A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)

Dicerna Pharmaceuticals

Status and phase

Terminated

Phase 1

Conditions

Primary Hyperoxaluria Type 1

Treatments

Other: Placebo

Drug: DCR-PH1

Study type

Interventional

Funder types

Industry

Identifiers

NCT02795325

DCR-PH1-101

Details and patient eligibility

About

A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)

Enrollment

41 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Male or female, at least 12 years of age
Diagnosis of PH1, confirmed by genotyping
24-hour urine oxalate excretion as defined in the protocol
eGFR ≥ 40 mL/min normalized to 1.73 m2 BSA
Written informed consent for adults (≥18 years old, or per local regulatory requirement); written informed assent for adolescents (12 to <18 years old, or per local regulatory requirement)

Exclusion criteria

Prior renal and/or hepatic transplantation
Participation in any clinical study involving administration of any investigational drug within the 30 days before enrollment
Pregnancy or lactation at the time of screening or enrollment
Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
Patients with a known history of human immunodeficiency virus (HIV) or active infection with hepatitis B virus or hepatitis C virus
Moderate to severe liver impairment
Liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) > 2 times upper limit of normal (ULN)
History of severe reaction to a liposomal product or a known hypersensitivity to lipid products.
Unable to collect required study samples or follow study procedures
No clinically significant health concerns