Status:
UNKNOWN
Suitability of Nitisinone in Alkaptonuria 2
Lead Sponsor:
University of Liverpool
Conditions:
Alkaptonuria
Eligibility:
All Genders
25+ years
Phase:
PHASE3
Brief Summary
This is a proposal to develop the orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). Thanks to our existing successful fundamental and clinical rese...
Eligibility Criteria
Inclusion
- A patient must fulfil the following criteria in order to be included in the study:
- 1\. Diagnosis of AKU Any Clinical manifestations of AKU, such as clinical ochronosis or chronic back/joint pain.
- 3\. Age ≥25 years. 4. Willing and able to visit the investigational site for study visits. 5. Signed written informed consent given.
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Exclusion
- The presence of any of the following will exclude a patient from inclusion in the study:
- Currently pregnant or lactating.
- Female patient of child-bearing potential not using a reliable method of contraception.
- Known allergy to nitisinone or any of the constituents of the investigational product.
- Current malignancy.
- Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).
- Unstable cardiovascular disease.
- Serum potassium \< 3.0 mmol/L.
- eGFR \< 60 mL/min .
- ALT \> 1.5 x upper limit of normal.
- Haemoglobin \< 10.0 g/dL.
- Platelets \< 100 x 109/L.
- Total white blood count \< 3.0 x 109/L or neutrophil count \< 1.5 x 109/L.
- History of alcohol or drug abuse.
- Participation in another clinical study within 3 months of randomization.
- Treatment with nitisinone within 60 days of randomization.
- Psychiatric or somatic illness that interferes with compliance or communication with health care personnel.
- Foreseeable inability to cooperate with given instructions or study procedures.
- Any other medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.
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Key Trial Info
Start Date :
April 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 2 2020
Estimated Enrollment :
140 Patients enrolled
Trial Details
Trial ID
NCT01916382
Start Date
April 1 2014
End Date
February 2 2020
Last Update
May 31 2018
Active Locations (1)
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1
Royal Liverpool Hospital
Liverpool, Merseyside, United Kingdom, L7 8XP