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ALK Inhibitor

Lorlatinib for Neuroblastoma

Phase 1
Waitlist Available
Research Sponsored by New Approaches to Neuroblastoma Therapy Consortium
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
All patients must have at least one of the following: Recurrent/progressive disease after the diagnosis of high-risk neuroblastoma, refractory disease, or persistent disease
Patients must have a life expectancy of at least 12 weeks and a Lansky (≤16 years) or Karnofsky (>16 years) score of at least 50
Timeline
Screening 3 weeks
Treatment Varies
Follow Up all toxicities from enrollment until completion of course 2 (day 56)
Awards & highlights

Study Summary

This trial is testing a new drug, called lorlatinib, to see if it is safe and effective in children with relapsed or refractory neuroblastoma. This is a Phase I trial, which means that the goal is to find the best dose of the drug to give. The trial will first test the drug in a small group of patients to see if it is safe. If it is, then the trial will move on to testing different doses of the drug in a larger group of patients.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Possible side effects include reactions related to liver function changes, blood cell counts alterations leading to increased infection risk, potential kidney issues, heart problems like altered heart rhythm or function, as well as general symptoms such as fatigue.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My neuroblastoma has come back, is not responding, or hasn't gone away.
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I am expected to live at least 12 weeks and can do some daily activities on my own.
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I have been diagnosed with neuroblastoma confirmed by tests.
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My neuroblastoma is classified as high-risk.
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My neuroblastoma cancer has spread to my bone marrow.
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My tumor has an ALK mutation confirmed by a certified test.
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I have recovered from side effects of my previous cancer treatments.
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I have a lesion that tested positive for neuroblastoma or ganglioneuroblastoma.
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I have a tumor that can be measured.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~all toxicities from enrollment until completion of course 2 (day 56)
This trial's timeline: 3 weeks for screening, Varies for treatment, and all toxicities from enrollment until completion of course 2 (day 56) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Describe Hematological Toxicities (A1 and B1)
Describe Hematological Toxicities (A2)
Describe Hematological Toxicities (B2)
+6 more
Secondary outcome measures
Overall Response A1 and B1
Overall Response A2
Overall Response B2
+3 more

Side effects data

From 2020 Phase 1 trial • 29 Patients • NCT03542305
13%
Dizziness
13%
Headache
13%
Skin laceration
13%
Oropharyngeal pain
13%
Upper respiratory tract infection
13%
Diarrhoea
100%
80%
60%
40%
20%
0%
Study treatment Arm
Normal Function
Mild Impairment
Moderate Impairment
Severe Impairment

Trial Design

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
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Topotecan
2017
Completed Phase 3
~2400
Lorlatinib
2018
Completed Phase 4
~440
Cyclophosphamide
1995
Completed Phase 3
~3780

Find a Location

Who is running the clinical trial?

Ronan Thompson FoundationUNKNOWN
New Approaches to Neuroblastoma Therapy ConsortiumLead Sponsor
16 Previous Clinical Trials
1,569 Total Patients Enrolled
16 Trials studying Neuroblastoma
1,569 Patients Enrolled for Neuroblastoma
University of Southern CaliforniaOTHER
899 Previous Clinical Trials
1,595,238 Total Patients Enrolled
1 Trials studying Neuroblastoma
4 Patients Enrolled for Neuroblastoma

Media Library

Lorlatinib (ALK Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT03107988 — Phase 1
Neuroblastoma Research Study Groups: Cohort B2 (Combined w/ chemotherapy), Cohort A2 (Adult and large BSA), Cohort A1 (Dose-finding), Cohort B1 (Expansion)
Neuroblastoma Clinical Trial 2023: Lorlatinib Highlights & Side Effects. Trial Name: NCT03107988 — Phase 1
Lorlatinib (ALK Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03107988 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any North American locations that are running this clinical experiment?

"As of now, 13 medical facilities are offering this trial. These include Children Hospital of Colorado in Aurora, Children's Hospital Los Angeles in Los Angeles and the renowned Children's Healthcare of Atlanta located in Atlanta."

Answered by AI

What are the main aims of this experimental investigation?

"This clinical trial will be measuring the primary outcome, MTD/RP2D determination A1 across a three day period. Secondary objectives include evaluating bone response with MIBG scans and FDG-PET scans following Curie scoring criteria, determining marrow response through morphological and immunohistologic evaluation of bilaterally obtained samples, as well as assessing overall responses according to NANT Response Criteria Version 2.0 definitions in order to determine complete or partial responders."

Answered by AI

To what extent is Lorlatinib detrimental to one's health?

"Lorlatinib's safety rating, on a 1-3 scale, is determined to be at the lower end of that range due to there being minimal clinical data pertaining to its efficacy or harm."

Answered by AI

Is there availability for participants in this trial currently?

"As per the data hosted on clinicaltrials.gov, this medical trial is still open for enrollment. This research was first posted in September of 2017 and has recently been updated as of May 5th 2022."

Answered by AI

Would I be able to participate in this research initiative?

"This trial is looking to recruit 65 people between the ages of one and 99 who have already been diagnosed with neuroblastoma. Additionally, potential participants must meet size criteria (lesions ≥ 10 mm or lymph nodes ≥ 15mm), possess an activating ALK mutation or amplification (>10 signals) and/or a fusion protein arising from chromosomal translocation, be classified as high-risk according to COG risk classification at registration time, present with recurrent/progressive disease after diagnosis; refractory disease with no response/stable disease since diagnosis; persistent disease without partial response since diagnosis; MIBG avid bone site(s); FD"

Answered by AI

How many individuals are currently involved in this experiment?

"Absolutely, according to the clinicaltrials.gov website, this trial is actively pursuing participants and began on September 5th 2017 with its latest update being recorded in May 2022. The medical study requires 65 patients from 13 sites for their research."

Answered by AI

Has Lorlatinib been subjected to any other scientific research?

"Lorlatinib was first studied by City of Hope Comprehensive Cancer Center over two decades ago. Currently, there are a total 1225 completed trials and 868 active studies being conducted across the world, with numerous happening in Aurora, Colorado."

Answered by AI

What pathologies are commonly remediated by Lorlatinib?

"Lorlatinib is the go-to treatment for malignant neoplasms, but has been found to assist with other afflictions such as multiple sclerosis, leukemia, myelocytic and acute sarcoma."

Answered by AI

Is this experiment allowing participants aged 20 and above to join?

"The research requirements state that prospective participants must be between one and ninety-nine years old. Per the database, there are 454 trials for minors and 703 studies available to those above 65."

Answered by AI
~6 spots leftby Dec 2024