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Suitability of Nitisinone in Alkaptonuria 2 (SONIA 2)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01916382
First Posted: August 5, 2013
Last Update Posted: January 13, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Professor Lakshminarayan Ranganath, University of Liverpool
  Purpose
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.

Condition Intervention Phase
Alkaptonuria Drug: Nitisinone Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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.

Resource links provided by NLM:


Further study details as provided by Professor Lakshminarayan Ranganath, University of Liverpool:

Primary Outcome Measures:
  • 24 houre Urine Homogentisic acid [ Time Frame: year 1 ]

Estimated Enrollment: 140
Study Start Date: April 2014
Estimated Study Completion Date: April 2018
Estimated Primary Completion Date: February 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Nitisinone
Homogentisic acid lowering drug intervention
Drug: Nitisinone
drug
Other Name: Orfadin
No Intervention: No treatment
comparrator

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   25 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01916382


Locations
United Kingdom
Royal Liverpool Hospital
Liverpool, Merseyside, United Kingdom, L7 8XP
Sponsors and Collaborators
University of Liverpool
Investigators
Principal Investigator: L Ranganath Royal Liverpool and Broadgreen University Hospitals NHS Trust
  More Information

Responsible Party: Professor Lakshminarayan Ranganath, Professor, University of Liverpool
ClinicalTrials.gov Identifier: NCT01916382     History of Changes
Other Study ID Numbers: SONIA 2
First Submitted: August 2, 2013
First Posted: August 5, 2013
Last Update Posted: January 13, 2016
Last Verified: August 2013

Keywords provided by Professor Lakshminarayan Ranganath, University of Liverpool:
Alkaptonuria, nitisinone,Ochronosis, Homogentisic acid

Additional relevant MeSH terms:
Alkaptonuria
Ochronosis
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Pathologic Processes
Nitisinone
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action