Phase 4 N=21

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

Hepatic Impairment

Bottom Line

View on ClinicalTrials.gov: NCT01767103 ↗

Enrolled (actual)

Serious AEs

4.8%

Results posted

Sep 2014

Primary outcome: Primary: Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide — 49.00; 36.26; 35.94; 69.03 ug/mL

Study Design & Population

Study type: Interventional
Phase: Phase 4
Interventions: Ferriprox® (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: ApoPharma
Primary completion: Apr 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide	49.00; 36.26; 35.94; 69.03; 56.91; 55.33	—
PRIMARY Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide	0.7500; 0.7500; 0.7500; 2.000; 3.000; 3.000	—
PRIMARY AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide	92.64; 82.02; 105.5; 337.3; 305.1; 305.9	—
PRIMARY T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide	1.994; 1.855; 2.180; 2.621; 2.518; 2.479	—
PRIMARY CumAe for Urine Deferiprone and Deferiprone 3-O-glucuronide	69.0; 59.5; 62.9; 6670; 6787; 5979	—
PRIMARY Fe% for Urine Deferiprone and Deferiprone 3-O-glucuronide	2.56; 2.18; 2.36; 109; 110; 97.7	—
SECONDARY Safety and Tolerability of Ferriprox in Subjects With or Without Hepatic Impairment.	0; 1; 3	—

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®.

Eligibility Criteria

Main Inclusion Criteria:

All subjects:

Adult males or females, 18 - 75 years of age (inclusive);
Body weight ≥ 50 kg;
Body mass index (BMI) between 19 and 32 kg/mE2 (inclusive);
Absolute neutrophil count (ANC) of >1.5x10E9/L ;

Healthy volunteers:

Medically healthy with clinically insignificant screening results (e.g., laboratory profiles, medical history, vital signs, physical examination);
Matched to hepatically impaired subjects in the study by age (+/- 10 years), sex and weight (+/- 15% BMI).

Hepatically impaired subjects:

Considered clinically stable in the opinion of the Investigator;
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:

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01767103). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.