Long Term Extension Study in Patients With Primary Hyperoxaluria

Brief Title

Long Term Extension Study in Patients With Primary Hyperoxaluria

Official 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

Brief Summary

      The proposed study is designed to provide patients previously enrolled in Phase 1 and 2
      studies of DCR-PHXC and their siblings (<18 years old) long-term access to DCR-PHXC, and to
      evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

The annual rate of decline in eGFR

Secondary Outcome

 The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal 12 lead electrocardiogram (ECG) readings

Condition

Primary Hyperoxaluria Type 1 (PH1)

Intervention

DCR-PHXC

Study Arms / Comparison Groups

 Open Label
Description:  Open label, monthly subcutaneous injection

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

50

Start Date

July 9, 2019

Completion Date

December 2023

Primary Completion Date

December 2023

Eligibility Criteria

        Key Inclusion Criteria:

        •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 (Levey & Stevens, 2010), or the multivariate equation
             by Schwartz in participants aged 6 to 17 years (Schwartz et al., 2012). In Japan, the
             formula by Uemura et al. will be used for participants aged 6 to 17 years, and the
             equation by Matsuo et al. will be used in participants aged ≥ 18 years (Uemura et al.,
             2014; Matsuo et al., 2009).

        Key Exclusion Criteria:

          -  Renal or hepatic transplantation (prior or planned within the study period)

          -  Plasma oxalate > 30 µmol/L

          -  Currently dialysis

          -  Documented evidence of clinical manifestations of systemic oxalosis
      

Gender

All

Ages

6 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT04042402

Organization ID

DCR-PHXC-301


Responsible Party

Sponsor

Study Sponsor

Dicerna Pharmaceuticals, Inc.


Study Sponsor

, , 


Verification Date

July 2021