A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

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Ornithine Transcarbamylase Deficiency
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Brief Title

A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

Official Title

A Single-Center Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve From Time Zero to 24 Hours and Rate of Ureagenesis in Healthy Adult Subjects

Brief Summary

      The objective of the study is to characterize 24-hour plasma ammonia levels, characterize
      urea production rates in healthy normal subjects.

Detailed Description

      During Part 1, eligible subjects will be asked to participate in 3 inpatient visits, each
      lasting up to 3 days (Day -1 to Day 2). Each visit will assess 24-hour ammonia levels in
      plasma and rate of urea production for 4 hours following ingestion of [1-13C]sodium acetate.
      Sodium acetate is used as a tracer to measure the rate of ureagenesis.

      During Part 2, eligible subjects will be asked to participate in 1 inpatient visit, lasting
      up to 3 days (Day -1 to Day 2). The visit will assess 24-hour ammonia levels in plasma and
      rate of urea production for 4 hours following ingestion of [1-13C]sodium acetate. Sodium
      acetate is used as a tracer to measure the rate of ureagenesis.

Study Type

Observational

Primary Outcome

Plasma Ammonia Area Under the Curve (AUC0-24)

Condition

Ornithine Transcarbamylase Deficiency

Intervention

No Intervention

Study Arms / Comparison Groups

 Part 1
Description:  30 eligible subjects will be asked to participate in 3 inpatient visits, each lasting up to 3 days (Day -1 to Day 2). Each visit will assess 24-hour ammonia levels in plasma and rate of urea production for 4 hours following ingestion of [1-13C]sodium acetate. Sodium acetate is used as a tracer to measure the rate of ureagenesis.

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

Other

Estimated Enrollment

120

Start Date

August 2, 2019

Completion Date

February 20, 2020

Primary Completion Date

February 20, 2020

Eligibility Criteria

        Inclusion Criteria:

          -  Body mass index between 18 and 30 kg/m2, inclusive.

        Exclusion Criteria:

          -  History of liver disease as evidenced by any of the following: portal hypertension,
             ascites, splenomegaly, esophageal varices, hepatic encephalopathy, or a liver biopsy
             with evidence of stage 3 fibrosis.

          -  Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the
             following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate
             aminotransferase (AST) greater than the upper limit of normal (ULN), total bilirubin
             >1.5 × ULN, alkaline phosphatase >2.5 × ULN. NOTE: the ALT and/or AST levels may be
             repeated.

          -  Subject has a history of gout.

          -  Plasma ammonia level that is not within normal limits at Screening in the opinion of
             the Investigator or Sponsor.

          -  Received any vaccine within 14 days prior to Screening.

          -  Pregnant, lactating, or intending to become pregnant at any time during the study.

          -  Blood transfusion within 8 weeks prior to Screening.diuretics, cyclophosphamide and
             other cytotoxic agents, tolbutamide, chlorpropamide, diazoxide, dichlorphenamide,
             pyrazinamide, probenecid, theophylline/aminophylline, riluzole, warfarin and other
             antithrombotic agents, supplements containing aluminum hydroxide, or iron supplements
             within 30 days of Part 1or Part 2.

Gender

All

Ages

18 Years - 55 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Medical Director, ,

Location Countries

United States

Location Countries

United States

Administrative Informations

NCT ID

NCT04269122

Organization ID

301NHV01

Responsible Party

Sponsor

Study Sponsor

Ultragenyx Pharmaceutical Inc

Study Sponsor

Medical Director, Study Director, Ultragenyx Pharmaceutical

Verification Date

March 2020