Efficacy and safety of 4 years treatment with nitisinone in patients with Alkaptonuria (AKU).

Phase 1

• Conditions: Alkaptonuria (AKU) - a serious, autosomal recessive, multisystem disorder.; MedDRA version: 20.0 Level: PT Classification code 10001689 Term: Alkaptonuria System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2013-001633-41-SK
• Lead Sponsor: niversity of Liverpool (UniLiv)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-10-14
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 140

Inclusion Criteria

1. Diagnosis of AKU.
2. 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 140
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Treatment with nitisinone within 3 months of randomization.
2. Participation in another clinical study within 3 months of randomization.
3. Known allergy to nitisinone or any of the constituents of the investigational product.
4. Female patient of child-bearing potential not using a reliable method of contraception.
5. Currently pregnant or lactating.
6. Current malignancy.
7. Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).
8. Unstable cardiovascular disease.
9. Clinically relevant lab abnormalities.
10. History of alcohol or drug abuse.
11. Psychiatric or somatic illness that interferes with compliance or communication with health care personnel.
12. Foreseeable inability to cooperate with given instructions or study procedures.
13. Any other medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified