Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Patients With Congenital Afibrinogenemia (IG0902)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02281500|
Recruitment Status : Recruiting
First Posted : November 2, 2014
Last Update Posted : July 5, 2019
|Condition or disease||Intervention/treatment||Phase|
|Congenital Afibrinogenemia||Biological: Human Plasma-Derived Fibrinogen Concentrate||Phase 1 Phase 2|
This study is a phase I-II, multi-center, prospective, open-label, single-arm, clinical trial to evaluate PK, efficacy, and safety of human plasma-derived fibrinogen concentrate (FIB Grifols) in adult and pediatric subjects with congenital afibrinogenemia.
Approximately 10 adult subjects (≥18 years) with congenital afibrinogenemia will be administered a single dose of FIB Grifols at 70 mg/kg body weight and will be followed for PK, efficacy, and safety assessments.
After the safety of fibrinogen concentrate FIB Grifols is assessed in at least 10 adult subjects and no safety issues are raised by the sponsor, the study will start to enroll approximately 10 pediatric subjects (<18 years) who will be dosed with study drug and followed for PK, efficacy, and safety assessments.
All enrolled subjects (both adult and pediatric) will have documented congenital fibrinogen deficiency manifested as afibrinogenemia but will not have received any fibrinogen-containing product therapy within the preceding 21 days before the infusion of study drug.
All subjects (both adult and pediatrics) will be infused with the investigational product at 70 mg/kg body weight. PK parameters that will be calculated from plasma fibrinogen levels measured at different time points include: incremental in vivo recovery [IVR], area under the curve (AUC) calculated as AUC from zero to 14 days (AUC^0-14days) and AUC from zero to infinity (AUC^0-∞), maximum plasma concentration (C^max), time to the observed maximum plasma concentration (t^max), half-life (t^1/2), mean residence time (MRT), volume of distribution (Vd), and clearance (Cl).
Hemostatic efficacy of the investigational product will be assessed by means of rotational thromboelastometry (ROTEM) measure of maximum clot firmness (MCF) at baseline and 1 hour post-infusion. Other thromboelastographic measures as well as standard coagulation tests will be also determined pre- and post-infusion.
Clinical safety, viral safety, and immunogenicity will be assessed in this clinical trial. Safety variables include adverse events (AEs), vital signs, physical assessments, laboratory tests, viral markers, and antibodies against human fibrinogen.
A monitoring plan will be implemented by the sponsor to carefully monitor and evaluate allergic/hypersensitivity reactions and thrombotic events during the study.
Stopping criteria have been established for immunogenic and thrombogenic events. If a single case of any these events is reported after a subject has been dosed with study drug, any further enrollment and dosing of subjects in the study will be suspended until the event can be adequately assessed by the sponsor. The enrollment and dosing will only resume if the sponsor deems it is safe to do so.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||22 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Multicenter, Prospective, Open-Label, Single-Arm Trial to Evaluate the Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Patients With Congenital Afibrinogenemia|
|Actual Study Start Date :||May 2016|
|Estimated Primary Completion Date :||November 2019|
|Estimated Study Completion Date :||December 2019|
Human Plasma-Derived Fibrinogen Concentrate Grifols (FIB Grifols)
Biological: Human Plasma-Derived Fibrinogen Concentrate
A sterile freeze-dried fibrinogen concentrate filled in vials containing 1 g of FIB Grifols. FIB Grifols contains 20 mg/ml of active substance fibrinogen when reconstituted.
Other Name: FIB Grifols
- Change in MCF measured by ROTEM [ Time Frame: Baseline to one hour post-infusion ]MCF, as a functional parameter of blood's ability to coagulate, provides an indirect measure of hemostatic efficacy of replacement treatment with fibrinogen concentrates in patients with fibrinogen deficiency.
- Difference (improvement) in Clotting time (CT) [ Time Frame: Baseline to one hour post-infusion ]
- Difference (improvement) in Clot Formation Time (CFT) [ Time Frame: Baseline to one hour post-infusion ]
- Difference (improvement) in Alpha angle (α) [ Time Frame: Baseline to one hour post-infusion ]
- Difference (improvement) in Prothrombin tme (PT) [ Time Frame: Baseline to one hour post-infusion ]
- Difference (improvement) in Thrombin tme (TT) [ Time Frame: Baseline to one hour post-infusion ]
- Difference (improvement) in Activated Partial Thromboplastin Time (aPTT) [ Time Frame: Baseline to one hour post-infusion ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02281500
|Contact: Jordi Navarrofirstname.lastname@example.org|
|Contact: Karen Ruckeremail@example.com|
|United States, New York|
|Northshore Long Island Jewish Medical Center||Recruiting|
|New Hyde Park, New York, United States, 11040|
|Contact: Joe Stanco 718-470-4434 firstname.lastname@example.org|
|Principal Investigator: Suchitra S. Acharya, MD|
|S.S. Institute of Medical Sciences and Research Centre||Recruiting|
|Davangere, Karnataka, India|
|Contact: Umesh V Kumar, SN +91-9739427409 email@example.com|
|Principal Investigator: N. K. Kalappanavar, MD|
|St. Johns Medical College and Hospital||Recruiting|
|Contact: Alekya Vemula, CRC 91 8897752905 firstname.lastname@example.org|
|Contact: Arpita Taran, CRC 91-9620719377 email@example.com|
|Principal Investigator: Cecil Rueben Ross, MD|
|Sahyadri Specialty Hospital||Recruiting|
|Contact: Manali Sapre, CRC +91-7756858299 firstname.lastname@example.org|
|Principal Investigator: Kannan Subramanian, MD|
|Agenzia per l'Emofilia Centro di Riferimento Regionale per le Coaugulopatie Congenite A.O. di Carreggi||Not yet recruiting|
|Firenze, Italy, 50134|
|Contact: Giancarlo Castamann, MD|
|Principal Investigator: Giancarlo Castamann, MD|
|Hôtel-Dieu de France Hospital||Recruiting|
|Contact: Claudia Khayat, MD 9611613027 email@example.com|
|Principal Investigator: Claudia Khayat, MD|
|Study Chair:||Flora Peyvandi, MD||Centro Emofilia & Trombosi Angelo Bianchi Bonomi|