Clinical trial NCT03093480: A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)
Official title: A Non-controlled, Open-Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects With Inhibitors Undergoing the First ITI Treatment
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemophilia A With Inhibitors
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: Swedish Orphan Biovitrum
First posted: 2017-03-28
Enrollment: 16
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary purpose of this study was to describe the time to tolerization (i.e., ITI success) with rFVIIIFc in participants within a maximum of 48 weeks (12 months) of ITI treatment.


Eligibility criteria: Inclusion Criteria:

* Ability of the participant or his legally authorized representative (e.g., parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local participant privacy regulations
* Male participants of any age diagnosed with severe hemophilia A (as confirmed from the medical record)
* Currently diagnosed with high titer inhibitors (historical peak greater than or equal to (\>=) 5 Bethesda units per milliliter (BU/mL), according to medical records)
* Previously treated with any plasma-derived or recombinant conventional or Extended Half-Life FVIII

Exclusion Criteria:

* Other coagulation disorder(s) in addition to hemophilia A
* Previous immune tolerance induction (ITI)
* History of hypersensitivity or anaphylaxis associated with any factor VIII (FVIII) administration
* Planned major surgery scheduled during the study unless deferred until after study completion (minor surgery such as tooth extraction or insertion/replacement of central venous access device is allowed)
* Abnormal renal function (serum creatinine \>1.5 milligram per deciliter (mg/dL) or 2 × upper limit of normal (ULN) for participant age based on local laboratory range) as assessed by local laboratory
* Serum alanine aminotransferase or aspartate aminotransferase \> 5 × upper limit of normal (ULN) as assessed by local laboratory

Primary outcomes:
measure: Time to Tolerization With rFVIIIFc
timeFrame: Up to 48 Weeks
Secondary outcomes:
measure: Number of Participants With Immune Tolerance Induction (ITI) Success
timeFrame: Up to 48 Weeks
measure: Number of Participants Who Experienced Relapse
timeFrame: Up to 48 weeks (16 weeks Tapering period and 32 weeks follow-up period)
measure: Annualized Bleeding Rates During ITI Period
timeFrame: Up to 48 weeks
measure: Annualized Bleeding Rates After ITI Period
timeFrame: Up to 48 weeks (16 weeks Tapering period and 32 weeks follow-up period)
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) as a Measure of Safety and Tolerability
timeFrame: Up to 2 Years
measure: Average Number of Days Missed From Work or School Per Month During ITI Period
timeFrame: Up to 48 weeks
measure: Average Number of Days Missed From Work or School Per Month After ITI Period
timeFrame: Up to 48 weeks (16 weeks Tapering period & 32 weeks Follow-up period)
measure: Annualized Number of Hospitalization Days During ITI Period
timeFrame: Up to 48 weeks
measure: Annualized Number of Hospitalization Days After ITI Period
timeFrame: Up to 48 weeks (16 weeks Tapering period & 32 weeks Follow-up period)
measure: Adherence to Treatment Regimen Overall Study Period
timeFrame: Up to 2 Years
measure: Annualized rFVIIIFc Consumption for Overall Study Period
timeFrame: Up to 2 Years
Other outcomes:
Locations:
city: Orange
country: United States
facility: Center for Inherited Blood Disorders
state: California
city: Aurora
country: United States
facility: University of Colorado Hemophilia & Thrombosis Center
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Hemophilia and Thrombosis Center
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Children's Hospital
state: Iowa
city: Detroit
country: United States
facility: Childrens Hospital of Michigan
state: Michigan
city: Dayton
country: United States
facility: Dayton Children's Hospital
state: Ohio
city: El Paso
country: United States
facility: El Paso Children's Hospital
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Houston
country: United States
facility: Gulf States Hemophilia and Thrombophilia Center
state: Texas
city: Milwaukee
country: United States
facility: Blood Center of Southeast Wisconsin
state: Wisconsin
city: Bruxelles
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Leuven
country: Belgium
facility: UZ Leuven
city: Plovdiv
country: Bulgaria
facility: UMHAT "Sv. Georgi", EAD
city: Sofia
country: Bulgaria
facility: UMHAT 'Tsaritsa Yoanna - ISUL', EAD
city: Vancouver
country: Canada
facility: Children's & Women's Health Centre of British Columbia
state: British Columbia
city: Hamilton
country: Canada
facility: McMaster Children's Hospital
state: Ontario
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Marseille
country: France
facility: Hôpital de la Timone
state: Bouches-Du-Rhône
city: Besançon
country: France
facility: CHU Besançon - Hôpital Jean Minjoz
state: Doubs
city: Toulouse
country: France
facility: CHU de Toulouse - Hôpital Purpan
state: Haute Garonne
city: Lille
country: France
facility: Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est)
state: Nord
city: Paris
country: France
facility: Hôpital Necker - Enfants Malades
city: Bonn
country: Germany
facility: Universitaetsklinikum Bonn AoeR
state: North Rhine-Westphalia
city: Milano
country: Italy
facility: Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
city: Napoli
country: Italy
facility: Azienda Ospedaliera Pediatrica Santobono Pausillipon
city: Vicenza
country: Italy
facility: Ospedale San Bortolo di Vicenza
city: Nagoya-shi
country: Japan
facility: Nagoya University Hospital
state: Aichi-Ken
city: Kawasaki
country: Japan
facility: St. Marianna University School of Medicine Hospital
state: Kanagawa-Ken
city: Kashihara-Shi
country: Japan
facility: Nara Medical University Hospital
state: Nara-Ken
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe
city: London
country: United Kingdom
facility: St Thomas' Hospital
state: Greater London
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
state: Oxfordshire
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Children
state: Strathclyde
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: