Long Term Extension Study in Patients With Primary Hyperoxaluria (PHYOX3)

Dicerna Pharmaceuticals

Status and phase: Invitation-only; Phase 3

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

Treatments: Drug: DCR-PHXC

Study type: Interventional

Funder types: Industry

Identifiers

NCT04042402

DCR-PHXC-301

Details and patient eligibility

About

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.

Enrollment: 75 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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, 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.

Key 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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75 participants in 1 patient group

Open Label

Experimental group

Description:

Open label, monthly subcutaneous injection

Treatment:

Drug: DCR-PHXC