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20010133 is an open-label, dose escalation study in pediatric patients with acute leukemias receiving myelotoxic therapy (high dose etoposide, cyclophosphamide and total body irradiation [TBI]) followed by hematopoietic stem cell transplant (HSCT). The study will evaluate the safety and pharmacokinetics of palifermin in pediatric patients. Three doses (40 μg/kg/day, 60 μg/kg/day, and 80 μg/kg/day) are to be evaluated in each age group (1 to 2, 3 to 11, and 12 to 16 years, respectively) using a conventional dose escalation design. Palifermin is administered for 3 consecutive days (Day -10 to Day -8, respectively) before the start of the conditioning regimen and for 3 consecutive days (Day 0 to Day +2) following HSCT. Patients will be enrolled simultaneously to each age group to identify a safe, well tolerated, efficacious dose in each age group. Patients will also be followed for secondary malignancies, progression-free survival (PFS) and overall survival (OS)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Palifermin Dose Escalation | Experimental | A 3 dose escalation design. Sucessive cohorts of patient (9 patients per group) will each be administered Palifermin as an IV bolus injection (40, 60 or 80 µg) once daily for 3 consecutive days before the start of conditioning regimen (chemotherapy and total body irradiation) and after HCST (Day -10, -9, -8 and Day 0, +1, +2 respectively). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Palifermin | Drug | Palifermin will be administered as an IV bolus injection (40, 60 or 80 µg/kg/day)once daily for 3 consecutive days before the start of conditioning regimen and after HCST (Day -10, -9, -8 and Day 0, +1, +2 respectively). |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Dose Limiting Toxicities (DLTs) | A DLT is appearance of side effects during treatment severe enough to prevent further increase in dosage or strength of treatment agent, or to prevent continuation of treatment at any dosage level. A DLT was defined as: Grade 3 or 4 AE [based on Cancer Therapy Evaluation Program Common Terminology Criteria for Adverse Events (CTCAE) v3.0] considered by the investigator to be related to palifermin with the exceptions: Grade 3 erythema, pruritus or rash that resolves within 7 days of the last dose of palifermin. The percentage of particiapnts with a DLT during the study was assessed. | Approximately 1 month duration (Day -10 through Day +16) |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Serum Palifermin Antibody Formation | The percentage of participants developing palifermin antibodies during the study was assessed. | Approximately 4 month duration (Through Day + 100 (+/- 40 days)) |
| Incidence of Severe Adverse Events (AEs) |
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Inclusion Criteria:
Acute lymphoblastic leukemia (ALL) or acute myeloid leukemia (AML) requiring HSCT
Age ≥ 1 and ≤ 16 years at screening
Lansky performance status > 60%
Candidate for allogeneic HSCT protocol:
Identification of an HLA-compatible donor per institutional standards
Assent from a minor (if the child is capable of giving assent) per Department of Health and Human Services (DHHS) guidelines listed in 21CFR 50.55 and local Institutional Review Board (IRB) standards.
Serum amylase and lipase: ≤ 1.2 x IULN
Negative serum/urine pregnancy test for females with childbearing potential within 4 days before administration of the first palifermin dose
Agreement by males and females of reproductive potential to use an effective means of contraception 30 days prior to enrollment through Day +30 (end of treatment)
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Maarten de Chateau, MD, PhD | Swedish Orphan Biovitrum | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arizona Cancer Center | Tucson | Arizona | United States | |||
| Loma Linda University |
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This phase 1 dose-escalation study to evaluate the safety and pharmacokinetics (PK) of palifermin in pediatric subjects with acute leukemias undergoing myeloblative therapy and allogeneic hematopoietic stem cell transplant (HSCT) was performed in 7 centers in USA between 2006 and 2011.
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| ID | Title | Description |
|---|---|---|
| FG000 | Palifermin 40 µg/kg/Day | Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts |
| FG001 | Palifermin 60 µg/kg/Day |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
| Total Body irradiation | Radiation |
|
| Chemotherapy | Drug | High dose etoposide, Cyclophosphamide |
|
The percentage of participants with a severe AE during the study was assessed. |
| Approximately 1 1/2 months duration (Through Day +30/End of Treatment) |
| Incidence of Laboratory Abnormalities | The percentage of participants with a laboratory value outside the normal ranges during the study. | Approximately 1 1/2 months duration (Through Day +30/End of Treatment) |
| Pharmacokinetics of Palifermin, Clearence (CL) After the 1st Intravenous (IV) Bolus Injection for Multiple Dose Levels | Clearence was estimated as dose divided by the area under serum concentration-time curve from time zero to infinity where the dose was given in amount palifermin actually administered. | Day -10 |
| Pharmacokinetics of Palifermin, Volume of Distribution at Steady State (Vss) After the 1st IV Bolus Injection for Multiple Dose Levels | Day -10 |
| Pharmacokinetics of Palifermin, Terminal Half-life (t½,z) After the 1st IV Bolus Injection for Multiple Dose Levels | The terminal half-life was calculated as ln(2)/lambda,z where lambda,z was estimated using at least three quantifiable serum concentrations of the terminal log-linear phase. | Day -10 |
| Pharmacokinetics of Palifermin, t½,z After the 3rd IV Bolus Injection for Multiple Dose Levels | The terminal half-life was calculated as ln(2)/lambda,z where lambda,z was estimated using at least three quantifiable serum concentrations of the terminal log-linear phase. | Day -8 |
| Pharmacokinetics of Palifermin, Area Under the Concentration Time Curve From Zero to the End of the Dosing Interval (AUCtau) After the 1st IV Bolus Injection for Multiple Dose Levels | The AUC was estimated using the linear/log trapezoidal method for AUC0-tau from time zero to the end of the dosing interval (24 hours (hrs) post-dose) Data collected at time points: 0, 2 minutes (min), 15 min, 30 min, 60 min, 2 hrs, 4 hrs, 6, hrs, 10, hrs and 24 hrs post-dose. | Day -10 |
| Pharmacokinetics of Palifermin, AUCtau After the 3rd IV Bolus Injection for Multiple Dose Levels | The AUC was estimated using the linear/log trapezoidal method for AUC0-tau from time zero to the end of the dosing interval (24 hours (hrs) post-dose) Data collected at time points: 0, 2 minutes (min), 15 min, 30 min, 60 min, 2 hrs, 4 hrs, 6, hrs, 10, hrs and 24 hrs post-dose. | Day -8 |
| Long-Term Follow-Up: Incidence of Secondary Malignancies | Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually) |
| Long-Term Follow-Up: Progression Free Survival | Progression free survival (PFS) was defined as the number of days between the date of first investigational product administration and the date when physical or radiological evidence of disease progression is determined or death (regardless of cause) | Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually) |
| Long-Term Follow-Up: Overall Survival | Overall survival was defined as the number of days from the date of first investigational product administration to the date of death (regardless of cause) | Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually) |
| Loma Linda |
| California |
| United States |
| Children´s Hospital | Los Angeles | California | United States |
| Regents of University of California | Los Angeles | California | United States |
| Children´s Hospital of Orange | Orange | California | United States |
| Children´s Memorial | Chicago | Illinois | United States |
| University of Texas | Dallas | Texas | United States |
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
| FG002 | Palifermin 80 µg/kg/Day | Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts |
| COMPLETED |
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| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Palifermin 40 µg/kg/Day | Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts |
| BG001 | Palifermin 60 µg/kg/Day | Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts |
| BG002 | Palifermin 80 µg/kg/Day | Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts |
| BG003 | 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, Categorical | Count of Participants | Participants |
| ||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | 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 | Incidence of Dose Limiting Toxicities (DLTs) | A DLT is appearance of side effects during treatment severe enough to prevent further increase in dosage or strength of treatment agent, or to prevent continuation of treatment at any dosage level. A DLT was defined as: Grade 3 or 4 AE [based on Cancer Therapy Evaluation Program Common Terminology Criteria for Adverse Events (CTCAE) v3.0] considered by the investigator to be related to palifermin with the exceptions: Grade 3 erythema, pruritus or rash that resolves within 7 days of the last dose of palifermin. The percentage of particiapnts with a DLT during the study was assessed. | Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin. | Posted | Number | percentage of participants | Approximately 1 month duration (Day -10 through Day +16) |
|
|
| ||||||||||||||||||||||||||||||||
| Secondary | Incidence of Serum Palifermin Antibody Formation | The percentage of participants developing palifermin antibodies during the study was assessed. | Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin. | Posted | Number | percentage of participants | Approximately 4 month duration (Through Day + 100 (+/- 40 days)) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Incidence of Severe Adverse Events (AEs) | The percentage of participants with a severe AE during the study was assessed. | Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin. | Posted | Number | percentage of participants | Approximately 1 1/2 months duration (Through Day +30/End of Treatment) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Incidence of Laboratory Abnormalities | The percentage of participants with a laboratory value outside the normal ranges during the study. | Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin. | Posted | Number | percentage of participants | Approximately 1 1/2 months duration (Through Day +30/End of Treatment) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetics of Palifermin, Clearence (CL) After the 1st Intravenous (IV) Bolus Injection for Multiple Dose Levels | Clearence was estimated as dose divided by the area under serum concentration-time curve from time zero to infinity where the dose was given in amount palifermin actually administered. | Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables. | Posted | Median | Full Range | mL/hr/kg | Day -10 |
| |||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetics of Palifermin, Volume of Distribution at Steady State (Vss) After the 1st IV Bolus Injection for Multiple Dose Levels | Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables. | Posted | Median | Full Range | mL/kg | Day -10 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetics of Palifermin, Terminal Half-life (t½,z) After the 1st IV Bolus Injection for Multiple Dose Levels | The terminal half-life was calculated as ln(2)/lambda,z where lambda,z was estimated using at least three quantifiable serum concentrations of the terminal log-linear phase. | Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables. | Posted | Median | Full Range | hour | Day -10 |
| |||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetics of Palifermin, t½,z After the 3rd IV Bolus Injection for Multiple Dose Levels | The terminal half-life was calculated as ln(2)/lambda,z where lambda,z was estimated using at least three quantifiable serum concentrations of the terminal log-linear phase. | Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables. | Posted | Median | Full Range | hour | Day -8 |
