Suitability of Nitisinone in Alkaptonuria 2 (SONIA 2)

May 30, 2018 updated by: Professor Lakshminarayan Ranganath, University of Liverpool

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.

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

140

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
    • Merseyside
      • Liverpool, Merseyside, United Kingdom, L7 8XP
        • Royal Liverpool Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

25 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

    -

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Nitisinone
Homogentisic acid lowering drug intervention
Drug: Nitisinone
drug
Other Names:
  • Orfadin
No Intervention: No treatment
comparrator

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
24 houre Urine Homogentisic acid
Time Frame: year 1
year 1

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Liverpool

Investigators

  • Principal Investigator: L Ranganath, Liverpool University Hospitals NHS Foundation Trust

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 1, 2014

Primary Completion (Actual)

February 1, 2016

Study Completion (Anticipated)

February 2, 2020

Study Registration Dates

First Submitted

August 2, 2013

First Submitted That Met QC Criteria

August 2, 2013

First Posted (Estimate)

August 5, 2013

Study Record Updates

Last Update Posted (Actual)

May 31, 2018

Last Update Submitted That Met QC Criteria

May 30, 2018

Last Verified

May 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SONIA 2

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Alkaptonuria

Clinical Trials on Nitisinone

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Norway

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe