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The purpose of this study is to compare the pharmacokinetics (blood levels) and safety of chimeric (ch) 14.18 manufactured by two independent drug makers (United Therapeutics [UTC] or the National Cancer Institute [NCI]).
This is a multi-center, randomized, open-label, two-sequence, cross-over study for eligible subjects with high-risk neuroblastoma to assess the comparability of ch14.18 manufactured with UTC drug product and ch14.18 manufactured with NCI drug product. Subjects will be randomly allocated to receive ch14.18 manufactured by UTC or NCI during Courses 1 and 2 followed by ch14.18 manufactured by other manufacturer (UTC or NCI) during Courses 3, 4, and 5.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sequence 1 | Experimental | UTC ch14.18 for two courses and NCI ch14.18 for three courses |
|
| Sequence 2 | Experimental | NCI ch14.18 for two courses and UTC ch14.18 for three courses |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ch14.18 -NCI | Biological | 25 mg/m^2/day IV for four consecutive days |
|
| Measure | Description | Time Frame |
|---|---|---|
| Area Under the Plasma Concentration Curve (AUC) | Twenty-two PK samples will be obtained at the following timepoints: Courses 1 and 3: Day: 0 Days: 3, 4, 5 and 6: post ch14.18 Days 7:10 to 14 hours post ch14.18 Days 9 to 11: Single sample Days 14 to 17: Single sample Courses 2 and 4: Day 0: Pre-IL-2 Day 7: Pre-ch14.18 Day 10 (Course 4 only): Post ch14.18 End of Treatment: Within 2 weeks post isotretinoin | PK samples obtained during Courses 1 and 3: Days 0, 3, 4, 5, 6, 7, 9, 10, 11, 14, 15, 16, 17; PK samples obtained during Courses 2 and 4: Days 0, 7, 10; End of Treatment |
| Peak Plasma Concentration (Cmax) | Twenty-two PK samples will be obtained at the following timepoints: Courses 1 and 3: Day: 0 Days: 3, 4, 5 and 6: post ch14.18 Days 7:10 to 14 hours post ch14.18 Days 9 to 11: Single sample Days 14 to 17: Single sample Courses 2 and 4: Day 0: Pre-IL-2 Day 7: Pre-ch14.18 Day 10 (Course 4 only): Post ch14.18 End of Treatment: Within 2 weeks post isotretinoin | PK samples obtained during Courses 1 and 3: Days 0, 3, 4, 5, 6, 7, 9, 10, 11, 14, 15, 16, 17; PK samples obtained during Courses 2 and 4: Days 0, 7, 10; End of Treatment |
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Inclusion Criteria:
Diagnosis of high-risk neuroblastoma
8 years of age or younger at diagnosis of high-risk neuroblastoma
Patients must have completed therapy including intensive induction followed by autologous stem cell transplantation (ASCT) and radiotherapy
* Radiotherapy may be waived for patients who either have small adrenal masses which are completely resected up front, or who never have an identifiable primary tumor
Must meet the International Neuroblastoma Response Criteria (INRC) for CR, VGPR, or PR for primary site, soft tissue metastases, and bone metastases AND must also meet the protocol specified criteria for bone marrow response as follows:
* No more than 10% tumor (of total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy
Patient who have no tumor seen on the prior bone marrow, and then have ≤ 10% tumor on any of the bilateral marrow aspirate/biopsy specimens done at pre-ASCT and/or pre-enrollment evaluation will also be eligible
No more than 12 months from starting the first induction chemotherapy after diagnosis to the date of ASCT
* For patients who became high-risk neuroblastoma after initial non-high risk disease, the 12 months period should start from the date of induction therapy for high-risk neuroblastoma to the date of ASCT
No progressive disease at time of registration except for protocol-specified bone marrow response
Adequate hematological, renal, hepatic, pulmonary and cardiac function
CNS toxicity < Grade 2
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Araz Marachelian, MD | Children's Hospital Los Angeles | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | United States | ||
| Children's Healthcare of Atlanta - Egleston |
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| ID | Title | Description |
|---|---|---|
| FG000 | Sequence 1 | UTC ch14.18 for two courses followed by NCI ch14.18 for three courses ch14.18 -NCI: 25 mg/m^2/day IV for four consecutive days ch14.18-UTC: 17.5 mg/m^2/day IV for four consecutive days Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF): GM-CSF will be administered SC at a dose of 250 mcg/m^2/day for 14 days during Courses 1, 3, and 5. Aldesleukin (IL-2): Aldesleukin (IL-2) will be administered IV at a dose of 3 MIU/m^2/day for the first week and at a dose of 4.5 MIU/m^2/day for the second week during Courses 2 and 4. Isotretinoin: Isotretinoin (13-cis-retinoic acid; ISOT) will be administered by mouth over six courses as follows: If weight > 12 kg: 80 mg/m^2/dose twice daily (total daily dose is 160 mg/m^2/day, divided twice daily). If weight ≤ 12 kg: 2.67 mg/kg/dose twice daily (total daily dose is 5.33 mg/kg/day, divided twice daily). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ch14.18-UTC | Biological | 17.5 mg/m^2/day IV for four consecutive days |
