Status:
COMPLETED
Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency
Lead Sponsor:
Arcturus Therapeutics, Inc.
Conditions:
Ornithine Transcarbamylase Deficiency
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in clinically stable patients (stable on standard of care treatment, e.g. diet ± ammonia scavengers) with ornithine ...
Detailed Description
This is a single ascending dose study of ARCT-810 in which approximately 12 (up to a maximum of 20) clinically stable patients with ornithine transcarbamylase deficiency (OTCD) are planned to be enrol...
Eligibility Criteria
Inclusion
- Adequate cognitive ability to consent and recall symptoms over a 1-week time period
- Males and females ≥18 years of age with documented diagnosis of ornithine transcarbamylase deficiency (OTCD) confirmed with genetic testing, or willing to consent to OTC gene sequencing and deletion/duplication testing
- Subject's ornithine transcarbamylase deficiency (OTCD) is stable as evidenced by
- no clinical symptoms of hyperammonemia AND
- an ammonia level \<100 µmol/L (170 µg/dL) at the screening evaluation
- Subjects must remain free from symptoms of hyperammonemia throughout the screening period.
- If using nitrogen ammonia scavenger therapy, must be on a stable regimen (no change in dose or frequency) for ≥ 28 days prior to providing informed consent and throughout the screening period
- Must have maintained a stable protein-restricted diet (+/- amino acid supplementation) for at least 28 days prior to providing informed consent and continue to maintain a stable diet for the duration of the study
- Good general health other than OTCD, in the opinion of the Investigator
- Willing to refrain from strenuous exercise/activity and alcohol for 72 hours before study visits
- Willingness to comply with procedures and visits
- Willingness to follow contraception guidelines
Exclusion
- History of clinically significant disease(s), in the opinion of the Investigator
- Clinically significant screening laboratory values
- Uncontrolled diabetes
- Clinically significant anemia
- Subjects who develop infection during screening must be asymptomatic for at least 7 days prior to dosing
- Unwillingness to comply with study requirements
- History of positive HIV, hepatitis C, or chronic hepatitis B
- Uncontrolled hypertension
- Malignancy within 5 years prior to study
- Treatment with another investigational drug, biological agent, or device within 30 days of screening, or 5 half-lives of investigational drug
- Treatment with any oligonucleotide or mRNA within 6 months of screening, with exceptions for some vaccinations and investigational treatments
- History of gene therapy, hepatocyte or mesenchymal stem cell transplantation
- Prior organ transplant
- History of severe allergic reaction to a liposomal product
- Recent history of, or current, drug or alcohol abuse
- Dependence on inhaled (smoked or vaped) or oral cannabis products
- Systemic corticosteroids within 6 weeks prior to screening
- Blood donation of 50 to 499 mL within 30 days of screening or of .499 mL within 60 days of screening
- Other conditions, in the opinion of the Investigator, that would make the subject unsuitable for participation
Key Trial Info
Start Date :
November 3 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 30 2024
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT04442347
Start Date
November 3 2020
End Date
April 30 2024
Last Update
August 2 2024
Active Locations (8)
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1
University of Florida
Gainesville, Florida, United States, 32608
2
M Health Fairview Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55454
3
The Mount Sinai Hospital
New York, New York, United States, 10029
4
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224