Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRD

• Status: Recruiting
• Trial end date: 2025-05-01
• Target enrollment: 0
• Participant gender: All

Summary

The aim of this study is to evaluate DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without dialysis.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Dicerna Pharmaceuticals, Inc.

Criteria

Inclusion Criteria:

1. Documented diagnosis of PH1 or PH2, confirmed by genotyping

2. Estimated GFR at Screening <30mL/min normalized to 1.73m^2 BSA

3. Plasma Oxalate >30μmol/L

4. For participants receiving dialysis, total duration must be less than 18 months

5. Male or Female

1. Male participants:

• A male participant with a female partner of childbearing potential must agree to use contraception during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period.

2. Female participants:

• A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:

Not a woman of childbearing potential (WOCBP).

• OR

• A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 12 weeks after the last dose of study intervention.

3. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

6. 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.

Exclusion Criteria:

1. Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Prior renal transplantation is allowed.

2. Documented evidence of severe systemic oxalosis, defined as overt signs of bone oxalate deposition in a plain x-ray of the left hand, as evidenced by large diffuse metaphyseal bands

3. Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact patient safety

4. Use of an RNAi drug, other DCR-PHXC, within the last 6 months

5. History of reactions to an oligonucleotide-based therapy

6. Participation in any clinical study in which they received an investigational medicinal product (IMP) other than DCR-PHXC within 4 months before Screening.

7. Liver function test abnormalities: ALT and/or AST >1.5 × ULN for age and gender

8. Positive anti-double-stranded deoxyribonucleic acid (anti-dsDNA) antibody test at Screening

9. Positive urine drug screen (to include at minimum: amphetamines, barbiturates, cocaine, opiates, and benzodiazepines). Urine drug screening is not required for participants ≤ 12 years of age. Exclusion for a positive screen is at the discretion of the Investigator.

10. Known hypersensitivity to DCR-PHXC or any of its ingredients

11. Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations