Clinical trials: Alkaptonuria
Tyrosinemia, Type I, Tyrosinemia, Type II
Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies
The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of Zero minis, a range of protein substitute tablets for use in the dietary management of children with either TYROSINAEMIA…
- Conditions
- Tyrosinemia, Type I, Tyrosinemia, Type II, Tyrosinemia, Type III, Alkaptonuria
- Trial name
- ZeroMinisMR (NCT06298292)
- Testing
- Zero minis (range of protein substitutes in tablet form)
- Volunteers
- 15 people
- Sponsor
- metaX Institut fuer Diatetik GmbH
Alkaptonuria
Dose Response Study of Nitisinone in Alkaptonuria
SONIA 1 is an international, multicentre, randomised, open-label, no-treatment controlled, parallel group, dose-response study to investigate the effect of once daily nitisinone on 24-hour urinary homogentisic acid excretion in…
- Trial name
- SONIA1 (NCT01828463)
- Testing
- Nitisinone
- Where
- United Kingdom
- Volunteers
- 40 people
- Sponsor
- University of Liverpool
Alkaptonuria
Individualised Gait Modification Strategies in Alkaptonuria Patients
This study evaluates the efficacy of a gait modification intervention using real-time biofeedback on reducing the knee joint loading in Alkaptonuria patients during treadmill walking.
- Trial name
- NCT04142671
- Testing
- Individualised gait modification intervention
- Volunteers
- 30 people
- Sponsor
- Liverpool John Moores University
Alkaptonuria
Long-Term Study of Nitisinone to Treat Alkaptonuria
This 3-year study will examine the safety and effectiveness of long-term use of nitisinone (Orfadin) for treating joint problems in patients with alkaptonuria, an inherited disease in which a compound called homogentisic acid…
- Trial name
- NCT00107783
- Testing
- Nitisinone (NTBC)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Alkaptonuria
Suitability of Nitisinone in Alkaptonuria 2
This is a proposal to develop the orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU).
- Trial name
- SONIA 2 (NCT01916382)
- Testing
- Nitisinone
- Where
- United Kingdom
- Volunteers
- 140 people
- Sponsor
- University of Liverpool
Alkaptonuria
Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria
Nitisinone is a potent inhibitor of the enzyme that catalyzes the formation of homogentisic acid, and should be an even more logical treatment for alkaptonuria than for tyrosinemia, for which it has been approved by the FDA.The…
- Trial name
- NCT01390077
- Testing
- Nitisinone
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of California, San Diego
Alkaptonuria, Homogentisic Acid
Questionnaire Follow=up Study Sonia 2
Survey of health of patients completing SONIA 2 study: A cross-sectional analysis by questionnaire
- Conditions
- Alkaptonuria, Homogentisic Acid, Homogentisate Dioxygenase, Alkaptonuria Severity Score Index
- Trial name
- QSonia (NCT04510142)
- Where
- United Kingdom
- Volunteers
- 24 people
- Sponsor
- Liverpool University Hospitals NHS Foundation Trust
Tyrosinemia, AKU
Evaluation of TYR Sphere in France
10 participants aged 3 years and over with tyrosinaemia or alkaptonuria will be prescribed Tyr sphere following an assessment of their individual needs by their dietitian.
- Trial name
- TYR sphere Fra (NCT04761588)
- Testing
- Tyr sphere
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Vitaflo International, Ltd
Tyrosinemias, Alkaptonuria
Evaluation of TYR Sphere
For 28 days, 20 patients with tyrosinaemia will take TYR sphere as part of the usual dietary management of their condition. Patients will record their adherence to recommended intakes each day, gastrointestinal symptoms (if any)…
- Conditions
- Tyrosinemias, Alkaptonuria, Tyrosinemia, Type I, Tyrosinemia, Type II
- Trial name
- NCT04196959
- Testing
- TYR sphere
- Where
- 2 sites in United Kingdom
- Volunteers
- 18 people
- Sponsor
- Vitaflo International, Ltd
Alkaptonuria
Study of Alkaptonuria
The purpose of this study is to gain a better understanding of alkaptonuria and collect medical data on patients who may later participate in new drug trials for this rare genetic disease.
- Trial name
- NCT00005909
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- National Human Genome Research Institute (NHGRI)