A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi Center Study in Patients with Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR PHXC Solution for Injection (subcutaneous use) - A 2-part study in NHV and PH patients

Dicerna Pharmaceutical Inc0 sites5 target enrollmentTBD

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Not specified
Sponsor: Dicerna Pharmaceutical Inc
Enrollment: 5
Status: Not yet recruiting
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

TBD

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional

Investigators

Sponsor

Dicerna Pharmaceutical Inc

Eligibility Criteria

Inclusion Criteria

•PH patients must meet all of the following criteria to be eligible for participation in this study.
•1\. Patient
•a. Understands the full nature and purpose of the study, including possible risks and side effects.
•b. Is willing and able to comply with all study procedures including collection of 24\-hr urine samples.
•c. Provides informed consent.
•2\. Male or female, at least 18 years of age at the time of obtaining informed consent. ;3\. Documented diagnosis of PH1 or PH2, confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
•4\. 24\-hr urine oxalate excretion \>\=0\.7 mmol on at least one of the two assessments conducted in the screening period, with less than 30% variation between both oxalate measurements.
•5\. eGFR \>\=30 mL/min normalized to 1\.73 m2 BSA calculated using the Modification of Diet in Renal Disease (MDRD) formula (Levey et al., 1999; National Kidney Foundation, 2002\).
•6\. Males, female patients of childbearing potential and female partners of male patients of childbearing potential must be willing to use a highly effective and approved contraceptive method(s) from the date of informed consent until 12 weeks after the last dose of IMP. A highly effective method of contraception is defined as fulfilling at least one of the following:
•a. Strict abstinence: When this is in line with the preferred and usual lifestyle of the patient. \[Periodic abstinence (e.g., calendar, ovulation, symptom\-thermal, post\-ovulation methods) and withdrawal are not acceptable methods of contraception.]

Exclusion Criteria

•PH patients meeting any of the following criteria will be excluded from this study:
•1\. Prior renal and/or hepatic transplantation.
•2\. Currently receiving dialysis.
•3\. Documented evidence of clinical manifestations of systemic oxalosis.
•4\. Participation in any clinical study where they received an investigational medical product within 4 months before enrollment. For IMPs with the potential to reduce Uox and/or plasma oxalate, these concentrations must have returned to historical baseline levels.
•a. If patient participated in an earlier cohort in this study (DCR\-PHXC\-101\), a minimum of 8 weeks must have elapsed prior to re\-enrollment and urinary oxalate excretion must have returned to \>\=80% of baseline.
•5\. Presence of any medical condition or co\-morbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to:
•a. severe intercurrent illness
•b. routine vaccination within 30 days prior to dosing and through EOS visit
•c. known causes of active liver disease/ injury or transaminase elevation (e.g., alcoholic liver disease, Nonalcoholic fatty liver disease/ steatohepatitis (NAFLD/NASH)