Trial record 5 of 5 for:    "Alkaptonuria"

Dose Response Study of Nitisinone in Alkaptonuria (SONIA1)

This study has been completed.
Sponsor:
Collaborator:
Royal Liverpool and Broadgreen University Hospitals NHS Trust
Information provided by (Responsible Party):
Professor Lakshminarayan Ranganath, University of Liverpool
ClinicalTrials.gov Identifier:
NCT01828463
First received: April 5, 2013
Last updated: June 10, 2014
Last verified: June 2014
  Purpose

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.


Condition Intervention Phase
Alkaptonuria
Drug: Nitisinone
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Pharmacokinetics/Dynamics Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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.

Resource links provided by NLM:


Further study details as provided by University of Liverpool:

Primary Outcome Measures:
  • dose of nitisinone that decreases urinary homogentisic acid to near normal [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]

Enrollment: 40
Study Start Date: May 2013
Study Completion Date: December 2013
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
No Intervention: no treatment
comparrator
Experimental: Nitisinone 1mg
interventional
Drug: Nitisinone
doses 1, 2, 4 & 8 mg plus no treatment arm
Experimental: Nitisinone 2mg
interventional
Drug: Nitisinone
doses 1, 2, 4 & 8 mg plus no treatment arm
Experimental: Nitisinone 4mg
interventional
Drug: Nitisinone
doses 1, 2, 4 & 8 mg plus no treatment arm
Experimental: Nitisinone 8mg
interventional
Drug: Nitisinone
doses 1, 2, 4 & 8 mg plus no treatment arm

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01828463

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

No publications provided

Responsible Party: Professor Lakshminarayan Ranganath, Consultant in Metabolic medicine, University of Liverpool
ClinicalTrials.gov Identifier: NCT01828463     History of Changes
Other Study ID Numbers: UoL000928
Study First Received: April 5, 2013
Last Updated: June 10, 2014
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

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
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 01, 2014