|
| Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) | Biological | GM-CSF will be administered SC at a dose of 250 mcg/m^2/day for 14 days during Courses 1, 3, and 5. |
|
| Aldesleukin (IL-2) | Biological | Aldesleukin (IL-2) will be administered IV at a dose of 3 MIU/m^2/day for the first week and at a dose of 4.5 MIU/m^2/day for the second week during Courses 2 and 4. |
|
| Isotretinoin | Drug | Isotretinoin (13-cis-retinoic acid; ISOT) will be administered by mouth over six courses as follows: If weight > 12 kg: 80 mg/m^2/dose twice daily (total daily dose is 160 mg/m^2/day, divided twice daily). If weight ≤ 12 kg: 2.67 mg/kg/dose twice daily (total daily dose is 5.33 mg/kg/day, divided twice daily). |
|
|
| Atlanta |
| Georgia |
| 30322 |
| United States |
| The 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) | Kansas City | Missouri | 64108 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63310 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| FG001 | Sequence 2 | NCI ch14.18 for two courses followed by UTC ch14.18 for three courses ch14.18 -NCI: 25 mg/m^2/day IV for four consecutive days ch14.18-UTC: 17.5 mg/m^2/day IV for four consecutive days Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF): GM-CSF will be administered SC at a dose of 250 mcg/m^2/day for 14 days during Courses 1, 3, and 5. Aldesleukin (IL-2): Aldesleukin (IL-2) will be administered IV at a dose of 3 MIU/m^2/day for the first week and at a dose of 4.5 MIU/m^2/day for the second week during Courses 2 and 4. Isotretinoin: Isotretinoin (13-cis-retinoic acid; ISOT) will be administered by mouth over six courses as follows: If weight > 12 kg: 80 mg/m^2/dose twice daily (total daily dose is 160 mg/m^2/day, divided twice daily). If weight ≤ 12 kg: 2.67 mg/kg/dose twice daily (total daily dose is 5.33 mg/kg/day, divided twice daily). |
| Completed Course 1 |
|
| Completed Course 2 |
|
| Completed Course 3 |
|
| Completed Course 4 |
|
| Completed Course 5 |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Twenty-eight subjects were enrolled in this study with all subjects receiving at least one dose of study therapy.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Sequence 1 | UTC ch14.18 for two courses followed by NCI ch14.18 for three courses ch14.18 -NCI: 25 mg/m^2/day IV for four consecutive days ch14.18-UTC: 17.5 mg/m^2/day IV for four consecutive days Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF): GM-CSF will be administered SC at a dose of 250 mcg/m^2/day for 14 days during Courses 1, 3, and 5. Aldesleukin (IL-2): Aldesleukin (IL-2) will be administered IV at a dose of 3 MIU/m^2/day for the first week and at a dose of 4.5 MIU/m^2/day for the second week during Courses 2 and 4. Isotretinoin: Isotretinoin (13-cis-retinoic acid; ISOT) will be administered by mouth over six courses as follows: If weight > 12 kg: 80 mg/m^2/dose twice daily (total daily dose is 160 mg/m^2/day, divided twice daily). If weight ≤ 12 kg: 2.67 mg/kg/dose twice daily (total daily dose is 5.33 mg/kg/day, divided twice daily). |
| BG001 | Sequence 2 | NCI ch14.18 for two courses followed by UTC ch14.18 for three courses ch14.18 -NCI: 25 mg/m^2/day IV for four consecutive days ch14.18-UTC: 17.5 mg/m^2/day IV for four consecutive days Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF): GM-CSF will be administered SC at a dose of 250 mcg/m^2/day for 14 days during Courses 1, 3, and 5. Aldesleukin (IL-2): Aldesleukin (IL-2) will be administered IV at a dose of 3 MIU/m^2/day for the first week and at a dose of 4.5 MIU/m^2/day for the second week during Courses 2 and 4. Isotretinoin: Isotretinoin (13-cis-retinoic acid; ISOT) will be administered by mouth over six courses as follows: If weight > 12 kg: 80 mg/m^2/dose twice daily (total daily dose is 160 mg/m^2/day, divided twice daily). If weight ≤ 12 kg: 2.67 mg/kg/dose twice daily (total daily dose is 5.33 mg/kg/day, divided twice daily). |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Full Range | 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 |
| ||||||||||||||||
| Pre-ASCT Response | At pre-ASCT evaluation, subjects must have met the International Neuroblastoma Response Criteria (INRC) for complete response (CR), very good partial response (VGPR) or partial response (PR) for primary site, soft tissue metastases, and bone metastases. Subjects who met those criteria were also required to meet the protocol specified criteria for bone marrow response. | Number | 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 | Area Under the Plasma Concentration Curve (AUC) | Twenty-two PK samples will be obtained at the following timepoints: Courses 1 and 3: Day: 0 Days: 3, 4, 5 and 6: post ch14.18 Days 7:10 to 14 hours post ch14.18 Days 9 to 11: Single sample Days 14 to 17: Single sample Courses 2 and 4: Day 0: Pre-IL-2 Day 7: Pre-ch14.18 Day 10 (Course 4 only): Post ch14.18 End of Treatment: Within 2 weeks post isotretinoin | Posted | Mean | Standard Deviation | mcg*hr/mL | PK samples obtained during Courses 1 and 3: Days 0, 3, 4, 5, 6, 7, 9, 10, 11, 14, 15, 16, 17; PK samples obtained during Courses 2 and 4: Days 0, 7, 10; End of Treatment |
|
|
| |||||||||||||||||||||||||||||
| Primary | Peak Plasma Concentration (Cmax) | Twenty-two PK samples will be obtained at the following timepoints: Courses 1 and 3: Day: 0 Days: 3, 4, 5 and 6: post ch14.18 Days 7:10 to 14 hours post ch14.18 Days 9 to 11: Single sample Days 14 to 17: Single sample Courses 2 and 4: Day 0: Pre-IL-2 Day 7: Pre-ch14.18 Day 10 (Course 4 only): Post ch14.18 End of Treatment: Within 2 weeks post isotretinoin | Posted | Mean | Standard Deviation | ng/mL | PK samples obtained during Courses 1 and 3: Days 0, 3, 4, 5, 6, 7, 9, 10, 11, 14, 15, 16, 17; PK samples obtained during Courses 2 and 4: Days 0, 7, 10; End of Treatment |
|
|
Adverse events (AEs) and serious adverse events (SAEs) were reported at the time of informed consent through 30 days after the last dose of study therapy.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | UTC ch14.18 | United Therapeutics Manufactured ch14.18 | 17 | 27 | 27 | 27 | ||
| EG001 | NCI ch14.18 | NCI Manufactured ch14.18 | 12 | 27 | 27 | 27 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Device Related Infection | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Gastroenteritis Viral | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Alpha Hemolytic Stretococcal Infection | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Bacteremia | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Bacterial Infection | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Enterobacter Sepsis | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Pneumonia Bacterial | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Streptococcal Bacteremia | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Disease Progression | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Malaise | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypokalemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypercalcemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypoalbuminemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hyponatremia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypophosphatemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Anemia | Blood and lymphatic system disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Anaphylactic Reaction | Immune system disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Serum Sickness | Immune system disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Mydriasis | Eye disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hyperbilirubinemia | Hepatobiliary disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Blood Creatinine Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Convulsion | Nervous system disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Renal Failure Acute | Renal and urinary disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Herpes Simplex | Skin and subcutaneous tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Capillary Leak Syndrome | Vascular disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (17.0) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Platelet Count Decreased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Alanine Aminotransferase Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Neutrophil Count Decreased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Lymphocyte Count Decreased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| White Blood Cell Count Decreased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Aspartate Aminotransferase Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Urine Output Decreased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypokalemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hyponatremia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypocalcemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hyperglycemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypophosphatemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypoalbuminemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Device Related Infection | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Gastroenteritis Viral | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Anemia | Blood and lymphatic system disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Pain in Extremity | Musculoskeletal and connective tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Cheilitis | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Abdominal Distension | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Lip Dry | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Lip Swelling | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Oropharyngeal Pain | Gastrointestinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Face Odema | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Malaise | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Chills | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Edema | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Edema Peripheral | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Injection Site Reaction | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Generalized Edema | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Localized Edema | General disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Blood Creatinine Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Blood Alkaline Phosphatase Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Blood Bilirubin Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| International Normalized Ratio Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Weight Decreased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Weight Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Activated Partial Thromboplastin Time Prolonged | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Electrocardiogram QT Prolonged | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Blood Chloride Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Blood Triglycerides Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Eosinophil Count Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Gamma-Glutamyltransferase Increased | Investigations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypertriglyceridemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Decreased Appetite | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypercalcemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypoglycemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypomagnesemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hyperkalemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Fluid Retention | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hyperphosphatemia | Metabolism and nutrition disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Dry Skin | Skin and subcutaneous tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Rash Maculo-Papular | Skin and subcutaneous tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Rhinorrea | Respiratory, thoracic and mediastinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Tachypnea | Respiratory, thoracic and mediastinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Upper Respiratory Tract Infection | Respiratory, thoracic and mediastinal disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Capillary Leak Syndrome | Vascular disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Sinus Tachycardia | Cardiac disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Clostridium Difficile Infection | Infections and infestations | MedDRA (17.0) | Non-systematic Assessment |
| |
| Urinary Retention | Renal and urinary disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hematuria | Renal and urinary disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Neck Pain | Musculoskeletal and connective tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Musculoskeletal Pain | Musculoskeletal and connective tissue disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Abnormal Behavior | Psychiatric disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Restlessness | Psychiatric disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Periorbital Edema | Eye disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Photophobia | Eye disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Vision Blurred | Eye disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hyperbilirubinemia | Hepatobiliary disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypoalbuminemia | Hepatobiliary disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA (17.0) | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (17.0) | Non-systematic Assessment |
| |
| Hypothyroidism | Endocrine disorders | MedDRA (17.0) | 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, except as provided below. 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 | |
|---|---|---|---|---|
| Allison Lim | United Therapeutics Corporation | 919-425-8799 | alim@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 |
| D016178 | Granulocyte-Macrophage Colony-Stimulating Factor |
| C082598 | aldesleukin |
| D007376 | Interleukin-2 |
| D015474 | Isotretinoin |
| ID | Term |
|---|---|
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
| D007378 | Interleukins |
| D008222 | Lymphokines |
| D012176 | Retinoids |
| D002338 | Carotenoids |
| D011090 | Polyenes |
| D000475 | Alkenes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D053138 | Cyclohexenes |
| D003510 | Cyclohexanes |
| D003516 | Cycloparaffins |
| D006840 | Hydrocarbons, Alicyclic |
| D006844 | Hydrocarbons, Cyclic |
| D013729 | Terpenes |
| D010860 | Pigments, Biological |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Very Good Partial Response (VGPR) |
|
| Partial Response (PR) |
|
|