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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-002333-34 | EudraCT Number |
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This study will investigate the pharmacokinetics (PK), efficacy, and safety of rIX-FP for the routine prophylaxis of bleeding episodes in male Chinese previously treated patients (PTPs) with hemophilia B (FIX activity of ≤ 2%). In addition to the scheduled rIX-FP prophylaxis regimen, subjects may also receive rIX-FP episodic (on-demand) treatment for breakthrough bleeding episodes and rIX-FP for the prophylaxis and treatment of bleeding in emergency surgical procedures.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rIX-FP | Experimental | Subjects will receive rIX-FP as an intravenous (IV) infusion for a minimum of 50 exposure days (EDs) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) | Biological | Lyophilized powder for solution for intravenous injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incremental recovery (IR) (plasma FIX activity) | Before, and at 30 minutes after the end of, rIX-FP infusion on Day 1 | |
| Maximum plasma concentration (Cmax) | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 | |
| Terminal elimination half-life (t1/2) of rIX-FP | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 | |
| Area under the concentration-time curve (AUC) | AUC from time zero to the last measurable concentration (plasma FIX activity) (AUC0-last), and AUC from time zero extrapolated to infinity (AUC0-inf). | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 |
| Clearance (Cl) of rIX-FP | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 | |
| Annualized spontaneous bleeding rate (AsBR) | AsBR for treated bleeding episodes, by prophylaxis regimen and overall | Up to 18 months |
| Number of subjects who develop an inhibitor to FIX | Up to 18 months after rIX-FP infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of area under the concentration-time curve extrapolated (%AUCExt) | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK) | |
| Area under the first moment versus time curve extrapolated to infinity (AUMC0-∞) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Program Director | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Beijing Children's Hospital | Beijing | Beijing Municipality | 100045 | China | ||
| Union Hospital Affiliated to Fujian Medical University |
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.
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Requests for IPD will generally be considered once review by major regulatory authorities (ie 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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| Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK) |
| Mean residence time (MRT) | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK) |
| Apparent volume of distribution during the terminal phase (Vz) | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK) |
| Apparent volume of distribution at steady-state (Vss) | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK) |
| Time to reach Cmax (Tmax) | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK) |
| Elimination rate constant (λz) | Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK) |
| Incremental recovery (IR) (plasma FIX activity) - Repeat PK | Before, and at 30 minutes after the end of, rIX-FP infusion at Week 26 |
| Maximum plasma concentration (Cmax) - Repeat PK | Before, and up to 336 hours after the end of, rIX-FP infusion at Week 26 |
| Terminal elimination half-life (t1/2) of rIX-FP - Repeat PK | Before, and up to 336 hours after the end of, rIX-FP infusion at Week 26 |
| Area under the concentration-time curve (AUC) - Repeat PK | AUC from time zero to the last measurable concentration (plasma FIX activity) (AUC0-last), and AUC from time zero extrapolated to infinity (AUC0-inf). | Before, and up to 336 hours after the end of, rIX-FP infusion at Week 26 |
| Clearance (Cl) of rIX-FP - Repeat PK | Before, and up to 336 hours after the end of, rIX-FP infusion at Week 26 |
| Annualized bleeding rate (ABR) | ABR for treated, untreated, and both treated and untreated bleeding episodes (spontaneous bleeding, traumatic bleeding, unknown bleeding, and total bleeding episodes), by prophylaxis regimen and overall | Up to 18 months after rIX-FP infusion |
| Annualized joint bleeding rate (AjBR) | AjBR for treated, untreated, and both treated and untreated bleeding episodes, by prophylaxis regimen and overall | Up to 18 months after rIX-FP infusion |
| Clinical evaluation of hemostatic efficacy for major bleeding episodes | The investigator will rate the efficacy of the rIX-FP treatment for major bleeding episodes based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or no efficacy", by prophylaxis regimen and overall | Up to 18 months after rIX-FP infusion |
| Change in target joints | At baseline and up to 18 months after rIX-FP infusion |
| Consumption of rIX-FP - number of rIX-FP infusions (doses) | Consumption of rIX-FP expressed as number of rIX-FP infusions (doses), for the prophylaxis regimens, episodic (on-demand) treatment for bleeding episodes (if any), and total (overall) treatment | Up to 18 months after rIX-FP infusion |
| Consumption of rIX-FP - IU/kg per subject per month | Consumption of rIX-FP expressed as total amount (IU/kg) per subject per month, for the prophylaxis regimens, episodic (on-demand) treatment for bleeding episodes (if any), and total (overall) treatment | Up to 18 months after rIX-FP infusion |
| Consumption of rIX-FP - IU/kg per subject per year | Consumption of rIX-FP expressed as total amount (IU/kg) per subject per year, for the prophylaxis regimens, episodic (on-demand) treatment for bleeding episodes (if any), and total (overall) treatment. | Up to 18 months after rIX-FP infusion |
| Number of bleeding episodes requiring rIX-FP to achieve hemostasis | Number of bleeding episodes requiring 1, ≤ 2, or > 2 infusions (doses) of rIX-FP to achieve hemostasis | Up to 18 months after rIX-FP infusion |
| Percentage of bleeding episodes requiring rIX-FP to achieve hemostasis | Percentage of bleeding episodes requiring 1, ≤ 2, or > 2 infusions (doses) of rIX-FP to achieve hemostasis | Up to 18 months after rIX-FP infusion |
| Number of subjects who develop antibodies against rIX-FP | Before, and up to 18 months after, rIX-FP infusion |
| Number of subjects who develop antibodies against Chinese hamster ovary host cell protein | Before, and up to 18 months after, rIX-FP infusion |
| The number of subjects with treatment emergent adverse events (TEAEs) related to rIX-FP | Up to 18 months after rIX-FP infusion |
| The percentage of subjects with treatment emergent adverse events (TEAEs) related to rIX-FP | Up to 18 months after rIX-FP infusion |
| Fuzhou |
| Fujian |
| 350001 |
| China |
| Nanfang Hospital of Southern Medical University | Guangzhou | Guangdong | 510515 | China |
| Affiliated Hospital of Guizhou Medical University | Guiyang | Guizhou | 550004 | China |
| North China University Of Science And Technology Affiliated Hospital | Tangshan | Hebei | 63000 | China |
| Jinan Central Hospital | Jinan | Shandong | 250013 | China |
| Hospital of Hematology, Chinese Academy of Medical Sciences | Tianjin | Tianjin Municipality | 300020 | China |
| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| 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 |
| D040181 | Genetic Diseases, X-Linked |
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| ID | Term |
|---|---|
| D000418 | Albumins |
| C000618231 | albutrepenonacog alfa |
| ID | Term |
|---|---|
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
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