo Oncology, Inc.
Industry Sponsor
Jacob Van Naarden
Loxo Oncology, Inc.
Chief Executive Officer since 2019
A.B. in Molecular Biology from Princeton University
Dr. Jennifer Low
Loxo Oncology, Inc.
Chief Medical Officer since 2014
MD and PhD from Georgetown University; Undergraduate degree from California Institute of Technology