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

Brief Title

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

Official 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

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)
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Area under the curve (AUC) of percent change from baseline in 24-hour urinary oxalate excretion between Day 90 and Day 180

Secondary Outcome

 Proportion of participants with a 24-hour Uox level < 0.46 mmol/24 hours or ≥ 0.46 - < 0.60 mmol/24 hours (adjusted per 1.73 m2 BSA in participants aged <18 years) on at least two consecutive study visits commencing at Day 90 and ending at Day 180

Condition

Primary Hyperoxaluria Type 1 (PH1)

Intervention

DCR-PHXC

Study Arms / Comparison Groups

 DCR-PHXC
Description:  Intervention, drug, DCR-PHXC

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

35

Start Date

October 28, 2019

Completion Date

June 29, 2021

Primary Completion Date

June 21, 2021

Eligibility Criteria

        Key Inclusion Criteria:

          -  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 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
      

Gender

All

Ages

6 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Alexandra Haagensen, MD, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT03847909

Organization ID

DCR-PHXC-201


Responsible Party

Sponsor

Study Sponsor

Dicerna Pharmaceuticals, Inc.


Study Sponsor

Alexandra Haagensen, MD, Study Director, Dicerna Pharmaceuticals, Inc.


Verification Date

July 2021