Primary Refractory NeuroblastomaPhase 2NCT00969722
A Study of MAb-3F8 Plus Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Versus 13-cis-Retinoic Acid (RA) Plus GM-CSF in Primary Refractory Neuroblastoma Patients
A Phase 2 study testing MAb-3F8, Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) and 13-cis-Retinoic Acid in people with Primary Refractory Neuroblastoma, run by United Therapeutics at 15 sites in United States.
Trial summary
This is a multicenter, randomized, controlled, open-label study. Patients meeting inclusion/exclusion criteria will be randomized (1:1) to receive two cycles of MAb-3F8 plus GM-CSF or RA plus GM-CSF. Read more about this study
- Who is it for?
- Ages 18 months to 13 years
- Conditions the study is for
- Primary Refractory Neuroblastoma
- What is being tested?
- MAb-3F8, Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) and 13-cis-Retinoic Acid
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 15 sitesUnited States
- Study dates
- Aug 2009to Aug 2010
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 months to 13 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Have a diagnosis of stage 4 neuroblastoma diagnosed in accordance with the International Neuroblastoma Staging System: either (a) histologic confirmation which may involve immunohistochemical, ultrastructural, and/or cytogenetic studies, or (b) elevated urinary catecholamines plus tumor cells/clumps in the bone marrow.
- Have evaluable disease or biopsy-proven stable disease in BM by histology or MIBG scan with MIBG-positive disease confined to the bone or bone marrow, plus urine catecholamine results, documented >3 weeks after conventional chemotherapy or >6 weeks after stem-cell transplantation. CT, MRI, or bone scan (if necessary) can be done at 2-3 weeks after conventional chemotherapy confirming that the chemotherapy, radiotherapy, and ABMT are not realistic curative options.
- Be between 18 months to 13 years old at diagnosis.
- Have recovered to grade 2 or better toxicities since their prior therapy.
- Must, if female of childbearing potential, be willing to use two forms of medically acceptable contraception (at least one barrier method) and have a negative pregnancy test at screening and monthly thereafter through the first four cycles of treatment.
- Have a performance score of at least 60 from Lansky Play Performance Scale if aged up to 16 years or at least 60 from Karnofsky Scale if aged more than 16 years.
- Have voluntarily agreed to participate.
Who cannot join
- Have measurable disease ≥ 1 cm assessed by CT or MRI.
- Have progressive disease (any new lesion; increase of any measurable lesion by >25%; or previous negative marrow positive for tumor).
- Have disease detectable in CNS (confirmed by CT or MRI of the brain at screening or within 8 weeks of randomization).
- Be receiving alternative therapy for the treatment of neuroblastoma, e.g. radiotherapy or chemotherapy within 3 weeks of randomization.
- Require additional therapy (such as radiotherapy) during the first two treatment cycles.
- Have detectable human anti-mouse antibody titers at screening.
Show all 10Show fewer
- Have received prior anti-GD2 investigational therapies.
- Have a history of allergies to mouse proteins.
- Have an active infection requiring IV infusion of antibiotics.
- Be currently receiving long-term chronic treatment with immunosuppressive drugs such as cyclosporine, adrenocorticotropic hormone (ACTH), or systemic corticosteroids.
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
Vermont Cancer Center
Burlington, Vermont, 05405
Nationwide Childrens Hospital
Columbus, Ohio, 43205
US Oncology
Dallas, Texas, 75230
Duke University Medical Center
Durham, North Carolina, 27705
The University of Texas M.D. Anderson Cancer Center
Houston, Texas, 77030
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
LSU Health Sciences Center; Children's Hospital
New Orleans, Louisiana, 70118
University of Oklahoma Cancer Center
Oklahoma City, Oklahoma, 73104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
University of Utah Medical Center
Salt Lake City, Utah, 84113
Rady Children's Hospital of San Diego
San Diego, California, 92123
All Children's Hospital in Florida
St. Petersburg, Florida, 33701
Children's Hospital at Montefiore
The Bronx, New York, 10467
Georgetown Medical Center
Washington D.C., District of Columbia, 20057
About this study
Official title: A Randomized Study of Monoclonal Antibody 3F8 Plus Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Compared to 13-cis-Retinoic Acid Plus GM-CSF in High Risk Stage 4, Primary Refractory Neuroblastoma Patients
This is a multicenter, randomized, controlled, open-label study. Patients meeting inclusion/exclusion criteria will be randomized (1:1) to receive two cycles of MAb-3F8 plus GM-CSF or RA plus GM-CSF. Patients who do not respond to their assigned treatment after two cycles may cross-over to receive the alternate treatment. Disease response and safety will be assessed in all patients after cycle 2 and after cycle 4.
- Conditions
- Primary Refractory Neuroblastoma
- Being tested
- MAb-3F8, Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF), 13-cis-Retinoic Acid
- Keywords
- Neuroblastoma, 3F8, Primary refractory
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MAb-3F8Biological
Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)Biological
13-cis-Retinoic AcidBiological
| Study group | Role | What they receive |
|---|---|---|
| ARM I | Experimental | Intravenous MAb-3F8 plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) Receives: Biological: MAb-3F8; Biological: Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) |
| ARM II | Active comparator | Oral 13-cis-Retinoic Acid (RA) plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) Receives: Biological: Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF); Biological: 13-cis-Retinoic Acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
To compare the proportion of patients achieving a complete bone marrow response measured by an absence of histological evidence of bone marrow disease and by MIBG scan after two cycles of treatment.
