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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03847909




Registration number
NCT03847909
Ethics application status
Date submitted
15/02/2019
Date registered
20/02/2019

Titles & IDs
Public title
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
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
Secondary ID [1] 0 0
DCR-PHXC-201
Universal Trial Number (UTN)
Trial acronym
PHYOX2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Hyperoxaluria Type 1 (PH1) 0 0
Primary Hyperoxaluria Type 2 (PH2) 0 0
Kidney Diseases 0 0
Urologic Diseases 0 0
Genetic Disease 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Kidney disease
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox
Timepoint [1] 0 0
From Day 90 to 180
Secondary outcome [1] 0 0
Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits
Timepoint [1] 0 0
From Day 90 to 180
Secondary outcome [2] 0 0
Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones
Timepoint [2] 0 0
Baseline, Day 180
Secondary outcome [3] 0 0
Percent Change From Baseline to Day 180 in the Number of Kidney Stones
Timepoint [3] 0 0
Baseline, Day 180
Secondary outcome [4] 0 0
Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only)
Timepoint [4] 0 0
Baseline, Day 180
Secondary outcome [5] 0 0
Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180
Timepoint [5] 0 0
Baseline, Day 180
Secondary outcome [6] 0 0
Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs)
Timepoint [6] 0 0
From Baseline up to Day 180
Secondary outcome [7] 0 0
Change From Baseline in Electrocardiogram (ECG): Heart Rate
Timepoint [7] 0 0
Baseline, Day 180
Secondary outcome [8] 0 0
Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval
Timepoint [8] 0 0
Baseline, Day 180
Secondary outcome [9] 0 0
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Timepoint [9] 0 0
Baseline up to Day 180
Secondary outcome [10] 0 0
Change From Baseline in Vital Signs: Height
Timepoint [10] 0 0
Baseline, Day 180
Secondary outcome [11] 0 0
Change From Baseline in Vital Signs: Weight
Timepoint [11] 0 0
Baseline, Day 180
Secondary outcome [12] 0 0
Change From Baseline in Vital Signs: Body Mass Index (BMI)
Timepoint [12] 0 0
Baseline, Day 180
Secondary outcome [13] 0 0
Change From Baseline in Vital Signs: Oral Body Temperature
Timepoint [13] 0 0
Baseline, Day 180
Secondary outcome [14] 0 0
Change From Baseline in Vital Signs: Heart Rate
Timepoint [14] 0 0
Baseline, Day 180
Secondary outcome [15] 0 0
Change From Baseline in Vital Signs: Respiratory Rate
Timepoint [15] 0 0
Baseline, Day 180
Secondary outcome [16] 0 0
Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure
Timepoint [16] 0 0
Baseline, Day 180
Secondary outcome [17] 0 0
Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase
Timepoint [17] 0 0
Baseline, Day 180
Secondary outcome [18] 0 0
Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine
Timepoint [18] 0 0
Baseline, Day 180
Secondary outcome [19] 0 0
Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin
Timepoint [19] 0 0
Baseline, Day 180
Secondary outcome [20] 0 0
Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea
Timepoint [20] 0 0
Baseline, Day 180
Secondary outcome [21] 0 0
Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6
Timepoint [21] 0 0
Baseline, Day 180
Secondary outcome [22] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes
Timepoint [22] 0 0
Baseline, Day 180
Secondary outcome [23] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration
Timepoint [23] 0 0
Baseline, Day 180
Secondary outcome [24] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit
Timepoint [24] 0 0
Baseline, Day 180
Secondary outcome [25] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume
Timepoint [25] 0 0
Baseline, Day 180
Secondary outcome [26] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin
Timepoint [26] 0 0
Baseline, Day 180
Secondary outcome [27] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils
Timepoint [27] 0 0
Baseline, Day 180
Secondary outcome [28] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes
Timepoint [28] 0 0
Baseline, Day 180
Secondary outcome [29] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes
Timepoint [29] 0 0
Baseline, Day 180
Secondary outcome [30] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte
Timepoint [30] 0 0
Baseline, Day 180
Secondary outcome [31] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes
Timepoint [31] 0 0
Baseline, Day 180
Secondary outcome [32] 0 0
Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes
Timepoint [32] 0 0
Baseline, Day 180
Secondary outcome [33] 0 0
Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity
Timepoint [33] 0 0
Baseline, Day 180
Secondary outcome [34] 0 0
Change From Baseline in Clinical Urinalysis Laboratory Tests: pH
Timepoint [34] 0 0
Baseline, Day 180
Secondary outcome [35] 0 0
Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC
Timepoint [35] 0 0
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
Secondary outcome [36] 0 0
Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC
Timepoint [36] 0 0
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

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
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/10/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
29/06/2021
Sample size
Target
Accrual to date
Final
35
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Herston
Recruitment hospital [2] 0 0
Clinical Trial Site - Parkville
Recruitment postcode(s) [1] 0 0
4029 - Herston
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
Minnesota
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
Canada
State/province [6] 0 0
Hamilton
Country [7] 0 0
France
State/province [7] 0 0
Bron
Country [8] 0 0
France
State/province [8] 0 0
Paris
Country [9] 0 0
Germany
State/province [9] 0 0
Bonn
Country [10] 0 0
Germany
State/province [10] 0 0
Heidelberg
Country [11] 0 0
Israel
State/province [11] 0 0
Jerusalem
Country [12] 0 0
Italy
State/province [12] 0 0
Roma
Country [13] 0 0
Japan
State/province [13] 0 0
Nagoya
Country [14] 0 0
Japan
State/province [14] 0 0
Tochigi
Country [15] 0 0
Japan
State/province [15] 0 0
Tokyo
Country [16] 0 0
Lebanon
State/province [16] 0 0
Beirut
Country [17] 0 0
Netherlands
State/province [17] 0 0
Amsterdam
Country [18] 0 0
New Zealand
State/province [18] 0 0
Auckland
Country [19] 0 0
Poland
State/province [19] 0 0
Bialystok
Country [20] 0 0
Romania
State/province [20] 0 0
Bucharest
Country [21] 0 0
Spain
State/province [21] 0 0
Barcelona
Country [22] 0 0
Spain
State/province [22] 0 0
Santa Cruz
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Birmingham
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novo Nordisk A/S
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Transparency (dept. 1452)
Address 0 0
Novo Nordisk A/S
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03847909