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

Phase 3Active Not RecruitingNCT06738485

CSL Behring60 enrolled

Overview

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%).

Study Type

INTERVENTIONAL

Allocation

Purpose

PREVENTION

Masking

NONE

Enrollment

Conditions

Congenital Hemophilia A

Interventions

Recombinant single-chain factor VIII (rVIII-SingleChain)BIOLOGICAL

Lyophilized powder for solution for intravenous injection

Eligibility

Sex: MALEMin age: 0 DaysMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (20)

The Second Hospital of Anhui Medical University

Hefei, Anhui, China

Outcomes

Primary Outcomes

Incremental Recovery (IR) of rVIII-SingleChain

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

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

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

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

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

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

Annualized Spontaneous Bleeding Rate (AsBR)

AsBR for treated bleeding episodes

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

Number of participants who develop FVIII inhibitors

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

Data from ClinicalTrials.gov

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Secondary Outcomes

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

Last Concentration (Clast) of rVIII-SingleChain

Last observed quantifiable plasma concentration

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

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

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

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

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

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

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

Annualized Bleeding Rate (ABR)

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

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

Hemostatic Efficacy for Major and Nonmajor Bleeding Episodes

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".

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

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

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

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

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

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

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

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

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

Number of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis

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

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

Percentage of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis

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

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

Number of participants who develop noninhibitory antibodies against rVIII-SingleChain

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

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

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

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

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

Number of TEAEs (events)

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