Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A (XTEND-Kids)
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| ClinicalTrials.gov Identifier: NCT04759131 |
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Recruitment Status :
Completed
First Posted : February 18, 2021
Last Update Posted : May 6, 2023
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Sponsor:
Bioverativ, a Sanofi company
Information provided by (Responsible Party):
Sanofi ( Bioverativ, a Sanofi company )
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Brief Summary:
Primary Objective:
- To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia A
Secondary Objectives:
- To evaluate the efficacy of BIVV001 as a prophylaxis treatment
- To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes
- To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes
- To evaluate the effect of BIVV001 prophylaxis on joint health outcomes
- To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes
- To evaluate the efficacy of BIVV001 for perioperative management
- To evaluate the safety and tolerability of BIVV001 treatment
- To assess the pharmacokinetics (PK) of BIVV001
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hemophilia A | Drug: efanesoctocog alfa (BIVV001) | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 75 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 3 Open-label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Pediatric Patients <12 Years of Age With Severe Hemophilia A |
| Actual Study Start Date : | February 19, 2021 |
| Actual Primary Completion Date : | January 18, 2023 |
| Actual Study Completion Date : | January 18, 2023 |
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: Prophylaxis
Participants will receive weekly dose of BIVV001 for 52 weeks.
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Drug: efanesoctocog alfa (BIVV001)
Pharmaceutical form:solution for injection Route of administration: Intravenous |
Primary Outcome Measures :
- Occurrence of inhibitor development [ Time Frame: baseline to 52 weeks ]The occurrence of inhibitor development (neutralizing antibodies directed against factor VIII [FVIII]) as determined via the Nijmegen modified Bethesda assay
Secondary Outcome Measures :
- Annualized bleeding rate (ABR) levels [ Time Frame: baseline to week 52 ]Annualized bleeding rate (ABR) for treated, for untreated, for all bleeding episodes.
- Annualized bleeding rate (ABR) by type of bleed [ Time Frame: baseline to week 52 ]ABR by type of bleed such as spontaneous or traumatic
- Annualized bleeding rate (ABR) by location of bleed [ Time Frame: baseline to week 52 ]ABR by location of bleed such as joint, muscle, internal, or skin/mucosa
- Percentage of participants who maintain FVIII activity above prespecified levels [ Time Frame: baseline to week 52 ]
- Number of injection and dose of BIVV001 to treat a bleeding episode [ Time Frame: baseline to week 52 ]
- Percentage of bleeding episodes treated with a single injection of BIVV001 [ Time Frame: baseline to week 52 ]
- Assessment of response to BIVV001 treatment of individual bleeding episodes [ Time Frame: 52 weeks ]Assessment of response to BIVV001 treatment of individual bleeding episodes based on the International Society on Thrombosis and Haemostasis (ISTH) 4-point response scale.
- Physician's global assessment of the participant's response based on BIVV001 treatment [ Time Frame: baseline to week 52 ]Physician's global assessment (PGA) of participant's response to BIVV001 treatment based on a 4-point response scale
- Total annualized BIVV001 consumption [ Time Frame: baseline to week 52 ]
- Annualized Joint Bleeding Rate (AJBR) [ Time Frame: baseline to week 52 ]
- Target joint resolution [ Time Frame: At week 52 ]Target joint resolution at week 52, based on ISTH criteria.
- Change in Hemophilia Joint Health Score (HJHS) total score and domain scores [ Time Frame: baseline to week 52 ]Change from baseline to week 52 in total score and domain scores (eg, swelling and strength) assessed by the HJHS.
- Changes in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL) total score [ Time Frame: baseline to week 52 ]Changes in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL) total score from baseline to Week 52 (≥ 4 years old and parent proxy for all ages)
- Changes in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL) physical health domain scores from [ Time Frame: baseline to week 52 ]Changes in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL) physical health domain scores from baseline to Week 52 (≥ 4 years old and parent proxy for all ages)
- Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment [ Time Frame: baseline to week 52 ]Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment on the ISTH 4 point response for surgical procedures scale
- Number of injections and dose to maintain hemostasis during perioperative period for major surgery [ Time Frame: baseline to week 52 ]
- Total BIVV001 consumption during perioperative period for major surgery [ Time Frame: baseline to week 52 ]
- Number of blood component transfusions used during perioperative period for major surgery [ Time Frame: baseline to week 52 ]
- Type of blood component transfusions used during perioperative period for major surgery [ Time Frame: baseline to week 52 ]
- Estimated blood loss during perioperative period for major surgery [ Time Frame: baseline to week 52 ]
- Number of participants with occurence of adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: baseline to week 52 ]Participants with ocurrence of adverse events (AEs) and serious adverse events (SAEs)
- Number of participants with occurrence of embolic and thrombotic events [ Time Frame: baseline to week 52 ]
- PK parameter: Maximum activity (Cmax) [ Time Frame: baseline to week 52 ]
- PK parameter: Elimination half-life (t1/2) [ Time Frame: baseline (day 1) ]
- PK parameter: Total clearance (CL) [ Time Frame: baseline (day 1) ]
- PK parameter: Total clearance at steady state (CLss) [ Time Frame: baseline (day 1) ]
- PK parameter:dose-normalized area under the activity-time curve (DNAUC) [ Time Frame: baseline (day 1) ]
- PK parameter: Area under the activity time curve (AUC) [ Time Frame: baseline (day 1) ]
- PK parameter: Volume of distribution at steady state (Vss) [ Time Frame: baseline (day 1) ]
- PK parameter: Mean residence time (MRT) [ Time Frame: baseline (day 1) ]
- PK parameter: Incremental recovery (IR) [ Time Frame: baseline to week 52 ]
- PK parameter: Trough activity (Ctrough) [ Time Frame: baseline to week 52 ]
- PK parameter: Time above predefined FVIII activity levels [ Time Frame: baseline (day 1) ]
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| Ages Eligible for Study: | up to 12 Years (Child) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion criteria :
- Participant must be younger than 12 years of age, at the time of signing the informed consent
- Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII as documented either by central laboratory testing at Screening or in historical medical records from a clinical laboratory demonstrating <1% FVIII coagulant activity (FVIII:C) or a documented genotype known to produce severe hemophilia A.
- Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs for patients aged 6-11 years and above 50 EDs for patients aged <6 years
- Weight above or equal to 10 kg.
Exclusion criteria:
- History of hypersensitivity or anaphylaxis associated with any FVIII product.
- History of a positive inhibitor (to FVIII) test defined as ≥0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant.
- Positive inhibitor test result, defined as ≥0.6 BU/mL at Screening.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
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): NCT04759131
Locations
Show 40 study locations
Sponsors and Collaborators
Bioverativ, a Sanofi company
Investigators
| Study Director: | Clinical Sciences & Operations | Sanofi |
| Responsible Party: | Bioverativ, a Sanofi company |
| ClinicalTrials.gov Identifier: | NCT04759131 |
| Other Study ID Numbers: |
EFC16295 2020-000769-18 ( EudraCT Number ) U1111-1244-0558 ( Other Identifier: UTN ) |
| First Posted: | February 18, 2021 Key Record Dates |
| Last Update Posted: | May 6, 2023 |
| Last Verified: | May 2023 |
| 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://vivli.org |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | 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 |