Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| EU CT Number | Other Identifier | 2023-503765-37-00 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CSL222 | Participants who received CSL222 and completed either the study CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891) will be followed up from Year 5 after administration of CSL222 in parent study up to Year 15. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV5-hFIXco-Padua | Genetic | Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs) | From Year 5 after administration of CSL222 in parent study up to Year 15 | |
| Percentage of Participants With SAEs and AESIs | From Year 5 after administration of CSL222 in parent study up to Year 15 | |
| Number of SAEs and AESIs | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Bleeding Rate (ABR) | The total, spontaneous, joint, and traumatic bleeds will be analyzed. | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes |
Not provided
Inclusion Criteria:
Received treatment with CSL222 in Study CSL222_2001 or Study CSL222_3001, and completed participation in Study CSL222_2001 or Study CSL222_3001 or at least 5 years have passed since receiving CSL222.
Exclusion Criteria:
Not Applicable
Not provided
Not provided
Adult male participants with hemophilia B who were previously treated with CSL222 in study CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Study Director | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| 10-15 Phoenix Childrens Hospital | Phoenix | Arizona | 85016 | United States | ||
| 10-14 Arkansas Children's Hospital - Pharmacology |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40188458 | Derived | von Drygalski A, Gomez E, Giermasz A, Castaman G, Key NS, Lattimore SU, Leebeek FWG, Miesbach WA, Recht M, Monahan PE, Le Quellec S, Pipe SW. Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. Blood Adv. 2025 Jul 22;9(14):3543-3552. doi: 10.1182/bloodadvances.2024015291. |
Not provided
Not provided
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 (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.
Not provided
Not provided
Not provided
Not provided
Not provided
| From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Endogenous Factor IX (FIX) Activity | Endogenous FIX activity (expressed as percentage [%]) will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay. | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Change from Baseline by Visit in Endogenous FIX Activity | Endogenous FIX activity (expressed as %) will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay. | At Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Annualized Consumption of FIX Replacement Therapy | The annualized consumption of FIX replacement therapy (excluding FIX replacement for invasive procedures) will be provided. | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Annualized Infusion Rate of FIX Replacement Therapy | Annualized infusion rate of FIX replacement therapy (excluding FIX replacement for invasive procedures) will be provided. | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Number of Participants Remaining Free of Continuous FIX Prophylaxis | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Percentage of Participants Remaining Free of Continuous FIX Prophylaxis | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Number of New Target Joints | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Percentage Resolution of Pre-existing Target Joints | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value | The EQ-5D-5L questionnaire consists of the EQ-5D-5L descriptive system and the EQ-5D visual analogue scale (VAS). The EQ-5D-5L descriptive system of health-related quality of life (QoL) consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single overall health state utility value. This utility value will be derived using the EQ-5D-5L Crosswalk Index Value Calculator v2.xls [Hernández Alava et al, 2023], using the United Kingdom value set. | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Change From Baseline in EQ-5D-5L Index Value | The EQ-5D-5L questionnaire consists of the EQ-5D-5L descriptive system and the EQ-5D VAS. The EQ-5D-5L descriptive system of health-related QoL consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single overall health state utility value. This utility value will be derived using the EQ-5D-5L Crosswalk Index Value Calculator v2.xls [Hernández Alava et al, 2023], using the United Kingdom value set. | Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15 |
| EQ-5D VAS Score | The EQ-5D VAS, which measures overall health status on a vertical VAS, ranges from 0 to 100. A higher score indicates better QoL. | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Change From Baseline in EQ-5D VAS Score | The EQ-5D VAS, which measures overall health status on a vertical VAS, ranges from 0 to 100. A higher score indicates better QoL. | Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score | The Hem-A-QoL consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a "not applicable" option). The total score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflecting a better QoL. | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Change From Baseline in Hem-A-QoL Total Score | The Hem-A-QoL consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a "not applicable" option). The total score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflecting a better QoL. | Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Little Rock |
| Arkansas |
| 72202 |
| United States |
| 10-22 Orthopaedic Institute for Children | Los Angeles | California | 90007 | United States |
| 10-21 Children's Hospital of Los Angeles | Los Angeles | California | 90027 | United States |
| 10-63 UC Davis Medical Center | Sacramento | California | 95817 | United States |
| 10-12 University of California, San Diego (UCSD) | San Diego | California | 92121 | United States |
| 10-25 University of Colorado Denver | Aurora | Colorado | 80045 | United States |
| 10-19 University of South Florida | Tampa | Florida | 33612 | United States |
| 10-10 University of Michigan Medical Center | Ann Arbor | Michigan | 48109 | United States |
| 10-16 University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| 10-18 Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| 10-20 Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| 10-28 University of Texas Health Science Center | Houston | Texas | 77030 | United States |
| 10-26 University of Utah | Salt Lake City | Utah | 84108 | United States |
| 10-62 Washington Institute for Coagulation | Seattle | Washington | 98104 | United States |
| 11-31 Cliniques Universitaires Saint-Luc | Brussels | 1200 | Belgium |
| 11-30 Universitair Ziekenhuis Leuven | Leuven | 3000 | Belgium |
| 12-32 Rigshospitalet | Copenhagen | 2100 | Denmark |
| 15-42 Vivantes Klinikum im Friedrichshain - Landsberger Allee | Berlin | 10249 | Germany |
| 19-56 St. James Hospital | Dublin | D08 A978 | Ireland |
| 13-33 Amsterdam Universitair Medische Centra (UMC) | Amsterdam-Zuidoost | 1105 AZ | Netherlands |
| 13-35 University Medical Center Groningen | Groningen | 9713 GZ | Netherlands |
| 13-34 Erasmus University Medical Center | Rotterdam | Netherlands |
| 13-36 Universitair Medisch Centrum Utrecht | Utrecht | 3584 CX | Netherlands |
| 16-43 Skane University Hospital | Malmö | SE-205 02 | Sweden |
| 18-52 Barts Health NHS Trust | London | England | E1 1FR. | United Kingdom |
| 18-54 Addenbrooke's Hospital | Cambridge | CB2 0QQ | United Kingdom |
| 18-53 University Hospital Southampton NHS Foundation Trust | Southampton | SO16 6YD | United Kingdom |
| 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 |
Not provided
Not provided