Treatment of Primary Hyperoxaluria Type 1 With Nedosiran (NCT05993416) | Clinical Trial Compass
NO_LONGER_AVAILABLENot Applicable
Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
Plain-language 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 nedosiran in this program until the drug is commercially available or until Novo Nordisk terminates the program, whichever comes first. Novo Nordisk may terminate the program at any time for any reason, including if the drug receives regulatory approval and becomes commercially available, or if the drug does not receive regulatory approval. Nedosiran will be given once a month with a thin needle in the thigh or abdomen. The study doctor will ask the participant to come to the clinic monthly. The study doctor may allow participant to take nedosiran at home for self-administration. The participant should let the doctor know if they are unable to make a visit so it can be rescheduled. Participants to inform the study doctor of any medications they are taking, including over the counter medicines, vitamins, and herbal medicines. If any medications change in dose, or new medications are added, participants should inform the study doctor. Study doctor should be informed of any new or continued health problems or any changes in the participant's health.
Who can participate
Age range
2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 physic…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Trial details
NCT IDNCT05993416
SponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk company