Status and phase
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About
The aim of this study is to evaluate DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without dialysis.
Full description
This is an open-label, repeat-dose, Phase 2 study of DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without dialysis.
Following the up-to-35- day screening period, participants will return to the clinic for monthly dosing visits through Day 180. Participants successfully completing the Day 180 visit will continue on to an extended follow-up period and receive open-label DCR-PHXC for an additional 3 years, or until DCR-PHXC is commercially available, whichever comes first. As participants in this extended treatment period will return to the clinic only every 3 months, participants and/or their caregivers may be trained in the at-home administration of DCR-PHXC or home health nurses may assist with administration of DCR -PHXC.
The total duration of the study is approximately 2 years from first participant, first visit, to last participant, last Day 180 visit, with up to an additional 3 years of extended follow-up.
Enrollment
Sex
Volunteers
Inclusion criteria
Four age groups of participants will be enrolled, in sequence:
. Documented diagnosis of PH1 or PH2, confirmed by genotyping
Estimated GFR at Screening <30mL/min normalized to 1.73m^2 BSA
Mean of 2 Plasma Oxalate >20μmol/L during screening
For participants receiving hemodialysis or peritoneal dialysis total duration of hemodialysis or peritoneal dialysis must be less than 18 months and hemodialysis or peritoneal dialysis regimen must have been stable for at least 2 weeks prior to Screening.
Body weight of:
Male or Female
Male participants:
Female participants:
Not a woman of childbearing potential (WOCBP).
Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Participant (and/or participant's parent or legal guardian if participant is a minor [defined as patient <18 years of age, or younger than the age of majority according to local regulations]) is capable of giving signed informed consent, which includes compliance with the requirement and restrictions listed in the informed consent form (ICF) and in the protocol.
Affiliated with or is a beneficiary of a health insurance system (if applicable per national regulations)
Exclusion criteria
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)
Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to:
Use of an RNAi drug, other DCR-PHXC, within the last 6 months
History of one or more of the following reactions to an oligonucleotide-based therapy:
Participation in any clinical study in which they received an investigational medicinal product (IMP) other than DCR-PHXC within 4 months before Screening.
Liver function test abnormalities: ALT and/or AST >1.5 × ULN for age and gender
Positive anti-double-stranded deoxyribonucleic acid (anti-dsDNA) antibody test at Screening
Known hypersensitivity to DCR-PHXC or any of its ingredients
Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations
Primary purpose
Allocation
Interventional model
Masking
17 participants in 1 patient group
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Medical Information
Data sourced from clinicaltrials.gov
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