Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products
Phase 3
Not yet recruiting
- Conditions
- Congenital Hemophilia A
- Interventions
- Biological: Recombinant single-chain factor VIII (rVIII-SingleChain)
- Registration Number
- NCT06738485
- Lead Sponsor
- CSL Behring
- Brief Summary
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%).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- Male
- Target Recruitment
- 60
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).
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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.
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Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description rVIII-SingleChain Recombinant single-chain factor VIII (rVIII-SingleChain) Participants will receive rVIII-SingleChain as an intravenous (IV) infusion for a minimum of 50 exposure days (EDs).
- Primary Outcome Measures
Name Time Method Maximum Concentration (Cmax) of rVIII-SingleChain Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1 Incremental Recovery (IR) of rVIII-SingleChain Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1 Area Under the Plasma Concentration Time Curve from Time Zero to the Last Measurable Concentration (AUC0-last) of rVIII-SingleChain Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1 Area Under the Plasma Concentration Time Curve from Time Zero to Infinity (AUC0-inf) of rVIII-SingleChain Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1 Half-life (t1/2) of rVIII-SingleChain Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1 Clearance (Cl) of rVIII-SingleChain Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1 Annualized Spontaneous Bleeding Rate (AsBR) Up to 29 weeks after rVIII-SingleChain administration AsBR for treated bleeding episodes
Number of participants who develop FVIII inhibitors During routine rVIII-SingleChain prophylaxis dosing, up to 29 weeks after rVIII-SingleChain administration.
- Secondary Outcome Measures
Name Time Method Time to reach maximum concentration (Tmax) of rVIII-SingleChain Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1 Last Concentration (Clast) of rVIII-SingleChain 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
IR (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat pharmacokinetic [PK]) Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28 Cmax (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat PK) Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28 AUC0-last of rVIII-SingleChain (Repeat PK) Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28 Consumption of rVIII-SingleChain - IU/kg per participant per month 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.
Consumption of rVIII-SingleChain - IU/kg per participant per year 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.
AUC0-inf of rVIII-SingleChain (Repeat PK) Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28 t1/2 of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK) Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28 Cl of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK) Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28 Annualized Bleeding Rate (ABR) 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)
Hemostatic Efficacy for Major and Nonmajor Bleeding Episodes 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".
Consumption of rVIII-SingleChain - number of infusions (doses) Up to 29 weeks after rVIII-SingleChain administration Number of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis 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
Percentage of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis 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
Number of participants who develop noninhibitory antibodies against rVIII-SingleChain Before, and up to 29 weeks after, rVIII-SingleChain administration Number of participants who develop antibodies against Chinese hamster ovary host cell protein Before, and up to 29 weeks after, rVIII-SingleChain administration Number of participants with Treatment-emergent Adverse Events (TEAEs), including related TEAEs, and serious adverse events (SAEs) Up to 33 weeks after rVIII-SingleChain administration Percentage of participants with TEAEs, including related TEAEs, and serious adverse events (SAEs) Up to 33 weeks after rVIII-SingleChain administration Number of TEAEs (events) Up to 33 weeks after rVIII-SingleChain administration