Clinical trial NCT06738485: Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products
Official 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Congenital Hemophilia A
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2024-12-17
Enrollment: 60
Sex: MALE
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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%).


Eligibility criteria: 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.

Primary outcomes:
measure: Incremental Recovery (IR) of rVIII-SingleChain
timeFrame: Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1
measure: Maximum Concentration (Cmax) of rVIII-SingleChain
timeFrame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
measure: Area Under the Plasma Concentration Time Curve from Time Zero to the Last Measurable Concentration (AUC0-last) of rVIII-SingleChain
timeFrame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
measure: Area Under the Plasma Concentration Time Curve from Time Zero to Infinity (AUC0-inf) of rVIII-SingleChain
timeFrame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
measure: Half-life (t1/2) of rVIII-SingleChain
timeFrame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
measure: Clearance (Cl) of rVIII-SingleChain
timeFrame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
measure: Annualized Spontaneous Bleeding Rate (AsBR)
timeFrame: Up to 29 weeks after rVIII-SingleChain administration
measure: Number of participants who develop FVIII inhibitors
timeFrame: During routine rVIII-SingleChain prophylaxis dosing, up to 29 weeks after rVIII-SingleChain administration.
Secondary outcomes:
measure: Time to reach maximum concentration (Tmax) of rVIII-SingleChain
timeFrame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
measure: Last Concentration (Clast) of rVIII-SingleChain
timeFrame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
measure: IR (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat pharmacokinetic [PK])
timeFrame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
measure: Cmax (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat PK)
timeFrame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
measure: AUC0-last of rVIII-SingleChain (Repeat PK)
timeFrame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
measure: AUC0-inf of rVIII-SingleChain (Repeat PK)
timeFrame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
measure: t1/2 of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)
timeFrame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
measure: Cl of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)
timeFrame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
measure: Annualized Bleeding Rate (ABR)
timeFrame: Up to 29 weeks after rVIII-SingleChain administration
measure: Hemostatic Efficacy for Major and Nonmajor Bleeding Episodes
timeFrame: Up to 29 weeks after rVIII-SingleChain administration
measure: Consumption of rVIII-SingleChain - number of infusions (doses)
timeFrame: Up to 29 weeks after rVIII-SingleChain administration
measure: Consumption of rVIII-SingleChain - IU/kg per participant per month
timeFrame: Up to 29 weeks after rVIII-SingleChain administration
measure: Consumption of rVIII-SingleChain - IU/kg per participant per year
timeFrame: Up to 29 weeks after rVIII-SingleChain administration
measure: Number of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis
timeFrame: Up to 29 weeks after rVIII-SingleChain administration
measure: Percentage of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis
timeFrame: Up to 29 weeks after rVIII-SingleChain administration
measure: Number of participants who develop noninhibitory antibodies against rVIII-SingleChain
timeFrame: Before, and up to 29 weeks after, rVIII-SingleChain administration
measure: Number of participants who develop antibodies against Chinese hamster ovary host cell protein
timeFrame: Before, and up to 29 weeks after, rVIII-SingleChain administration
measure: Number of participants with Treatment-emergent Adverse Events (TEAEs), including related TEAEs, and serious adverse events (SAEs)
timeFrame: Up to 33 weeks after rVIII-SingleChain administration
measure: Percentage of participants with TEAEs, including related TEAEs, and serious adverse events (SAEs)
timeFrame: Up to 33 weeks after rVIII-SingleChain administration
measure: Number of TEAEs (events)
timeFrame: Up to 33 weeks after rVIII-SingleChain administration
Other outcomes:
Locations:
city: Hefei
country: China
facility: The Second Hospital of Anhui Medical University
state: Anhui
city: Beijing
country: China
facility: Beijing Children's Hospital
state: Beijing
city: Fuzhou
country: China
facility: Fujian Medical University Union Hospital
state: Fujian
city: Guangzhou
country: China
facility: Guangzhou Women and Children's Medical Center
state: Guangdong
city: Guangzhou
country: China
facility: Nanfang Hospital of Southern Medical University
state: Guangdong
city: Liuzhou
country: China
facility: Liuzhou People's Hospital
state: Guangxi
city: Tianjin
country: China
facility: Hospital of Hematology, Chinese Academy of Medical Sciences
state: Hebei
city: Changsha
country: China
facility: Hunan Provincial Children's Hospital
state: Hunan
city: Xuzhou
country: China
facility: XuZhou Children's Hospital
state: Jiangsu
city: Xining
country: China
facility: Quinghai Provincial People's Hospital
state: Qinghai
city: Qingdao
country: China
facility: Qingdao Women and Children's Hospital
state: Shandong
Study officials:
affiliation: CSL Behring
name: Study Director
role: STUDY_DIRECTOR
References: