XTEND-1 and XTEND-Kids Trials | Sanofi

First-in-class ALTUVIIIO: XTEND-1 clinical trial¹

In previously treated patients (PTPs) who had received Factor VIII replacements^2*

XTEND-1 Pivotal Trial²

The Prior Factor VIII Prophylaxis Group included the first female patient with severe hemophilia A in a pivotal Factor VIII clinical trial^1-15‡

XTEND-1 Study Design²:

A multicenter, open-label, nonrandomized trial in adults and adolescents ≥12 years (158 males and 1 female) with severe hemophilia A, including an intrapatient comparison in patients who had participated in a prospective observational study prior to enrollment in XTEND-1 study.

*Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived Factor VIII or cryoprecipitate for at least 150 exposure days for patients ≥12 years.¹⁶
†Efficacy of prophylaxis was evaluated in patients with at least 26 weeks of exposure to ALTUVIIIO (N=128).²
‡Pivotal trials for Recombinate™ (Antihemophilic Factor) did not present demographic data based on sex.¹²

XTEND-1 Endpoints^1,16

Primary Endpoint:

Mean annualized bleed rate (ABR)

Secondary Endpoints:

Intrapatient ABR comparison (key secondary endpoint)

Perioperative management

Treatment of bleeds

Quality of life (physical health and pain)

Joint health outcomes

Pharmacokinetics

Safety and tolerability

XTEND-1: A large group of 159 diverse patients were studied¹

Diverse patient population (including 1 female participant)

99% Male
~1% Female

61% White
19% Not reported or other
18% Asian
2% Black or African American

81% 18-64 years
16% 12-17 years
3% ≥65 years

See how ALTUVIIIO performed

ALTUVIIIO has efficacy data in the prophylaxis, on-demand, and perioperative settings for adult and adolescent patients.

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Learn how ALTUVIIIO was studied

The XTEND-Kids trial studied ALTUVIIIO in pediatric patients <12 years old.

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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.

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Indication

Important Safety Information

IU=international unit.

References: 1. von Drygalski A, et al. N Engl J Med. 2023;388(4):310-318. 2. ALTUVIIIO Prescribing Information. Bioverativ Therapeutics Inc. Waltham, MA. 3. Advate Prescribing Information. Takeda Pharmaceuticals U.S.A., Inc. Lexington, MA. 4. Adynovate Prescribing Information. Takeda Pharmaceuticals U.S.A., Inc. Lexington, MA. 5. Afstyla Prescribing Information. CSL Behring LLC. Kankakee, IL. 6. Eloctate Prescribing Information. Bioverativ Therapeutics Inc. Waltham, MA. 7. Esperoct Prescribing Information. Novo Nordisk Inc. Plainsboro, NJ. 8. Jivi Prescribing Information. Bayer HealthCare LLC. Whippany, NJ. 9. Kovaltry Prescribing Information. Bayer HealthCare LLC. Whippany, NJ. 10. Novoeight Prescribing Information. Novo Nordisk Inc. Plainsboro, NJ. 11. Nuwiq Prescribing Information. Octapharma USA, Inc. Paramus, NJ. 12. Recombinate Prescribing Information. Takeda Pharmaceuticals U.S.A., Inc. Lexington, MA. 13. Clinicaltrials.gov. NCT00543439 (Xyntha prophylaxis). Sexes Eligible for Study. Accessed May 15, 2024. https://classic.clinicaltrials.gov/ct2/show/NCT00543439 14. Clinicaltrials.gov. NCT00141843 (Xyntha bioequiv). Sexes Eligible for Study. Accessed May 15, 2024. https://clinicaltrials.gov/ct2/show/NCT00141843 15. Clinicaltrials.gov. NCT00243659 (Xyntha surgery). Sexes Eligible for Study. Accessed May 15, 2024. https://clinicaltrials.gov/ct2/show/NCT00243659 16. von Drygalski A, et al. Supplementary Appendix. N Engl J Med. 2023;388(4):310-318.

Adult & Adolescent Study - Trial Design

First-in-class ALTUVIIIO: XTEND-1 clinical trial1

In previously treated patients (PTPs) who had received Factor VIII replacements2*

XTEND-1 Pivotal Trial2

XTEND-1 Study Design2:

XTEND-1 Endpoints1,16

Primary Endpoint:

Secondary Endpoints:

XTEND-1: A large group of 159 diverse patients were studied1

Diverse patient population (including 1 female participant)

See how ALTUVIIIO performed

Learn how ALTUVIIIO was studied

Indication

Limitation of Use

Important Safety Information

Contraindications

Warnings and Precautions

Adverse Reactions

Indication

Important Safety Information

First-in-class ALTUVIIIO: XTEND-1 clinical trial¹

In previously treated patients (PTPs) who had received Factor VIII replacements^2*

XTEND-1 Pivotal Trial²

XTEND-1 Study Design²:

XTEND-1 Endpoints^1,16

XTEND-1: A large group of 159 diverse patients were studied¹