Status:
NO_LONGER_AVAILABLE
Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
Lead Sponsor:
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Conditions:
Primary Hyperoxaluria Type 1 (PH1)
Eligibility:
All Genders
2+ years
Brief Summary
The purpose of this program is to provide participants with access to an investigational drug, nedosiran, for treatment of primary hyperoxaluria type 1 (PH1). Eligible participants may receive nedosir...
Eligibility Criteria
Inclusion
- Early Access Eligibility Criteria:
- Written, signed, and dated informed patient/parent consent; and for patients who were minors, age-appropriate assent (performed according to local regulations)
- Male or female patients at least 2 years of age
- Genetically-confirmed diagnosis of PH1
- Accessible for treatment and follow-up and be able to comply with treatment monitoring requirements
- Not eligible for an ongoing RNAi therapy trial and not participating simultaneously in any interventional clinical research study
- Patients with PH1 that are not satisfactorily treated with current standard of care; i.e., 24-hour urinary oxalate (Uox) excretion ≥ 0.7 (millimoles) mmol for participants 18 years and older, or greater or lesser than (≥) 0.7 mmol per 1.73 metre square (m\^2) body surface area (BSA) for participants less than 18 years of age
- Estimated GFR at screening ≥ 30 millilitres per minute (mL/min) normalized to 1.73 m\^2 BSA
- No renal or hepatic transplantation; prior or planned within the treatment period
- No documented evidence of clinical manifestations of systemic oxalosis (including pre existing retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
- Patient not currently on dialysis
- Plasma oxalate ≤ 30 micromoles per litre (μmol/L)
- Female patients not breastfeeding or pregnant
- The potential benefit for the individual patient justifies the potential risks of treatment as per prescribing physician judgment
Exclusion
Key Trial Info
Start Date :
Trial Type :
EXPANDED_ACCESS
End Date :
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT05993416
Last Update
July 22 2025
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