Clinical trial NCT01767103: An Open-label, Non-randomized, Parallel Group Study in Subjects With Mild and Moderate Hepatic Insufficiency and Healthy Volunteers
Official title: An Open-Label Study to Compare the Pharmacokinetic Profiles of a Single Dose of Ferriprox® in Subjects With Impaired Hepatic Function and Healthy Volunteers
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hepatic Impairment
Keywords: Hepatic Impairment
Liver Impairment
Liver Disease
Ferriprox®
LI
DFP
Deferiprone
Lead sponsor: ApoPharma
Sponsor class: INDUSTRY
First posted: 2013-01-14
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL

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.

Eligibility criteria: Main 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.

Main 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;

Primary outcomes:
measure: Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
timeFrame: 24-hour interval
measure: Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
timeFrame: 24-hour interval
measure: AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide
timeFrame: 24-hour interval
measure: T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide
timeFrame: 24-hour interval
measure: CumAe for Urine Deferiprone and Deferiprone 3-O-glucuronide
timeFrame: 24-hour interval
measure: Fe% for Urine Deferiprone and Deferiprone 3-O-glucuronide
timeFrame: 24-hour interval
Secondary outcomes:
measure: Safety and Tolerability of Ferriprox in Subjects With or Without Hepatic Impairment.
timeFrame: Time of dosing until 48 hours post-dose
Other outcomes:
Locations:
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Orlando
country: United States
facility: Orlando Clinical Research Center
state: Florida
Study officials:
affiliation: ApoPharma
name: Fernando Tricta, MD
role: STUDY_CHAIR
References: