Long Term Extension Study in Patients With Primary Hyperoxaluria

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

75 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-07-09

Study Completion Date

2030-04-01

Brief Summary

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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.

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Detailed Description

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Conditions

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Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 2 (PH2) Kidney Diseases Urologic Diseases Genetic Disease Primary Hyperoxaluria Type 3 (PH3)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Open Label

Open label, monthly subcutaneous injection

Group Type EXPERIMENTAL

DCR-PHXC

Intervention Type DRUG

Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection

Interventions

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DCR-PHXC

Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection

Intervention Type DRUG

Other Intervention Names

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Nedosiran

Eligibility Criteria

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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, 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.

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

Eligible Sex

ALL

Accepts Healthy Volunteers

No