An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (subcutaneous use) in Patients with Primary Hyperoxaluria
- Conditions
- HyperoxaluriaPrimary Hyperoxaluria100274241002160510038430
- Registration Number
- NL-OMON54596
- Lead Sponsor
- Dicerna Pharmaceuticals Inc (a Novo Nordisk Company)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 8
ROLL-OVER PARTICIPANTS AND THEIR PEDIATRIC SIBLINGS
1. Participants starting at birth are eligible for this study.
2.Documented diagnosis of PH, confirmed by genotyping (historically available
genotype information is acceptable for study eligibility)
3. Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of
DCR-PHXC or is the sibling of a participant who either successfully completed a
Dicerna study of DCR-PHXC successfully completed a Dicerna Pharmaceuticals,
Inc. study of DCR-PHXC.
a. For participants rolling over from a multidose study of DCR-PHXC, enrollment
should occur within a window of 25 to 75 days from the last dose of study
intervention. In order to minimize any gap in administration of DCR-PHXC, every
effort should be made to enroll participants as soon as all assessments from
the previous study have been completed. It should be noted if the participant
was required to repeat the end-of-study (EOS) 24-hour Uox collection for
violation of completeness criteria.
4. Estimated GFR at screening >= 30 mL/min normalized to 1.73 m2 BSA,calculated
using the equations found in Section8.2.4.1 (Protocol). For infants aged less
than 12 months, serum creatinine below The 97.5th percentile of a healthy
population (Boer et al. 2010)
In the Netherlands, children aged 0 to 5 must have PH1 to be eligible for
enrollment. Pediatric patients with PH2 or PH3 are not eligible for enrollment,
as the efficacy of DCR-PHXC has not yet been established in patients with PH2
or PH3.
ROLL-OVER PARTICIPANTS AND THEIR PEDIATRIC SIBLING
1. Prior renal or hepatic transplantation; or planned transplantation within
the study period
2. Currently receiving dialysis
3. Documented evidence of clinical manifestations of systemic oxalosis.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary Endpoint:<br /><br>The annual rate of decline in eGFR in participants with PH1<br /><br><br /><br><br /><br>Key Secondary Endpoint:<br /><br>1. The incidence and severity of treatment-emergent adverse events (TEAE) and<br /><br>serious adverse events (SAE)<br /><br>2. Change from Baseline in 12-lead electrocardiogram (ECG), physical<br /><br>examination findings, vital signs, and clinical laboratory tests (hematology,<br /><br>chemistry, coagulation parameters, and urinalysis)<br /><br><br /><br><br /><br></p><br>
- Secondary Outcome Measures
Name Time Method