Study investigating the pharmacokinetic properties (to see how active the study drug is in your blood and how long it takes for the study drug to get out of your blood) , efficacy and safety of FIB Grifols in subjects with congenital fibrinogen deficiency
- Conditions
- Congenital AfibrinogenemiaMedDRA version: 20.0Level: LLTClassification code 10052651Term: AfibrinogenaemiaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2013-004343-23-IT
- Lead Sponsor
- INSTITUTO GRIFOLS, S.A.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 22
1 Male or female subjects less than 70 years old.
2 Sign the written Informed Consent Form (ICF), or the subject's parent
or legal guardian signs the ICF where applicable, and the Subject
Authorization Form (SAF) where applicable. Pediatric subjects, as
defined by local regulations, will be asked to sign an age appropriate
assent form.
3 Subjects diagnosed with congenital fibrinogen deficiency manifested
as afibrinogenemia.
4 Subjects with a fibrinogen level undetectable, or equal or less than 30
mg/dL determined by both Clauss and antigen methods at baseline.
5 Female subjects of child-bearing potential must have a negative test
for pregnancy blood or urine human chorionic gonadotropin (HCG-based
assay) at baseline
6 Female subjects of child-bearing potential and their partners have
agreed to practice contraception using a method of proven reliability
(i.e. hormonal methods, barrier methods, intrauterine devices methods,
or abstinence) to prevent a pregnancy during the course of the clinical
trial.
7 Subjects must be willing to comply with all aspects of the clinical trial
protocol, including blood sampling, for the whole duration of the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1
1 Subjects who received any fibrinogen-containing product within 21
days prior to Day 0 Visit.
2 Subjects who present with active bleeding within 10 days prior to
infusion on Day 0.
3 Subjects with acquired (secondary) fibrinogen deficiency.
4 Subjects diagnosed with dysfibrinogenemia.
5 Subjects with documented history of deep vein thrombosis (DVT),
pulmonary embolism, or arterial thrombosis within
1 year prior to enrolment in this clinical trial.
6 Subjects with known antibodies against fibrinogen.
7 Subjects with a history of severe anaphylactic reactions or reactions
to any blood-derived product.
8 Subjects with a history of intolerance to any component of the
investigational product.
9 Subjects with a documented history of IgA deficiency and antibodies
against IgA.
10 Females who are pregnant or breastfeeding.
11 Subjects with renal impairment (i.e. serum creatinine exceeds more
than 2.0 times the upper limit of normal [ULN]) at baseline.
12 Subjects with aspartate aminotransferase (AST) or alanine
aminotransferase (ALT) levels exceeding more than 2.5 times the ULN at
baseline.
13 Subjects with a history of chronic alcoholism or illicit drug addiction
in the preceding 12 months prior to enrollment in this clinical trial.
14 Subjects with any medical condition which is likely to interfere with
the evaluation of the study drug and/or the satisfactory conduct of the
clinical trial according to the investigator's judgment.
15 Subjects who received aspirin-containing products and nonsteroidal
anti-inflammatory drugs (NSAIDs) within 7 days prior to the Day 0 Visit.
16 Subjects currently receiving, or having received within 3 months
prior to enrolment into this clinical trial, any investigational drug or
device.
17 Subjects who were previously administered the investigational
product FIB Grifols during this clinical trial (i.e. every subject can only
participate in the study once).
18 Subjects who are unlikely to adhere to the protocol requirements,
are likely to be uncooperative, or are unable to provide a storage serum
sample prior to investigational drug infusion.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the pharmacokinetics, efficacy, and safety of human plasmaderived fibrinogen concentrate FIB Grifols after a single-dose 70 mg/kg body weight administration.;Secondary Objective: NA;Primary end point(s): -AUC0-14days (Area under the curve from zero to 14 days)<br>-AUC0-8 (Area under the curve from zero to infinity)<br>-Cmax (Maximum plasma concentration)<br>-tmax (Time to the observed maximum plasma concentration)<br>-t1/2 (Half-life)<br>-MRT (Mean residence time)<br>-Vd (Volume of distribution)<br>-Cl (Clearance)<br>-IVR (In vivo recovery)<br>-Change in MCF (Maximum clot firmness) measured by ROTEM (Rotational thromboelastometry);Timepoint(s) of evaluation of this end point: At Baseline,0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216 and 336 hours post-infusion
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Difference (improvement) in other thromboelastographic parameters:<br>Clotting time (CT)<br>Clot formation time (CFT)<br>Alpha angle (a)<br>-Difference (improvement) in standard coagulation variables from baseline to 1-hour post-infusion.<br>Prothrombin time (PT)<br>Thrombin time (TT)<br>Activated partial thromboplastin time (aPTT);Timepoint(s) of evaluation of this end point: Baseline (prior to infusion), one hour after completion of infusion