A study to assess the safety and effectiveness of the medicine called Nitisinone 2 and 5 mg once daily among patients who have the rare genetic disease named Alkaptonuria.
Phase 3
- Conditions
- Health Condition 1: E702- Disorders of tyrosine metabolism
- Registration Number
- CTRI/2024/03/063760
- Lead Sponsor
- INDIAN COUNCIL OF MEDICAL RESEARCH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Yet Recruiting
- Sex
- Not specified
- Target Recruitment
- 0
Inclusion Criteria
Patients harboring HGD pathogenic variant.
Any Clinical manifestations of AKU, such as clinical ochronosis or chronic back/joint pain.
Age 18 – 45 years.
Willing and able to visit the investigational site for study visits.
Signed written informed consent given.
Exclusion Criteria
Currently pregnant or lactating.
Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).
•Unstable cardiovascular disease.
History of alcohol or drug abuse.
Psychiatric or somatic illness that interferes with compliance or communication with health care personnel.
Any other medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 90-95% reduction in the urine and serum HGA levels after 3 months of treatment <br/ ><br>Timepoint: Three months
- Secondary Outcome Measures
Name Time Method Secondary Outcome/s: <br/ ><br> <br/ ><br>Effect of Nitisinone on clinical parameters will be clearly understood. <br/ ><br> <br/ ><br>The safe & effective dose for Indian population will be identified. <br/ ><br> <br/ ><br>Prevention of debilitating joint disabilities by early initiation of the treatment.Timepoint: 6 months to one year