Study on Repotrectinib for Children and Young Adults with Advanced Solid Tumors with ALK, ROS1, or NTRK1-3 Alterations

1 1

What is this study about?

This clinical trial is focused on studying the effects of a medication called Repotrectinib (TPX-0005) in children and young adults who have advanced or metastatic cancers. These cancers are characterized by specific genetic changes known as ALK, ROS1, or NTRK1-3 alterations. The purpose of the study is to evaluate how well Repotrectinib works in treating these types of cancers.

The study involves taking Repotrectinib either as a hard capsule or an oral solution. Participants will be monitored to assess the safety and tolerability of the medication, which means checking for any side effects and how well the body handles the drug. The study will also look at the medication’s ability to reduce or control the growth of tumors. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects.

Throughout the study, participants will have regular check-ups and tests to monitor their health and the progress of their cancer. The study aims to gather information over several years to understand the long-term effects and benefits of Repotrectinib for treating these specific genetic types of cancer in young patients. This research is important for developing new treatments that can improve outcomes for those affected by these challenging conditions.

1 Enrollment

Upon joining the study, the participant will be enrolled in either Phase 1 or Phase 2, depending on age and previous treatment history.

Participants must have a confirmed diagnosis of advanced solid tumors with specific genetic alterations.

2 Initial Assessment

Participants will undergo a series of assessments to confirm eligibility, including laboratory tests and imaging studies to measure the disease.

These assessments ensure that participants meet the study’s inclusion criteria.

3 Treatment Administration

Participants will receive repotrectinib, which is administered orally.

The medication is available in two forms: a hard capsule and an oral solution.

The dosage and frequency will be determined based on the participant’s specific cohort and phase of the study.

4 Regular Monitoring

Participants will attend regular outpatient visits for monitoring.

These visits will include laboratory tests and imaging to assess the response to treatment and monitor for any side effects.

5 Evaluation of Treatment Response

The effectiveness of the treatment will be evaluated using criteria such as tumor size reduction and overall response rate.

Participants will be assessed for any changes in their condition, including potential improvements or progression of the disease.

6 Completion of Study Participation

Participants will complete the study after a predetermined period or if they meet specific criteria for discontinuation.

Final assessments will be conducted to gather data on the long-term effects and outcomes of the treatment.

Who Can Join the Study?

Participants must have advanced solid tumors. These are cancers that have spread and are not limited to one area.
Participants must have a specific genetic change called NTRK+ or ROS1 gene fusions. This means their cancer has a particular alteration in its DNA.
Participants must have tried at least one but no more than two previous treatments called TRK TKIs and either did not respond, their disease got worse, or they could not tolerate the treatment.
Participants must have a measurable disease. This means the cancer can be measured by doctors using specific criteria.
Participants must have certain blood test results within normal limits, including levels of white blood cells, platelets, hemoglobin, and kidney function.
Participants must be able to attend outpatient visits and undergo laboratory testing and study procedures.
Participants must have recovered from any side effects of previous treatments to a mild level, except for hair loss.
Participants must not have received certain treatments like chemotherapy, radiation, or specific antibodies within a specified time before starting the study drug.
Participants with primary CNS tumors (tumors in the brain or spinal cord) or metastases must be stable and on a stable or decreasing dose of steroids for at least 7 days before joining the study.
Participants must have a performance status score of at least 50, which means they can perform daily activities with some help.
Participants must have a life expectancy of at least 12 weeks.
Participants must be between the ages of 12 and 25 years for Phase 2 of the study.
Participants, or their parents or guardians, must sign a consent form agreeing to participate in the study.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Institut Gustave Roussy	Villejuif	France
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Hospital Universitario Hm Sanchinarro	Madrid	Spain
Hospital Universitario De Navarra	Pamplona	Spain

Other Sites

Site Name	City	Country
Hospital Infantil Universitario Nino Jesus	Madrid	Spain
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Pellegrin Hospital	Bordeaux	France
Virgen del Rocío University Hospital	Sevilla	Spain
Rigshospitalet	Copenhagen	Denmark
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Csetdr Lxwv Bfjrul	Lyon	France
Ajkexgrfxe Paecvnyh Hslfvjwj Do Mwxelsjpq	Marseille	France
Ccpctw Hsrhdplvvjf Rvtrhrcf Dyfbozuusdzfsa	Angers	France
Hlmislgq Vlvu dzutlnjl	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Denmark	Recruiting	01.02.2023
France	Recruiting	01.02.2023
Italy	Recruiting	01.02.2023
Spain	Recruiting	01.02.2023

Trial locations

Investigated drugs:

Repotrectinib

Repotrectinib is a medication being studied for its ability to treat certain types of cancer in children and young adults. It is designed to target and block specific proteins that are involved in the growth and spread of cancer cells. This medication is being tested to see if it can help shrink tumors or slow their growth in patients whose cancers have specific genetic changes known as ALK, ROS1, or NTRK1-3 alterations.

Advanced Solid Tumors – Advanced solid tumors refer to a group of cancers that originate in solid organs or tissues and have progressed to a more severe stage. These tumors can occur in various parts of the body, such as the lungs, liver, pancreas, or colon. As they advance, they may grow larger, invade nearby tissues, and potentially spread to other parts of the body through the bloodstream or lymphatic system. The progression of these tumors can lead to symptoms such as pain, swelling, or dysfunction of the affected organ. The specific symptoms and progression depend on the tumor’s location and the organs involved.

Trial ID:

2023-506464-14-00

Protocol code:

TPX-0005-07

NCT ID:

NCT04094610

Trial Phase:

Human Pharmacology (Phase I) – Other

Other Trials to Consider

#1 Clinical Trials Search in Europe

Study on Repotrectinib for Children and Young Adults with Advanced Solid Tumors with ALK, ROS1, or NTRK1-3 Alterations

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?