Suitability of Nitisinone in Alkaptonuria 2

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE3

Total Enrollment

140 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-04-30

Study Completion Date

2020-02-02

Brief Summary

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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.

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Detailed Description

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Conditions

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Alkaptonuria

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Nitisinone

Homogentisic acid lowering drug intervention

Group Type EXPERIMENTAL

Nitisinone

Intervention Type DRUG

drug

No treatment

comparrator

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Nitisinone

drug

Intervention Type DRUG

Other Intervention Names

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Orfadin

Eligibility Criteria

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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.

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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.

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Minimum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No