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Treatment of Primary Hyperoxaluria Type 1 With Nedosiran

Primary Purpose

Primary Hyperoxaluria Type 1 (PH1)

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
Nedosiran
Sponsored by
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Primary Hyperoxaluria Type 1 (PH1)

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All Sexes

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    August 6, 2023
    Last Updated
    August 7, 2023
    Sponsor
    Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05993416
    Brief Title
    Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
    Official Title
    Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    August 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

    4. Oversight

    5. Study Description

    Brief 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Primary Hyperoxaluria Type 1 (PH1)

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Nedosiran
    Intervention Description
    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.

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    6 Years
    Eligibility Criteria
    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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Medical Information
    Phone
    +44 20 39 408 111
    Email
    Nedosiran@tannerpharma.com

    12. IPD Sharing Statement

    Learn more about this trial

    Treatment of Primary Hyperoxaluria Type 1 With Nedosiran

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