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NCT Number: NCT06738485

Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products

For bridging the available global clinical data of rVIII-SingleChain, with the Chinese population, the aim of this study in China is to investigate the pharmacokinetics (PK) of rVIII-SingleChain after an initial and repeat dose and to assess efficacy and safety during 2 to 3 times weekly prophylaxis treatment with rVIII-SingleChain in male Chinese PTPs with severe hemophilia A (FVIII activity less than [<] 1%).

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Key information

Conditions

Congenital Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders

Age range

0 day–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

The Second Hospital of Anhui Medical University, Hefei, Anhui, China

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • • Male Chinese participants <= 65 years of age.
  • • Participants with severe hemophilia A (FVIII activity < 1%).
  • • Participants who have received FVIII products for >= 150 EDs (>= 6 years of age) or >= 50 EDs (< 6 years of age).

Exclusion criteria

  • • Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or hamster protein.
  • • Known congenital or acquired coagulation disorder other than congenital FVIII deficiency.
  • • Currently receiving intravenous (IV) immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment.
  • • Receiving any cryoprecipitate, whole blood, or plasma within 30 days before administration of rVIII-SingleChain.
  • • Use of traditional or herbal Chinese medicine(s) with an impact on hemophilia, including coagulation, within 28 days before Day 1 and / or refusal to abstain from these during the study until the end of the participant's participation in the study.

Treatment and study plan

Recombinant single-chain factor VIII (rVIII-SingleChain)

Biological

Lyophilized powder for solution for intravenous injection

Other names: CSL627

Primary outcomes

  1. Incremental Recovery (IR) of rVIII-SingleChain

    Time frame: Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1

  2. Maximum Concentration (Cmax) of rVIII-SingleChain

    Time frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  3. Area Under the Plasma Concentration Time Curve from Time Zero to the Last Measurable Concentration (AUC0-last) of rVIII-SingleChain

    Time frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  4. Area Under the Plasma Concentration Time Curve from Time Zero to Infinity (AUC0-inf) of rVIII-SingleChain

    Time frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  5. Half-life (t1/2) of rVIII-SingleChain

    Time frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  6. Clearance (Cl) of rVIII-SingleChain

    Time frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  7. Annualized Spontaneous Bleeding Rate (AsBR)

    Time frame: Up to 29 weeks after rVIII-SingleChain administration

    AsBR for treated bleeding episodes

  8. Number of participants who develop FVIII inhibitors

    Time frame: During routine rVIII-SingleChain prophylaxis dosing, up to 29 weeks after rVIII-SingleChain administration.

Secondary outcomes

  1. Time to reach maximum concentration (Tmax) of rVIII-SingleChain

    Time frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  2. Last Concentration (Clast) of rVIII-SingleChain

    Time frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

    Last observed quantifiable plasma concentration

  3. IR (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat pharmacokinetic [PK])

    Time frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28

  4. Cmax (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat PK)

    Time frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28

  5. AUC0-last of rVIII-SingleChain (Repeat PK)

    Time frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28

  6. AUC0-inf of rVIII-SingleChain (Repeat PK)

    Time frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28

  7. t1/2 of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)

    Time frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28

  8. Cl of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)

    Time frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28

  9. Annualized Bleeding Rate (ABR)

    Time frame: Up to 29 weeks after rVIII-SingleChain administration

    Total ABR for treated, untreated, and both treated and untreated bleeding episodes (spontaneous, traumatic, and unknown cause)

  10. Hemostatic Efficacy for Major and Nonmajor Bleeding Episodes

    Time frame: Up to 29 weeks after rVIII-SingleChain administration

    The investigator will rate the efficacy of the rVIII-SingleChain treatment for major and nonmajor bleeding episodes based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or no efficacy".

  11. Consumption of rVIII-SingleChain - number of infusions (doses)

    Time frame: Up to 29 weeks after rVIII-SingleChain administration

  12. Consumption of rVIII-SingleChain - IU/kg per participant per month

    Time frame: Up to 29 weeks after rVIII-SingleChain administration

    rVIII-SingleChain consumption (IU/kg) per participant per month, for the weekly regimen, on-demand treatment, and total treatment.

  13. Consumption of rVIII-SingleChain - IU/kg per participant per year

    Time frame: Up to 29 weeks after rVIII-SingleChain administration

    rVIII-SingleChain consumption (IU/kg) per participant per year, for the weekly regimen, on-demand treatment, and total treatment.

  14. Number of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis

    Time frame: Up to 29 weeks after rVIII-SingleChain administration

    Number of bleeding episodes requiring 1, <= 2, or > 2 infusions (doses) of rVIII-SingleChain to achieve hemostasis

  15. Percentage of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis

    Time frame: Up to 29 weeks after rVIII-SingleChain administration

    Percentage of bleeding episodes requiring 1, <= 2, or > 2 infusions (doses) of rVIII-SingleChain to achieve hemostasis

  16. Number of participants who develop noninhibitory antibodies against rVIII-SingleChain

    Time frame: Before, and up to 29 weeks after, rVIII-SingleChain administration

  17. Number of participants who develop antibodies against Chinese hamster ovary host cell protein

    Time frame: Before, and up to 29 weeks after, rVIII-SingleChain administration

  18. Number of participants with Treatment-emergent Adverse Events (TEAEs), including related TEAEs, and serious adverse events (SAEs)

    Time frame: Up to 33 weeks after rVIII-SingleChain administration

  19. Percentage of participants with TEAEs, including related TEAEs, and serious adverse events (SAEs)

    Time frame: Up to 33 weeks after rVIII-SingleChain administration

  20. Number of TEAEs (events)

    Time frame: Up to 33 weeks after rVIII-SingleChain administration

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

A Phase 3, Open-label, Multicenter, Pharmacokinetics, Efficacy, and Safety Study of a Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Previously Treated Patients (PTPs) With Hemophilia A

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Dec 17, 2024
Registry last updated
Apr 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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