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)
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| ClinicalTrials.gov Identifier: NCT03093480 |
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Recruitment Status :
Active, not recruiting
First Posted : March 28, 2017
Last Update Posted : July 2, 2020
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Sponsor:
Bioverativ, a Sanofi company
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Sanofi ( Bioverativ, a Sanofi company )
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Brief Summary:
The primary purpose of this study is 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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hemophilia A With Inhibitors | Biological: rFVIIIFc | Phase 4 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 16 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| 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 |
| Actual Study Start Date : | December 8, 2017 |
| Estimated Primary Completion Date : | February 15, 2021 |
| Estimated Study Completion Date : | February 15, 2021 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Hemophilia
MedlinePlus related topics:
Hemophilia
| Arm | Intervention/treatment |
|---|---|
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Experimental: Recombinant coagulation factor VIII Fc (rFVIIIFc)
Participants will receive rFVIIIFc at a dose of 200 international units (IU)/kilogram (kg) as once daily injections or divided on several injections per day at the discretion of the Investigator, starting at baseline visit up to maximum of 48 Weeks in ITI Period. Participants who meet the criteria for immune tolerance induction (ITI) success will enter the tapering period and will receive rFVIIIFc at a dose adjusted according to Investigator judgment based on the FVIII activity levels and with the aim of tapering the rFVIIIFc dose to reach a prophylactic dosing regimen within 16 weeks (4 months). Follow-Up will be 32 weeks under an adjusted prophylactic regimen according to Investigator judgment.
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Biological: rFVIIIFc
rFVIIIFc 200 IU/kg/day in ITI Period, 50 or 100 IU/kg (adjusted according to Investigator judgement) in tapering Period, and prophylactic regimen in Follow-Up period as powder for injection administered intravenously.
Other Name: ELOCTATE/ELOCTA |
Primary Outcome Measures :
- Time to Tolerization With rFVIIIFc [ Time Frame: Up to 48 Weeks ]Time required for participants to achieve ITI success where ITI success is defined as achieving all 3 of the following criteria: confirmed negative titers consisting of 2 consecutive negative inhibitor assessments within 2 weeks (less than [<] 0.6 Bethesda units/milliliter [mL] by the Nijmegen-modified Bethesda assay); incremental recovery (IR) greater than or equal to (>=) 66 percent (%) of the expected IR in 2 consecutive assessments; half-life (t½) >= 7 hours.
Secondary Outcome Measures :
- Number of Participants With Immune Tolerance Induction (ITI) Success [ Time Frame: Up to 48 Weeks ]Number of participants who achieve ITI success where ITI success is defined as achieving all 3 of the following criteria: confirmed negative titers consisting of 2 consecutive negative inhibitor assessments within 2 weeks (<0.6 Bethesda units/mL by the Nijmegen-modified Bethesda assay); incremental recovery (IR) >= 66% of the expected IR at 2 consecutive assessments; half-life (t½) >=7 hours.
- Number of Participants Who Experience Relapse [ Time Frame: Approximately 48 Weeks ]Number of Participants with ITI success who reaches the criteria for relapse (defined as confirmed positive inhibitor titer >= 0.6 BU/mL or abnormal recovery after tolerance is achieved, and t½ less than [<] 7 hours) will be evaluated during the Tapering or Follow-Up Periods.
- Number of Bleeding Episodes During ITI, Tapering and Follow-Up periods [ Time Frame: Up to 2 Years ]A bleeding episode started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode.
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) as a Measure of Safety and Tolerability [ Time Frame: Up to 2 Years ]An AE is any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition.
- Number of Days Away From Work or School [ Time Frame: Up to 2 Years ]Number of days missed from school or work will be summarized descriptively.
- Number of Hospitalization Days [ Time Frame: Up to 2 Years ]Number of hospitalization days will be summarized descriptively.
- Adherence to Treatment Regimen [ Time Frame: Up to 2 Years ]Defined as percentage of administered doses versus planned doses.
- Consumption of rFVIIIFc [ Time Frame: Up to 2 Years ]Consumption will be assessed based on amount of administered study treatment.
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| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
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
Information from the National Library of Medicine
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): NCT03093480
Locations
Show 37 study locations
Sponsors and Collaborators
Bioverativ, a Sanofi company
Swedish Orphan Biovitrum
Investigators
| Study Director: | Clinical Sciences & Operations | Sanofi |
| Responsible Party: | Bioverativ, a Sanofi company |
| ClinicalTrials.gov Identifier: | NCT03093480 |
| Other Study ID Numbers: |
LPS16473 2017-000373-36 ( EudraCT Number ) 997HA402 ( Other Identifier: Bioverativ Therapeutics Inc. ) |
| First Posted: | March 28, 2017 Key Record Dates |
| Last Update Posted: | July 2, 2020 |
| Last Verified: | May 2020 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com/ |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | No |
Additional relevant MeSH terms:
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Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |