Lorlatinib for Neuroblastoma

Recruiting in Palo Alto (17 mi)

+14 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: New Approaches to Neuroblastoma Therapy Consortium

No Placebo Group

Breakthrough Therapy

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?This trial tests Lorlatinib, a new drug that blocks proteins helping cancer grow, in children whose neuroblastoma has returned or not responded to other treatments. The goal is to find the best dose and see how well it works.

Eligibility Criteria

This trial is for children and adults with high-risk neuroblastoma that's come back or hasn't responded to treatment. They must have certain types of tumor cells in their bone marrow, a life expectancy over 12 weeks, and good organ function. They can't have had lorlatinib before but other ALK inhibitors are okay. No recent cancer treatments or uncontrolled illnesses.

Inclusion Criteria

My neuroblastoma has come back, is not responding, or hasn't gone away.

Patients must have specific imaging criteria for MIBG avid or non-avid tumors

I am expected to live at least 12 weeks and can do some daily activities on my own.

+8 more

Exclusion Criteria

I do not have any ongoing or uncontrolled infections.

I have thought about suicide recently and have attempted it before.

I have chosen not to participate in the NANT 2004-05 study.

+7 more

Participant Groups

The study tests Lorlatinib alone and with chemotherapy (Topotecan, Cyclophosphamide) in patients whose neuroblastoma has relapsed or is resistant to treatment. It starts by finding the safest dose of Lorlatinib (Phase 1), then expands to more patients once the right dose is found.

4Treatment groups

Experimental Treatment

Group I: Cohort B2 (Combined w/ chemotherapy)Experimental Treatment4 Interventions

Lorlatinib will be given orally once daily continuously for 28 days, at the RP2D defined by cohort A1. Lorlatinib should be administered at least one hour prior to conventional chemotherapy (Cyclophosphamide and Topotecan) on days 1-5 of each cycle.

Group II: Cohort B1 (Expansion)Experimental Treatment1 Intervention

Lorlatinib will be given orally once daily continuously for 28 days at the RP2D defined by cohort A1. This cohort will not begin enrollment until the recommended phase 2 dose is established from the dose escalation cohort A1.

Group III: Cohort A2 (Adult and large BSA)Experimental Treatment1 Intervention

Lorlatinib will be given at the adult recommended phase 2 dose (RP2D) of 100 mg orally once daily continuously for 28 days.

Group IV: Cohort A1 (Dose-finding)Experimental Treatment1 Intervention

Lorlatinib will be given orally once daily continuously for 28 days. The dose level of lorlatinib will be assigned at the time of study registration. The starting dose for cohort A1 is 45 mg/m2/dose

Lorlatinib is already approved in United States, European Union, Japan, Canada for the following indications:

🇺🇸 Approved in United States as Lorbrena for: