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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:
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights
Study 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.Check my eligibility
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.See study design
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.
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%
42600-Hypoalbuminemia
1%
71500-Respiratory failure
1%
26600-Duodenal obstruction
1%
69700-Rash maculo-papular
1%
23000-Confusion
1%
66300-Pneumonitis
1%
34000-Fibrinogen decreased
1%
45800-INR increased
1%
73900-Serum amylase increased
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
Busulfan
2008
Completed Phase 3
~1120
Cyclophosphamide
1995
Completed Phase 3
~3780
Carboplatin
2014
Completed Phase 3
~6670
Topotecan Hydrochloride
2013
Completed Phase 3
~6120
Autologous Hematopoietic Stem Cell Transplantation
2017
Completed Phase 3
~1730
Vincristine Sulfate
2005
Completed Phase 3
~10110
External Beam Radiation Therapy
2006
Completed Phase 3
~3070
Lorlatinib
2018
Completed Phase 4
~440
Sargramostim
2008
Completed Phase 4
~710
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
~17850
Melphalan Hydrochloride
2016
Completed Phase 2
~70
Isotretinoin
2019
Completed Phase 4
~3520
Therapeutic Conventional Surgery
2005
Completed Phase 3
~9850
Thiotepa
2008
Completed Phase 3
~2150
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)NIH
13,654 Previous Clinical Trials
40,932,429 Total Patients Enrolled
205 Trials studying Neuroblastoma
53,004 Patients Enrolled for Neuroblastoma
Children's Oncology GroupLead Sponsor
453 Previous Clinical Trials
236,976 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.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Based on the information I have provided, can I join this test group?
"This study is investigating a treatment for ganglioneuroblastoma in patients that are between 365 days and 30 years old. A total of 658 individuals will be enrolled in the trial."
Answered by AI
What types of diseases or conditions does Autologous Hematopoietic Stem Cell Transplantation commonly help?
"Autologous Hematopoietic Stem Cell Transplantation is a medical procedure with potential to cure malignant neoplasms. Additionally, it has shown efficacy in treating other conditions such as high risk neuroblastoma, kaposi's sarcoma aids related, and merkel cell cancer."
Answered by AI
Are there other similar investigations to this one?
"As of now, there are 2386 open medical studies related to Autologous Hematopoietic Stem Cell Transplantation in 87 different countries. The first trial was sponsored by Alfacell and completed in 1997. That study only had 300 participants and only reached Phase 3 in terms of drug approval. In the 23 years since 1997, there have been 3657 trials in total."
Answered by AI
Does the FDA sanction Autologous Hematopoietic Stem Cell Transplantation?
"Autologous hematopoietic stem cell transplantation is a phase 3 trial, meaning that there is some data supporting efficacy and multiple rounds of data supporting safety. Therefore, our team at Power estimates the safety of this treatment to be a 3."
Answered by AI
What is the frequency with which Autologous Hematopoietic Stem Cell Transplantation has been used in previous clinical trials?
"The first clinical trial studying Autologous Hematopoietic Stem Cell Transplantation was completed in 1997 at Spectrum Health Hospital - Butterworth Campus. In total, there have been 3657 completed clinical trials as of today. There are 2386 trials that are ongoing, with a significant amount of these taking place in Saint Louis, Missouri."
Answered by AI
Are individuals below the age of 80 allowed to participate in this experiment?
"As this study only accepts participants that meet specific eligibility requirements, the minimum age an individual can be is 365 days old, with a maximum age of 30."
Answered by AI
How many guinea pigs are testing this new drug?
"In order to carry out this research, 658 willing participants that meet the necessary requirements are needed. Patients have the opportunity to take part in this study at different sites such as Washington University School of Medicine in Saint Louis, Missouri and University of Minnesota/Masonic Cancer Center in Minneapolis, Minnesota."
Answered by AI
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