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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01769001
Registration number
NCT01769001
Ethics application status
Date submitted
31/08/2012
Date registered
5/09/2012
Date last updated
13/09/2023
Titles & IDs
Public title
Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
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Scientific title
A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Months to Adult Fabry Patients
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Secondary ID [1]
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PB-102-F01 & PB-102-F02
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
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0
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Condition category
Condition code
Human Genetics and Inherited Disorders
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0
0
0
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Other human genetics and inherited disorders
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Metabolic and Endocrine
0
0
0
0
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Metabolic disorders
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Metabolic and Endocrine
0
0
0
0
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Other metabolic disorders
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Cancer
0
0
0
0
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Leukaemia - Acute leukaemia
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Cancer
0
0
0
0
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Leukaemia - Chronic leukaemia
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Cancer
0
0
0
0
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Children's - Leukaemia & Lymphoma
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Cancer
0
0
0
0
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Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
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Cancer
0
0
0
0
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Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Cytarabine
Treatment: Drugs - Dasatinib
Treatment: Drugs - Daunorubicin Hydrochloride
Treatment: Drugs - Dexamethasone
Treatment: Drugs - Doxorubicin Hydrochloride
Treatment: Drugs - Etoposide
Treatment: Drugs - Hydrocortisone Sodium Succinate
Other interventions - Laboratory Biomarker Analysis
Treatment: Drugs - Leucovorin Calcium
Treatment: Drugs - Mercaptopurine
Treatment: Drugs - Methotrexate
Treatment: Drugs - Pegaspargase
Treatment: Drugs - Prednisone
Treatment: Other - Radiation Therapy
Treatment: Drugs - Thioguanine
Treatment: Drugs - Vincristine Sulfate
Experimental: 0.2 mg/kg - PRX-102 0.2 mg/kg every 2 weeks
Experimental: 1 mg/kg - PRX-102 1 mg/kg every 2 weeks
Experimental: 2 mg/kg - PRX-102 2 mg/kg every 2 weeks
Treatment: Drugs: Cytarabine
Given IT, IV, or SC
Treatment: Drugs: Dasatinib
Given PO
Treatment: Drugs: Daunorubicin Hydrochloride
Given IV
Treatment: Drugs: Dexamethasone
PO or IV
Treatment: Drugs: Doxorubicin Hydrochloride
Given IV
Treatment: Drugs: Etoposide
Given IV
Treatment: Drugs: Hydrocortisone Sodium Succinate
Given IT
Other interventions: Laboratory Biomarker Analysis
Correlative studies
Treatment: Drugs: Leucovorin Calcium
Given PO or IV
Treatment: Drugs: Mercaptopurine
Given PO
Treatment: Drugs: Methotrexate
Given IT and IV
Treatment: Drugs: Pegaspargase
Given IV
Treatment: Drugs: Prednisone
Given PO or IV
Treatment: Other: Radiation Therapy
Undergo radiation therapy
Treatment: Drugs: Thioguanine
Given PO
Treatment: Drugs: Vincristine Sulfate
Given IV
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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0
Other interventions
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Intervention code [3]
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Treatment: Other
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Adverse Events
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Assessment method [1]
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Reportings of adverse events reported by the patient and from monitoring with clinical laboratory, physical examination and ECG. Results represent the number of AEs that were considered possibly, probably, or definitely related to treatment.
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Timepoint [1]
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12 months
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Primary outcome [2]
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DFS of Non-DS HR Post-induction Patients Receiving Intrathecal (IT) Methotrexate (MTX) Compared With Patients Receiving Intrathecal Triple Therapy (ITT) on a Modified Berlin-Frankfurt-Munster (MBFM) Interim Maintenance High-dose Methotrexate Backbone
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Assessment method [2]
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DFS time is defined as time from randomization end of induction to first event (relapse, second malignant neoplasm, death) or date of last contact for patients who are event-free. Compared using 2-sided log rank test, alpha = 5%. (Completed effective March 19, 2018)
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Timepoint [2]
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At 5 years
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Primary outcome [3]
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DFS of Non-DS VHR Post-Induction Patients Who Receive a Modified MBFM-IMHDM Regimen That Contains a Second IM (Control Arm) Compared to Patients Receive the Cyclophosphamide + Etoposide Containing Regimen (Experimental Arm 1)
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Assessment method [3]
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(Completed effective February 15, 2017) DFS time is defined as time from randomization end of induction to first event (relapse, second malignant neoplasm, death) or date of last contact for patients who are event-free. Compared using a 1-sided log rank test, alpha of 2.5%.
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Timepoint [3]
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0
At 4 years
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Secondary outcome [1]
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Toxicity and Tolerability of Post-induction Age-adjusted ITT Compared to Age-adjusted IT MTX in Children With HR B-ALL
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Assessment method [1]
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(Completed effective March 19, 2018) Percentage of patients who experienced Grade 3 or higher Toxicity Assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0, will be estimated for each randomized arm.
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Timepoint [1]
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Up to 10 years
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Secondary outcome [2]
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Toxicity and Tolerability of Experimental Arm 1 and Control Arm in Patients With VHR B-ALL
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Assessment method [2]
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Percentage of patients who experienced Grade 3 or higher Toxicity Assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0, will be estimated for each randomized arm. (Closed effective February 15, 2017)
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Timepoint [2]
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Up to 10 years
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Secondary outcome [3]
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Induction Mortality in Patients With DS and HR B-ALL Treated With Modified Induction
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Assessment method [3]
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Percentage of deaths in induction will be calculated.
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Timepoint [3]
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At 1 month
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Secondary outcome [4]
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5-year DFS in Patients With Down Syndrome (DS) and HR B-ALL Treated With Modified Induction and Post-Induction Therapy Regimen With MBFM-IMIDM
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Assessment method [4]
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DFS time is defined as time from end of induction to first event (relapse, second malignant neoplasm, death) or date of last contact for patients who are event-free. 5-year DFS will be estimated using the Kaplan-Meier method.
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Timepoint [4]
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At 5 years
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Secondary outcome [5]
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DFS for Children and Young Adults With Ph-like B-ALL and a Predicted Tyrosine Kinase Inhibitor (TKI)-Sensitive Mutation Treated With Dasatinib Plus MBFM-IMHDM
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Assessment method [5]
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DFS time is defined as time from randomization end of induction to first event (relapse, second malignant neoplasm, death) or date of last contact for patients who are event-free. 4-year DFS will be estimated using the Kaplan-Meier method.
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Timepoint [5]
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Up to 4 years
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Secondary outcome [6]
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Toxicity and Tolerability of MBFM-interim Maintenance Intermediate Dose Methotrexate (IMIDM) in Children With Down Syndrome
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Assessment method [6]
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Percentage of patients who experienced Grade 3 or higher Toxicity Assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0, will be estimated.
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Timepoint [6]
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Up to 10 years
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Secondary outcome [7]
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Overall Survival (OS) Rate for HR B-ALL Patients, Overall and by Randomized Arm
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Assessment method [7]
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OS time is defined as time from enrollment to death or date of last contact for patients who are alive. 5-year OS will be estimated for the two randomized arms using the Kaplan-Meier method.
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Timepoint [7]
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At 5 years
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Secondary outcome [8]
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Overall Survival (OS) Rate for VHR B-ALL Patients, Overall and by Randomized Arm.
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Assessment method [8]
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OS of VHR-B-ALL post-Induction patients who receive a modified MBFM-IMHDM regimen that contains a second IM (Control Arm) compared to patients receive the cyclophosphamide + etoposide containing regimen (Experimental Arm 1). OS time is defined as time from enrollment to death or date of last contact for patients who are alive. 4-year OS will be estimated for the two randomized arms using the Kaplan-Meier method.
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Timepoint [8]
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At 4 years
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Secondary outcome [9]
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Incidence of Osteonecrosis (ON) Defined by Magnetic Resonance (MR) Imaging in Children, Adolescents, and Young Adults 10 Years of Age and Greater
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Assessment method [9]
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Incidence of ON among patients who have submitted MRI screening data will be estimated.
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Timepoint [9]
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Up to 10 years
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Secondary outcome [10]
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The Prevalence of Cognitive Deficits Measured by CogState (Domain: Working Memory), in Children (Ages 6 to < 13 Years) With HR- and VHR B-ALL at 1 Year Off Therapy
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Assessment method [10]
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The percentage of patients with abnormal results (Z = -1.5) in working memory measured by CogState will be estimated.
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Timepoint [10]
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Up to 10 years
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Secondary outcome [11]
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The Prevalence of Cognitive Deficits Measured by CogState (Domain: Executive Function), in Children (Ages 6 to < 13 Years) With HR- and VHR B-ALL at 1 Year Off Therapy
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Assessment method [11]
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The percentage of patients with abnormal results (Z = -1.5) in executive function measured by CogState will be estimated.
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Timepoint [11]
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Up to 10 years
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Secondary outcome [12]
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The Prevalence of Cognitive Deficits Measured by CogState (Domain: Visual Learning), in Children (Ages 6 to < 13 Years) With HR- and VHR B-ALL at 1 Year Off Therapy
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Assessment method [12]
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The percentage of patients with abnormal results (Z = -1.5) in visual learning measured by CogState will be estimated.
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Timepoint [12]
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Up to 10 years
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Secondary outcome [13]
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The Prevalence of Cognitive Deficits Measured by CogState (Domain: Processing Speed), in Children (Ages 6 to < 13 Years) With HR- and VHR B-ALL at 1 Year Off Therapy
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Assessment method [13]
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The percentage of patients with abnormal results (Z = -1.5) in processing speed measured by CogState will be estimated.
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Timepoint [13]
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Up to 10 years
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Secondary outcome [14]
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The Prevalence of Cognitive Deficits Measured by CogState (Domain: Visual Attention), in Children (Ages 6 to < 13 Years) With HR- and VHR B-ALL at 1 Year Off Therapy
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Assessment method [14]
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The percentage of patients with abnormal results (Z = -1.5) in visual attention measured by CogState will be estimated.
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Timepoint [14]
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Up to 10 years
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Eligibility
Key inclusion criteria
* Symptomatic adult Fabry patients (=18 yrs)
* Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2 nmol/hr/ml, LLN in leucocytes=32 nmol/hr/mg/protein)
* Females: historical genetic test results consistent with Fabry mutations
* Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit
* Patients who have never received enzyme replacement therapy (ERT) in the past, or patients who have not received ERT in the past 6 months and have a negative anti alpha galactosidase antibody test
* eGFR = 60 mL/min/1.73m2
* The patient signs informed consent
* Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Participation in any trial of an investigational drug within 30 days prior to study screening
* Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7)
* History of dialysis or renal transplantation
* Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
* Severe myocardial fibrosis by MRI (=2 late-enhancement [LE] positive left ventricular segments) (Weidemann et al. 2009)
* History of clinical stroke
* Pregnant or nursing
* Presence of HIV and/or HBsAg and/or Hepatitis C infections
* Known allergies to ERT
* Known allergy to Gadolinium based contrast agents
* Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient's compliance with the requirements of the study
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/10/2012
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
6/03/2016
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Sample size
Target
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Accrual to date
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Final
18
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
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Recruitment hospital [1]
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Royal Melbourne Hospital - Victoria Park
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Recruitment hospital [2]
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John Hunter Children's Hospital - Hunter Regional Mail Centre
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Recruitment hospital [3]
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Royal Brisbane and Women's Hospital - Herston
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Recruitment hospital [4]
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Royal Children's Hospital-Brisbane - Herston
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Recruitment hospital [5]
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Queensland Children's Hospital - South Brisbane
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Recruitment hospital [6]
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Women's and Children's Hospital-Adelaide - North Adelaide
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Recruitment hospital [7]
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Monash Medical Center-Clayton Campus - Clayton
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Recruitment hospital [8]
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Royal Children's Hospital - Parkville
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Recruitment hospital [9]
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Princess Margaret Hospital for Children - Perth
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Recruitment hospital [10]
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Perth Children's Hospital - Perth
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Recruitment postcode(s) [1]
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3050 - Victoria Park
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Recruitment postcode(s) [2]
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2310 - Hunter Regional Mail Centre
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Recruitment postcode(s) [3]
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4029 - Herston
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Recruitment postcode(s) [4]
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4101 - South Brisbane
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Recruitment postcode(s) [5]
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5006 - North Adelaide
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Recruitment postcode(s) [6]
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3168 - Clayton
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Recruitment postcode(s) [7]
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3052 - Parkville
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Recruitment postcode(s) [8]
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6008 - Perth
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Recruitment postcode(s) [9]
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6009 - Perth
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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United States of America
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Georgia
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United States of America
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Iowa
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United States of America
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Kansas
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Maryland
