Treatment of Primary Hyperoxaluria Type 1 With Nedosiran

August 7, 2023 updated by: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
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 Overview

Status

Available

Conditions

Intervention / Treatment

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

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 6 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

August 6, 2023

First Submitted That Met QC Criteria

August 7, 2023

First Posted (Actual)

August 15, 2023

Study Record Updates

Last Update Posted (Actual)

August 15, 2023

Last Update Submitted That Met QC Criteria

August 7, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DCR-PHXC-401

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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