NCT05993416

Treatment of Primary Hyperoxaluria Type 1 With Nedosiran

• Status: No Longer Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: —

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.

Conditions

• Primary Hyperoxaluria Type 1 (PH1)

Interventions

Type	Name	Description
DRUG	Nedosiran	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

• First posted: 2023-08-15
• Last updated: 2025-07-22