Clinical 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

Phase 1

• Conditions: Patients with congenital afibrinogenemia or severe congenital hypofibrinogenemia.; MedDRA version: 14.1Level: PTClassification code 10016075Term: Factor I deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2011-004154-25-IT
• Lead Sponsor: Biotest AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-02-20
• Last Update Posted: 20 June 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1.Congenital afibrinogenemia or severe congenital hypofibrinogenemia defined by plasma fibrinogen activity = 0.5 g/l and antigen = 0.5 g/l
2.Male or female
3.Age 6 to 75 years, with the first ten patients will be 18 years or older
4.Presumed to be compliant with the study procedures and to terminate the study as scheduled
5.Willing and able to be hospitalized for 3 days for the pharmaco-kinetic assessment
6.Willing and able to be hospitalized - if required - in case of interventions (e.g., surgical procedures, major bleeds)
7.Written informed consent by the patient, his/her parents or by the patient's legal / authorized representative as applicable

Are the trial subjects under 18? yes
Number of subjects for this age range: 6
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

General exclusion criteria:
1.Known congenital dysfibrinogenemia
2.Known bleeding disorder other than congenital fibrinogen deficiency
3.History of esophageal variceal bleeding
4.Known presence or history of venous/arterial thrombosis or thromboembolic event in the preceding 6 months
5.Known presence or history of fibrinogen inhibitory antibodies
6.Known presence or history of hypersensitivity to human fibrinogen or human plasma proteins e.g., immunoglobulins, vaccines
7.Known positive serology for HIV-1 and HIV-2
8.Clinically relevant biochemical or hematological findings (except due to underlying disease or emergency bleeding) outside the normal range (at the investigator's discretion)
9.Clinically relevant pathological findings in physical examination including electrocardiogram (ECG)
10.Treatment with any fibrinogen concentrate and/or fibrinogen-containing product within 2 weeks prior to infusion of BT524
11.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
12.Recent vaccination (at least 3 weeks)
13.Body weight below 22 kg
14.End stage disease
15.Abuse of drugs
16.Unable to understand and follow the study requirements
17.Participation in another interventional clinical study within 30 days before entering the study or during the study
18.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)
19.Any other condition that, to the investigator's judgment, could have an impact on patient's safety or the study results

PK-specific exclusion criteria:
20.Elective surgery during the 14 day PK blood sampling period
21.Acute infection
22.Clinically relevant increase or decrease in body temperature
23.Actively bleeding or anticipated bleeding (including female menorrhea) at the time point of or within 7 days prior to infusion of BT524
24.Surgery within 7 days prior to infusion of BT524
25.Immobilization within 7 days prior to infusion of BT524
26.Intake of alcohol or significantly increased intake of caffeine containing products within 24 hours prior to infusion of BT524
27.Blood donation or comparable blood loss within 60 days prior to infusion of BT524
28.Excessive physical exercise (extreme sports activities, sauna) within 72 hours prior to infusion of BT524

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified