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Evaluation of Nitisinone in the Indian Patients of Hereditary Tyrosinemia Type 1

Pharmacokinetic and Dynamic Correlation for optimum therapy of NTBC for the Treatment of Indian Patients with Hereditary Tyrosinemia Type 1

Status
Recruiting
Phases
Unknown
Study type
Interventional
Source
CTRI
Registry ID
CTRI/2023/05/052626
Enrollment
15
Registered
2023-05-15
Start date
Unknown
Completion date
Unknown
Last updated
2026-02-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Health Condition 1: E72- Other disorders of amino-acid metabolism

Interventions

Intervention1: 2-[2-nitro-4-trifluoromethylbenzoyl]-1,3-cyclohexanedione (NTBC, nitisinone): NTBC (Nitisinone) at a dose of 1 mg/kg/day orally, daily for 36 months

Sponsors

Department of Science & Technology DST
Lead Sponsor

Eligibility

Inclusion criteria

Inclusion criteria: Molecularly and/or biochemically proven patients with hereditary tyrosinemia type 1

Exclusion criteria

Exclusion criteria: Poor compliance

Design outcomes

Primary

MeasureTime frame
Pharmacokinetic profile for the NTBC in Indian patients with Hereditary Tyrosinemia type 1Timepoint: 0, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, and 36 months—

Secondary

MeasureTime frame
Pharmacodynamic profile for the NTBC in Indian patients with Hereditary Tyrosinemia type 1Timepoint: 0, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, and 36 months—

Countries

India

Contacts

Public ContactThirumurthy Velpandian

All India Institute of Medical Sciences

tvelpandian@hotmail.com

Outcome results

None listed

Source: CTRI (via WHO ICTRP) · Data processed: Feb 7, 2026