Clinical Trials in Australia / NCT04042402
Active, not recruiting
Phase 3
Long Term Extension Study in Patients With Primary Hyperoxaluria
NCT04042402 · tracked via the Priya Life Science Australia tracker
Condition(s) studied
Primary Hyperoxaluria Type 1 (PH1)Primary Hyperoxaluria Type 2 (PH2)Kidney DiseasesUrologic DiseasesGenetic DiseasePrimary Hyperoxaluria Type 3 (PH3)
Investigational drug(s) / intervention(s)
DCR-PHXC: Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection
Study summary
The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC and their siblings (\<18 years old) long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.
Eligibility
Key Inclusion Criteria:
•Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC.
OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. Siblings must be younger than 18 years of age and must have genetically confirmed PH.
* 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.
* Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 body surface area (BSA), calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) formula in participants aged ≥ 18 years, or the multivariate equation by Schwartz in participants aged 12 months to 17 years. In Japan, the cystatin C-based Uemura formula will be used for participants aged 12 months to \<2 years, the creatinine-based Uemura formula by will be used for participants aged 2 to 17 years, and the equation by Matsuo will be used in participants aged ≥ 18 years.
Key Exclusion Criteria:
* Renal or hepatic transplantation (prior or planned within the study period)
* Plasma oxalate \> 30 µmol/L
* Currently on dialysis
* Documented evidence of clinical manifestations of systemic oxalosis
Primary outcome measure(s)
- The annual rate of decline in eGFR in participants with PH1 — Annual change from baseline
To evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1
Trial sites (23)
| Facility | City | Region | Status |
|---|---|---|---|
| Clinical Research Site | San Francisco | California | |
| Clinical Trial Site | Boston | Massachusetts | |
| Clinical Trial Site | Rochester | Minnesota | |
| Clinical Trial Site | New York | New York | |
| Clinical Research Site | Herston | Queensland | |
| Clinical Trial Site | Melbourne | Australia | |
| Clinical Research Site | Hamilton | Ontario | |
| Clinical Trial Site | Bron | France | |
| Clinical Trial Site | Paris | France | |
| Clinical Trial Site | Bonn | Germany | |
| Clinical Trial Site | Heidelberg | Germany | |
| Clinical Research Site | Roma | Italy | |
| Clinical Trial Site | Fukuoka | Japan | |
| Clinical Trial Site | Nagoya | Japan | |
| Clinical Trial Site | Tokyo | Japan | |
| Clinical Trial Site | Beirut | Lebanon | |
| Clinical Trial Site | Amsterdam | Netherlands | |
| Clinical Trial Site | Tromsø | Norway | |
| Clinical Research Site | Barcelona | Spain | |
| Clinical Trial Site | Barcelona | Spain | |
| Clinical Trial Site | Ankara | Turkey (Türkiye) | |
| Clinical Trial Site | Hampstead | London | |
| Clinical Trial Site | Birmingham | United Kingdom |
Other trials for the same condition
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04042402 on ClinicalTrials.gov ↗ ← All trials in Australia