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Dose Response Study of Nitisinone in Alkaptonuria (SONIA1)

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.
 
ClinicalTrials.gov Identifier: NCT01828463
Recruitment Status : Completed
First Posted : April 10, 2013
Last Update Posted : June 11, 2014
Sponsor:
Collaborator:
Liverpool University Hospitals NHS Foundation Trust
Information provided by (Responsible Party):
Professor Lakshminarayan Ranganath, University of Liverpool

Tracking Information
First Submitted Date  ICMJE April 5, 2013
First Posted Date  ICMJE April 10, 2013
Last Update Posted Date June 11, 2014
Study Start Date  ICMJE May 2013
Actual Primary Completion Date October 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 8, 2013)
dose of nitisinone that decreases urinary homogentisic acid to near normal [ Time Frame: 4 weeks ]
Original Primary Outcome Measures  ICMJE
 (submitted: April 5, 2013)
dose of nitisinone that decreases urinary homgenistic acid to near normal [ Time Frame: 4 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Dose Response Study of Nitisinone in Alkaptonuria
Official Title  ICMJE An International, Multicentre, Randomised, Open-label, No-treatment Controlled, Parallel-group, Dose-response Study to Investigate the Effect of Once Daily Nitisinone on 24-hour Urinary Homogentisic Acid Excretion in Patients With Alkaptonuria After 4 Weeks Treatment.
Brief Summary SONIA 1 is an international, multicentre, randomised, open-label, no-treatment controlled, parallel group, dose-response study to investigate the effect of once daily nitisinone on 24-hour urinary homogentisic acid excretion in patients with alkaptonuria after 4-weeks treatment. They study will identify the optimal dose to decrease urine homogentisic acid to near normal levels.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Alkaptonuria
Intervention  ICMJE Drug: Nitisinone
doses 1, 2, 4 & 8 mg plus no treatment arm
Study Arms  ICMJE
  • No Intervention: no treatment
    comparrator
  • Experimental: Nitisinone 1mg
    interventional
    Intervention: Drug: Nitisinone
  • Experimental: Nitisinone 2mg
    interventional
    Intervention: Drug: Nitisinone
  • Experimental: Nitisinone 4mg
    interventional
    Intervention: Drug: Nitisinone
  • Experimental: Nitisinone 8mg
    interventional
    Intervention: Drug: Nitisinone
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 5, 2013)
40
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 2013
Actual Primary Completion Date October 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion criteria

A subject must fulfil the following criteria in order to be included in the study:

  1. Diagnosis of alkaptonuria verified by documented elevated urinary homogentisic acid excretion.
  2. Age ≥18 years.
  3. Willing and able to visit the investigational site for study visits.
  4. Signed written informed consent obtained.

Exclusion criteria

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

  1. Non-alkaptonuria causes of ochronosis.
  2. Currently pregnant or lactating.
  3. Known allergy to nitisinone or any of the constituents of the investigational product.
  4. Use of a protein-restricted diet
  5. Dietary habits or use of homeopathic therapies that interfere with tyrosine catabolism.
  6. Current keratopathy, contact lens use or uncontrolled glaucoma.
  7. Current malignancy.
  8. Uncontrolled hypertension (blood pressure greater than 180 systolic or greater than 95 diastolic).
  9. Electrocardiogram changes indicative of myocardial infarction, arrhythmia, tachycardia, bradycardia, left bundle branch block.
  10. Chest radiographic abnormalities, including an infiltrative, mass, congestive heart failure, embolism, atelectasis.
  11. Serum potassium < 3.0 mmol/L.
  12. eGFR < 60 mL/min.
  13. Any hepatic enzymes greater than 3 x upper limit of normal.
  14. Haemoglobin < 10.0 g/dL.
  15. Platelets less than 100 x 109/L.
  16. WBC less than 3.0 x 109/L.
  17. ESR greater than 100 mm/h.
  18. History of alcohol or drug abuse.
  19. Participation in another clinical trial within 3 months of randomisation.
  20. Treatment with nitisinone within 3 months of randomisation
  21. Psychiatric illness or neurological disease that interferes with compliance or communication with health care personnel.
  22. Any other medical condition which in the opinion of the investigator makes the subject unsuitable for inclusion.
  23. Foreseeable inability to cooperate with given instructions or study procedures.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01828463
Other Study ID Numbers  ICMJE UoL000928
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Professor Lakshminarayan Ranganath, University of Liverpool
Study Sponsor  ICMJE University of Liverpool
Collaborators  ICMJE Liverpool University Hospitals NHS Foundation Trust
Investigators  ICMJE
Principal Investigator: L Ranganath Royal Liverpool & Broadgreen University Hospitals NHS Truts
PRS Account University of Liverpool
Verification Date June 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP