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Trial details
Phase 3 study to evaluate the efficacy and safety of human plasma-derived fibrinogen concentrate (FIB Grifols) in subjects with congenital afibrinogenaemia and severe hypofibrinogenemia
Current status:
Approved
|
Date registered:
21/03/2021
Trial version(s)
Current: 14/07/2020
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Main Information
Primary registry identifying number
LBCTR2021024523
Protocol number
GC1801
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
NA
Type of registration
Prospective
Date of registration in national regulatory agency
14/07/2020
Primary sponsor
Instituto Grifols, S.A.
Primary sponsor: Country of origin
Spain
Public title
Phase 3 study to evaluate the efficacy and safety of human plasma-derived fibrinogen concentrate (FIB Grifols) in subjects with congenital afibrinogenaemia and severe hypofibrinogenemia
Acronym
NA
Scientific title
A prospective, multicenter, open-label, single-arm study to evaluate the efficacy and safety of human plasma-derived fibrinogen concentrate (FIB Grifols) in subjects with congenital afibrinogenaemia and severe hypofibrinogenemia requiring either ondemand treatment for acute bleeding or surgical prophylaxis
Acronym
NA
Brief summary of the study: English
This is a phase 3, multi-center, prospective, open-label, single-arm, clinical trial to be carried out in subjects with congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia in order to evaluate the efficacy and safety of human plasma-derived fibrinogen concentrate (FIB Grifols)
Brief summary of the study: Arabic
هذه دراسة سريرية مستقبلية في المرحلة الثالثة، متعددة المراكز، مفتوحة التسمية, أحادية المجموعة يتم إجراؤها لدى الأشخاص الذين يعانون من نقص الفيبرينوجين الخلقي الذي يتجلى على أنه فبرينوجين الدم المنعدم أو نقص فيبرينوجين الدم الوخيم من أجل تقييم فعالية وسلامة مركز الفيبرينوجين المشتق من البلازما البشرية (FIB Grifols)
Health conditions/problem studied: Specify
Congenital fibrinogen deficiency (afibrinogenemia or severe hypofibrinogenemia)
Interventions: Specify
Experimental: FIB Grifols FIB Grifols will be administered via slow intravenous (IV) infusion at a rate not to exceed 5 mL/minute. Dosing will be individually calculated for each subject based on the target plasma fibrinogen level according to the type of bleeding, measured actual plasma fibrinogen level before infusion, and body weight. The IP will be administered according to the nominal potency of the product. The duration of FIB Grifols treatment is 1 day with additional IV infusions administered according to daily assessments, clinical trial protocol guidance, and investigator’s judgment.
Key inclusion and exclusion criteria: Inclusion criteria
A subject must meet all the following inclusion criteria to be eligible for participation in this study. 1. Male or female subject between 6 to 70 years of age. 2. Signed and dated written ICF, or the subject’s parent or legal guardian signs and dates 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. Diagnosed with congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia (fibrinogen <50 mg/dL) and expected to require treatment for acute bleeding (either spontaneous or after trauma [defined as any accidental event leading to acute bleeding]), or prophylaxis of bleeding before a surgical intervention or invasive procedure. 4. Fibrinogen level < 50 mg/dL determined by Clauss method at baseline (sample drawn within 24 hours prior to infusion on Infusion 1 Day 1 Visit). 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 (sample drawn within 24 hours prior to infusion on Infusion 1 Day 1 Visit). a. Female subjects/partners of child-bearing potential include any female who has experienced menarche and who has not undergone successful surgical sterilization (eg, hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or are not postmenopausal (post menopausal is defined as amenorrhea for >12 consecutive months or women on hormone replacement therapy with documented serum follicle stimulating hormone level <35 mIU/mL). Even women who are using oral, implanted or injectable contraceptive hormones, mechanical products such as an intrauterine device [IUD], or barrier methods (eg, diaphragm, condoms, spermicides) to prevent pregnancy, or are practicing abstinence or where their partner is sterile (eg, vasectomy), should be considered to be of child bearing potential. 6. Willing to comply with all aspects of the clinical trial protocol, including blood sampling, for the entire duration of the study.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
6
Key inclusion and exclusion criteria: Age maximum
70
Key inclusion and exclusion criteria: Exclusion criteria
Exclusion Criteria A subject meeting any of the following exclusion criteria is NOT eligible for participation in the study. 1. Has acquired (secondary) fibrinogen deficiency. 2. Diagnosed with dysfibrinogenemia. 3. Has known antibodies against fibrinogen. 4. Has history of anaphylaxis or severe systemic response to any drug or blood-derived product. 5. Has history of intolerance to any component of the IP. 6. Documented history of immunoglobulin A (IgA) deficiency and antibodies against IgA. 7. Is a female who is pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (eg, oral, injectable, or implantable hormonal methods of contraception, placement of an IUD or intrauterine system, condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinancea) throughout the study. a. True abstinance: when this is in line with the preferred and usual lifestyle of the subject (Periodic abstinance [eg, calendar, ovulation, symptothermal, post-ovulation methods], declaration of abstinance for the duration of a trial, and withdrawal are not acceptable methods of contraception. 8. Has any medical condition which is likely to interfere with the evaluation of the IP and/or the satisfactory conduct of the clinical trial according to the investigator’s judgment. 9. Has congenital or acquired bleeding disorders other than congenital fibrinogen deficiency. 10. Has life expectancy of less than 6 months. 11. Received FRT within 21 days prior to the Screening Visit. 12. Receiving, or having received within 3 months prior to the Screening Visit of this clinical trial, any investigational drug or device. 13. Is unlikely to adhere the protocol requirements, or is likely to be uncooperative, or unable to provide a storage sample prior to IP infusion.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
Single Arm Study
Study design: Masking
Open (masking not used)
Study design: Control
N/A
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
No
Name of IMP
FIB Grifols
Type of IMP
Plasma derived
Pharmaceutical class
Human plasma-derived fibrinogen concentrate
Therapeutic indication
Congenital fibrinogen deficiency (afibrinogenemia or severe hypofibrinogenemia)
Therapeutic benefit
The intended benefit of fibrinogen concentrates is to avoid hemorrhagic manifestations in subjects with congenital fibrinogen deficiency.
Biospecimen retention
None retained
Biospecimen description
NA
Target sample size
32
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
12/10/2020
Date of study closure: Type
Anticipated
Date of study closure: Date
31/12/2023
Recruitment status
Pending
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
NA
Additional data URL
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Food and Drug Administration
IND number 15811
Sources of Monetary or Material Support
Name
Instituto Grifols, S.A.
Secondary Sponsors
Name
NA
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Aziz
Zoghbi
MCT-CRO
Lebanon
+961 71 008 269
zog_az@mct-cro.com
Country Oversight and Manageme nt Africa, Levant and GCC
Scientific
Claudia
Khayat
Hotel Dieu de France Hospital
Lebanon
+961 3 704 864
claudiakhayat@yahoo.fr
Principle Investigator
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Hotel Dieu de France Hospital
Dr. Claudia Khayat
Pediatric Hematology/Oncology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
30/06/2020
Nancy Alam
nancy.alam@usj.edu.lb
+961 1 421 000 ext 2335
Countries of Recruitment
Name
India
Bulgaria
Lebanon
Turkey
United States of America
United Kingdom
Health Conditions or Problems Studied
Condition
Code
Keyword
Congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia
Fibrinolysis-affecting drugs (T45.6)
afibrinogenemia or severe hypofibrinogenemia
Interventions
Intervention
Description
Keyword
Experimental: FIB Grifols
FIB Grifols will be administered via slow intravenous (IV) infusion at a rate not to exceed 5 mL/minute. Dosing will be individually calculated for each subject based on the target plasma fibrinogen level according to the type of bleeding, measured actual plasma fibrinogen level before infusion, and body weight. The IP will be administered according to the nominal potency of the product. The duration of FIB Grifols treatment is 1 day with additional IV infusions administered according to daily assessments, clinical trial protocol guidance, and investigator’s judgment.
FIB Grifols, IV
Primary Outcomes
Name
Time points
Measure
overall clinical assessment of the hemostatic efficacy of FIB Grifols
In case of bleeding episode: At the end of treatment Observation period visit. In case of surgery: End of surgery and at the end of treatment observational period visit
The primary efficacy endpoint is the overall clinical assessment of the hemostatic efficacy of FIB Grifols in treating all documented acute bleeding episodes or in preventing excessive bleeding during and after all documented surgical procedures according to a 4-point scale. Hemostatic efficacy will be rated as excellent, good, moderate, or none. The primary efficacy endpoint will be assessed by the IEAC.
Key Secondary Outcomes
Name
Time points
Measure
Overall clinical assessment of the hemostatic efficacy of FIB Grifols
In case of bleeding episode: At the end of treatment Observation period visit. In case of surgery: End of surgery and at the end of treatment observational period visit
Overall clinical assessment of the hemostatic efficacy of FIB Grifols in treating the first documented acute bleeding episode or in preventing excessive bleeding during and after the first documented surgical procedure according to a 4 point scale. This secondary efficacy endpoint will be assessed by the IEAC.
Overall clinical assessment of the hemostatic efficacy of FIB Grifols
In case of bleeding episode: At the end of treatment Observation period visit. In case of surgery: End of surgery and at the end of treatment observational period visit
Overall clinical assessment of the hemostatic efficacy of FIB Grifols in treating the first acute bleeding episode as assessed by the principal investigator at each trial site according to a 4-point scale
Overall clinical assessment of the hemostatic efficacy of FIB Grifols
In case of bleeding episode: At the end of treatment Observation period visit. In case of surgery: End of surgery and at the end of treatment observational period visit
Overall clinical assessment of the hemostatic efficacy of FIB Grifols in treating all acute bleeding episodes as assessed by the principal investigator at each trial site according to a 4-point scale
Overall clinical assessment of the hemostatic efficacy of FIB Grifols
In case of bleeding episode: At the end of treatment Observation period visit. In case of surgery: End of surgery and at the end of treatment observational period visit
Clinical assessment of the hemostatic efficacy of FIB Grifols in preventing excessive bleeding intra-operatively for all surgical procedures as assessed by the surgeon (defined as the licensed medical professional performing the invasive procedure [eg, dentist]) according to a 4-point scale
Overall clinical assessment of the hemostatic efficacy of FIB Grifols
In case of bleeding episode: At the end of treatment Observation period visit. In case of surgery: End of surgery and at the end of treatment observational period visit
Clinical assessment of the hemostatic efficacy of FIB Grifols in preventing excessive bleeding after all surgical procedures (ie, post-operative) as assessed by the principal investigator at each trial site according to a 4-point scale
Difference in MCF
From pre-infusion to 1 hour after the end of each infusion
Measured by ROTEM
Difference in MCF
From pre-infusion to 4 hours after the end of each infusion
Measured by ROTEM
Difference (improvement) in Thrombin Time
From pre-infusion to 1 hour after the end of each infusion
Difference (improvement) in Thrombin Time
Difference (improvement) in Prothrombin Time
From pre-infusion to 1 hour after the end of each infusion
Difference (improvement) in Prothrombin Time
Difference (improvement) in activated Partial Thromboplastin Time
From pre-infusion to 1 hour after the end of each infusion
Difference (improvement) in activated Partial Thromboplastin Time
Difference in plasma fibrinogen levels
From pre-infusion to 1 hour after the end of each infusion
By Clauss and ELISA
Difference in plasma fibrinogen levels
From pre-infusion to 4 hours after the end of each infusion
By Clauss and ELISA
Difference in plasma fibrinogen levels
From pre-infusion to 24 hours after the end of each infusion
By Clauss and ELISA
Incremental IVR
During the first 4 hours of sampling after the end of each infusion of FIB Grifols calculated using concentration of plasma fibrinogen
Measured by both Clauss method (functional) and ELISA method (immunologic) for subjects with acute bleeding episodes or need for additional post-operative FIB Grifols infusion
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial