Nitisinone

DRUG12 trials

Sponsors

University of California, San Diego, Swedish Orphan Biovitrum, University of Liverpool, National Eye Institute (NEI), Cycle Pharmaceuticals Ltd.

Conditions

AlbinismAlkaptonuriaHereditary Tyrosinemia, Type IVision Loss

Phase 1

Taste and Palatability of Orfadin Suspension

CompletedNCT01734889

Swedish Orphan BiovitrumHereditary Tyrosinemia, Type I

Start: 2012-10-31End: 2013-03-31Updated: 2014-11-06

Nitisinone for Type 1B Oculocutaneous Albinism

CompletedNCT01838655

National Eye Institute (NEI)Albinism, Vision Loss

Start: 2013-04-16End: 2017-02-07Updated: 2019-02-26

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

CompletedNCT02750332

Cycle Pharmaceuticals Ltd.Hereditary Tyrosinemia, Type I

Start: 2015-11-30End: 2016-01-31Updated: 2017-03-15

Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin

CompletedNCT02750709

Cycle Pharmaceuticals Ltd.Hereditary Tyrosinemia, Type I

Start: 2015-10-31End: 2016-01-31Updated: 2017-03-15

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

CompletedNCT02750345

Cycle Pharmaceuticals Ltd.Hereditary Tyrosinemia, Type I

Start: 2016-03-31End: 2016-05-31Updated: 2017-06-14

Phase 2

Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria

CompletedNCT01390077

University of California, San DiegoAlkaptonuria

Start: 2011-01-31End: 2016-07-31Updated: 2021-04-22

Dose Response Study of Nitisinone in Alkaptonuria

CompletedNCT01828463

University of LiverpoolAlkaptonuria

Start: 2013-05-31End: 2013-12-31Updated: 2014-06-11

Phase 3

Suitability of Nitisinone in Alkaptonuria 2

University of LiverpoolAlkaptonuria

Start: 2014-04-30End: 2020-02-02Target: 140Updated: 2018-05-31

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

CompletedNCT02323529

Swedish Orphan BiovitrumHereditary Tyrosinemia, Type I

Start: 2014-12-31End: 2015-09-30Updated: 2015-11-11

Unknown Phase

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

CompletedNCT02320084

Swedish Orphan BiovitrumHereditary Tyrosinemia, Type I

Start: 2013-09-30End: 2019-09-30Updated: 2024-09-19

Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

Sutphin DrugsHereditary Tyrosinemia, Type I

Start: 2019-11-01End: 2021-03-31Target: 4Updated: 2019-10-03

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

WithdrawnNCT06227429

Swedish Orphan BiovitrumHereditary Tyrosinemia, Type I

Start: 2025-09-30End: 2027-06-30Updated: 2025-12-23

Related Papers

Nitisinone Treatment Affects Biomarkers of Bone and Cartilage Remodelling in Alkaptonuria Patients

International Journal of Molecular Sciences2023-07-016 citations

Radiological evolution of spinal disease in alkaptonuria and the effect of nitisinone

RMD Open2022-10-017 citations

Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial.

2020-08-1884 citations

One-year pilot study on the effects of nitisinone on melanin in patients with OCA-1B.

2019-01-2420 citations

Open-Label Single-Sequence Crossover Study Evaluating Pharmacokinetics, Efficacy, and Safety of Once-Daily Dosing of Nitisinone in Patients with Hereditary Tyrosinemia Type 1.

2017-06-234 citations