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Tests treatment safety and results for OTC Deficiency
Official title A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
ClinicalTrials.gov ID: NCT06488313
What this study is testing
What is ARCT-810?
ARCT-810 is an investigational medicine, being studied as a potential treatment for otc deficiency.
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
- Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
- 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
Ages 12 and older
You may be able to join if
- Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent.
- Males and Females aged ≥12 years, at Screening.
- Documented clinical diagnosis of OTC deficiency.
- History of symptomatic hyperammonemia or elevated plasma ammonia or glutamine with clinical stability for at least 1 month prior to Screening.
- Medically managed for OTC deficiency and receiving a stable protein-restricted diet, dietary supplements, and/or ammonia scavenger regimen (if...
You likely can't join if
- Uncontrolled hypertension.
- Symptoms of infection for at least 7 days prior to dosing.
- Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully...
- History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 2 years.
- History of any organ transplant.
- History of severe allergic reaction to a liposomal or PEG-containing product.
See the full eligibility criteria
- Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent.
- Males and Females aged ≥12 years, at Screening.
- Documented clinical diagnosis of OTC deficiency.
- History of symptomatic hyperammonemia or elevated plasma ammonia or glutamine with clinical stability for at least 1 month prior to Screening.
- Medically managed for OTC deficiency and receiving a stable protein-restricted diet, dietary supplements, and/or ammonia scavenger regimen (if applicable) for at least 28 days.
- Good general health with no clinically significant abnormal findings that would interfere with study procedures (including plasma ammonia within participant's historical range).
- Must be willing to adhere to contraception guidelines.
- Uncontrolled hypertension.
- Symptoms of infection for at least 7 days prior to dosing.
- Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
- History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 2 years.
- History of any organ transplant.
- History of severe allergic reaction to a liposomal or PEG-containing product.
- History of congenital or acquired cardiac disorders.
- Abuse of medications, illicit drugs or alcohol.
- Blood donation of 50 to 499 mL within 30 days of screening or of \>499 mL within 60 days of screening.
- Clinically significant laboratory abnormalities on screening labs including INR \>1.5, eGFR\< 60 mL/min/1.73m2 or positive test results for HIV, HBV, or HCV.
- Inadequately controlled diabetes.
- Clinically significant anemia.
- Changes in maintenance therapies for OTC deficiency with 28 days prior to dosing.
- Medical history requiring continuous or intermittent systemic corticosteroid administration.
- Receipt of inhibitors of urea synthesis or drugs that significantly affect renal clearance.
- Recent treatment with another investigational drug, biological agent, or device.
- Treatment with any oligonucleotide (siRNA or mRNA) within 6 months prior to screening. COVID-19 vaccines are not exclusionary.
- Involved in study conduct or an immediate family member of an individual involved in the study.
- Participated in another dosing cohort of the study.
- Any other conditions, in the opinion of the investigator, that would interfere with participation.
The study team makes the final eligibility decision.
Where it's taking place
- Chevy Chase, Maryland, United States
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, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Chevy Chase, Maryland, United States. 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.