Clinical trials: Homogentisic Acid
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
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
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)