Recombinant single-chain factor VIII (rVIII-SingleChain)
Recombinant single-chain factor VIII (rVIII-SingleChain): Lyophilized powder for solution for intravenous injection
Study 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
Sex
MALE
Min age
0 Days
Max age
65 Years
Healthy volunteers
No
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 outcome measure(s)
Incremental Recovery (IR) of rVIII-SingleChain — Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1
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
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.
Trial sites (20)
Facility
City
Region
Status
The Second Hospital of Anhui Medical University
Hefei
Anhui
Beijing Children's Hospital
Beijing
Beijing Municipality
Children's Hospital of Chongqing Medical University
Chongqing
Chongqing Municipality
Fujian Medical University Union Hospital
Fuzhou
Fujian
Guangzhou Women and Children's Medical Center
Guangzhou
Guangdong
Nanfang Hospital of Southern Medical University
Guangzhou
Guangdong
Liuzhou People's Hospital
Liuzhou
Guangxi
North China University of Science and Technology Affiliated Hospital
Tangshan
Hebei
Hospital of Hematology, Chinese Academy of Medical Sciences
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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