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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04042402




Registration number
NCT04042402
Ethics application status
Date submitted
10/07/2019
Date registered
2/08/2019

Titles & IDs
Public title
Long Term Extension Study in Patients With Primary Hyperoxaluria
Scientific title
An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria
Secondary ID [1] 0 0
DCR-PHXC-301
Universal Trial Number (UTN)
Trial acronym
PHYOX3
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
Primary Hyperoxaluria Type 3 (PH3) 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

Experimental: Open Label - Open label, monthly subcutaneous injection


Treatment: Drugs: DCR-PHXC
Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The annual rate of decline in eGFR in participants with PH1
Timepoint [1] 0 0
Annual change from baseline
Secondary outcome [1] 0 0
The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal 12 lead electrocardiogram (ECG) readings
Timepoint [1] 0 0
TEAEs and SAEs are evaluated monthly for 6 years
Secondary outcome [2] 0 0
The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal physical examination findings
Timepoint [2] 0 0
TEAEs and SAEs are evaluated monthly for 6 years
Secondary outcome [3] 0 0
The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal vital signs
Timepoint [3] 0 0
TEAEs and SAEs are evaluated monthly for 6 years
Secondary outcome [4] 0 0
The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs related to abnormal clinical laboratory tests (hematology, chemistry, coagulation parameters, and urinalysis)
Timepoint [4] 0 0
TEAEs and SAEs are evaluated monthly for 6 years
Secondary outcome [5] 0 0
To identify the proportion of participants with normalized or near-normalized 24 hour urinary oxalate (Uox)
Timepoint [5] 0 0
24 hour urine collections (if applicable) are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.
Secondary outcome [6] 0 0
To identify the percentage of participants with spot urinary oxalate-to-creatinine ratio = the ULN or = 1.5 x ULN
Timepoint [6] 0 0
Spot urine collections are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.
Secondary outcome [7] 0 0
To assess the effect of DCR-PHXC on stone events in patients with PH
Timepoint [7] 0 0
Evaluated yearly for 6 years
Secondary outcome [8] 0 0
To assess the effect of DCR-PHXC on stone burden grade in patients with PH
Timepoint [8] 0 0
Evaluated yearly for 6 years
Secondary outcome [9] 0 0
To assess the effect of DCR-PHXC in nephrocalcinosis grade in patients with PH
Timepoint [9] 0 0
Evaluated yearly for 6 years
Secondary outcome [10] 0 0
To evaluate the incidence of chronic kidney disease (CKD) and end-stage renal disease (ESRD) in participants with PH
Timepoint [10] 0 0
eGFR is evaluated monthly for 6 months (or quarterly for multidose rollovers), quarterly for 2 1/2 years, and every 6 months for 3 years after that.
Secondary outcome [11] 0 0
Change from Baseline in the Short Form (36) Health Survey (SF-36®) in PH1, PH2, and PH3 participant subgroups
Timepoint [11] 0 0
Surveys are administered at screening, Day 180, yearly for 3.5 years, then at Month 72 (EOS).
Secondary outcome [12] 0 0
Change from Baseline in the EQ-5D-5L™ in adults in PH1, PH2, and PH3 participant subgroups
Timepoint [12] 0 0
Surveys are administered at screening, Day 180, yearly for 3.5 years, then at Month 72 (EOS).
Secondary outcome [13] 0 0
Change from Baseline in the Pediatric Quality of Life Inventory (PedsQL™) in children in PH1, PH2, and PH3 participant subgroups
Timepoint [13] 0 0
Surveys are administered at screening, Day 180, yearly for 3.5 years, then at Month 72 (EOS).
Secondary outcome [14] 0 0
To assess the efficacy of DCR PHXC in reducing Uox burden in patients with PH: TWS AUC
Timepoint [14] 0 0
Monthly for 4 months (D90 through D180)
Secondary outcome [15] 0 0
To assess the long-term efficacy of DCR PHXC in reducing Uox burden in patients with PH
Timepoint [15] 0 0
24 hour urine collections (if applicable) are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.
Secondary outcome [16] 0 0
To assess the long-term efficacy of DCR-PHXC in reducing Uox burden in patients with PH
Timepoint [16] 0 0
Spot urine collections are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.

Eligibility
Key inclusion criteria
Key

•Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC.

OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. Siblings must be younger than 18 years of age and must have genetically confirmed PH.

* For participants rolling over from a multidose study of DCR-PHXC, enrollment should occur within a window of 25 to 75 days from the last dose of study intervention.
* Estimated GFR at screening = 30 mL/min normalized to 1.73 m2 body surface area (BSA), calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) formula in participants aged = 18 years, or the multivariate equation by Schwartz in participants aged 12 months to 17 years. In Japan, the cystatin C-based Uemura formula will be used for participants aged 12 months to <2 years, the creatinine-based Uemura formula by will be used for participants aged 2 to 17 years, and the equation by Matsuo will be used in participants aged = 18 years.

Key
Minimum age
No limit
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)
* Plasma oxalate > 30 µmol/L
* Currently on dialysis
* Documented evidence of clinical manifestations of systemic oxalosis

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/07/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2030
Actual
Sample size
Target
75
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Clinical Research Site - Herston
Recruitment hospital [2] 0 0
Clinical Trial Site - Melbourne
Recruitment postcode(s) [1] 0 0
4029 - Herston
Recruitment postcode(s) [2] 0 0
3052 - Melbourne
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
Canada
State/province [5] 0 0
Ontario
Country [6] 0 0
France
State/province [6] 0 0
Bron
Country [7] 0 0
France
State/province [7] 0 0
Paris
Country [8] 0 0
Germany
State/province [8] 0 0
Bonn
Country [9] 0 0
Germany
State/province [9] 0 0
Heidelberg
Country [10] 0 0
Italy
State/province [10] 0 0
Roma
Country [11] 0 0
Japan
State/province [11] 0 0
Fukuoka
Country [12] 0 0
Japan
State/province [12] 0 0
Nagoya
Country [13] 0 0
Japan
State/province [13] 0 0
Tokyo
Country [14] 0 0
Lebanon
State/province [14] 0 0
Beirut
Country [15] 0 0
Netherlands
State/province [15] 0 0
Amsterdam
Country [16] 0 0
Norway
State/province [16] 0 0
Tromsø
Country [17] 0 0
Spain
State/province [17] 0 0
Barcelona
Country [18] 0 0
Turkey
State/province [18] 0 0
Ankara
Country [19] 0 0
United Kingdom
State/province [19] 0 0
London
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Birmingham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Verity Rawson, MB.CHB
Address 0 0
Dicerna, A Novo Nordisk Company
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?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04042402