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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03847909
Registration number
NCT03847909
Ethics application status
Date submitted
15/02/2019
Date registered
20/02/2019
Date last updated
22/05/2024
Titles & IDs
Public title
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
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Scientific title
A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria
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Secondary ID [1]
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DCR-PHXC-201
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Universal Trial Number (UTN)
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Trial acronym
PHYOX2
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Primary Hyperoxaluria Type 1 (PH1)
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Primary Hyperoxaluria Type 2 (PH2)
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Kidney Diseases
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Urologic Diseases
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Genetic Disease
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Condition category
Condition code
Renal and Urogenital
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0
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Kidney disease
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Renal and Urogenital
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Other renal and urogenital disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - DCR-PHXC
Treatment: Drugs - Sterile Normal Saline (0.9% NaCl)
Experimental: DCR-PHXC - Intervention, drug, DCR-PHXC
Placebo Comparator: Placebo - Sterile Normal Saline (0.9% NaCl) - Placebo, sterile normal saline (0.9% NaCl) for subcutaneous (SC) injection
Treatment: Drugs: DCR-PHXC
Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection
Treatment: Drugs: Sterile Normal Saline (0.9% NaCl)
Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo
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Intervention code [1]
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Treatment: Drugs
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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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AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox
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Assessment method [1]
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The AUC of 24-hour urinary oxalate (Uox) from Day 90 to Day 180, based on percent change from baseline, was compared between the active treatment group and placebo group. A multiple imputation approach was used to handle missing Uox data and then calculate the AUC.
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Timepoint [1]
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From Day 90 to 180
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Secondary outcome [1]
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Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits
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Assessment method [1]
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Percentage of participants whose 24-hour Uox values normalized or near-normalized on at least 2 consecutive visits are presented. Normalization of Uox was defined as less than (<) 0.46 millimole per 24 hours (mmol/24 hours) and near normalization was defined as greater than or equal to (>=) 0.46 to < 0.60 mmol/24 hours (values adjusted per 1.73 square meter [1.73 m^2] body surface area [BSA] in participants aged <18 years).
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Timepoint [1]
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From Day 90 to 180
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Secondary outcome [2]
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Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones
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Assessment method [2]
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Percent change from baseline to Day 180 in the summed surface area measured in millimetre square (mm^2) of kidney stones is presented.
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Timepoint [2]
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Baseline, Day 180
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Secondary outcome [3]
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Percent Change From Baseline to Day 180 in the Number of Kidney Stones
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Assessment method [3]
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Percent change from baseline to Day 180 in the number of kidney stones is presented.
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Timepoint [3]
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Baseline, Day 180
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Secondary outcome [4]
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Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only)
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Assessment method [4]
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Percent change from baseline to Day 180 in plasma oxalate (for adults only) is presented.
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Timepoint [4]
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Baseline, Day 180
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Secondary outcome [5]
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Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180
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Assessment method [5]
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Monthly rate of eGFR change is presented. eGFR was calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) and creatinine-based equation.
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Timepoint [5]
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Baseline, Day 180
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Secondary outcome [6]
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Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs)
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Assessment method [6]
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Number of TEAEs and TESAEs are presented. An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An SAE was defined as any untoward medical occurrence that, at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalisation, results in persistent disability/incapacity or is a congenital anomaly/birth defect. TEAE was defined as any AE with an onset date/time on or after administration (including any partial administration) of the first dose of study intervention and through the study completion date from the end of study case report form (CRF).
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Timepoint [6]
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From Baseline up to Day 180
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Secondary outcome [7]
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Change From Baseline in Electrocardiogram (ECG): Heart Rate
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Assessment method [7]
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Change from baseline in heart rate is presented.
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Timepoint [7]
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Baseline, Day 180
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Secondary outcome [8]
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Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval
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Assessment method [8]
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Change from baseline in PR interval, QRS duration, QT interval, QTcB interval, QTcF interval and RR interval is presented.
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Timepoint [8]
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Baseline, Day 180
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Secondary outcome [9]
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Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
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Assessment method [9]
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Number of participants who had most abnormal post-baseline shift in physical examination are presented. Physical examination shifts were categories into 4 categories: 1) missing; 2) normal; 3) abnormal-not clinically significant (NCS) and 4) abnormal-clinically significant (CS). Each category was presented according body systems including: 1) eyes, ears, nose and throat; 2) chest/respiratory; 3) heart/cardiovascular; 4) gastrointestinal/liver; 5) musculoskeletal/extremities; 6) dermatological/skin; 7) thyroid/neck; 8) lymph nodes; 9) neurological.
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Timepoint [9]
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Baseline up to Day 180
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Secondary outcome [10]
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Change From Baseline in Vital Signs: Height
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Assessment method [10]
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Change from baseline to Day 180 in height is presented.
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Timepoint [10]
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Baseline, Day 180
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Secondary outcome [11]
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Change From Baseline in Vital Signs: Weight
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Assessment method [11]
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Change from baseline to Day 180 in weight is presented.
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Timepoint [11]
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Baseline, Day 180
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Secondary outcome [12]
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Change From Baseline in Vital Signs: Body Mass Index (BMI)
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Assessment method [12]
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Change from baseline to Day 180 in BMI is presented.
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Timepoint [12]
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Baseline, Day 180
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Secondary outcome [13]
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Change From Baseline in Vital Signs: Oral Body Temperature
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Assessment method [13]
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Change from baseline to Day 180 in oral body temperature is presented.
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Timepoint [13]
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Baseline, Day 180
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Secondary outcome [14]
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Change From Baseline in Vital Signs: Heart Rate
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Assessment method [14]
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Change from baseline to Day 180 in heart rate is presented.
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Timepoint [14]
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Baseline, Day 180
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Secondary outcome [15]
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Change From Baseline in Vital Signs: Respiratory Rate
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Assessment method [15]
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Change from baseline to Day 180 in respiratory rate is presented.
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Timepoint [15]
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Baseline, Day 180
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Secondary outcome [16]
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Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure
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Assessment method [16]
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Change from baseline to Day 180 in systolic and diastolic blood pressure is presented.
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Timepoint [16]
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Baseline, Day 180
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Secondary outcome [17]
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Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase
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Assessment method [17]
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Change from baseline to Day 180 in alanine aminotransferase, aspartate aminotransferase, glutamate dehydrogenase, gamma glutamyl transferase, alkaline phosphatase, lactate dehydrogenase and creatine kinase are presented.
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Timepoint [17]
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Baseline, Day 180
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Secondary outcome [18]
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Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine
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Assessment method [18]
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Change from baseline to Day 180 in bilirubin, direct bilirubin and creatinine are presented.
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Timepoint [18]
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Baseline, Day 180
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Secondary outcome [19]
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Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin
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Assessment method [19]
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Change from baseline to Day 180 in protein and albumin are presented.
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Timepoint [19]
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Baseline, Day 180
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Secondary outcome [20]
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Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea
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Assessment method [20]
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Change from baseline to Day 180 in sodium, chloride, potassium and urea are presented.
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Timepoint [20]
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Baseline, Day 180
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Secondary outcome [21]
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Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6
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Assessment method [21]
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Change from baseline to Day 180 in vitamin B6 is presented.
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Timepoint [21]
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Baseline, Day 180
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Secondary outcome [22]
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Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes
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Assessment method [22]
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Change from baseline to Day 180 in erythrocytes is presented.
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Timepoint [22]
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Baseline, Day 180
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Secondary outcome [23]
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Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration
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Assessment method [23]
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Change from baseline to Day 180 in hemoglobin and erythrocytes mean corpuscular hemoglobin concentration are presented.
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Timepoint [23]
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Baseline, Day 180
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Secondary outcome [24]
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Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit
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Assessment method [24]
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Change from baseline to Day 180 in hematocrit is presented.
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Timepoint [24]
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Baseline, Day 180
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Secondary outcome [25]
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Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume
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Assessment method [25]
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Change from baseline to Day 180 in ery. mean corpuscular volume and mean platelet volume are presented.
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Timepoint [25]
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Baseline, Day 180
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Secondary outcome [26]
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Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin
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Assessment method [26]
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Change from baseline to Day 180 in erythrocytes mean corpuscular hemoglobin is presented.
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Timepoint [26]
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Baseline, Day 180
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Secondary outcome [27]
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Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils
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Assessment method [27]
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Change from baseline to Day 180 in reticulocytes, platelets, leukocytes, lymphocytes, monocytes, eosinophils, basophils, neutrophils are presented.
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Timepoint [27]
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Baseline, Day 180
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Secondary outcome [28]
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Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes
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Assessment method [28]
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Change from baseline to Day 180 in lymphocytes/leukocytes is presented.
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Timepoint [28]
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Baseline, Day 180
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Secondary outcome [29]
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Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes
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Assessment method [29]
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Change from baseline to Day 180 in monocytes/leukocytes is presented.
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Timepoint [29]
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Baseline, Day 180
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Secondary outcome [30]
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Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte
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Assessment method [30]
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Change from baseline to Day 180 in eosinophils/leukocytes is presented.
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Timepoint [30]
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Baseline, Day 180
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Secondary outcome [31]
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Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes
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Assessment method [31]
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Change from baseline to Day 180 in basophils/leukocytes is presented.
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Timepoint [31]
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Baseline, Day 180
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Secondary outcome [32]
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Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes
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Assessment method [32]
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Change from baseline to Day 180 in neutrophils/leukocytes is presented.
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Timepoint [32]
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Baseline, Day 180
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Secondary outcome [33]
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Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity
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Assessment method [33]
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Change from baseline to Day 180 in urine specific gravity is presented.
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Timepoint [33]
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Baseline, Day 180
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Secondary outcome [34]
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Change From Baseline in Clinical Urinalysis Laboratory Tests: pH
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Assessment method [34]
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Change from baseline to Day 180 in urine pH is presented.
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Timepoint [34]
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Baseline, Day 180
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Secondary outcome [35]
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Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC
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Assessment method [35]
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The Cmax was defined as the maximum observed plasma concentration during a dosing interval. Data for this endpoint is reported only for adults and adolescent participants from PK population.
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Timepoint [35]
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For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours (hrs) postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose
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Secondary outcome [36]
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Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC
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Assessment method [36]
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AUC0-last was defined as the area under the curve from time of administration to the last measurable concentration. Data for this endpoint is reported only for adults and adolescent participants from PK population.
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Timepoint [36]
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For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose
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Eligibility
Key inclusion criteria
Key
- Capable and willing to provide written informed consent or assent
- Documented diagnosis of PH1 or PH2, confirmed by genotyping
- Must meet the 24 hour urine oxalate excretion requirements
- Less than 20% variation between the two 24-hour urinary creatinine excretion values
derived from the two 24-hour urine collections in the screening period
- Estimated GFR at screening = 30 mL/min normalized to 1.73 m2 BSA
Key
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Minimum age
6
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
- Renal or hepatic transplantation (prior or planned within the study period)
- Currently on dialysis or anticipated requirement for dialysis during the study period
- Plasma oxalate >30 µmol/L
- Documented evidence of clinical manifestations of systemic oxalosis (including
pre-existing retinal, heart, or skin calcifications, or history of severe bone pain,
pathological fractures, or bone deformations)
- Use of an RNA interference (RNAi) drug within the last 6 months
- Participation in any clinical study in which you received an investigational medicinal
product (IMP) within 4 months before Screening
- Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT) and/or
aspartate aminotransferase (AST) >1.5 times upper limit of normal (ULN) for age and
gender
- Inability or unwillingness to comply with study procedures
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled 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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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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
28/10/2019
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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
29/06/2021
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Sample size
Target
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Accrual to date
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Final
35
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Clinical Trial Site - Herston
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Recruitment hospital [2]
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Clinical Trial Site - Parkville
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Recruitment postcode(s) [1]
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4029 - Herston
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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0
United States of America
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State/province [1]
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0
California
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Country [2]
0
0
United States of America
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State/province [2]
0
0
Massachusetts
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Minnesota
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Country [4]
0
0
United States of America
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State/province [4]
0
0
New York
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Country [5]
0
0
United States of America
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State/province [5]
0
0
Pennsylvania
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Country [6]
0
0
Canada
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State/province [6]
0
0
Hamilton
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Country [7]
0
0
France
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State/province [7]
0
0
Bron
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Country [8]
0
0
France
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State/province [8]
0
0
Paris
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Country [9]
0
0
Germany
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State/province [9]
0
0
Bonn
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Country [10]
0
0
Germany
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State/province [10]
0
0
Heidelberg
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Country [11]
0
0
Israel
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State/province [11]
0
0
Jerusalem
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Country [12]
0
0
Italy
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State/province [12]
0
0
Roma
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Country [13]
0
0
Japan
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State/province [13]
0
0
Nagoya
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Country [14]
0
0
Japan
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State/province [14]
0
0
Tochigi
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Country [15]
0
0
Japan
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State/province [15]
0
0
Tokyo
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Country [16]
0
0
Lebanon
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State/province [16]
0
0
Beirut
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Country [17]
0
0
Netherlands
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State/province [17]
0
0
Amsterdam
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Country [18]
0
0
New Zealand
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State/province [18]
0
0
Auckland
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Country [19]
0
0
Poland
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State/province [19]
0
0
Bialystok
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Country [20]
0
0
Romania
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State/province [20]
0
0
Bucharest
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Country [21]
0
0
Spain
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State/province [21]
0
0
Barcelona
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Country [22]
0
0
Spain
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State/province [22]
0
0
Santa Cruz
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Country [23]
0
0
United Kingdom
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State/province [23]
0
0
Birmingham
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Country [24]
0
0
United Kingdom
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State/province [24]
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0
London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Novo Nordisk A/S
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and
Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03847909
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Transparency (dept. 1452)
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Address
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Novo Nordisk A/S
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Country
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0
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Phone
0
0
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Fax
0
0
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Email
0
0
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Contact person for public queries
Name
0
0
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Address
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0
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Country
0
0
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Phone
0
0
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Fax
0
0
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Email
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0
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03847909
Download to PDF