Suitability of Nitisinone in Alkaptonuria 2

Phase 3

• Conditions: Alkaptonuria
• Interventions: Drug: Nitisinone

• Registration Number: NCT01916382
• Lead Sponsor: University of Liverpool

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 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-08-02
• Study First Submitted QC: 2013-08-02
• Study First Posted: 2013-08-05
• Study Start: 2014-04
• Primary Completion: 2016-02
• Status Verified: 2018-05
• Last Update Submitted: 2018-05-30
• Last Update Posted: 2018-05-31
• Study Completion: 2020-02-02

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 140

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.

2. 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Nitisinone	Nitisinone	Homogentisic acid lowering drug intervention

Primary Outcome Measures

Name	Time	Method
24 houre Urine Homogentisic acid	year 1

Trial Locations

Locations (1)

Royal Liverpool Hospital; 🇬🇧 Liverpool, Merseyside, United Kingdom