Trial record 3 of 5 for:    "Alkaptonuria"

Suitability of Nitisinone in Alkaptonuria 2 (SONIA 2)

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified August 2013 by University of Liverpool
Sponsor:
Information provided by (Responsible Party):
Professor Lakshminarayan Ranganath, University of Liverpool
ClinicalTrials.gov Identifier:
NCT01916382
First received: August 2, 2013
Last updated: August 5, 2013
Last verified: August 2013
  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
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: 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 University of Liverpool:

Primary Outcome Measures:
  • 24 houre Urine Homogentisic acid [ Time Frame: year 1 ] [ Designated as safety issue: No ]

Estimated Enrollment: 140
Study Start Date: November 2013
Estimated Study Completion Date: April 2018
Estimated Primary Completion Date: April 2015 (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

Ages Eligible for Study:   25 Years and older
Genders Eligible for Study:   Both
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.

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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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  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: NCT01916382

Locations
United Kingdom
Royal Liverpool Hospital Not yet recruiting
Liverpool, Merseyside, United Kingdom, L7 8XP
Contact: Helen Bygott    0151 706 4197    helen.bygott@rlbuht.nhs.uk   
Sponsors and Collaborators
University of Liverpool
Investigators
Principal Investigator: L Ranganath Royal Liverpool & Broadgreen University Hospitals NHS Trust
  More Information

No publications provided

Responsible Party: Professor Lakshminarayan Ranganath, Professor, University of Liverpool
ClinicalTrials.gov Identifier: NCT01916382     History of Changes
Other Study ID Numbers: SONIA 2
Study First Received: August 2, 2013
Last Updated: August 5, 2013
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by 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
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 19, 2014