Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT04113772
• Date of registration: 01/10/2019
• Prospective Registration: Yes
• Primary sponsor: Sutphin Drugs
• Public title: Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine Orfadin
• Scientific title: Orfadin and Nitinosine Study
• Date of first enrolment: November 1, 2019
• Target sample size: 4
• Recruitment status: Not yet recruiting
• URL: https://clinicaltrials.gov/show/NCT04113772
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Single (Participant).
• Phase: N/A

Countries of recruitment

India

Contacts

• Name: Ajai Prakash

Key inclusion & exclusion criteria

Inclusion Criteria:

- All HT-1 patients receiving Orfadin treatment are eligible for entry.

- Male and female patients of all ages diagnosed with HT-1.

- Stable lab values, including liver values <2 ULN (ALP, ALT, AST, bilirubin, INR).

- Women of childbearing potential willing to use adequate contraception

- Signed informed consent/assent.

Exclusion Criteria:

- Any medical condition which in the opinion of the investigator makes the patient
unsuitable for inclusion.

- Enrollment in another concurrent clinical interventional study within three months
prior to inclusion in this study.

- Pregnant women.

- Lactating women. .Known hepatitis B, hepatitis C or HIV infection.

- Foreseeable inability to cooperate with given instructions or study procedures.



Age minimum: 18 Years
Age maximum: 50 Years
Gender: All

Health Condition(s) or Problem(s) studied

Hereditary Tyrosinemia, Type I

Intervention(s)

Drug: Orfadin

Drug: Nitisinone

Primary Outcome(s)

Succinylacetone level [Time Frame: 8 weeks]

Secondary ID(s)

Global CRO Orfadin

Source(s) of Monetary Support

Please refer to primary and secondary sponsors