Overview
Sponsor-declared trial summary
Phase
Phase II and Phase III (Integrated)
Status
Ongoing, recruitment ended
Participants planned
56
Countries
6
Sites
10
Hemophilia B
To assess the long-term safety in adult male subjects with hemophilia B who were treated with CSL222 in Study CSL222_2001 or Study CSL222_3001
Key facts
- Sponsor
- CSL Behring LLC
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 30 Sep 2024 → ongoing
- Decision date (initial)
- 2024-03-05
- Transition trial
- No
- Low-intervention
- Yes
- Rare-disease indication
- Yes
- Vulnerable population
- No
External identifiers
- EU CT number
- 2023-503765-37-00
- ClinicalTrials.gov
- NCT05962398
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
To assess the long-term safety in adult male subjects with hemophilia B who were treated with CSL222 in Study CSL222_2001 or Study CSL222_3001
Conditions and MedDRA coding
Hemophilia B
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | LLT | 10018939 | Haemophilia B (Factor IX) | 10010331 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Long-term Follow-up This is an open-label, long-term follow-up extension study enrolling subjects who participated in Study CSL222_2001 or Study CSL222_3001. Subjects will be monitored for long-term safety and efficacy from approximately 5 years until 15 years after treatment with CSL222 in either parent study.
|
Not Applicable | None | 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. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- 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.
- Provided written informed consent.
- Willing and able to adhere to all protocol requirements.
Exclusion criteria 1
- There are no exclusion criteria in this study
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 3
- Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)
- Percentage of Participants with SAEs and AESIs
- Number of SAEs and AESIs
Secondary endpoints 16
- Annualized Bleeding Rate (ABR)
- Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes
- Endogenous Factor IX (FIX) Activity
- Change from Baseline by Visit in Endogenous FIX Activity
- Annualized Consumption of FIX Replacement Therapy
- Annualized Infusion Rate of FIX Replacement Therapy
- Number of Participants Remaining Free of Continuous FIX Prophylaxis
- Percentage of Participants Remaining Free of Continuous FIX Prophylaxis
- Number of New Target Joints
- Percentage Resolution of Pre-existing Target Joints
- EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value
- Change From Baseline in EQ-5D-5L Index Value
- EQ-5D VAS Score
- Change From Baseline in EQ-5D VAS Score
- Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score
- Change From Baseline in Hem-A-QoL Total Score
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Hemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion
PRD10234072 · Product
- Active substance
- Etranacogene Dezaparvovec
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- IV INFUSION
- Max daily dose
- 2 millilitre(s)/kilogram
- Max total dose
- 2 millilitre(s)/kilogram
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- NOTASSIGN — -
- Marketing authorisation
- EU/1/22/1715/001
- MA holder
- CSL BEHRING GMBH
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/18/1999
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
CSL Behring LLC
- Sponsor organisation
- CSL Behring LLC
- Address
- 1020 1st Avenue
- City
- King Of Prussia
- Postcode
- 19406-1310
- Country
- United States
Scientific contact point
- Organisation
- CSL Behring LLC
- Contact name
- Trial Registration Coordinator
Public contact point
- Organisation
- CSL Behring LLC
- Contact name
- Trial Registration Coordinator
Third parties 10
| Organisation | City, country | Duties |
|---|---|---|
| Block Clinical Inc. ORG-100048643
|
San Diego, United States | Other |
| Unilabs A/S ORG-100032351
|
Copenhagen Oe, Denmark | Laboratory analysis |
| Everest Clinical Research Corporation ORG-100041734
|
Markham, Canada | Data management, E-data capture |
| Medpace Inc. ORG-100026760
|
Cincinnati, United States | On site monitoring, Code 12, Code 13, Other, Code 5, Code 8 |
| Precision For Medicine Inc. ORG-100041895
|
Frederick, United States | Laboratory analysis |
| Inseption Group LLC ORG-100041732
|
Lansdale, United States | Other |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| MEDPACE LABORATORIES ORG-100042942
|
Leuven, Belgium | Laboratory analysis |
| ProtaGene CGT GmbH ORG-100041450
|
Heidelberg, Germany | Laboratory analysis |
| Medpace Reference Laboratories LLC ORG-100041727
|
Cincinnati, United States | Laboratory analysis |
Locations
6 EU/EEA countries · 10 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Ongoing, recruitment ended | 6 | 2 |
| Denmark | Ongoing, recruitment ended | 3 | 1 |
| Germany | Ongoing, recruitment ended | 2 | 1 |
| Ireland | Ongoing, recruitment ended | 3 | 1 |
| Netherlands | Ongoing, recruitment ended | 12 | 4 |
| Sweden | Ongoing, recruitment ended | 2 | 1 |
| Rest of world
United Kingdom, United States
|
— | 28 | — |
Investigational sites
Cliniques Universitaires Saint-Luc
Hematology, Hippokrateslaan 10, Batiment 54, Sint-Lambrechts-Woluwe
UZ Leuven
Vascular Medicine, Herestraat 49, 3000, Leuven
Rigshospitalet
Department of Hematology, Blegdamsvej 9, 2100, Copenhagen Oe
Vivantes Netzwerk fuer Gesundheit GmbH
Klinikum für Angiologie/Hömostaseologie, Landsberger Allee 49, Friedrichshain, Berlin
Academisch Medisch Centrum
Internal and Vascular Medicine & Haemophilia, Meibergdreef 9, 1105 AZ, Amsterdam
Universitair Medisch Centrum Groningen
Haematology, Hanzeplein 1, 9713 GZ, Groningen
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Haematology, Dr. Molewaterplein 40, 3015 GD, Rotterdam
Universitair Medisch Centrum Utrecht
Van Creveldkliniek, Heidelberglaan 100, 3584 CX, Utrecht
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Belgium | 2024-10-29 | 2024-10-29 | 2025-01-28 | ||
| Denmark | 2024-10-23 | 2024-10-23 | 2024-12-18 | ||
| Germany | 2025-03-10 | 2025-03-10 | 2025-11-27 | ||
| Ireland | 2025-02-12 | 2025-02-12 | 2025-03-05 | ||
| Netherlands | 2024-09-30 | 2024-09-30 | 2025-04-07 | ||
| Sweden | 2025-01-21 | 2025-01-21 | 2025-01-28 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 91 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_CSL222_3003_Protocol_2023-503765-37-00_english_redacted | Amendment3 |
| Protocol (for publication) | D4_CSL222_3003_EQ-5D-5L Paper Self-Complete_dan-DK | 1.1 |
| Protocol (for publication) | D4_CSL222_3003_EQ-5D-5L Paper Self-Complete_deu_DE | 1 |
| Protocol (for publication) | D4_CSL222_3003_EQ-5D-5L Paper Self-Complete_dut-BE | 1.2 |
| Protocol (for publication) | D4_CSL222_3003_EQ-5D-5L Paper Self-Complete_fr-BE | 1.1 |
| Protocol (for publication) | D4_CSL222_3003_EQ-5D-5L Paper Self-Complete_nld-NL | 1.1 |
| Protocol (for publication) | D4_CSL222_3003_Patient Facing Documents_Bleed Diary_dut-BE | 2.0 |
| Protocol (for publication) | D4_CSL222_3003_Patient Facing Documents_Bleed Diary_fr-BE | 2.0 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_bleeddiary_deu_DE | 1.0 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_BleedDiary_nld-NL | 2.0 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_FactorIXUseDiary_dan-DK | 2.0 |
| Protocol (for publication) | D4_CSL222_3003_Patient-facing-Documents_FactorIXUseDiary_dut-BE | 2.0 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_FactorIXUseDiary_fre-BE | 2.0 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_FactorIXusediary_nld-NL | 2.0 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_FIXuse_deu_DE | 1.0 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_Haem-A-QoL_dan-DK | 1 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_Haem-A-QoL_deu_DE | 1 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_Haem-A-Qol_dut-BE | 1 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_Haem-A-QoL_fre-BE | 1 |
| Protocol (for publication) | D4_CSL222_3003_Patient-Facing-Documents_Heam-A-Qol_nld-NL | 1 |
