Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine (Orfadin)

Orfadin and Nitinosine Study

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

Study Overview

• Status: Unknown
• Conditions: Hereditary Tyrosinemia, Type I
• Intervention / Treatment: Drug: Orfadin; Drug: Nitisinone

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 nitinosine

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: Interventional
• Enrollment (Anticipated): 4
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Ajai Prakash; Phone Number: 7185260310; Email: ajaiprakashny@gmail.com

Study Locations

• India
  • Gujarat
    • Navsari, Gujarat, India, 396421
      • Lifein Multi-Specialty Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 48 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Orfandin .5 mgm/kg mgm bid; Two participant will receive .5 mgm/kg mgm of orfadin	Drug: Orfadin; Orfadin
Experimental: Nitinosine .5 mgm/kg bid; Two participant will receive .5 mgm/kg of nitinosine	Drug: Nitisinone; Measure bio equivalency/efficacy of nitinosine and orfadin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Succinylacetone level	Succinylacetone level will be measured every 2 weeks for eight weeks	8 weeks

Collaborators and Investigators

• Sponsor: Sutphin Drugs

Study record dates

Study Major Dates

• Study Start (Anticipated): November 1, 2019
• Primary Completion (Anticipated): December 31, 2020
• Study Completion (Anticipated): March 31, 2021

Study Registration Dates

• First Submitted: October 1, 2019
• First Submitted That Met QC Criteria: October 2, 2019
• First Posted (Actual): October 3, 2019

Study Record Updates

• Last Update Posted (Actual): October 3, 2019
• Last Update Submitted That Met QC Criteria: October 2, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Tyrosinemias; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Nitisinone
• Other Study ID Numbers: Global CRO Orfadin

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No