Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

Brief Title

Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

Official Title

Orfadin and Nitinosine Study

Brief Summary

      he purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product are
      bioequivalent to the reference product Orfadin 10 mg

Detailed Description

      Detailed Description:

      The specific aim is to conduct a randomized, single dose, three-period crossover
      bioequivalence study in at least 18 healthy male and female subjects at a single study center
      to evaluate the in vivo performance of two formulations of Nitisinone 10 mg and the reference
      product Orfadin under fasting.

      A total of 4 healthy female and male volunteers (age 18 to 55 years old) will be entered into
      the study. Volunteers will be determined to be free of significant medical conditions as
      assessed by medical history, physical examination, and blood and urine tests. Volunteers will
      be randomly allocated to a treatment sequence, before administration of investigational

      Determination of succinylacetone (SA) in blood (serum/plasma) and/or urine will be performed.
      Results from samples analyzed at the central laboratory, including determination of
      nitisinone, will be used in the evaluation of pharmacokinetics, efficacy and safety during
      the two treatment periods.

Study Type


Primary Outcome

Succinylacetone level


Hereditary Tyrosinemia, Type I



Study Arms / Comparison Groups

 Orfandin .5 mgm/kg mgm bid
Description:  Two participant will receive .5 mgm/kg mgm of orfadin


* 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


Estimated Enrollment


Start Date

November 1, 2019

Completion Date

March 31, 2021

Primary Completion Date

December 31, 2020

Eligibility Criteria

        Inclusion Criteria:

          -  All HT-1 patients receiving Orfadin treatment are eligible for entry.

          -  Male and female patients of all ages diagnosed with HT-1.

          -  Stable lab values, including liver values <2 ULN (ALP, ALT, AST, bilirubin, INR).

          -  Women of childbearing potential willing to use adequate contraception

          -  Signed informed consent/assent.

        Exclusion Criteria:

          -  Any medical condition which in the opinion of the investigator makes the patient
             unsuitable for inclusion.

          -  Enrollment in another concurrent clinical interventional study within three months
             prior to inclusion in this study.

          -  Pregnant women.

          -  Lactating women. .Known hepatitis B, hepatitis C or HIV infection.

          -  Foreseeable inability to cooperate with given instructions or study procedures.




18 Years - 50 Years

Accepts Healthy Volunteers



, 7185260310, [email protected]

Location Countries


Location Countries


Administrative Informations



Organization ID

Global CRO Orfadin

Responsible Party


Study Sponsor

Sutphin Drugs

Study Sponsor

, , 

Verification Date

October 2019