| Protocol (for publication) | D4_Patient facing documents_DE_FollowUpBleedDiary_CSL Behring LLC | 2.0 |
| Protocol (for publication) | D4_Patient facing documents_DE_FollowUpFactorIXUseDiary_CSL Behring LLC | 2.0 |
| Protocol (for publication) | D4_Patient facing documents_DK_FollowUpBleedDiary_CSL Behring LLC | 2.0 |
| Protocol (for publication) | D4_Patient facing documents_IE_FollowUpBleedDiary_CSL Behring LLC | 2.0 |
| Protocol (for publication) | D4_Patient facing documents_IE_FollowUpFactorIXUseDiary_CSL Behring LLC | 2.0 |
| Protocol (for publication) | D4_Patient facing documents_PROMIS-29_DE_CSL Behring LLC_statement_for publication | N/A |
| Protocol (for publication) | D4_Patient facing documents_PROMIS-29_DK_CSL Behring LLC_statement_for publication | N/A |
| Protocol (for publication) | D4_Patient facing documents_PROMIS-29_DUT-FLE_CSL Behring LLC_statement_for publication | N/A |
| Protocol (for publication) | D4_Patient facing documents_PROMIS-29_EN_CLS Behring LLC_statement_for publication | N/A |
| Protocol (for publication) | D4_Patient facing documents_PROMIS-29_FR_CSL Behring LLC_statement_for publication | N/A |
| Protocol (for publication) | D4_Patient facing documents_PROMIS-29_SE_CSL Behring LLC_statement_for publication | N/A |
| Protocol (for publication) | D4_Patient facing documents_SE_FollowUpBleedDiary_CSL Behring LLC | 2.0 |
| Protocol (for publication) | D4_Patient facing documents_SE_FollowUpFactorIXUseDiary_CSL Behring LLC | 2 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Belgium_CSL Behring LLC | 3 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_DE_CSL Behring LLC | 3 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_DK_CSL Behring LLC | 4 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_IE_CSL Behring LLC | 3 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Netherlands_CSL Behring LLC | 3.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_SE_CSLBehringLLC | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_ Brochure_CSLBehringLLC | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_BE-DUT_CSL Behring LLC | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_BE-FR_CSL Behring LLC | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_CSL Behring LLC | 3 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_CSL Behring LLC | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_CSL Behring LLC | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment Material_Brochure_DE_CSL Behring LLC | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_adults_CSL Behring LLC | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Data Processor_CSL Behring_Clean | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Future Research_DE_CSL Behring LLC | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_LTSFU ICF_BE-DUT_CSL Behring LLC | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_LTSFU ICF_BE-FR_CSL Behring LLC | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_LTSFU ICF_CSLBehringLLC | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_LTSFU ICF_ENG_CSL Behring LLC | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_LTSFU_CSL Behring LLC | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_LTSFU_CSL Behring LLC_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_LTSFU_DE_CSL Behring LLC_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Adult_CSL Behring LLC_redacted | 5.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_BE-DUT_CSL Behring LLC | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_BE-FR_CSL Behring LLC | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_CSL Behring LLC_redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_CSLBehringLLC | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_ENG_CSL Behring LLC | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_DE_CSL Behring LLC_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Optional Future Research_CSL Behring LLC_redacted | 4.