| |||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetics of Palifermin, Area Under the Concentration Time Curve From Zero to the End of the Dosing Interval (AUCtau) After the 1st IV Bolus Injection for Multiple Dose Levels | The AUC was estimated using the linear/log trapezoidal method for AUC0-tau from time zero to the end of the dosing interval (24 hours (hrs) post-dose) Data collected at time points: 0, 2 minutes (min), 15 min, 30 min, 60 min, 2 hrs, 4 hrs, 6, hrs, 10, hrs and 24 hrs post-dose. | Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables. | Posted | Median | Full Range | ng*hr/mL | Day -10 |
| |||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetics of Palifermin, AUCtau After the 3rd IV Bolus Injection for Multiple Dose Levels | The AUC was estimated using the linear/log trapezoidal method for AUC0-tau from time zero to the end of the dosing interval (24 hours (hrs) post-dose) Data collected at time points: 0, 2 minutes (min), 15 min, 30 min, 60 min, 2 hrs, 4 hrs, 6, hrs, 10, hrs and 24 hrs post-dose. | Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables. | Posted | Median | Full Range | ng*hr/mL | Day -8 |
| |||||||||||||||||||||||||||||||||
| Secondary | Long-Term Follow-Up: Incidence of Secondary Malignancies | Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin. | Posted | Number | percentage of participants | Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually) |
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Long-Term Follow-Up: Progression Free Survival | Progression free survival (PFS) was defined as the number of days between the date of first investigational product administration and the date when physical or radiological evidence of disease progression is determined or death (regardless of cause) | Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin. | Posted | Median | Full Range | months | Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually) |
| |||||||||||||||||||||||||||||||||
| Secondary | Long-Term Follow-Up: Overall Survival | Overall survival was defined as the number of days from the date of first investigational product administration to the date of death (regardless of cause) | Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin. | Posted | Median | Full Range | months | Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually) |
|
Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
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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 | All Subjects | All subjects from each age group (1-2, 3-11, 12-16 years) | 5 | 27 | 27 | 27 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Enterococcal infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Septic chock | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Urine output decreased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Venoocclusive disease | Vascular disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Convulsion | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Ascites | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Gingival hyperplasia | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Lip dry | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Oesophagitis | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Oral pain | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Perianal erythema | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Proctalgia | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Catheter related complication | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Catheter site pain | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Chills | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Face oedema | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Fatigue | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Irritability | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Oedema | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Pain | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Hepatomegaly | Hepatobiliary disorders | MedDRA (12.0) | Systematic Assessment |
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| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA (12.0) | Systematic Assessment |
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| Acute graft versus host disease in skin | Immune system disorders | MedDRA (12.0) | Systematic Assessment |
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| Alpha haemolytic streptococcal infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Bacteraemia | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Clostridium difficile colitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Enterococcal sepsis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Fungal infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Parotitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Urinary tract infection enterococcal | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Excoriation | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Blood bilirubin increased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Occult blood positive | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Weight decreased | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Weight increased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Anorexia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Fluid retention | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Hyponatraemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| bone pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Renal tubular acidosis | Renal and urinary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Scrotal erythema | Reproductive system and breast disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Testicular pain | Reproductive system and breast disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Skin disorder | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (12.0) | Systematic Assessment |
|
The original protocol included a long term follow-up (LTFU) phase up to 10 years. This was reduced in a protocol amendment to up to the time when the last enrolled subject had completed the day +100 follow-up.
The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits sponsor a limited period of time to review material discussing trial results (approximately up to 90 days). Sponsor may remove confidential information, but authors have final control and approval of publication content. For multi-center studies, investigator agrees not to publish any results before the first multi-center publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Program Director | Swedish Orphan Biovitrum | +46 8 697 20 00 | clinical@sobi.com |
| ID | Term |
|---|---|
| D007938 | Leukemia |
| D013280 | Stomatitis |
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D015470 | Leukemia, Myeloid, Acute |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009059 | Mouth Diseases |
| D009057 | Stomatognathic Diseases |
| D007945 | Leukemia, Lymphoid |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D007951 | Leukemia, Myeloid |
Not provided
Not provided
| ID | Term |
|---|---|
| D051523 | Fibroblast Growth Factor 7 |
| D014916 | Whole-Body Irradiation |
| D004358 | Drug Therapy |
| ID | Term |
|---|---|
| D005346 | Fibroblast Growth Factors |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
| D011878 | Radiotherapy |
| D013812 | Therapeutics |
| D008919 | Investigative Techniques |
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
|
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|
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| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
|---|
| Participants |
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| Units | Counts |
|---|---|
| Participants |
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All subjects from each age group (1-2, 3-11, 12-16 years) |
|
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All subjects from each age group (1-2, 3-11, 12-16 years)
|
|