Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Sponsor decision
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The purpose of this study was to assess the incidence of human anti-chimeric antibody (HACA) in high-risk neuroblastoma patients treated with Unituxin combination therapy.
This was a multi-center, non-randomized, open-label study in patients with high-risk neuroblastoma to assess the immunogenicity, safety and tolerability of Unituxin combination therapy. Patients enrolled in the study were prescribed Unituxin for the treatment of high-risk neuroblastoma.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dinutuximab administered for 5 cycles | High-risk neuroblastoma patient treated with Unituxin as standard of care |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dinutuximab | Drug | Unituxin was administered along with cytokines according to the prescribing information |
|
| Measure | Description | Time Frame |
|---|---|---|
| To Determine the Incidence of Human Anti-chimeric Antibody (HACA) in High-risk Neuroblastoma Patients Treated With Unituxin Combination Therapy. | Seven blood samples were collected at the following time points for the evaluation of HACA levels:
| Approximately 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| To Determine the Incidence of Targeted Immune-related Adverse Events (AEs) During Treatment With Dinutuximab Combination Therapy in High-risk Neuroblastoma Subjects. | The incidence of targeted immune-related adverse events (AEs) during treatment with dinutuximab combination therapy in high-risk neuroblastoma subjects were summarized and listed. | Approximately 6 months |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
High-risk neuroblastoma patients prescribed Unituxin
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Ami Desai, MD | Children's Hospital of Philadelphia | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Of Alabama At Birmingham | Birmingham | Alabama | 35233 | United States | ||
| Arkansas Children's Hospital |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Dinutuximab Administered for 5 Cycles | High-risk neuroblastoma patient treated with Unituxin as standard of care Dinutuximab: Unituxin was administered along with cytokines according to the prescribing information |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
| To Determine the Incidence of Neutralizing Antibody (NAb) in Patients With Human Anti-chimeric Antibody (HACA) Positive Samples. | Incidence of neutralizing antibody (NAb) in patients with human anti-chimeric antibody (HACA) positive samples was summarized and listed. | Approximately 6 months |
| To Determine the Effect of HACA on Dinutuximab Plasma Concentrations. | Ten blood samples were collected at the following time points for the evaluation of dinutuximab plasma concentrations:
An additional 3 blood samples were obtained for the evaluation of dinutuximab plasma concentrations. Each of these blood samples was obtained immediately following the fourth dinutuximab infusion in Courses 1, 3, and 5. | Approximately 6 months |
| Little Rock |
| Arkansas |
| 72202 |
| United States |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States |
| Children's Hospital of Orange County | Orange | California | 92868 | United States |
| Rady Children's Hospital- San Diego | San Diego | California | 92123 | United States |
| University of Chicago | Chicago | Illinois | 60637 | United States |
| Dana Farber Cancer Institute | Boston | Massachusetts | 02215 | United States |
| University of Michigan - C S Mott Children's Hospital | Ann Arbor | Michigan | 48109 | United States |
| Children's Hospitals and Clinics of Minnesota - Minneapolis | Minneapolis | Minnesota | 55404 | United States |
| Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Carolinas Medical Center / Levine Children's Hospital | Charlotte | North Carolina | 28203 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Penn State Hershey Children's Hospital | Hershey | Pennsylvania | 17033 | United States |
| Vanderbilt-Ingram Cancer Center | Nashville | Tennessee | 37232 | United States |
| Cook Children's Health Care System | Fort Worth | Texas | 76104 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety Population
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Dinutuximab Administered for 5 Cycles | High-risk neuroblastoma patient treated with Unituxin as standard of care Dinutuximab: Unituxin was administered along with cytokines according to the prescribing information |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Age at Enrollment | Mean | Standard Deviation | years |
| |||||||||||||||||||||
| Age, Customized | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | participants |
| |||||||||||||||||||||||
| Time since Diagnosis | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Treatment of Neuroblastoma | Count of Participants | Participants |
| |||||||||||||||||||||||
| Pre-Autologous Stem Cell Transplantation (ASCT) Response | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | To Determine the Incidence of Human Anti-chimeric Antibody (HACA) in High-risk Neuroblastoma Patients Treated With Unituxin Combination Therapy. | Seven blood samples were collected at the following time points for the evaluation of HACA levels:
| Outcome measure data were not collected. Sponsor was required to run this Phase 4 study for EU marketing approval. However, Sponsor terminated the study 15 Dec 2016 and application withdrawn (accepted by European Commission 20 Mar 2017). | Posted | Approximately 6 months |
|
| |||||||||||||||||||
| Secondary | To Determine the Incidence of Targeted Immune-related Adverse Events (AEs) During Treatment With Dinutuximab Combination Therapy in High-risk Neuroblastoma Subjects. | The incidence of targeted immune-related adverse events (AEs) during treatment with dinutuximab combination therapy in high-risk neuroblastoma subjects were summarized and listed. | Outcome measure data were not collected. Safety data from the 12 subjects dosed prior to study termination were collected and summary level data were reported (eg, total number of subjects affected and total number of events for dinutuximab-treated subjects). Please refer to the adverse event tables for results. | Posted | Approximately 6 months |
|
| |||||||||||||||||||
| Secondary | To Determine the Incidence of Neutralizing Antibody (NAb) in Patients With Human Anti-chimeric Antibody (HACA) Positive Samples. | Incidence of neutralizing antibody (NAb) in patients with human anti-chimeric antibody (HACA) positive samples was summarized and listed. | Outcome measure data were not collected. Sponsor was required to run this Phase 4 study for EU marketing approval. However, Sponsor terminated the study 15 Dec 2016 and application withdrawn (accepted by European Commission 20 Mar 2017). | Posted | Approximately 6 months |
|
| |||||||||||||||||||
| Secondary | To Determine the Effect of HACA on Dinutuximab Plasma Concentrations. | Ten blood samples were collected at the following time points for the evaluation of dinutuximab plasma concentrations:
An additional 3 blood samples were obtained for the evaluation of dinutuximab plasma concentrations. Each of these blood samples was obtained immediately following the fourth dinutuximab infusion in Courses 1, 3, and 5. | Outcome measure data were not collected. Sponsor was required to run this Phase 4 study for EU marketing approval. However, Sponsor terminated the study 15 Dec 2016 and application withdrawn (accepted by European Commission 20 Mar 2017). | Posted | Approximately 6 months |
|
|
The Treatment Phase of the study lasted on average approximately 180 days. From Screening until the completion of study termination assessments, subjects participated in the study for approximately 220 days. However, the Sponsor terminated Study DIV-NB-401 on 15 December 2016; all safety data were collected for each subject from screening until the subject's last scheduled visit.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Dinutuximab Administered for 5 Cycles | High-risk neuroblastoma patient treated with Unituxin as standard of care Dinutuximab: Unituxin was administered along with cytokines according to the prescribing information | 0 | 12 | 0 | 12 | 10 | 12 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Adverse drug reaction | General disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Face oedema | General disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Oedema | General disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Allergic cough | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Stridor | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Capillary leak syndrome | Vascular disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Lip swelling | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Mydriasis | Eye disorders | MedDRA (18.0) | Non-systematic Assessment |
|
Institution and/or Principal Investigator agree not to publish or publicly present any interim results of the Study without the prior written consent of Sponsor, not to be unreasonably withheld or delayed. Institution and/or Principal Investigator further agree to provide Sponsor with drafts of any such publication or presentation for review and approval no less than 30 days prior to submission for publication or the date of public presentation.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Odette Jordan, MD, PMP | United Therapeutics Corp. | 919-425-5606 | ojordan@unither.com |
| ID | Term |
|---|---|
| D009447 | Neuroblastoma |
| ID | Term |
|---|---|
| D018241 | Neuroectodermal Tumors, Primitive, Peripheral |
| D018242 | Neuroectodermal Tumors, Primitive |
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009380 | Neoplasms, Nerve Tissue |
Not provided
Not provided
| ID | Term |
|---|---|
| C112746 | dinutuximab |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Single stem cell transplant |
|
| Tandem stem cell transplant |
|
| Radiotherapy treatment |
|
| Other cancer-related treatment |
|
| Partial response (PR) |
|
| Mixed response (MR) |
|
| No response (NR) |
|
| Progressive disease (PD) |
|
|