An Open-label, Non-randomized, Parallel Group Study in Subjects With Mild and Moderate Hepatic Insufficiency and Healthy Volunteers

NCT ID: NCT01767103

Last Updated: 2014-09-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-01-31
• Study Completion Date: 2014-04-30

Brief Summary

Multi-center, non-randomized, open-label, single-dose, parallel group study to determine the effect of impaired hepatic function on the PK of deferiprone and its 3-O-glucuronide metabolite following a single oral dose of 33mg/kg Ferriprox®.

Detailed Description

Post-marketing study to evaluate the effect of impaired hepatic function on the pharmacokinetics (PK) of deferiprone and its 3-O-glucuronide metabolite and on the safety of Ferriprox® in subjects with mild and moderate hepatic impairment as compared to healthy volunteers.

Conditions

Hepatic Impairment

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Blinding Strategy: NONE

Study Groups

Normal Hepatic Function (healthy volunteers)

Healthy volunteers with normal hepatic function received a single 33 mg/kg dose of Ferriprox®.

Group Type: EXPERIMENTAL

Ferriprox®

Intervention Type: DRUG

Mild Hepatic Failure

Subjects with mild hepatic failure as defined by the Child-Pugh Class C: 5-6 points received a single 33 mg/kg dose of Ferriprox®.

Group Type: EXPERIMENTAL

Ferriprox®

Intervention Type: DRUG

Moderate Hepatic Failure

Subjects with moderate hepatic failure as defined by the Child-Pugh Class B: 7-9 points received a single 33 mg/kg dose of Ferriprox®.

Group Type: EXPERIMENTAL

Ferriprox®

Intervention Type: DRUG

Interventions

Ferriprox®

Intervention Type: DRUG

Other Intervention Names

DFP; Deferiprone; L1

Eligibility Criteria

Inclusion Criteria

All subjects:

1. Adult males or females, 18 - 75 years of age (inclusive);

2. Body weight ≥ 50 kg;

3. Body mass index (BMI) between 19 and 32 kg/mE2 (inclusive);

4. Absolute neutrophil count (ANC) of >1.5x10E9/L ;

Healthy volunteers:

1. Medically healthy with clinically insignificant screening results (e.g., laboratory profiles, medical history, vital signs, physical examination);

2. Matched to hepatically impaired subjects in the study by age (+/- 10 years), sex and weight (+/- 15% BMI).

Hepatically impaired subjects:

1. Considered clinically stable in the opinion of the Investigator;

2. Subjects with different degrees of impaired hepatic function as assessed by a Child-Pugh classification score: mild (Class A: 5-6 points) and moderate (Class B: 7-9 points) impaired hepatic function.

Exclusion Criteria

1. For subjects with hepatic impairment, fluctuating or rapidly deteriorating hepatic function as indicated by clinical and/or laboratory signs of hepatic impairment (e.g. advanced ascites, infection of ascites, fever, or active gastrointestinal bleeding).

2. Evidence of liver impairment in healthy volunteers: hepatitis B and C; or aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin, clotting factors, serum protein that is considered clinically significant by the Investigator;

3. History or presence of significant clinically unstable respiratory, cardiovascular, pulmonary, hepatic (except for subjects assigned to one of the hepatically impaired groups), renal, hematologic, gastrointestinal, endocrine (except for subjects with hepatic impairment with clinically stable and treated diabetes, hypertension and thyroid disorders), immunologic, dermatologic, neurologic, or psychiatric disease;

4. Disorders or surgery of the gastrointestinal tract which may interfere with drug absorption or may otherwise influence the PK of the investigational medicinal product (e.g. resections of the small or large intestine, febrile conditions, chronic diarrhea, chronic vomiting, clinically unstable endocrine disease, severe infections, acute inflammations, etc.);

5. Received a pharmacological agent in another clinical trial within 28 days prior to the first dose of the study;

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

ApoPharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Fernando Tricta, MD

Role: STUDY_CHAIR

ApoPharma

Locations

University of Miami

Miami, Florida, United States

Orlando Clinical Research Center

Orlando, Florida, United States

Countries

United States

Other Identifiers

LA40-0412

Identifier Type: -

Identifier Source: org_study_id