Status:
COMPLETED
Dose Response Study of Nitisinone in Alkaptonuria
Lead Sponsor:
University of Liverpool
Collaborating Sponsors:
Liverpool University Hospitals NHS Foundation Trust
Conditions:
Alkaptonuria
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
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 hom...
Eligibility Criteria
Inclusion
- Inclusion criteria
- A subject must fulfil the following criteria in order to be included in the study:
- Diagnosis of alkaptonuria verified by documented elevated urinary homogentisic acid excretion.
- Age ≥18 years.
- Willing and able to visit the investigational site for study visits.
- Signed written informed consent obtained.
- Exclusion criteria
- The presence of any of the following will exclude a subject from inclusion in the study:
- Non-alkaptonuria causes of ochronosis.
- Currently pregnant or lactating.
- Known allergy to nitisinone or any of the constituents of the investigational product.
- Use of a protein-restricted diet
- Dietary habits or use of homeopathic therapies that interfere with tyrosine catabolism.
- Current keratopathy, contact lens use or uncontrolled glaucoma.
- Current malignancy.
- Uncontrolled hypertension (blood pressure greater than 180 systolic or greater than 95 diastolic).
- Electrocardiogram changes indicative of myocardial infarction, arrhythmia, tachycardia, bradycardia, left bundle branch block.
- Chest radiographic abnormalities, including an infiltrative, mass, congestive heart failure, embolism, atelectasis.
- Serum potassium \< 3.0 mmol/L.
- eGFR \< 60 mL/min.
- Any hepatic enzymes greater than 3 x upper limit of normal.
- Haemoglobin \< 10.0 g/dL.
- Platelets less than 100 x 109/L.
- WBC less than 3.0 x 109/L.
- ESR greater than 100 mm/h.
- History of alcohol or drug abuse.
- Participation in another clinical trial within 3 months of randomisation.
- Treatment with nitisinone within 3 months of randomisation
- Psychiatric illness or neurological disease that interferes with compliance or communication with health care personnel.
- Any other medical condition which in the opinion of the investigator makes the subject unsuitable for inclusion.
- Foreseeable inability to cooperate with given instructions or study procedures.
Exclusion
Key Trial Info
Start Date :
May 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2013
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT01828463
Start Date
May 1 2013
End Date
December 1 2013
Last Update
June 11 2014
Active Locations (1)
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1
Royal Liverpool Hospital
Liverpool, Merseyside, United Kingdom, L7 8XP