Primary Hyperoxaluria Type 1 (PH1)NCT05993416
Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
A study testing Nedosiran in people with Primary Hyperoxaluria Type 1 (PH1), run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company.
Trial 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… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Primary Hyperoxaluria Type 1 (PH1)
- What is being tested?
- Nedosiran
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- Child, Adult, Older adult
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
About this study
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.
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Being tested
- Nedosiran
- Medical subject headings
- Primary hyperoxaluria type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NedosiranDrug
In adults and in adolescents (aged 12-17 years) weighing greater than or equal to (>=) 50 kilograms (kg), Nedosiran will be administered via subcutaneous (SC) injection once monthly at a dose of 170 milligrams (mg). In adults and adolescents weighing < 50 kg, nedosiran will be administered once monthly at a dose of 136 mg. The dose for participants aged 6 to 11 years will be 3.5 mg/kg monthly, not to exceed 170 mg.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Hyperoxaluria Type 1 (PH1)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05993416
- Sponsor's protocol ID
- DCR-PHXC-401
- Status last verified
- Jul 2025
- First submitted
- 6 Aug 2023
- Last updated
- 22 Jul 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
- ClinicalTrials.gov ID
- NCT05993416
- Acronym / sponsor ID
- DCR-PHXC-401
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyIndustry (pharma, biotech, devices)
- First posted
- 15 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NedosiranIn adults and in adolescents (aged 12-17 years) weighing greater than or equal to (>=) 50 kilograms (kg), Nedosiran will be administered via subcutaneous (SC) injection once monthly at a dose of 170 milligrams (mg). In adults and adolescents weighing < 50 kg, nedosiran will be administered once monthly at a dose of 136 mg. The dose for participants aged 6 to 11 years will be 3.5 mg/kg monthly, not to exceed 170 mg. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Nedosiran in people with Primary Hyperoxaluria Type 1 (PH1), run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 22 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2025.