NCT04113772

Brief Summary

he purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product are bioequivalent to the reference product Orfadin 10 mg

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
4

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Nov 2019

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 1, 2019

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 3, 2019

Completed
29 days until next milestone

Study Start

First participant enrolled

November 1, 2019

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2020

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2021

Completed
Last Updated

October 3, 2019

Status Verified

October 1, 2019

Enrollment Period

1.2 years

First QC Date

October 1, 2019

Last Update Submit

October 2, 2019

Conditions

Hereditary Tyrosinemia, Type I

Outcome Measures

Primary Outcomes (1)

  • Succinylacetone level

    Succinylacetone level will be measured every 2 weeks for eight weeks

    8 weeks

Study Arms (2)

Orfandin .5 mgm/kg mgm bid

ACTIVE COMPARATOR

Two participant will receive .5 mgm/kg mgm of orfadin

Drug: Orfadin

Nitinosine .5 mgm/kg bid

EXPERIMENTAL

Two participant will receive .5 mgm/kg of nitinosine

Drug: Nitisinone

Interventions

NitisinoneDRUG

Measure bio equivalency/efficacy of nitinosine and orfadin

Nitinosine .5 mgm/kg bid
OrfadinDRUG

Orfadin

Orfandin .5 mgm/kg mgm bid

Eligibility Criteria

Age18 Years - 50 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • Pregnant women.
  • Lactating women. .Known hepatitis B, hepatitis C or HIV infection.
  • Foreseeable inability to cooperate with given instructions or study procedures.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Lifein Multi-Specialty Hospital

Navsari, Gujarat, 396421, India

Location

MeSH Terms

Conditions

Tyrosinemias

Interventions

nitisinone

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

Ajai Prakash

CONTACT

7185260310ajaiprakashny@gmail.com

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 1, 2019

First Posted

October 3, 2019

Study Start

November 1, 2019

Primary Completion

December 31, 2020

Study Completion

March 31, 2021

Last Updated

October 3, 2019

Record last verified: 2019-10

Data Sharing

IPD Sharing
Will not share

Locations

IN(1)