Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients (ITI)
|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: NCT02479087|
Recruitment Status : Recruiting
First Posted : June 23, 2015
Last Update Posted : June 23, 2015
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia A||Drug: Plasma-derived FVIII/VWF concentrate||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||IMMUNE TOLERANCE INDUCTION, BY FACTOR VIII CONCENTRATE CONTAINING VON WILLEBRAND FACTOR, IN SEVERE OR MODERATE HAEMOPHILIA A PATIENTS WITH INHIBITORS|
|Study Start Date :||January 2015|
|Estimated Primary Completion Date :||January 2019|
|Estimated Study Completion Date :||January 2020|
Experimental: Plasma-derived FVIII/VWF concentrate
The drug will be delivered through intravenous slow infusion/injection. The starting dosage can vary between the minimum dosage of 50 IU/Kg 3 times a week up to a maximum of 200 IU/kg per day.
This starting dosage will be decided by the Principal Investigator according to patient's condition and other variables.
The initial dosage can be then adjusted on the base of response.
Drug: Plasma-derived FVIII/VWF concentrate
The investigational treatment is with lyophilized plasma-derived Factor VIII. The product belongs to the factor VIII concentrates class, containing also VW Factor in an average ratio VW/VIII of > 1: 4.5.
The product is as a powder and a solvent solution for continuous infusion of Factor VIII. The specific activity of Factor VIII is of approximately 80 IU/mg protein.
The number of units of FVIII administered is expressed in International Units (IU), which are consistent with current WHO standards for products containing Factor VIII. The activity of Factor VIII in plasma is expressed either as a percentage (compared to normal human plasma) or in International Units (compared to the international standard for FVIII in plasma).
Other Name: Emoclot
- Efficacy: evaluation of the success of IT induction [ Time Frame: Up to33 months ]Success:Inhibitor disappearance/reduction to <0.6 BU/ml with FVIII activity recovery of at least ≥ 66% within 33 months of treatment ; Partial Success: inhibitor reduction to <5 BU/ml with clinical response to FVIII treatment, not followed by an inhibitor increase to values >5 BU/ml for a treatment period of 6 months on demand, or for 12 months of prophylactic treatment; No Response (Failure):Failure in relation to the above criteria defining complete response and partial response within 33 months, OR a reduction of the concentration of the inhibitor, less than 20%, compared to the peak of inhibitor in the IT, in every period of 6 months after the first 3 months of treatment. This implies that 9 months represents the minimum period of treatment and 33 months the maximum possible duration of ITI without success, OR patient withdrawal from the study for any reason.
- Safety (adverse events) [ Time Frame: Up to 33 months ]Description and incidence of adverse events during the course of prophylactic treatment, with severity, correlation with the investigational product and final outcome.
- Analysis of treatment compliance [ Time Frame: Up to 33 months ]Description of the patient's adherence to the optimal prolonged treatment.
- Efficacy evaluation - Time to achieve ITI [ Time Frame: Up to 33 months ]Time to achieve the complete or partial response (as defined in the primary outcome measure).
- Evaluation of the cost of therapy [ Time Frame: Up to 33 months ]Recording of overall amount of direct costs of therapy.
- Efficacy evaluation - IT persistence [ Time Frame: Up to 33 months+ 12 months FU ]Absence of relapse, assessed at 12 months from IT achievement
- Efficacy evaluation - FVIII genetic defect role in IT achievement [ Time Frame: Up to 33 months ]Role of FVIII mutations in influencing IT achievement
- Efficacy evaluation - Role of an immediate IT to delayed IT in IT induction. [ Time Frame: Up to 33 months ]Time elapsing between the onset of the inhibitor and the beginning of treatment and its importance for induction of IT in achieving primary endpoints.
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): NCT02479087
|Contact: Pier Mannuccio Mannucci, MD||+39 firstname.lastname@example.org|
|Contact: Elena Santagostino, MD||+39 email@example.com|
|Ain Shams Pediatric hospital, Ain Shams University||Not yet recruiting|
|Contact: Mohssen El-Alfy, MD +20 1000864343 firstname.lastname@example.org|
|Contact: Shereen Abdel Ghany, MD +20 1200533484 email@example.com|
|Principal Investigator: Mohssen El-Alfy, MD|
|Sub-Investigator: Shereen Abdel Ghany, MD|
|Sub-Investigator: Neveen Gamal, MD|
|Almoneera Pediatric Cairo University Hospital (Abu El- Reesh)||Not yet recruiting|
|Contact: Amal El-Beshlawy, MD +20 1223124674 firstname.lastname@example.org|
|Contact: Sonia Aldof, MD +20 1223431046 email@example.com|
|Principal Investigator: Amal El-Beshlawy, MD|
|Sub-Investigator: Sonia Aldof, MD|
|Sub-Investigator: Shaymaa Mohamed, MD|
|St. John's Medical College Hospital||Recruiting|
|Bangalore, India, 560034|
|Contact: Cecil Ross, MD +91 9448493705 firstname.lastname@example.org|
|Contact: Sita Lakshmi, MD +91 8022065178 email@example.com|
|Principal Investigator: Cecil Ross, MD|
|Sub-Investigator: Sita Lakshmi, MD|
|Sub-Investigator: Fulton D'souza, MD|
|All India Institute of Medical Sciences||Recruiting|
|New Delhi, India, 110029|
|Contact: Tulika Seth, MD +91 9868397236 firstname.lastname@example.org|
|Contact: Renu Saxena, MD +91 1126594670 email@example.com|
|Principal Investigator: Tulika Seth, MD|
|Sub-Investigator: Renu Saxena, MD|
|Sub-Investigator: Vandana Sharma, MD|
|Study Chair:||Pier Mannuccio Mannucci, MD||IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico|
|Study Director:||Flora Peyvandi, MD||Università di Milano, IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico|
|Study Director:||Elena Santagostino, MD||Centro Emofilia e Trombosi Angela Bianchi Bonomi, IRCCS Fondazione Ospedale Maggiore Policlinico|