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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02795325
• Date of registration: 18/05/2016
• Prospective Registration: No
• Primary sponsor: Dicerna Pharmaceuticals, Inc.
• Public title: A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
• Scientific title: A Phase 1 Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
• Date of first enrolment: May 13, 2016
• Target sample size: 41
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02795325
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Germany, Netherlands

Key inclusion & exclusion criteria

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



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primary Hyperoxaluria Type 1

Intervention(s)

Other: Placebo

Drug: DCR-PH1

Primary Outcome(s)

The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs) [Time Frame: Through Day 29]

Secondary Outcome(s)

The effect of DCR-PH1 on urine oxalate levels [Time Frame: Through Day 29]

Profile of pharmacokinetics (PK) of DCR-PH1 - t½ [Time Frame: Through Day 29]

Profile of pharmacokinetics (PK) of DCR-PH1 - AUC [Time Frame: Through Day 29]

Profile of pharmacokinetics (PK) of DCR-PH1 - tmax [Time Frame: Through Day 29]

Profile of pharmacokinetics (PK) of DCR-PH1 - Cmax [Time Frame: Through Day 29]

The effect of DCR-PH1 on urine glycolate levels [Time Frame: Through Day 29]

The effect of DCR-PH1 on plasma glycolate levels [Time Frame: Through Day 29]

The effect of DCR-PH1 on plasma oxalate levels [Time Frame: Through Day 29]

Secondary ID(s)

DCR-PH1-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors