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Anti-sense Oligonucleotide
ARCT-810 for OTC Deficiency
Phase 2
Recruiting
Research Sponsored by Arcturus Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Documented clinical diagnosis of OTC deficiency
Must not have
Uncontrolled hypertension
History of any organ transplant
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 85
Awards & highlights
No Placebo-Only Group
Summary
"This trial will test the safety and effects of different doses of ARCT-810 in teenagers and adults with OTC deficiency."
Who is the study for?
This trial is for adolescents and adults aged 12 or older with Ornithine Transcarbamylase Deficiency (OTCD). Participants must be on a stable diet and/or medication regimen for OTCD, in good health, and willing to follow the study's procedures. They cannot have severe allergies to liposomal/PEG products, drug/alcohol abuse history, certain infections or uncontrolled conditions like hypertension.
What is being tested?
The trial tests multiple doses of ARCT-810 to see how it affects people with OTCD. It looks at safety and how the body responds to the treatment (pharmacodynamics). The goal is to find out if this new intervention can help manage symptoms related to OTCD more effectively.
What are the potential side effects?
While specific side effects are not listed here, participants will be monitored for any adverse reactions due to ARCT-810. This could include allergic reactions given its liposomal/PEG content or other common drug-related side effects such as nausea, headache, fatigue or injection site reactions.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with OTC deficiency.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My blood pressure is not well-controlled.
Select...
I have had an organ transplant.
Select...
I have been diagnosed with severe anemia.
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I haven't had cancer in the last 5 years, except for certain skin cancers or cervical cancer that was treated.
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I haven't had gene or liver stem cell therapy in the last 2 years.
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I have a history of heart problems since birth or developed later.
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My diabetes is not well-controlled.
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I am taking medication that affects my kidney function or urea synthesis.
Select...
I need to take corticosteroids regularly.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ day 85
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 85
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Incidence, severity and dose-relationship of adverse events (AEs)
Secondary study objectives
Fasting plasma ammonia
Plasma Glutamine
Plasma pharmacokinetics
+2 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: ARCT-810Experimental Treatment1 Intervention
Participants will receive up to 5 IV infusions of ARCT-810 administered at 14-day intervals.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
ARCT-810
2020
Completed Phase 1
~50
Find a Location
Who is running the clinical trial?
Arcturus Therapeutics, Inc.Lead Sponsor
12 Previous Clinical Trials
22,261 Total Patients Enrolled