XTEND-Kids Pediatric Study | Sanofi

XTEND-Kids clinical trial^1,2

A 52-week, multinational, multicenter, single-arm, open-label Phase 3 trial was conducted in previously treated patients <12 years with severe hemophilia A

^* A subject in the <6 age group who had a positive inhibitor result at baseline (pre-exposure to study drug) and was withdrawn after 3 exposure days was excluded.¹
^† A subject in the 6 to <12 age group who received an intense consolidation treatment (2 to 3 infusions per week) for 15 weeks, after 2 traumatic hip joint bleeds was excluded from the efficacy analysis as the subject did not receive the weekly prophylaxis treatment as specified in the protocol for an extended period.¹

Primary Endpoint³:

The occurrence of inhibitor development

Secondary Endpoints³:

ABR for treated bleeds by type and location

Pharmacokinetics

Perioperative management

Safety and tolerability

Treatment of bleeds

Learn how ALTUVIIIO performed

ALTUVIIIO has efficacy data in the prophylaxis, on-demand, and perioperative settings for children <12 years.

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Safety from the pediatric study

Find out more about the ALTUVIIIO safety profile in the XTEND-Kids clinical trial.

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Indication

ALTUVIIIO^® [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl] is a von Willebrand Factor (VWF) independent recombinant DNA-derived, Factor VIII concentrate indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for:

Routine prophylaxis to reduce the frequency of bleeding episodes
On-demand treatment & control of bleeding episodes
Perioperative management of bleeding

Limitation of Use

ALTUVIIIO is not indicated for the treatment of von Willebrand disease.

Important Safety Information

Contraindications

ALTUVIIIO is contraindicated in patients who have had severe hypersensitivity reactions, including anaphylaxis, to the product or its excipients.

Warnings and Precautions

Allergic-type hypersensitivity reactions, including anaphylaxis, may occur with ALTUVIIIO. Allergic-type hypersensitivity reactions were not reported in the clinical trials. Advise patients to discontinue use of ALTUVIIIO if hypersensitivity symptoms occur and contact a physician and/or seek immediate emergency care.
Formation of neutralizing antibodies (inhibitors) to Factor VIII are possible following administration of ALTUVIIIO. Neutralizing antibodies were not reported in the clinical trials. Monitor all patients for the development of Factor VIII inhibitors by appropriate clinical observations and laboratory tests.
If assessment of plasma Factor VIII activity is needed, it is recommended to use a validated one-stage clotting assay. The ALTUVIIIO Factor VIII activity level is overestimated by the chromogenic assay and a specific ellagic acid-based aPTT reagent in one-stage clotting assay by approximately 2.5-fold. If these assays are used, divide the result by 2.5 to approximate the patient's ALTUVIIIO Factor VIII activity level.

Adverse Reactions

The most common adverse reactions (>10% of subjects) reported in clinical trials were headache and arthralgia.

Please see full Prescribing Information.

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IndicationImportant Safety Information

ABR=annualized bleed rate; IU=international unit.

References: 1. ALTUVIIIO Prescribing Information. Bioverativ Therapeutics Inc. Waltham, MA. 2. Data on file, May 2023. 3. Malec L, et al. Efanesoctocog Alfa Prophylaxis for Previously Treated Patients <12 Years of Age With Severe Hemophilia A. Oral presentation at: 2023 International Society on Thrombosis and Haemostasis (ISTH); June, 2023; Montreal, Canada.

Pediatric Study - Trial Design

XTEND-Kids clinical trial1,2