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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-001026-34 | EudraCT Number |
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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rVIII-SingleChain | Experimental | Participants will receive rVIII-SingleChain as an intravenous (IV) infusion for a minimum of 50 exposure days (EDs). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Recombinant single-chain factor VIII (rVIII-SingleChain) | Biological | Lyophilized powder for solution for intravenous injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) | AsBR for treated bleeding episodes | Up to 29 weeks after rVIII-SingleChain administration |
| Number of participants who develop FVIII inhibitors |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Second Hospital of Anhui Medical University | Hefei | Anhui | 230000 | China | ||
| Beijing Children's Hospital |
CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Requests for IPD will generally be considered once review by major regulatory authorities (i.e., FDA, EMA) is complete and the primary publication is available.
Proposed research should seek to answer a previously unanswered important medical or scientific question.
Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.
If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
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| During routine rVIII-SingleChain prophylaxis dosing, up to 29 weeks after rVIII-SingleChain administration. |
Last observed quantifiable plasma concentration
| 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]) | 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 |
| 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) | Total ABR for treated, untreated, and both treated and untreated bleeding episodes (spontaneous, traumatic, and unknown cause) | 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". | Up to 29 weeks after rVIII-SingleChain administration |
| Consumption of rVIII-SingleChain - number of infusions (doses) | 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. | 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. | 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 | 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 | Up to 29 weeks after rVIII-SingleChain administration |
| 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 |
| Beijing |
| Beijing Municipality |
| 100045 |
| China |
| Children's Hospital of Chongqing Medical University | Chongqing | Chongqing Municipality | 400014 | China |
| Fujian Medical University Union Hospital | Fuzhou | Fujian | 350001 | China |
| Guangzhou Women and Children's Medical Center | Guangzhou | Guangdong | 510623 | China |
| Nanfang Hospital of Southern Medical University | Guangzhou | Guangdong | 515399 | China |
| Liuzhou People's Hospital | Liuzhou | Guangxi | 545006 | China |
| North China University of Science and Technology Affiliated Hospital | Tangshan | Hebei | 63000 | China |
| Hospital of Hematology, Chinese Academy of Medical Sciences | Tianjin | Hebei | 300020 | China |
| Henan children's hospital Zhengzhou children's Hospital | Zhengzhou | Henan | 450053 | China |
| Hunan Provincial Children's Hospital | Changsha | Hunan | 410021 | China |
| Nanjing Children's Hospital | Nanjing | Jiangsu | 210008 | China |
| XuZhou Children's Hospital | Xuzhou | Jiangsu | 221006 | China |
| Jiangxi Provincial People's Hospital | Nanchang | Jiangxi | 212028 | China |
| Quinghai Provincial People's Hospital | Xining | Qinghai | 81000 | China |
| Jinan Central Hospital | Jinan | Shandong | 250013 | China |
| Qingdao Women and Children's Hospital | Qingdao | Shandong | 266071 | China |
| Shanghai Jiaotong University School of Medicine, Ruijin Hospital | Shanghai | Shanghai Municipality | 200020 | China |
| The second affiliated hospital of Kunming Medical University | Kunming | Yunan | 650011 | China |
| The Children's Hospital Zhejiang University School of Medicine | Hangzhou | Zhejiang | 310005 | China |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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