Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors

This research study is testing a new oral medicine called Selpercatinib (also known as LOXO-292) for children and young people aged 6 months to 21 years. It's for those who have advanced solid tumours or brain tumours that haven't responded to standard treatments. Specifically, the study is looking for participants whose tumours have a particular genetic change called a RET alteration. The study is split into two parts: first, finding the right dose of the medicine, and then, expanding to treat more people once that dose is known. Conditions being studied include medullary thyroid cancer, infantile myofibromatosis, other sarcomas, and papillary thyroid cancer, among others. The aim is to see how safe and effective this new treatment is for these specific cancers.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Eli Lilly and Company

Enrolment target

Start

13 Jun 2019

Estimated completion

01 May 2029

Medullary Thyroid Cancer Infantile Myofibromatosis Infantile Fibrosarcoma Papillary Thyroid Cancer Soft Tissue Sarcoma

What is this study about?

This study is investigating a new medicine called Selpercatinib, also known by its research name LOXO-292. This medicine comes in a pill form that you take by mouth. It's being tested in children and young people, from 6 months old up to 21 years old, who have certain types of advanced cancers. These cancers include specific solid tumours or brain tumours that have not gotten better with usual treatments.

The key to joining this study is that the cancer must have a particular genetic change called a 'RET alteration'. Think of this as a specific switch in the cancer cells that the new medicine is designed to target. The study aims to see if Selpercatinib can help treat these cancers by focusing on this specific change. It's important to remember that this is a research study, and we're still learning about this medicine.

The study has two main parts. The first part is about carefully figuring out the best and safest dose of Selpercatinib for children and young people. Once that dose is determined, the second part of the study will involve more participants to further check how well the medicine works and if it's safe for different types of tumours with this RET alteration.

Key takeaways

This study is for children and young people (ages 6 months to 21 years) with specific advanced cancers.
The treatment is a new oral medicine called Selpercatinib, targeting a 'RET alteration' in cancer cells.
It's a two-part study: first finding the right dose, then expanding to more participants.
Participation involves taking the medicine and having regular check-ups with blood tests and scans.
The study aims to assess the safety and effectiveness of Selpercatinib for these specific cancers.

Who may be eligible?

To be considered for this study, participants generally need to have an advanced solid tumour or a brain tumour that hasn't responded to standard treatments. A key requirement is that the tumour must show a specific genetic change called a 'RET alteration', which can be found in a sample from the tumour or a blood test.

Participants should also be well enough to take part, meaning their overall health, including their heart, liver, kidney, and blood functions, needs to be adequate. For brain tumours, participants must have been stable without needing more steroids for at least 7 days before starting the study medicine. They should also be able to take medicine by mouth or through a feeding tube.

There are also reasons why someone might not be able to join. These include having had major surgery recently (within two weeks), serious heart problems, uncontrolled infections, or certain thyroid or calcium imbalances. Also, those who are pregnant or breastfeeding, or who have previously taken a similar RET-targeting medicine, cannot participate.

Quick self-check

Do I have an advanced solid tumour or a brain tumour that hasn't responded to standard treatment?
Has my tumour been tested and found to have a 'RET alteration'?
Am I between 6 months and 21 years old?
Am I generally well enough to take part, with good heart, liver, and kidney function?
Have I been stable (for brain tumours) without needing more steroids for at least 7 days?
Have I had any major surgery recently (within the last two weeks)?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you would be taking the study medicine, Selpercatinib, which is taken by mouth, either as pills or a liquid suspension. In the first part of the study, the dose of the medicine will be carefully increased for small groups of participants until the safest and most effective dose is found. After that, more participants will join the second part of the study, taking the dose that was determined.

Throughout the study, you'll have regular hospital visits for check-ups. These visits will involve various tests, such as blood tests, scans, and other assessments, to monitor your health, how your body is handling the medicine, and whether the medicine is affecting your tumour. Each 'cycle' of treatment is 28 days long, and these cycles will continue as long as the medicine is helping and you are tolerating it well. The total duration of participation will vary for each individual, depending on how they respond to the treatment.

Potential risks and benefits

Participating in a clinical trial offers potential benefits, such as access to a new medicine not yet widely available that might help treat your specific cancer. It also contributes to medical knowledge, which could help future patients. However, like all medicines, Selpercatinib may have side effects, which can range from mild to serious. These will be carefully monitored throughout the study. You will be closely watched by the study team, and they will explain all known potential risks in detail. Remember, your participation is voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (26)

Childrens Hospital of Los Angeles
Los Angeles, United States
The Children's Hospital for Cancer and Blood Disorders
Aurora, United States
Nemours Children's Health
Orlando, United States
Dana-Farber Cancer Institute
Boston, United States
University of Minnesota Hospital
Minneapolis, United States
Memorial Sloan Kettering Cancer Center
New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, United States
Children's Hospital of Philadelphia
Philadelphia, United States
St. Jude Children's Research Hospital
Memphis, United States
University of Texas Southwestern Medical Center at Dallas
Dallas, United States
Texas Childrens Hospital
Houston, United States
Seattle Children's Hospital Research Foundation
Seattle, United States

+14 more sites — see the official record for the full list.

Common questions

What is a 'RET alteration'?

A RET alteration is a specific change in the genes within the cancer cells. This medicine is designed to target only cancers that have this particular genetic change.

What does 'advanced solid or primary CNS tumor' mean?

This means a cancer that has either spread from where it started (advanced) or a tumour that began in the brain or spinal cord (primary Central Nervous System, or CNS) and has not responded to other treatments.

Is Selpercatinib a new type of treatment?

Yes, Selpercatinib is a new medicine being studied. It works differently from traditional chemotherapy by specifically targeting the RET alteration in cancer cells.

How long would I need to be in the study?

The length of time you participate can vary. You would continue as long as the medicine is helping you and you are not experiencing severe side effects. Each treatment cycle is 28 days.

Will I know if I'm getting the active medicine or a placebo?

This study is 'open-label', meaning both you and your study doctor will know that you are receiving the real study medicine, Selpercatinib. There is no placebo used in this trial.