Locally Advanced Solid Tumors study: Oral repotrectinib
Official title A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 Alterations
ClinicalTrials.gov ID: NCT04094610
What this study is testing
What is Oral repotrectinib (TPX-0005)?
An investigational oral medication being studied for advanced or metastatic solid tumors in children and young adults with ALK, ROS1, or NTRK gene alterations. It is designed to inhibit abnormal kinase activity associated with these gene alterations.
Also referred to as repotrectinib.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Phase 1 will look at how safe and tolerable repotrectinib is at different doses and find the best dose for kids. Phase 2 will see if the drug can shrink tumors in these patients.
- Who it's for
- Children and young adults with advanced or metastatic solid tumors, lymphoma, or primary brain tumors that have ALK, ROS1, or NTRK1‑3 gene changes.
- What's involved
- Participants will receive repotrectinib at varying doses, attend regular clinic visits, and have blood and imaging tests to monitor safety and tumor response.
- Time commitment
- The study lasts several months, with multiple clinic visits and tests scheduled throughout the treatment period.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 25
You may be able to join if
- Documented genetic ROS1 point mutation, fusion, or amplification or NTRK1-3 fusion as identified by local testing in a Clinical Laboratory...
- Phase 1: Age \<12 years; Phase 2: Age 12- 25 years
- Prior cytotoxic chemotherapy is allowed.
- Prior immunotherapy is allowed.
- Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer therapy to National Cancer Institute Common Terminology Criteria...
You likely can't join if
- people with neuroblastoma with only bone marrow disease evaluable by bone marrow aspiration only.
- Major surgery within 14 days (2 weeks) of start of repotrectinib treatment.
- Known active infections requiring ongoing treatment (bacterial, fungal, viral including HIV positivity).
- Gastrointestinal disease (e.g., Crohn's disease, ulcerative colitis, or short gut syndrome) or other malabsorption syndromes that would impact drug...
- Mean resting corrected QT interval (ECG interval measured from the onset of the QRS complex to the end of the T wave) for heart rate (QTc) \> 480...
- Any clinically important abnormalities in rhythm, conduction, or morphology of resting ECG (e.g., complete left bundle branch block, third degree...
See the full eligibility criteria
- Documented genetic ROS1 point mutation, fusion, or amplification or NTRK1-3 fusion as identified by local testing in a Clinical Laboratory Improvement Amendments (CLIA) laboratory in the US or equivalently accredited...
- Phase 1: Age \<12 years; Phase 2: Age 12- 25 years
- Prior cytotoxic chemotherapy is allowed.
- Prior immunotherapy is allowed.
- Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer therapy to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 4.03 Grade less than or equal...
- All people must have measurable disease by RECIST v1.1 or Response Assessment in Neuro-Oncology (RANO) criteria at time of enrollment.
- people with a primary CNS tumor or CNS metastases must be neurologically stable on a stable or decreasing dose of steroids for at least 7 days prior to enrollment.
- people must have a Lansky (\< 16 years) or Karnofsky (≥ 16 years) score of at least 50.
- Life expectancy greater than or equal to 12 weeks, in the investigator's opinion.
- Adequate hematologic, renal and hepatic function. Phase 2
- Cohort Specific
- Cohort 1: people with NTRK fusion gene positive (NTRK+) advanced solid tumors (including primary CNS tumors), that are tropomyosin receptor kinase (TRK) TKI naïve;
- Cohort 2: people with NTRK+ advanced solid tumors (including primary CNS tumors), that are TRK TKI pre-treated;
- Cohort 3: people with advanced solid tumors with ROS1 gene fusions or other ROS1 aberrations (including amplifications and point mutations) with measurable disease.
- people in Cohorts 1 and 2 must have prospectively confirmed measurable disease by BICR prior to enrollment. Key
- people with neuroblastoma with only bone marrow disease evaluable by bone marrow aspiration only.
- Major surgery within 14 days (2 weeks) of start of repotrectinib treatment. Central venous access (Broviac, Mediport, etc.) placement does not meet criteria for major surgery.
- Known active infections requiring ongoing treatment (bacterial, fungal, viral including HIV positivity).
- Gastrointestinal disease (e.g., Crohn's disease, ulcerative colitis, or short gut syndrome) or other malabsorption syndromes that would impact drug absorption.
- Mean resting corrected QT interval (ECG interval measured from the onset of the QRS complex to the end of the T wave) for heart rate (QTc) \> 480 msec obtained from three ECGs, using the screening clinic ECG...
- Any clinically important abnormalities in rhythm, conduction, or morphology of resting ECG (e.g., complete left bundle branch block, third degree heart block, second degree heart block, PR interval \> 250 msec)
- Any factors that increase the risk of QTc prolongation or risk of arrhythmic events such as heart failure, congenital long QT syndrome, family history of long QT syndrome, or any concomitant medication known to prolong...
- Peripheral neuropathy of CTCAE ≥grade 2.
- people being treated with or anticipating the need for treatment with strong CYP3A4 inhibitors or inducers.
- Any potential allergies to repotrectinib and/or its excipients.
The study team makes the final eligibility decision.
Where it is enrolling
- Los Angeles, California, United States
- Aurora, Colorado, United States
- Atlanta, Georgia, United States
- Scarborough, Maine, United States
- Boston, Massachusetts, United States
- St Louis, Missouri, United States
- Charlotte, North Carolina, United States
- Philadelphia, Pennsylvania, United States
- Memphis, Tennessee, United States
- Dallas, Texas, United States
- Houston, Texas, United States
- Randwick, New South Wales, Australia
- South Brisbane, Queensland, Australia
- Nedlands, Western Australia, Australia
- Calgary, Alberta, Canada
- Edmonton, Alberta, Canada
- Ottawa, Ontario, Canada
- Montreal, Quebec, Canada
- Copenhagen, Denmark
- Angers, France
+ 18 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 25 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Los Angeles, California, United States; Aurora, Colorado, United States; Atlanta, Georgia, United States; Scarborough, Maine, United States; Boston, Massachusetts, United States; St Louis, Missouri, United States and 32 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.