Study on the Effects of Selpercatinib in Children with Advanced RET-Altered Solid Tumors or Primary Central Nervous System Tumors

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What is this study about?

This clinical trial is focused on studying the effects of a medication called Selpercatinib in children and young adults with advanced tumors. These tumors are either solid or located in the central nervous system, such as the brain or spinal cord, and have a specific change in their genes known as RET-altered. The purpose of the study is to understand how safe the medication is and how well it works in treating these types of tumors.

The study is divided into two phases. In the first phase, the main goal is to determine the safety of Selpercatinib and identify any side effects that might occur. In the second phase, the focus is on evaluating how well the tumors respond to the treatment. This involves measuring the size of the tumors to see if they shrink or disappear after taking the medication. The study will involve taking Selpercatinib in different forms, such as tablets or oral solutions, and it will be administered by mouth.

Participants in the study will be monitored closely to track their response to the treatment and any side effects they may experience. The study aims to provide valuable information on the potential benefits and risks of using Selpercatinib for treating these specific types of tumors in young patients. The trial is expected to continue until late 2024, with the hope of finding an effective treatment option for those affected by these challenging conditions.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history, current health status, and any previous treatments.

Eligibility criteria include being between 12 and 21 years old, having a specific type of tumor, and meeting certain health requirements.

2 treatment initiation

The treatment involves taking the study drug, selpercatinib, which is an oral medication. It is available in tablet or oral solution form.

The dosage and frequency of administration are determined by the study protocol and the patient’s specific condition.

3 monitoring and evaluation

Regular monitoring is conducted to assess the safety and effectiveness of the treatment. This includes physical exams, blood tests, and imaging studies.

The primary goal is to evaluate the safety profile of selpercatinib and to measure the response to treatment using specific criteria.

4 response assessment

The response to the treatment is assessed using established criteria to determine if there is a complete or partial response to the medication.

This assessment is conducted by an independent review committee to ensure objectivity.

5 continuation or adjustment of treatment

Based on the response assessment, the treatment plan may be continued or adjusted. This decision is made by the study team in consultation with the patient.

The study aims to continue until the estimated end date in October 2024, unless specific circumstances require earlier termination.

Who Can Join the Study?

Pediatric patients aged between 12 and 21 years with a solid or primary central nervous system (CNS) tumor that has come back, gotten worse, or did not respond to available treatments. This includes patients who would need major surgery to remove the tumor.
Patients must have a certain level of blood health, including enough white blood cells, platelets, and hemoglobin, without needing recent medical support like growth factors or transfusions.
Patients must have a certain level of liver and pancreas health, with specific limits on liver enzymes and bilirubin levels. Patients with certain liver conditions may have different requirements.
Patients must have a certain level of kidney health, measured by how well the kidneys filter blood.
Patients must be able to follow the study’s treatment plan, including attending clinic visits and undergoing lab tests.
Patients who can have children must agree to use two effective methods of birth control.
Patients must be able to swallow capsules or take medicine through a tube if needed.
Patients and their parents or guardians must understand and agree to the study’s terms by signing a consent form.
There must be evidence of a specific change in the RET gene in the tumor or blood, identified through tests.
Patients with primary CNS tumors or brain metastasis must have stable neurological symptoms and not need increasing doses of steroids to manage symptoms before joining the study.
An imaging study must be done within 28 days before starting treatment, with a stable dose of steroids if needed.
The original diagnosis or relapse must be confirmed through a tissue sample, except for certain brain tumors.
Patients must have a disease that can be measured or evaluated.
Patients aged 16 and older must have a Karnofsky performance score of at least 50, and those younger than 16 must have a Lansky score of at least 50. These scores measure the patient’s ability to perform daily activities.
Patients must have recovered from the side effects of previous cancer treatments to a certain level.
A sample of the tumor tissue must be available for the study.

Who Cannot Join the Study?

Patients who have not been diagnosed with advanced RET-altered solid tumors or primary central nervous system tumors. These are specific types of cancer that affect certain parts of the body or brain.
Patients who are not within the specified age range for the study. The study is designed for certain age groups, and those outside this range cannot participate.
Patients who are unable to take the study medication, Selpercatinib, due to allergies or other medical reasons. This medication is a treatment being tested in the study.
Patients who have other medical conditions that might interfere with the study or make participation unsafe. This includes conditions that could worsen with the study treatment.
Patients who are currently participating in another clinical trial. Being in more than one study at a time can affect the results and safety.
Patients who are pregnant or breastfeeding. The effects of the study treatment on unborn babies or nursing infants are not known.
Patients who have not recovered from previous treatments or surgeries. The body needs to be in a stable condition to participate safely in the study.
Patients who have a history of certain heart conditions. These conditions could be worsened by the study treatment.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Institut Gustave Roussy	Villejuif	France
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country
Rigshospitalet	Copenhagen	Denmark
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Hdyeweyy Vptm dfojjetq	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	28.02.2019
France	Not recruiting	28.02.2019
Germany	Not recruiting	28.02.2019
Italy	Not recruiting	28.02.2019
Spain	Not recruiting	28.02.2019

Trial locations

Investigated drugs:

Selpercatinib

Selpercatinib is an oral medication used in this clinical trial. It is a RET inhibitor, which means it targets and blocks a specific protein called RET that can contribute to the growth of certain types of tumors. This medication is being studied to see if it is safe and effective for treating pediatric patients with advanced solid tumors or primary central nervous system tumors that have alterations in the RET gene. The trial aims to understand how well the medication works in shrinking or controlling these tumors.

Investigated diseases:

RET-Altered Solid Tumors – These are a group of cancers characterized by changes in the RET gene, which can lead to uncontrolled cell growth. These tumors can occur in various parts of the body, including the lungs and thyroid. As the disease progresses, the tumors may grow larger and potentially spread to other areas. Symptoms can vary depending on the tumor’s location and size, often causing pain or discomfort. The progression of these tumors can be unpredictable, with some growing slowly while others may advance more rapidly.

Primary Central Nervous System Tumors – These tumors originate in the brain or spinal cord and can affect both children and adults. They can be benign or malignant, with varying growth rates and potential to spread within the central nervous system. Symptoms often depend on the tumor’s location and size, potentially causing headaches, seizures, or neurological deficits. As they progress, these tumors can interfere with normal brain or spinal cord function. The impact on the patient can vary widely, from minimal symptoms to significant neurological impairment.

Trial ID:

2023-507703-63-00

Protocol code:

LOXO-RET-18036

NCT ID:

NCT03899792

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on the Effects of Selpercatinib in Children with Advanced RET-Altered Solid Tumors or Primary Central Nervous System Tumors

What is this study about?

Who Can Join the Study?

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