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United States of America
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North Carolina
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United States of America
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Pennsylvania
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United States of America
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Texas
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Virginia
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Paraguay
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Asuncion
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Serbia
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Belgrade
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Spain
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Zaragoza
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United Kingdom
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London
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United States of America
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Alabama
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Alaska
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Arizona
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Arkansas
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Colorado
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Connecticut
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Delaware
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District of Columbia
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Florida
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Hawaii
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Idaho
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Illinois
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Indiana
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Kentucky
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Louisiana
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Maine
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Massachusetts
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Michigan
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Minnesota
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Mississippi
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Missouri
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Nebraska
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Nevada
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New Hampshire
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New Jersey
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New Mexico
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New York
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North Dakota
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Ohio
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Oklahoma
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Oregon
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Rhode Island
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South Carolina
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South Dakota
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Tennessee
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Utah
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Vermont
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Washington
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West Virginia
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Wisconsin
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Canada
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Alberta
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Canada
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British Columbia
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0
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Canada
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Manitoba
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Country [57]
0
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Canada
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0
Newfoundland and Labrador
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Country [58]
0
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Canada
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State/province [58]
0
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Nova Scotia
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Country [59]
0
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Canada
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State/province [59]
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Ontario
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Country [60]
0
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Canada
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0
0
Quebec
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Country [61]
0
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Canada
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0
0
Saskatchewan
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Country [62]
0
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Ireland
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Co Dublin
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Country [63]
0
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New Zealand
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Auckland
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Country [64]
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New Zealand
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State/province [64]
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Christchurch
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0
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Puerto Rico
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State/province [65]
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San Juan
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0
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Switzerland
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Geneva
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Country [67]
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Switzerland
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State/province [67]
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Lausanne
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Protalix
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Commercial sector/industry
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Chiesi Farmaceutici S.p.A.
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Ethics approval
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Summary
Brief summary
This is the first human treatment with PRX-102, an enzyme being developed as a long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (alpha galactosidase deficiency). The safety, tolerability, and exploratory efficacy will be evaluated in this study of increasing doses. Patients will be treated with infusions every two weeks for 12 months.
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Trial website
https://clinicaltrials.gov/study/NCT01769001
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Trial related presentations / publications
Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, Barisoni L, Colvin RB, Jennette CJ, Maegawa G, Boyadjiev SA, Gonzalez D, Nicholls K, Tuffaha A, Atta MG, Rup B, Charney MR, Paz A, Szlaifer M, Alon S, Brill-Almon E, Chertkoff R, Hughes D. Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial. J Inherit Metab Dis. 2019 May;42(3):534-544. doi: 10.1002/jimd.12080. Epub 2019 Apr 8. Salzer WL, Burke MJ, Devidas M, Dai Y, Hardy KK, Kairalla JA, Gore L, Hilden JM, Larsen E, Rabin KR, Zweidler-McKay PA, Borowitz MJ, Wood B, Heerema NA, Carroll AJ, Winick N, Carroll WL, Raetz EA, Loh ML, Hunger SP. Impact of Intrathecal Triple Therapy Versus Intrathecal Methotrexate on Disease-Free Survival for High-Risk B-Lymphoblastic Leukemia: Children's Oncology Group Study AALL1131. J Clin Oncol. 2020 Aug 10;38(23):2628-2638. doi: 10.1200/JCO.19.02892. Epub 2020 Jun 4.
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Public notes
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Contacts
Principal investigator
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Einat Almon, PhD
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Protalix Biotherapeutics
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
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Citations or Other Details
Journal
Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, ...
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Results are available at
https://clinicaltrials.gov/study/NCT01769001
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