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A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 (PHYOX2)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03847909
Recruitment Status : Recruiting
First Posted : February 20, 2019
Last Update Posted : January 13, 2020
Sponsor:
Information provided by (Responsible Party):
Dicerna Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE February 15, 2019
First Posted Date  ICMJE February 20, 2019
Last Update Posted Date January 13, 2020
Actual Study Start Date  ICMJE October 28, 2019
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 18, 2019)
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 [ Time Frame: 3 months (Last 3 months of the 6 month treatment period) ]
Four measurements of percent change from baseline in 24-hour urinary oxalate are combined to determine a single AUC value
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 18, 2019)
  • 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 [ Time Frame: 3 months (Last 3 months of the 6 month treatment period) ]
    Four measurements of percent change from baseline in 24-hour urinary oxalate-to-creatinine ratio are combined to determine a single AUC value
  • 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 [ Time Frame: At least 2 months starting at Day 90 and through Day 180 ]
  • 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 [ Time Frame: 7 months ]
  • 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 [ Time Frame: 7 months ]
  • TWS AUC of percent change from baseline in plasma oxalate [ Time Frame: 3 months (Last 3 months of the 6 month treatment period) ]
    Four measurements of percent change from baseline in plasma oxalate are combined to determine a single AUC value
  • Frequency of Adverse events (AE) from baseline to study completion [ Time Frame: 7 months ]
  • Frequency of serious adverse events (SAE) from baseline to study completion [ Time Frame: 7 months ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
Official Title  ICMJE 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
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)
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Primary Hyperoxaluria Type 1 (PH1)
  • Primary Hyperoxaluria Type 2 (PH2)
  • Kidney Diseases
  • Urologic Diseases
  • Genetic Disease
Intervention  ICMJE
  • Drug: DCR-PHXC
    Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection
  • Drug: 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
Study Arms  ICMJE
  • Experimental: DCR-PHXC
    Intervention, drug, 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
    Intervention: Drug: Sterile Normal Saline (0.9% NaCl)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 18, 2019)
36
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2021
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Willing to provide written informed consent or assent
  • Confirmation of PH1 and PH2 disease
  • Must meet the 24 hour urine oxalate excretion requirements

    - Page 3 of 5 -

  • Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 BSA

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Info 617-621-8097 medicalinfo@dicerna.com
Listed Location Countries  ICMJE Australia,   Canada,   France,   Germany,   Israel,   Italy,   Netherlands,   New Zealand,   Poland,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03847909
Other Study ID Numbers  ICMJE DCR-PHXC-201
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Dicerna Pharmaceuticals, Inc.
Study Sponsor  ICMJE Dicerna Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Dicerna Pharmaceuticals, Inc.
Verification Date September 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP