Health Condition 1: E702- Disorders of tyrosine metabolism
Conditions
Interventions
Intervention1: Nitisinone Capsules 2 mg: Each hard-gelatin capsule contains 2 mg of Nitisinone once daily oral for 6 months
Control Intervention1: Nitisinone Capsules 5 mg: Each hard-gelatin capsule c
Sponsors
INDIAN COUNCIL OF MEDICAL RESEARCH
Eligibility
Inclusion criteria
Inclusion criteria: Patients harboring HGD pathogenic variant. Any Clinical manifestations of AKU, such as clinical ochronosis or chronic back/joint pain. Age 18 ââ?¬â?? 45 years. Willing and able to visit the investigational site for study visits. Signed written informed consent given.
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 90-95% reduction in the urine and serum HGA levels after 3 months of treatment Timepoint: Three months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Outcome/s: Effect of Nitisinone on clinical parameters will be clearly understood. The safe & effective dose for Indian population will be identified. Prevention of debilitating joint disabilities by early initiation of the treatment.Timepoint: 6 months to one year | — |
Countries
India
Contacts
Public ContactDr. Sumita Danda
Christian Medical College, Vellore
Outcome results
None listed