- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT01767103
An Open-label, Non-randomized, Parallel Group Study in Subjects With Mild and Moderate Hepatic Insufficiency and Healthy Volunteers
An Open-Label Study to Compare the Pharmacokinetic Profiles of a Single Dose of Ferriprox® in Subjects With Impaired Hepatic Function and Healthy Volunteers
Studieoversigt
Detaljeret beskrivelse
Undersøgelsestype
Tilmelding (Faktiske)
Fase
- Fase 4
Kontakter og lokationer
Studiesteder
-
-
Florida
-
Miami, Florida, Forenede Stater, 33136
- University of Miami
-
Orlando, Florida, Forenede Stater, 32809
- Orlando Clinical Research Center
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
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;
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Tildeling: Ikke-randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
---|---|
Eksperimentel: Normal Hepatic Function (healthy volunteers)
Healthy volunteers with normal hepatic function received a single 33 mg/kg dose of Ferriprox®.
|
Andre navne:
|
Eksperimentel: 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®.
|
Andre navne:
|
Eksperimentel: 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®.
|
Andre navne:
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
---|---|---|
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Tidsramme: 24-hour interval
|
Cmax was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment.
Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.
|
24-hour interval
|
Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Tidsramme: 24-hour interval
|
Tmax was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment.
Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.
|
24-hour interval
|
AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide
Tidsramme: 24-hour interval
|
AUC (area under the curve) from zero to infinity was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment.
Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.
|
24-hour interval
|
T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide
Tidsramme: 24-hour interval
|
T1/2 was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment.
Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.
|
24-hour interval
|
CumAe for Urine Deferiprone and Deferiprone 3-O-glucuronide
Tidsramme: 24-hour interval
|
Cumulative amount of deferiprone and deferiprone 3-O-glucuronide excreted in the urine. Urine samples were collected at the intervals of -2 to 0 hours pre-dose, and 0-2, 2-4, 4-8, 8-12 ,and 12- 24 hours post-dose. Note: For unknown reasons, the urine samples for one subject in the moderate hepatic failure group had low or zero volume and there were no measurable levels of deferiprone or its metabolite in any of the samples. Accordingly, the urine PK results were derived both with and without this subject's data, and are here presented without them (i.e., N=6 rather than 7). |
24-hour interval
|
Fe% for Urine Deferiprone and Deferiprone 3-O-glucuronide
Tidsramme: 24-hour interval
|
Cumulative fraction of Ferriprox dose excreted in urine as deferiprone or deferiprone 3-O-glucuronide. Urine samples were collected at the intervals of -2 to 0 hours pre-dose, and 0-2, 2-4, 4-8, 8-12 ,and 12- 24 hours post-dose. Note: For unknown reasons, the urine samples for one subject in the moderate hepatic failure group had low or zero volume and there were no measurable levels of deferiprone or its metabolite in any of the samples. Accordingly, the urine PK results were derived both with and without this subject's data, and are here presented without them (i.e., N=6 rather than 7). |
24-hour interval
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
---|---|---|
Safety and Tolerability of Ferriprox in Subjects With or Without Hepatic Impairment.
Tidsramme: Time of dosing until 48 hours post-dose
|
The number of participants who experienced adverse events following a single dose of Ferriprox, between the time of dosing and the follow-up visit (including any changes of clinical significance in physical examinations, vital signs, 12-lead ECG, and clinical laboratory tests).
|
Time of dosing until 48 hours post-dose
|
Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Studiestol: Fernando Tricta, MD, ApoPharma
Datoer for undersøgelser
Studer store datoer
Studiestart
Primær færdiggørelse (Faktiske)
Studieafslutning (Faktiske)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Skøn)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Skøn)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- LA40-0412
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .
Kliniske forsøg med Nedsat leverfunktion
-
Dart NeuroScience, LLCAfsluttetAge-Associated Memory Impairment (AAMI)Forenede Stater
-
Charité Neurocure AG FlöelUkendtAfasi | Anomi (ord-finding impairment)Tyskland
-
Johns Hopkins UniversityNational Institute on Aging (NIA)RekrutteringSøvnforstyrrelser | AMCI - Amnestic Mild Cognitive ImpairmentForenede Stater
Kliniske forsøg med Ferriprox®
-
ApoPharmaAfsluttetOverbelastning af jern | Seglcellesygdom | Andre anæmierForenede Stater, Det Forenede Kongerige, Egypten, Canada, Saudi Arabien
-
University Hospital, LilleApoPharmaAfsluttetALS (amyotrofisk lateral sklerose) | Overbelastning af jernFrankrig
-
Children's Hospital of PhiladelphiaApoPharmaGodkendt til markedsføringOverbelastning af jernForenede Stater
-
Ente Ospedaliero Ospedali GallieraAktiv, ikke rekrutterendeFerrochelerende behandling hos patienter ramt af neurodegeneration med hjernejernakkumulation (NBIA)Overbelastning af jern | Neurodegenerativ sygdomItalien
-
Chiesi Canada CorpIkke længere tilgængeligPantothenatkinase-associeret neurodegeneration
-
Sheba Medical CenterHadassah Medical Organization; Kaplan Medical Center; Tel Aviv Medical Center og andre samarbejdspartnereAfsluttetMyelodysplastisk syndrom med lavgradige læsioner | Overbelastning af jern på grund af gentagne transfusioner af røde blodlegemerIsrael
-
Ain Shams UniversityUkendtBeta-thalassæmi majorEgypten
-
ApoPharmaAfsluttet
-
ApoPharmaAfsluttet
-
ApoPharmaAfsluttet