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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04113772
Recruitment Status : Unknown
Verified October 2019 by Sutphin Drugs.
Recruitment status was:  Not yet recruiting
First Posted : October 3, 2019
Last Update Posted : October 3, 2019
Information provided by (Responsible Party):
Sutphin Drugs

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

Condition or disease Intervention/treatment Phase
Hereditary Tyrosinemia, Type I Drug: Nitisinone Drug: Orfadin Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 4 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Official Title: Orfadin and Nitinosine Study
Estimated Study Start Date : November 1, 2019
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : March 31, 2021

Arm Intervention/treatment
Active Comparator: Orfandin .5 mgm/kg mgm bid
Two participant will receive .5 mgm/kg mgm of orfadin
Drug: 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

Primary Outcome Measures :
  1. Succinylacetone level [ Time Frame: 8 weeks ]
    Succinylacetone level will be measured every 2 weeks for eight weeks

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04113772

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Contact: Ajai Prakash 7185260310 ajaiprakashny@gmail.com

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Lifein Multi-Specialty Hospital
Navsari, Gujarat, India, 396421
Sponsors and Collaborators
Sutphin Drugs
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Responsible Party: Sutphin Drugs
ClinicalTrials.gov Identifier: NCT04113772    
Other Study ID Numbers: Global CRO Orfadin
First Posted: October 3, 2019    Key Record Dates
Last Update Posted: October 3, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action