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Suitability of Nitisinone in Alkaptonuria 2

NCT01916382 · Status: UNKNOWN · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 140

Last updated 2018-05-31

No results posted yet for this study

Summary

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.

Conditions

  • Alkaptonuria

Interventions

DRUG

Nitisinone

drug

Sponsors & Collaborators

  • University of Liverpool

    lead OTHER

Principal Investigators

  • L Ranganath · Liverpool University Hospitals NHS Foundation Trust

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-04-30
Primary Completion
2016-02-29
Completion
2020-02-02

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01916382 on ClinicalTrials.gov