Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With OTC Deficiency

Brief Title

Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

Official Title

A Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Clinically Stable Patients With Ornithine Transcarbamylase Deficiency

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 Transcarbamylase Deficiency.
    

Detailed Description

      This is a single ascending dose study of ARCT-810 in which approximately 12 (up to a maximum
      of 20) subjects are planned to be enrolled. The length of each study participant is
      approximately 8 weeks from screening to last study visit. The study comprises an up to 4-week
      Screening and a 4-week Diet Run-in period to run concurrently followed by a 1-day dosing
      period and a 28-day Post Treatment Period.

      Study participants will be allocated to one of the three different study groups (also called
      cohorts), to test different doses of ARCT-810. There will be 4 participants in each group.
      Within each cohort, subjects will be randomized 3 :1 to receive ARCT-810 or placebo as an IV
      infusion.
    

Study Phase

Phase 1

Study Type

Interventional


Primary Outcome

Incidence, severity and dose-relationship of adverse events (AEs)

Secondary Outcome

 Change in area under the curve after single dose of ARCT-810

Condition

Ornithine Transcarbamylase Deficiency

Intervention

ARCT-810

Study Arms / Comparison Groups

 ARCT-810
Description:  Ascending single doses of ARCT-810 administered intravenously

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

12

Start Date

November 3, 2020

Completion Date

December 2021

Primary Completion Date

November 2021

Eligibility Criteria

        Inclusion Criteria:

          -  Males and females ≥18 years of age with documented diagnosis of late onset OTC
             deficiency confirmed with genetic testing.

          -  Subject's Ornithine Transcarbamylase deficiency (OTCD) is stable as evidenced by a) no
             clinical symptoms of hyperammonemia and b) 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 for ≥ 28 days
             prior to signing informed consent and must remain on the same regimen throughout the
             screening period

          -  Must have maintained a stable protein restricted diet +/- amino acid supplementation
             for at least 28 days prior to signing informed consent and continue to maintain a
             stable diet for the duration of the study

          -  No clinically significant abnormal findings on medical history, clinical laboratory
             test results (other than ammonia) vital sign measurements, 12-lead ECG results, or
             physical examination

          -  Males must be surgically sterile or willing to use adequate contraception; females
             must be post-menopausal, surgically sterile or willing to use adequate contraception

        Exclusion Criteria:

          -  History of clinically significant disease(s) (other than OTCD)

          -  Abnormal hepatic enzymes, significant renal impairment, clinically significant anemia
             or uncontrolled diabetes

          -  Blood Pressure greater than 160/100 mm Hg

          -  Malignancy within 5 years prior to study

          -  Treatment with another investigational drug, biological agent, or device within one
             month of screening, or 5 half-lives of investigational drug, whichever is longer

          -  Gene therapy within 1 year prior to screening

          -  Prior organ transplant

          -  Positive viral serology test results for HIV type 1 or 2 antibodies, hepatitis B
             surface antigen (HBsAg) or hepatitis C virus (HCV) antibody
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, 858-900-2660, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04442347

Organization ID

ARCT- 810-02


Responsible Party

Sponsor

Study Sponsor

Arcturus Therapeutics, Inc.


Study Sponsor

, , 


Verification Date

November 2021