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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03847909
Ethics application status
Date submitted
15/02/2019
Date registered
20/02/2019
Date last updated
19/07/2019

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
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Renal and Urogenital 0 0 0 0
Other renal and urogenital 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
Time-weighted standardized (TWS) area under the curve (AUC) of percent change from baseline in 24-hour urinary oxalate excretion between Day 90 and Day 180 - Four measurements of percent change from baseline in 24-hour urinary oxalate are combined to determine a single AUC value
Timepoint [1] 0 0
3 months (Last 3 months of the 6 month treatment period)
Secondary outcome [1] 0 0
TWS AUC of percent change from baseline in 24-hour urinary oxalate-to-creatinine ratio (value/upper limit of normal [ULN]) between Day 90 and Day 180 - Four measurements of percent change from baseline in 24-hour urinary oxalate-to-creatinine ratio are combined to determine a single AUC value
Timepoint [1] 0 0
3 months (Last 3 months of the 6 month treatment period)
Secondary outcome [2] 0 0
Proportion of participants with a 24-hour Uox level < 0.46 mmol/24 hours or = 0.46 - < 0.60 mmol/24 hours at at least two consecutive study visits commencing at Day 90 and ending at Day 180
Timepoint [2] 0 0
At least 2 months starting at Day 90 and through Day 180
Secondary outcome [3] 0 0
Proportion of participants with 24-hour Uox levels in each of 4 quartile ranges (< 1.1 mmol, 1.1 to < 1.6 mmol, 1.6 to = 2.4 mmol, and > 2.4 mmol/24 hours from baseline to Day 180
Timepoint [3] 0 0
7 months
Secondary outcome [4] 0 0
TWS AUC of percent change from baseline in 24-hour urinary oxalate-to-creatinine ratio (value/upper limit of normal [ULN]) between baseline and Day 180
Timepoint [4] 0 0
7 months
Secondary outcome [5] 0 0
TWS AUC of percent change from baseline in plasma oxalate - Four measurements of percent change from baseline in plasma oxalate are combined to determine a single AUC value
Timepoint [5] 0 0
3 months (Last 3 months of the 6 month treatment period)
Secondary outcome [6] 0 0
Frequency of Adverse events (AE) from baseline to study completion
Timepoint [6] 0 0
7 months
Secondary outcome [7] 0 0
Frequency of serious adverse events (SAE) from baseline to study completion
Timepoint [7] 0 0
7 months

Eligibility
Key inclusion criteria
Key

- Willing to provide written informed consent or assent

- Confirmation of PH1 and PH2 disease

- Must meet the 24 hour urine oxalate excretion requirements

- Estimated GFR at screening = 30 mL/min normalized to 1.73 m2 BSA

Key
Minimum age
6 Years
Maximum age
No limit
Gender
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

- Documented evidence of clinical manifestations of systemic oxalosis

- 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

- 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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Dicerna Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

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)
Trial website
https://clinicaltrials.gov/show/NCT03847909
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Amanda Gentile
Address 0 0
Country 0 0
Phone 0 0
(617) 621-8097
Fax 0 0
Email 0 0
agentile@dicerna.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03847909