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Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency

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ClinicalTrials.gov Identifier: NCT02065882
Recruitment Status : Unknown
Verified July 2019 by Biotest.
Recruitment status was:  Recruiting
First Posted : February 19, 2014
Last Update Posted : July 25, 2019
Sponsor:
Collaborators:
ICON plc
SYNLAB Analytics & Services Germany GmbH
Phoenix Clinical Research
Accovion GmbH
Information provided by (Responsible Party):
Biotest

Tracking Information
First Submitted Date  ICMJE February 3, 2014
First Posted Date  ICMJE February 19, 2014
Last Update Posted Date July 25, 2019
Study Start Date  ICMJE March 2013
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 17, 2014)
Terminal Elimination Half-life (t1/2) for fibrinogen antigen, [ Time Frame: Prior to the initial dose on day 1, at the end of the infusion and 0.5, 1, 2, 4, 8 hours post dose, 24, 48,96, 168, 240h and 336 hours post-dose ]
The primary objective of this study is to investigate the 14 day single-dose pharmacokinetics of BT524 following intravenous (IV) infusion in patients with congenital afibrinogenemia or severe congenital hypofibrinogenemia (part I).
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 17, 2014)
Terminal Elimination Half-life (t1/2) for fibrinogen activity [ Time Frame: Prior to the initial dose on day 1, at the end of the infusion and 0.5, 1, 2, 4, 8 hours post dose, 24, 48,96, 168, 240 and 336 hours post-dose ]
Secondary objectives are to investigate the 14 day single-dose pharmacodynamics of BT524, and the surrogate efficacy and safety of BT524 in part I of the study. In addition, the hemostatic efficacy, surrogate efficacy, and safety of single and/or repetitive administrations for on-demand prophylaxis (ODP) and/or on-demand treatment (ODT) of bleeding events will be investigated.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency
Official Title  ICMJE A Prospective, Open-label, Phase I/III Study Investigating Pharmacokinetic Properties of BT524 and Efficacy and Safety of BT524 in the Treatment and Prophylaxis of Bleeding in Patients With Congenital Fibrinogen Deficiency
Brief Summary The purpose of the study is to investigate pharmacokinetics, efficacy and safety of BT524 in patients with congenital fibrinogen deficiency.
Detailed Description The present study is designed as a prospective, open-label, multicentre, phase I/III study investigating the 14 day single-dose pharmacokinetic properties, efficacy and safety of BT524 following intravenous administration in the treatment or prophylaxis of bleeding in patients with congenital afibrinogenemia or severe congenital hypofibrinogenemia.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Congenital Afibrinogenemia
  • Congenital Hypofibrinogenemia
Intervention  ICMJE Biological: BT524 (fibrinogen concentrate from human plasma)
single intravenous infusion
Study Arms  ICMJE Experimental: BT524
Single intravenous infusion of a fixed dose of 70 mg BT524 per kilogram body weight (BW)
Intervention: Biological: BT524 (fibrinogen concentrate from human plasma)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: July 24, 2019)
36
Original Estimated Enrollment  ICMJE
 (submitted: February 17, 2014)
20
Estimated Study Completion Date  ICMJE November 2020
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Known congenital afibrinogenemia or severe congenital hypofibrinogenemia
  • Plasma fibrinogen activity ≤ 0.5 g/l and antigen ≤ 0.5 g/l
  • Male or female
  • Age 0 to 75 years, with the first ten patients will be 18 years or
  • Presumed to be compliant with the study procedures and to terminate the study as scheduled
  • Willing and able to be hospitalized for 3 days for the pharmacokinetic assessment (if applicable)
  • Willing and able to be hospitalized - if required - in case of interventions (e.g., surgical procedures, major bleeds)
  • Written informed consent by the patient, his/her parents or by the patient's legal / authorized representative as applicable

Exclusion Criteria:

  • Known congenital dysfibrinogenemia
  • Known bleeding disorder other than congenital fibrinogen deficiency
  • History of esophageal variceal bleeding
  • Known presence or history of venous/arterial thrombosis or thromboembolic event in the preceding 6 months
  • Known presence or history of fibrinogen inhibitory antibodies
  • Known presence or history of hypersensitivity to human fibrinogen or human plasma proteins e.g., immunoglobulins, vaccines or hypersensitivity to any of the excipients
  • Known positive serology for HIV-1 and HIV-2
  • Clinically relevant biochemical or hematological findings (except due to underlying disease or emergency bleeding) outside the normal range (at the investigator's discretion)
  • Clinically relevant pathological findings in physical examination including electrocardiogram (ECG)
  • Treatment with any fibrinogen concentrate and/or fibrinogen-containing product within 2 weeks prior to infusion of BT524
  • Concomitant medication interacting relevantly with the coagulation system (e.g., low molecular weight heparin, unfractioned heparin, factor Xa inhibitors, factor IIa inhibitors or PY12 inhibitors) within 2 weeks prior to infusion of BT524
  • Recent vaccination (within 3 weeks prior to infusion)
  • Body weight (BW) below 22 kg for patients ≥ 6 years; BW below the 5th percentile of the normal range for children < 6 years (refers to local standard)
  • End stage disease
  • Abuse of drugs
  • Unable to understand and follow the study requirements
  • Participation in another interventional clinical study within 30 days before entering the study or during the study
  • Pregnant/ nursing woman, or woman of childbearing potential not using reliable/ effective contraceptive method(s) during the study and at least one month after the last administration of study drug (e.g., oral/ injectable/ implantable/ insertable/ topical hormonal contraceptives, intrauterine devices, female sterilization, partner's vasectomy or condoms)
  • Any other condition that, to the investigator's judgment, could have an impact on patient's safety or the study results
  • Elective surgery during the 14 day PK blood sampling period
  • Acute infection
  • Clinically relevant increase or decrease in body temperature
  • Actively bleeding or anticipated bleeding (including female menorrhea) at the time point of or within 7 days prior to infusion of BT524
  • Surgery within 7 days prior to infusion of BT524
  • Immobilization within 7 days prior to infusion of BT524
  • Intake of alcohol or significantly increased intake of caffeine containing products within 24 hours prior to infusion of BT524
  • Blood donation or comparable blood loss within 60 days prior to infusion of BT524
  • Excessive physical exercise (extreme sports activities, sauna) within 72 hours prior to infusion of BT524
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 75 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Egypt,   Germany,   Italy,   Lebanon,   Tunisia
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02065882
Other Study ID Numbers  ICMJE Biotest 984
2011-004154-25 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Biotest
Study Sponsor  ICMJE Biotest
Collaborators  ICMJE
  • ICON plc
  • SYNLAB Analytics & Services Germany GmbH
  • Phoenix Clinical Research
  • Accovion GmbH
Investigators  ICMJE
Principal Investigator: Claudia Djambas Khayat, MD Hôtel Dieu de France, Dept. of Pediatrics
PRS Account Biotest
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP