Not recruitingRare disease

Study of Avapritinib for Children with Solid Tumors Linked to KIT or PDGFRA Signaling

Verified siteInvestigational

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called Avapritinib in children with certain types of solid tumors. These tumors are dependent on specific signals known as KIT or PDGFRA. Solid tumors are abnormal masses of tissue that can occur in various parts of the body. The study aims to understand how safe and effective Avapritinib is for treating these tumors in young patients.

The study is divided into two parts. In the first part, researchers will determine the appropriate dose of Avapritinib for children and assess its safety. In the second part, the focus will be on evaluating how well the medication works in shrinking or controlling the tumors. Participants will take Avapritinib orally, either as a film-coated tablet or a hard capsule. Throughout the study, doctors will monitor the children's health and any side effects they may experience.

The goal of this research is to find a potential new treatment option for children with these specific types of tumors, especially when other treatments have not been successful. By participating in this study, researchers hope to gather valuable information that could lead to better care and outcomes for young patients with these challenging conditions.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the patient will be informed about the trial's purpose and procedures. The patient must provide written consent to participate, which may also require parental or legal guardian consent, depending on local regulations.
Step 2
Initial assessment
The patient will undergo an initial assessment to confirm eligibility. This includes verifying age, diagnosis of a solid tumor dependent on KIT or PDGFRA signaling, and other health criteria. The patient must have at least one measurable lesion as defined by specific medical criteria.
Step 3
Treatment phase part 1
The patient will begin treatment with avapritinib, administered orally in the form of a film-coated tablet or hard capsule. The dosage will be determined based on safety and tolerability assessments. The primary goal is to establish the recommended dose for the next phase.
Step 4
Monitoring and evaluation part 1
Throughout Part 1, the patient will be closely monitored for any adverse effects and the effectiveness of the treatment. Regular assessments will be conducted to evaluate the patient's response to the medication.
Step 5
Treatment phase part 2
In Part 2, the patient will continue receiving avapritinib at the dose determined in Part 1. The focus will be on assessing the anti-tumor activity of the medication using specific evaluation criteria.
Step 6
Monitoring and evaluation part 2
The patient will undergo regular evaluations to monitor the incidence and severity of any adverse events. The effectiveness of the treatment will be assessed through objective response rates and other measures.
Step 7
Completion of the study
Upon completion of the study, the patient's overall response to the treatment will be evaluated. The study is estimated to conclude by September 30, 2025.

Who can join the trial?

7 criteria

The patient must be between 2 and less than 18 years old at the time of signing the consent form.
The patient must have a confirmed diagnosis of a solid tumor, including tumors in the brain, that has a specific genetic change in PDGFRA or KIT genes. This tumor must have worsened despite standard treatments, and no other treatment options are available. If the tumor only has an increase in PDGFRA or KIT genes, approval from the study sponsor is needed. Alternatively, the patient may have a specific type of brain tumor called DMG-H3K27a that has not responded to standard treatments or for which no beneficial standard treatment exists, as determined by the doctor.
If the patient has a disease affecting the brain or spinal cord, they should be on a stable or decreasing dose of corticosteroids (a type of medication) for at least 7 days before starting the study medication, with no plans to increase the dose.
The patient must have at least one tumor that can be measured. If they have received radiation therapy, at least one measurable tumor must not have been treated with radiation, or it must have clearly worsened since being treated with radiation, and it must be at least 12 weeks since the radiation was given to any target tumor.
The patient must have a performance score of at least 50 on the Lansky scale (for those under 16 years old) or the Karnofsky scale (for those 16 years and older). These scales measure the patient's ability to perform daily activities.
The patient must agree to use birth control methods that are consistent with local regulations. Male patients must either have had a vasectomy, agree to use condoms, practice true abstinence, or have a female partner who cannot become pregnant. Female patients must agree to use effective birth control, have a male partner who uses a condom, practice true abstinence, or have a male partner who has had a vasectomy with confirmed absence of sperm.
The patient or their legal guardian must be able to provide written consent before any study-specific screening procedures are done. The need for parental or legal guardian consent will be determined by local, regional, or national guidelines.

Who cannot join the trial?

10 criteria

Patients who have a type of cancer that is not dependent on KIT or PDGFRA signaling. These are specific proteins that can affect how cancer grows.
Patients who are not within the age range specified for the study. The study is for children, so adults cannot participate.
Patients who are not able to follow the study procedures or take the study medication as required.
Patients who have other medical conditions that might interfere with the study or make it unsafe for them to participate.
Patients who are pregnant or breastfeeding, as the study medication might affect the baby.
Patients who are currently participating in another clinical trial or have recently taken part in one.
Patients who have had a recent surgery or are planning to have surgery during the study period.
Patients who have allergies or reactions to the study medication or similar medications.
Patients who have certain abnormal lab test results that might indicate other health issues.
Patients who have a history of certain mental health conditions that might affect their ability to participate in the study.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Fondazione I.R.C.C.S. Istituto Neurologico Besta
Milan, Italy
Verified
Institut Gustave Roussy
Villejuif, France
Verified
Medical University Of Vienna
Vienna, Austria

Trial locations

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Investigated drugs

Avapritinib is a medication being studied for its potential to treat pediatric patients with solid tumors that rely on specific signaling pathways, namely KIT or PDGFRA. The trial aims to determine the appropriate dose for further studies and to evaluate how safe and tolerable the medication is for children. Additionally, the study seeks to understand how effective avapritinib is in reducing tumor size or slowing tumor growth in these patients.

Avapritinib

What is already known about the treatment

Avapritinib – Avapritinib is administered orally and is currently being studied in clinical trials for its safety and effectiveness in treating pediatric patients with solid tumors that rely on KIT or PDGFRA signaling. It is in the investigational stage, with ongoing research to determine its optimal dosing and therapeutic potential. The main therapeutic indication for avapritinib is its use in tumors that are dependent on specific signaling pathways. At the molecular level, avapritinib works by inhibiting the activity of certain proteins involved in tumor growth and survival. It is classified pharmacologically as a tyrosine kinase inhibitor, targeting specific enzymes that play a role in cancer cell proliferation.

Investigated diseases

Solid Tumors Dependent on KIT or PDGFRA Signaling – These are a group of tumors that rely on specific signaling pathways involving the KIT or PDGFRA genes for their growth and survival. These tumors can occur in various parts of the body and are characterized by the abnormal proliferation of cells. The progression of these tumors is often driven by mutations in the KIT or PDGFRA genes, which lead to continuous activation of signaling pathways that promote cell division and tumor growth. Over time, these tumors may increase in size and potentially spread to other areas of the body. The behavior and growth rate of these tumors can vary depending on the specific mutations and the location of the tumor. Understanding the genetic basis of these tumors is crucial for developing targeted therapies.