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Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXC.
EU CTIS ID: 2024-512259-19-00
What this study is testing
To assess the efficacy of DCR-PHXC in lowering Pox in participants with PH1 and severe renal impairment, with or without hemodialysis or peritoneal dialysis.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Four age groups of participants will be enrolled: a. adults and adolescents (aged ≥ 12 years); b. children 6 to 11 years of age c. children 2 to 5 years of age and d. infants and newborns from birth to < 2 years of age.
- 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).
- Documented diagnosis of PH1 confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
- Estimated GFR at Screening < 30 mL/min normalized to 1.73 m2 BSA. For infants aged less than 12 months, serum creatinine above the 97.5th percentile of a healthy population.
You likely can't join if
- Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Renal transplantation planned in the 6 months from Day 1. Prior renal transplantation is allowed.
- Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations detailed in Section 5.3.
- 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.
- Use of an RNAi drug, other than DCR-PHXC, within the last 6 months.
- History of one or more of the following reactions to an oligonucleotide-based therapy.
See the full eligibility criteria
Who can join
- Four age groups of participants will be enrolled: a. adults and adolescents (aged ≥ 12 years); b. children 6 to 11 years of age c. children 2 to 5 years of age and d. infants and newborns from birth to < 2 years of age.
- 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).
- Documented diagnosis of PH1 confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
- Estimated GFR at Screening < 30 mL/min normalized to 1.73 m2 BSA. For infants aged less than 12 months, serum creatinine above the 97.5th percentile of a healthy population.
- Mean of 2 plasma oxalate values > 20 μmol/L during Screening.
- For participants receiving hemodialysis or peritoneal dialysis, total duration of hemodialysis or peritoneal dialysis must be less than or equal to 24 months and hemodialysis or peritoneal dialysis regimen must have been stable for at least 2 weeks prior to Screening.
- Male or female.
- A male participant with a female partner of childbearing potential must agree to use contraception, as detailed in Section 10.5.2, during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period.
- 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), OR A WOCBP who agrees to follow the contraceptive guidance in Section 10.5.2.2 for the 4 weeks prior to randomization, 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 period.
- 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 signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
What rules you out
- Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Renal transplantation planned in the 6 months from Day 1. Prior renal transplantation is allowed.
- Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations detailed in Section 5.3.
- 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.
- Use of an RNAi drug, other than DCR-PHXC, within the last 6 months.
- History of one or more of the following reactions to an oligonucleotide-based therapy.
- Participation in any clinical study in which they received an investigational medicinal product (IMP) other than DCR-PHXC within 4 months or 5 times the half-life of the drug (whichever is longer) 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.
The study team makes the final eligibility decision.
Where it's taking place
- Lebanon
- United States
- United Arab Emirates
- United Kingdom
- Morocco
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, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Lebanon; United States; United Arab Emirates; United Kingdom; Morocco. 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 EU CTIS; the study team decides eligibility.