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

3. September 2014 aktualisiert von: ApoPharma

An Open-Label Study to Compare the Pharmacokinetic Profiles of a Single Dose of Ferriprox® in Subjects With Impaired Hepatic Function and Healthy Volunteers

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®.

Studienübersicht

Status

Abgeschlossen

Intervention / Behandlung

Detaillierte Beschreibung

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.

Studientyp

Interventionell

Einschreibung (Tatsächlich)

21

Phase

  • Phase 4

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienorte

    • Florida
      • Miami, Florida, Vereinigte Staaten, 33136
        • University of Miami
      • Orlando, Florida, Vereinigte Staaten, 32809
        • Orlando Clinical Research Center

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

18 Jahre bis 75 Jahre (Erwachsene, Älterer Erwachsener)

Akzeptiert gesunde Freiwillige

Ja

Studienberechtigte Geschlechter

Alle

Beschreibung

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;

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Zuteilung: Nicht randomisiert
  • Interventionsmodell: Parallele Zuordnung
  • Maskierung: Keine (Offenes Etikett)

Waffen und Interventionen

Teilnehmergruppe / Arm
Intervention / Behandlung
Experimental: Normal Hepatic Function (healthy volunteers)
Healthy volunteers with normal hepatic function received a single 33 mg/kg dose of Ferriprox®.
Andere Namen:
  • L1
  • DFP
  • Deferipron
Experimental: 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®.
Andere Namen:
  • L1
  • DFP
  • Deferipron
Experimental: 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®.
Andere Namen:
  • L1
  • DFP
  • Deferipron

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Safety and Tolerability of Ferriprox in Subjects With or Without Hepatic Impairment.
Zeitfenster: 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

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

Ermittler

  • Studienstuhl: Fernando Tricta, MD, ApoPharma

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn

1. Januar 2013

Primärer Abschluss (Tatsächlich)

1. April 2014

Studienabschluss (Tatsächlich)

1. April 2014

Studienanmeldedaten

Zuerst eingereicht

9. Januar 2013

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

10. Januar 2013

Zuerst gepostet (Schätzen)

14. Januar 2013

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Schätzen)

10. September 2014

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

3. September 2014

Zuletzt verifiziert

1. April 2014

Mehr Informationen

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