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Tests treatment safety and results for Primary Hyperoxaluria Type 1
Official title Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
ClinicalTrials.gov ID: NCT04580420
What this study is testing
What is DCR-PHXC?
DCR-PHXC is an investigational medicine, given as a monthly, being studied as a potential treatment for primary hyperoxaluria type 1.
Also referred to as Nedosiran.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Adults
You may be able to join if
- Note: Currently, the DCR-PHXC-204 study is only enrolling potential participants under 6 years of age.
- Four age groups of participants will be enrolled:
- adults and adolescents (aged ≥ 12 years)
- children 6 to 11 years of age
- children 2 to 5 years of age
You likely can't join if
- Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Prior renal transplantation is allowed.
- Documented evidence of clinical manifestations of severe systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history...
- Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact patient safety...
- Severe intercurrent illness
- Known causes of active liver disease/injury (e.g., alcoholic liver disease, nonalcoholic fatty liver disease/steatohepatitis)
- Non-PH related conditions contributing to renal insufficiency
See the full eligibility criteria
- Note: Currently, the DCR-PHXC-204 study is only enrolling potential participants under 6 years of age.
- Four age groups of participants will be enrolled:
- adults and adolescents (aged ≥ 12 years)
- children 6 to 11 years of age
- children 2 to 5 years of age
- infants and newborns from birth to \< 2 years of age
- . Documented diagnosis of PH1, confirmed by genotyping
- Estimated GFR at Screening \<30mL/min normalized to 1.73m\^2 BSA
- Mean of 2 Plasma Oxalate \>20μmol/L during screening
- For participants receiving hemodialysis or peritoneal dialysis total duration of hemodialysis or peritoneal dialysis must be less than 24 months and hemodialysis or peritoneal dialysis regimen must have been stable for...
- Male or Female
- Male participants:
- A male participant with a female partner of childbearing potential must agree to use contraception during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from...
- Female participants:
- A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies: Not a woman of childbearing potential (WOCBP).
- A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 12 weeks after the last dose of study intervention and agrees to refrain from harvesting/freezing eggs during this...
- Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Participant (and/or participant's parent or legal guardian if participant is a minor [defined as patient \<18 years of age, or younger than the age of majority according to local regulations]) is capable of giving...
- Adolescents (12 to \< 18 years of age, or older than 12 years but younger than the age of majority according to local regulations) must be able to provide written assent for participation.
- For children younger than 12 years of age, assent will be based on local regulations
- Affiliated with or is a beneficiary of a health insurance system (if applicable per national regulations)
- Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Prior renal transplantation is allowed.
- Documented evidence of clinical manifestations of severe systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
- Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to:
- Severe intercurrent illness
- Known causes of active liver disease/injury (e.g., alcoholic liver disease, nonalcoholic fatty liver disease/steatohepatitis)
- Non-PH related conditions contributing to renal insufficiency
- Physician concerns about intake of drugs of abuse or excessive alcohol intake, or history of excessive alcohol intake in the 2 years prior to enrollment (defined as ≥ 21 units of alcohol per week in men and ≥ 14 units...
- Use of an RNAi drug, other DCR-PHXC, within the last 6 months
- History of one or more of the following reactions to an oligonucleotide-based therapy:
- Severe thrombocytopenia (platelet count ≤ 100,000/µL)
- Hepatotoxicity, defined as alanine transaminase (ALT) or aspartate transaminase (AST) \> 3 times the upper limit of normal (ULN) and total bilirubin \> 2 × ULN or international normalized ratio (INR) \>1.5
- Severe flu-like symptoms leading to discontinuation of therapy
- Localized skin reaction from the injection (graded severe) leading to discontinuation of therapy
- Coagulopathy/clinically significant prolongation of clotting time
- Participation in any clinical study in which they received an investigational medicinal product (IMP) other than DCR-PHXC within 4 months before Screening.
- Liver function test abnormalities: ALT and/or AST \>1.5 × ULN for age and gender
- Positive anti-double-stranded deoxyribonucleic acid (anti-dsDNA) antibody test at Screening
- Known hypersensitivity to DCR-PHXC or any of its ingredients
- Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations
The study team makes the final eligibility decision.
Where it's taking place
- San Francisco, California, United States
- Boston, Massachusetts, United States
- Rochester, Minnesota, United States
- New York, New York, United States
- Bron, France
- Paris, France
- Bonn, Germany
- Heidelberg, Germany
- Roma, Italy
- Beirut, Lebanon
- Casablanca, Morocco
- Oradea, Romania
- Barcelona, Spain
- Santa Cruz de Tenerife, Spain
- Dubai, United Arab Emirates
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, not specified. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Francisco, California, United States; Boston, Massachusetts, United States; Rochester, Minnesota, United States; New York, New York, United States; Bron, France; Paris, France and 10 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.