Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk company
About this trial
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
Eligibility criteria
Qualifiers
adults and adolescents (aged ≥ 12 years)
children 6 to 11 years of age
children 2 to 5 years of age
infants and newborns from birth to < 2 years of age
Disqualifiers
Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Prior renal transplantation is allowed.
Documented evidence of clinical manifestations of severe systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
Severe intercurrent illness
Known causes of active liver disease/injury (e.g., alcoholic liver disease, nonalcoholic fatty liver disease/steatohepatitis)
Trial design
Treatments tested in this trial
- DCR-PHXC
Treatment groups
28 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov