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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
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 belowSelect...
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Event free survival (EFS) (Arm A, B, D, and E)
Secondary outcome measures
EFS (Arm C)
Incidence of adverse events
Overall survival (OS)
+1 moreSide effects data
From 2022 Phase 3 trial • 665 Patients • NCT0056756784%
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%
26600-Duodenal obstruction
1%
42600-Hypoalbuminemia
1%
34000-Fibrinogen decreased
1%
71500-Respiratory failure
1%
69700-Rash maculo-papular
1%
73900-Serum amylase increased
1%
23000-Confusion
1%
45800-INR increased
1%
66300-Pneumonitis
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
Trial Design
5Treatment groups
Experimental Treatment
Group I: Arm E (lorlatinib, chemotherapy, HSCT, EBRT)Experimental Treatment15 Interventions
See Arm E in detailed description.
Group II: Arm D (chemotherapy, HSCT, EBRT)Experimental Treatment15 Interventions
See Arm D in detailed description.
Group III: Arm C (Iobenguane I-131, chemotherapy, BuMel, HSCT, EBRT)Experimental Treatment17 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 Treatment16 Interventions
See Arm B in detailed description.
Group V: Arm A (chemotherapy, HSCT, EBRT)Experimental Treatment15 Interventions
See Arm A in detailed description.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
External Beam Radiation Therapy
2006
Completed Phase 3
~3150
Topotecan Hydrochloride
2013
Completed Phase 3
~6120
Vincristine Sulfate
2005
Completed Phase 3
~10160
Autologous Hematopoietic Stem Cell Transplantation
2017
Completed Phase 3
~1880
Busulfan
2008
Completed Phase 3
~930
Carboplatin
2014
Completed Phase 3
~6670
Cyclophosphamide
1995
Completed Phase 3
~3770
Lorlatinib
2018
Completed Phase 4
~440
Sargramostim
2008
Completed Phase 4
~850
Dexrazoxane Hydrochloride
2013
Completed Phase 1
~50
Etoposide Phosphate
2011
Completed Phase 2
~160
Cisplatin
2013
Completed Phase 3
~1940
Dinutuximab
2009
Completed Phase 3
~710
Doxorubicin Hydrochloride
2019
Completed Phase 3
~17860
Melphalan Hydrochloride
2016
Completed Phase 2
~70
Isotretinoin
2019
Completed Phase 4
~3520
Therapeutic Conventional Surgery
2005
Completed Phase 3
~9870
Thiotepa
2008
Completed Phase 3
~2140
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)NIH
13,748 Previous Clinical Trials
40,958,677 Total Patients Enrolled
205 Trials studying Neuroblastoma
52,960 Patients Enrolled for Neuroblastoma
Children's Oncology GroupLead Sponsor
456 Previous Clinical Trials
239,063 Total Patients Enrolled
63 Trials studying Neuroblastoma
31,145 Patients Enrolled for Neuroblastoma
Steven DuBoisPrincipal InvestigatorChildren's Oncology Group
1 Previous Clinical Trials
35 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have a condition where my bone marrow doesn't produce enough blood cells.I cannot receive targeted radiopharmaceutical therapy due to my health conditions.My cancer is in an advanced stage with a specific genetic change.I was diagnosed with a certain stage of cancer before it spread, and I haven't had chemotherapy.I am a child aged 10 to less than 13 years with a specific health measure.I have been diagnosed with neuroblastoma or ganglioneuroblastoma.I am a boy aged 13-15 with a score of 1.5 or a girl with a score of 1.4.My creatinine clearance rate is at least 1.7 (male) or 1.4 (female).I can safely undergo a procedure to collect stem cells from my blood.My tumor is stage L2 without MYCN amplification.My child was diagnosed with a specific type of cancer before it spread, and it has now progressed without treatment.I have high-risk disease with limited prior treatment or emergency care only.I was diagnosed between the ages of 1 and 30.My child is between 2 and 6 years old with a specific health measurement.My cancer shows a high level of MYCN gene activity.My neuroblastoma is stage M with specific features.My cancer is at a specific stage (L2) and has a certain genetic feature (MYCN amplified).I am not pregnant and can take a pregnancy test if needed.I am older than 547 days.My kidney function is normal or near normal.My child is between 1 and 2 years old with a required level.My bilirubin levels are within the normal range for my age.I am a child aged 6 to less than 10 years.
Research Study Groups:
This trial has the following groups:- Group 1: Arm C (Iobenguane I-131, chemotherapy, BuMel, HSCT, EBRT)
- Group 2: Arm E (lorlatinib, chemotherapy, HSCT, EBRT)
- Group 3: Arm A (chemotherapy, HSCT, EBRT)
- Group 4: Arm D (chemotherapy, HSCT, EBRT)
- Group 5: Arm B (Iobenguane I-131, chemotherapy, HSCT, EBRT)
Awards:
This trial has 2 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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