Measured: two years
Secondary outcomes (1)
A comparison in treatment arms for disease response as measured by CT/MRI scan and urine catecholamines, MIBG extent of disease scores, disease response in cross-over patients.
Measured: two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2009 (estimated)
- Study startAug 2009
- Primary completionAug 2010
Why it stopped: Lack of enrollment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Refractory NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- United Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Peter E. Zage, M.D., Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00969722
- Sponsor's protocol ID
- 3F8-NB-201
- Status last verified
- Feb 2013
- First submitted
- 31 Aug 2009
- Last updated
- 8 Mar 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Mar 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Study of Monoclonal Antibody 3F8 Plus Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Compared to 13-cis-Retinoic Acid Plus GM-CSF in High Risk Stage 4, Primary Refractory Neuroblastoma Patients
- ClinicalTrials.gov ID
- NCT00969722
- Acronym / sponsor ID
- 3F8-NB-201
- Conditions
- Primary Refractory Neuroblastoma
- Keywords
- Neuroblastoma, 3F8, Primary refractory
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of enrollment.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- United TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Aug 2009
- Primary completion
- Aug 2010 (actual)
- First posted
- 1 Sep 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, randomized, controlled, open-label study. Patients meeting inclusion/exclusion criteria will be randomized (1:1) to receive two cycles of MAb-3F8 plus GM-CSF or RA plus GM-CSF. Patients who do not respond to their assigned treatment after two cycles may cross-over to receive the alternate treatment. Disease response and safety will be assessed in all patients after cycle 2 and after cycle 4.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ARM I | Experimental | Biological: MAb-3F8, Biological: Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)Intravenous MAb-3F8 plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) |
| ARM II | Active comparator | Biological: Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF), Biological: 13-cis-Retinoic AcidOral 13-cis-Retinoic Acid (RA) plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) |
Who may qualify
- Ages
- 18 months to 13 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Have a diagnosis of stage 4 neuroblastoma diagnosed in accordance with the International Neuroblastoma Staging System: either (a) histologic confirmation which may involve immunohistochemical, ultrastructural, and/or cytogenetic studies, or (b) elevated urinary catecholamines plus tumor cells/clumps in the bone marrow.
- Have evaluable disease or biopsy-proven stable disease in BM by histology or MIBG scan with MIBG-positive disease confined to the bone or bone marrow, plus urine catecholamine results, documented >3 weeks after conventional chemotherapy or >6 weeks after stem-cell transplantation. CT, MRI, or bone scan (if necessary) can be done at 2-3 weeks after conventional chemotherapy confirming that the chemotherapy, radiotherapy, and ABMT are not realistic curative options.
- Be between 18 months to 13 years old at diagnosis.
- Have recovered to grade 2 or better toxicities since their prior therapy.
- Must, if female of childbearing potential, be willing to use two forms of medically acceptable contraception (at least one barrier method) and have a negative pregnancy test at screening and monthly thereafter through the first four cycles of treatment.
Key exclusion criteria (10 listed)
- Have measurable disease ≥ 1 cm assessed by CT or MRI.
- Have progressive disease (any new lesion; increase of any measurable lesion by >25%; or previous negative marrow positive for tumor).
- Have disease detectable in CNS (confirmed by CT or MRI of the brain at screening or within 8 weeks of randomization).
- Be receiving alternative therapy for the treatment of neuroblastoma, e.g. radiotherapy or chemotherapy within 3 weeks of randomization.
- Require additional therapy (such as radiotherapy) during the first two treatment cycles.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MAb-3F8 | Biological | ARM I |
| Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) | Biological | ARM I, ARM II |
| 13-cis-Retinoic Acid | Biological | ARM II |
Primary outcome
To compare the proportion of patients achieving a complete bone marrow response measured by an absence of histological evidence of bone marrow disease and by MIBG scan after two cycles of treatment.
Time frame: two years
1 secondary outcome
A comparison in treatment arms for disease response as measured by CT/MRI scan and urine catecholamines, MIBG extent of disease scores, disease response in cross-over patients.
Time frame: two years
Results and safety
The study was stopped early (reason given: Lack of enrollment.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Mar 2013.
Related trials for Primary Refractory Neuroblastoma
Active, not recruitingPhase 2
Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)
Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm
Active, not recruitingPhase 2
Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma
Active, not recruitingPhase 2
Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)
Advanced Malignant Solid Neoplasm, Recurrent Ependymal Tumor
Active, not recruitingPhase 2
Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma
Active, not recruitingPhase 2
Larotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial)
Advanced Malignant Solid Neoplasm, Recurrent Ependymoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Peter E. Zage, M.D. principal investigator
M.D. Anderson Cancer Center
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MAb-3F8, Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) and 13-cis-Retinoic Acid in people with Primary Refractory Neuroblastoma, run by United Therapeutics at 15 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Mar 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Lack of enrollment.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To compare the proportion of patients achieving a complete bone marrow response measured by an absence of histological evidence of bone marrow disease and by MIBG scan after two cycles of treatment. (time frame: two years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Mar 2013.