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Lorlatinib + Standard Therapy for Neuroblastoma

Phase 3
Waitlist Available
Led By Steven DuBois
Research Sponsored by Children's Oncology Group
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients with INRG stage MS disease with MYCN amplification
Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; the following disease groups are eligible:
Must not have
Patients with bone marrow failure syndromes
Patients for whom targeted radiopharmaceutical therapy would be contraindicated due to underlying medical disorders
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is testing a new drug, lorlatinib, in combination with the standard treatment for neuroblastoma or ganglioneuroblastoma, compared to the standard treatment alone.

Who is the study for?
This trial is for young patients up to 30 years old with high-risk neuroblastoma or ganglioneuroblastoma. They must have certain levels of kidney and liver function, no prior systemic therapy (with some exceptions), and can't be pregnant or breastfeeding. Patients need a confirmed diagnosis and meet specific criteria based on their disease stage and features.
What is being tested?
The study tests adding iobenguane I-131 or lorlatinib to standard therapy in treating high-risk neuroblastoma. Iobenguane I-131 delivers radiation directly to cancer cells, while lorlatinib blocks enzymes that help tumor growth. The goal is to see if these additions improve treatment outcomes.
What are the potential side effects?
Potential side effects include radiation-related risks from iobenguane I-131, such as damage to healthy cells near the tumor, and typical chemotherapy side effects like nausea, hair loss, fatigue, infection risk increase due to lowered blood cell counts.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My cancer is in an advanced stage with a specific genetic change.
Select...
I have been diagnosed with neuroblastoma or ganglioneuroblastoma.
Select...
I am a boy aged 13-15 with a score of 1.5 or a girl with a score of 1.4.
Select...
I can safely undergo a procedure to collect stem cells from my blood.
Select...
I was diagnosed between the ages of 1 and 30.
Select...
My cancer shows a high level of MYCN gene activity.
Select...
My cancer is at a specific stage (L2) and has a certain genetic feature (MYCN amplified).
Select...
I am older than 547 days.
Select...
My kidney function is normal or near normal.
Select...
My bilirubin levels are within the normal range for my age.
Select...
I am a child aged 6 to less than 10 years.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have a condition where my bone marrow doesn't produce enough blood cells.
Select...
I cannot receive targeted radiopharmaceutical therapy due to my health conditions.
Select...
My tumor is stage L2 without MYCN amplification.
Select...
I am not pregnant and can take a pregnancy test if needed.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Event free survival (EFS) (Arm A, B, D, and E)
Secondary study objectives
EFS (Arm C)
Incidence of adverse events
Overall survival (OS)
+1 more

Side effects data

From 2022 Phase 3 trial • 665 Patients • NCT00567567
84%
58300-Neutrophil count decreased
70%
65800-Platelet count decreased
20%
43100-Hypokalemia
18%
44800-Infections and infestations - Other specify
18%
33300-Febrile neutropenia
17%
88500-White blood cell decreased
13%
13200-Anemia
9%
55600-Mucositis oral
7%
42700-Hypocalcemia
7%
13500-Anorexia
7%
11600-Alanine aminotransferase increased
7%
41400-Hyperglycemia
6%
15000-Aspartate aminotransferase increased
6%
43300-Hyponatremia
6%
73700-Sepsis
6%
53700-Lymphocyte count decreased
4%
25700-Diarrhea
3%
57600-Nausea
3%
65900-Pleural effusion
3%
37500-GGT increased
3%
41600-Hyperkalemia
3%
10300-Abdominal pain
3%
20500-Catheter related infection
3%
59700-Oral pain
2%
38900-Hearing impaired
2%
43900-Hypoxia
2%
17200-Blood and lymphatic system disorders - Other specify
2%
14900-Ascites
2%
75700-Small intestinal obstruction
2%
87900-Vomiting
2%
43600-Hypotension
1%
34000-Fibrinogen decreased
1%
23000-Confusion
1%
42600-Hypoalbuminemia
1%
71500-Respiratory failure
1%
45800-INR increased
1%
73900-Serum amylase increased
1%
26600-Duodenal obstruction
1%
66300-Pneumonitis
1%
69700-Rash maculo-papular
1%
58000-Neoplasms benign malignant and unspecified (incl cysts and polyps) - Other specify
1%
56600-Myelitis
1%
75600-Small intestinal mucositis
1%
66800-Postoperative hemorrhage
1%
43500-Hypophosphatemia
1%
37300-Generalized muscle weakness
1%
81200-Treatment related secondary malignancy
1%
31200-Esophagitis
1%
83100-Urinary tract infection
1%
24100-Creatinine increased
1%
11100-Acute kidney injury
1%
62600-Pelvic pain
1%
65300-Pharyngolaryngeal pain
1%
31900-Eye disorders - Other specify
1%
10900-Activated partial thromboplastin time prolonged
1%
11800-Alkaline phosphatase increased
1%
42500-Hyperuricemia
1%
17400-Blood bilirubin increased
1%
63100-Pericardial effusion
1%
72700-Right ventricular dysfunction
1%
37200-General disorders and administration site conditions - Other specify
1%
40000-Hepatic failure
1%
88200-Weight gain
1%
41300-Hypercalcemia
1%
54900-Metabolism and nutrition disorders - Other specify
1%
71000-Renal and urinary disorders - Other specify
1%
69000-Pulmonary hypertension
1%
20100-Cardiac disorders - Other specify
1%
22100-Colitis
1%
44200-Ileal obstruction
1%
81900-Typhlitis
1%
33900-Fever
1%
35500-Gallbladder pain
1%
40600-Hepatobiliary disorders - Other specify
1%
66500-Portal hypertension
1%
12000-Allergic reaction
1%
13100-Anaphylaxis
1%
44700-Immune system disorders - Other specify
1%
13400-Anorectal infection
1%
25600-Device related infection
1%
29500-Enterocolitis infectious
1%
53100-Lung infection
1%
62500-Pelvic infection
1%
75200-Skin infection
1%
82300-Upper respiratory infection
1%
14500-Arterial injury
1%
15300-Ataxia
1%
38800-Headache
1%
63900-Peripheral motor neuropathy
1%
11300-Adult respiratory distress syndrome
1%
29700-Epistaxis
1%
78100-Stridor
1%
68400-Pruritus
1%
51700-Left ventricular systolic dysfunction
1%
27800-Dyspnea
1%
58100-Nervous system disorders - Other specify
1%
29000-Encephalopathy
1%
42100-Hypertension
1%
24700-Dehydration
1%
43200-Hypomagnesemia
1%
31800-Extrapyramidal disorder
1%
52600-Lipase increased
1%
10700-Acidosis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Tandem HST (CEM), Randomly Assigned
Single HST (CEM)
Not Assigned

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

5Treatment groups
Experimental Treatment
Group I: Arm E (lorlatinib, chemotherapy, HSCT, EBRT)Experimental Treatment22 Interventions
See Arm E in detailed description.
Group II: Arm D (chemotherapy, HSCT, EBRT)Experimental Treatment22 Interventions
See Arm D in detailed description.
Group III: Arm C (Iobenguane I-131, chemotherapy, BuMel, HSCT, EBRT)Experimental Treatment24 Interventions
See Arm C in detailed description. Closed to accrual as of 12/17/20.
Group IV: Arm B (Iobenguane I-131, chemotherapy, HSCT, EBRT)Experimental Treatment24 Interventions
See Arm B in detailed description.
Group V: Arm A (chemotherapy, HSCT, EBRT)Experimental Treatment22 Interventions
See Arm A in detailed description.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Biospecimen Collection
2004
Completed Phase 3
~2020
Bone Marrow Aspiration and Biopsy
2016
Completed Phase 1
~40
Positron Emission Tomography
2011
Completed Phase 2
~2200
Topotecan Hydrochloride
2013
Completed Phase 3
~6270
External Beam Radiation Therapy
2006
Completed Phase 3
~3300
Echocardiography
2013
Completed Phase 4
~11580
Vincristine Sulfate
2005
Completed Phase 3
~10270
Computed Tomography
2017
Completed Phase 2
~2740
Magnetic Resonance Imaging
2017
Completed Phase 3
~1160
Multigated Acquisition Scan
2015
Completed Phase 3
~270
Autologous Hematopoietic Stem Cell Transplantation
2017
Completed Phase 3
~2090
Busulfan
2008
Completed Phase 4
~1710
Carboplatin
2014
Completed Phase 3
~6120
Cyclophosphamide
2010
Completed Phase 4
~2310
Lorlatinib
2018
Completed Phase 4
~530
Sargramostim
2006
Completed Phase 4
~880
Dexrazoxane Hydrochloride
2013
Completed Phase 1
~50
Etoposide Phosphate
2011
Completed Phase 2
~160
Cisplatin
2013
Completed Phase 3
~3120
Dinutuximab
2009
Completed Phase 3
~710
Doxorubicin Hydrochloride
2019
Completed Phase 3
~17860
Melphalan Hydrochloride
2016
Completed Phase 2
~130
Isotretinoin
2019
Completed Phase 4
~3520
Therapeutic Conventional Surgery
2005
Completed Phase 3
~9890
Thiotepa
2008
Completed Phase 3
~2120

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)NIH
13,920 Previous Clinical Trials
41,016,210 Total Patients Enrolled
206 Trials studying Neuroblastoma
52,913 Patients Enrolled for Neuroblastoma
Children's Oncology GroupLead Sponsor
460 Previous Clinical Trials
239,313 Total Patients Enrolled
63 Trials studying Neuroblastoma
31,104 Patients Enrolled for Neuroblastoma
Steven DuBoisPrincipal InvestigatorChildren's Oncology Group
1 Previous Clinical Trials
35 Total Patients Enrolled

Media Library

Autologous Hematopoietic Stem Cell Transplantation Clinical Trial Eligibility Overview. Trial Name: NCT03126916 — Phase 3
Neuroblastoma Research Study Groups: Arm C (Iobenguane I-131, chemotherapy, BuMel, HSCT, EBRT), Arm E (lorlatinib, chemotherapy, HSCT, EBRT), Arm A (chemotherapy, HSCT, EBRT), Arm D (chemotherapy, HSCT, EBRT), Arm B (Iobenguane I-131, chemotherapy, HSCT, EBRT)
Neuroblastoma Clinical Trial 2023: Autologous Hematopoietic Stem Cell Transplantation Highlights & Side Effects. Trial Name: NCT03126916 — Phase 3
Autologous Hematopoietic Stem Cell Transplantation 2023 Treatment Timeline for Medical Study. Trial Name: NCT03126916 — Phase 3
~159 spots leftby Sep 2026
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