Suitability of Nitisinone in Alkaptonuria 2

Alkaptonuria Clinical Trial

— SONIA 2  

Official title:

An International, Multicentre, Randomised, Evaluator-blind, No-treatment Controlled, Parallel-group Study to Assess the Efficacy and Safety of Once Daily Nitisinone in Patients With Alkaptonuria After 12 Months of Treatment, Followed by an Additional 36 Month Treatment Period.

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT01916382
• Other study ID #: SONIA 2
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: April 2014
• Est. completion date: February 2, 2020

Study information

• Verified date: May 2018
• Source: University of Liverpool
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 research (cell models, animal models, natural history studies), we are now ready for this final stage of clinical development of nitisinone for AKU: a phase 3 clinical trial to prove efficacy. The results of DevelopAKUre will allow us to make the case to the European Medicines Agency for marketing authorisation of nitisinone for AKU, thereby contributing to the goal of the International Rare Diseases Research Consortium of developing 200 new therapies by 2020.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 140
• Est. completion date: February 2, 2020
• Est. primary completion date: February 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 25 Years and older

Eligibility

Inclusion Criteria:

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.

Exclusion Criteria:

The presence of any of the following will exclude a patient from inclusion in the study:

1. Currently pregnant or lactating.

2. Female patient of child-bearing potential not using a reliable method of contraception.

3. Known allergy to nitisinone or any of the constituents of the investigational product.

4. Current malignancy.

5. Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).

6. Unstable cardiovascular disease.

7. Serum potassium < 3.0 mmol/L.

8. eGFR < 60 mL/min .

9. ALT > 1.5 x upper limit of normal.

10. Haemoglobin < 10.0 g/dL.

11. Platelets < 100 x 109/L.

12. Total white blood count < 3.0 x 109/L or neutrophil count < 1.5 x 109/L.

13. History of alcohol or drug abuse.

14. Participation in another clinical study within 3 months of randomization.

15. Treatment with nitisinone within 60 days of randomization.

16. Psychiatric or somatic illness that interferes with compliance or communication with health care personnel.

17. Foreseeable inability to cooperate with given instructions or study procedures.

18. Any other medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.

Related Conditions & MeSH terms

• Alkaptonuria

Intervention

Drug:
• Nitisinone
drug

Locations

Country	Name	City	State
United Kingdom	Royal Liverpool Hospital	Liverpool	Merseyside

Sponsors (1)

Lead Sponsor	Collaborator
University of Liverpool

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	24 houre Urine Homogentisic acid		year 1