Nitisinone

Generic Name: Nitisinone

Brand Names: Nityr, Orfadin

Drug Type: Small Molecule

Chemical Formula: C_₁₄H_₁₀F_₃NO_₅

CAS Number: 104206-65-7

Unique Ingredient Identifier: K5BN214699

Background: Nitisinone is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase. It is used in the treatment of hereditary tyrosinemia type 1. It is sold under the brand name Orfadin.

Indication: Used as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1.

Associated Conditions: Tyrosinemia Type 1

Associated Therapies: -

Clinical Trials

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

Recruiting

Conditions: Hereditary Tyrosinemia, Type I

Interventions: Drug: Nitisinone

First Posted Date: 2024-01-26

Last Posted Date: 2024-02-07

Lead Sponsor: Swedish Orphan Biovitrum

Target Recruit Count: 15

Registration Number: NCT06227429

Locations: 🇨🇳
Swedish Orphan Biovitrum Research Site, Wuhan, China

Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

Not Applicable

Conditions: Hereditary Tyrosinemia, Type I

Interventions: Drug: Orfadin
Drug: Nitisinone

First Posted Date: 2019-10-03

Last Posted Date: 2019-10-03

Lead Sponsor: Sutphin Drugs

Target Recruit Count: 4

Registration Number: NCT04113772

Locations: 🇮🇳
Lifein Multi-Specialty Hospital, Navsari, Gujarat, India

Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)

Phase 1

Completed

Conditions: Hereditary Tyrosinemia, Type I

Interventions: Drug: Nitisinone

First Posted Date: 2016-04-25

Last Posted Date: 2017-03-15

Lead Sponsor: Cycle Pharmaceuticals Ltd.

Target Recruit Count: 20

Registration Number: NCT02750332

Locations: 🇿🇦
Bloemfontein Early Phase Clinical Unit, PAREXEL International (South Africa), Bloemfontein, Free State, South Africa

Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)

Phase 1

Completed

Conditions: Hereditary Tyrosinemia, Type I

Interventions: Drug: Nitisinone
Drug: Nitisinone Baked Tablet
Drug: Orfadin

First Posted Date: 2016-04-25

Last Posted Date: 2017-06-14

Lead Sponsor: Cycle Pharmaceuticals Ltd.

Target Recruit Count: 24

Registration Number: NCT02750345

Locations: 🇿🇦
Bloemfontein Early Phase Clinical Unit, PAREXEL International (South Africa), Bloemfontein, Free State, South Africa

Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin

Phase 1

Completed

Conditions: Hereditary Tyrosinemia, Type I

Interventions: Drug: Nitisinone
Drug: Nitisinone 10 mg Tablet High Compritol
Drug: Orfadin

First Posted Date: 2016-04-25

Last Posted Date: 2017-03-15

Lead Sponsor: Cycle Pharmaceuticals Ltd.

Target Recruit Count: 24

Registration Number: NCT02750709

Locations: 🇿🇦
Bloemfontein Early Phase Clinical Unit, PAREXEL International (South Africa), Bloemfontein, Free State, South Africa

Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1

Phase 3

Completed

Conditions: Hereditary Tyrosinemia, Type I

Interventions: Drug: Nitisinone

First Posted Date: 2014-12-23

Last Posted Date: 2015-11-11

Lead Sponsor: Swedish Orphan Biovitrum

Target Recruit Count: 18

Registration Number: NCT02323529

Locations: 🇸🇪
Swedish Orphan Biovitrum Investigational site, Gothenburg, Sweden
🇩🇪
Swedish Orphan Biovitrum Investigational Site, Reutlingen, Germany

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care

Completed

Conditions: Hereditary Tyrosinemia, Type I

Interventions: Drug: Nitisinone

First Posted Date: 2014-12-19

Last Posted Date: 2020-01-14

Lead Sponsor: Swedish Orphan Biovitrum

Target Recruit Count: 315

Registration Number: NCT02320084

Locations: 🇬🇧
Swedish Orphan Biovitrum Investigational Site, Sheffield, United Kingdom
🇬🇧
Swedish Oprhan Biovitrum Investigational Site, Manchester, United Kingdom

Suitability of Nitisinone in Alkaptonuria 2

Phase 3

Conditions: Alkaptonuria

Interventions: Drug: Nitisinone

First Posted Date: 2013-08-05

Last Posted Date: 2018-05-31

Lead Sponsor: University of Liverpool

Target Recruit Count: 140

Registration Number: NCT01916382

Locations: 🇬🇧
Royal Liverpool Hospital, Liverpool, Merseyside, United Kingdom

Nitisinone for Type 1B Oculocutaneous Albinism

Phase 1

Completed

Conditions: Albinism
Vision Loss

Interventions: Drug: Nitisinone

First Posted Date: 2013-04-24

Last Posted Date: 2019-02-26

Lead Sponsor: National Eye Institute (NEI)

Target Recruit Count: 5

Registration Number: NCT01838655

Locations: 🇺🇸
National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States

Dose Response Study of Nitisinone in Alkaptonuria

Phase 2

Completed

Conditions: Alkaptonuria

Interventions: Drug: Nitisinone

First Posted Date: 2013-04-10

Last Posted Date: 2014-06-11

Lead Sponsor: University of Liverpool

Target Recruit Count: 40

Registration Number: NCT01828463

Locations: 🇬🇧
Royal Liverpool Hospital, Liverpool, Merseyside, United Kingdom

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