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Safety Only Follow Up_CSL Behring LLC_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Travel ICF_CSL Behring LLC | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Travel ICF_DE_CSL Behring LLC | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Convenience Programme_Intro Letter_CSL Behring | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Convenience Programme_IRB Screenshots_CSL Behring | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Convenience Programme_Travel Policy_CSL Behring | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Data Processor Consent_DE_CSLBehringLLC | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Data Processor Consent_DU_CSLBehringLLC | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Data Processor Consent_EN_CSLBehringLLC | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Data Processor Consent_FR_CSLBehringLLC | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_GP Letter_CSL Behring | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Optional Contact Form_CSLBehringLLC | 1 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Optional_Contact_Card_CSL Berhing LLC | 1 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient_Alert_Card_CSL Behring LLC | 2 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_PatientAlertCard_CSLBehringLLC | 2 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Your rights_CSL Behring LLC | NA |
| Synopsis of the protocol (for publication) | D1_CSL222_3003_Protocol synopsis_2023-503765-37-00_BE-deu | Amendment3 |
| Synopsis of the protocol (for publication) | D1_CSL222_3003_Protocol synopsis_2023-503765-37-00_BE-dut | Amendment3 |
| Synopsis of the protocol (for publication) | D1_CSL222_3003_Protocol synopsis_2023-503765-37-00_BE-fre | Amendment3 |
| Synopsis of the protocol (for publication) | D1_CSL222_3003_Protocol synopsis_2023-503765-37-00_english | Amendment3 |
| Synopsis of the protocol (for publication) | D1_CSL222_3003_Protocol synopsis_2023-503765-37-00_NL-dut | Amendment3 |
| Synopsis of the protocol (for publication) | D1_CSL222_3003_Protocol-Synopsis_2023-503765-37-00_sw | Amendment2 |
| Synopsis of the protocol (for publication) | D1_Protocol_Synopsis_2023-503765-37_EN_CSL Behring LLC_Tracked Changes | Amendment3 |
| Synopsis of the protocol (for publication) | D1_Protocol_Synopsis_DE_2023-503765-37_ CSL Behring LLC_Tracked Changes | Amendment3 |
| Synopsis of the protocol (for publication) | D1_Protocol_Synopsis_DUT_2023-503765-37_CSL Behring LLC_TC | Amendment3 |
| Synopsis of the protocol (for publication) | D1_Protocol_Synopsis_DUT_2023-503765-37_CSL Behring LLC_TC | Amendment3 |
| Synopsis of the protocol (for publication) | D1_Protocol_Synopsis_FR_2023-503765-37_CSL Behring LLC_TC | Amendment3 |
Application history
19 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-10-27 | Netherlands | Acceptable 2024-03-04
|
2024-03-04 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-03-12 | Netherlands | Acceptable 2024-03-04
|
2024-03-12 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2024-03-15 | Netherlands | Acceptable 2024-03-04
|
2024-03-15 |
| 4 | SUBSEQUENT ADDITION OF MSC | APP-4 | 2024-03-27 | Acceptable 2024-03-04
|
2024-06-24 | |
| 5 | SUBSEQUENT ADDITION OF MSC | APP-5 | 2024-03-27 | Acceptable 2024-03-04
|
2024-06-21 | |
| 6 | SUBSEQUENT ADDITION OF MSC | APP-6 | 2024-03-27 | Acceptable 2024-03-04
|
2024-06-17 | |
| 7 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-04-04 | Acceptable | 2024-06-24 | |
| 8 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-04-04 | Netherlands | Acceptable | 2024-04-19 |
| 9 | SUBSTANTIAL MODIFICATION | SM-3 | 2024-04-04 | Acceptable | 2024-05-17 | |
| 10 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2024-06-24 | Acceptable | 2024-06-24 | |
| 11 | SUBSTANTIAL MODIFICATION | SM-4 | 2024-08-09 | Netherlands | Acceptable 2024-11-14
|
2024-11-14 |
| 12 | NON SUBSTANTIAL MODIFICATION | NSM-4 | 2025-02-28 | Netherlands | Acceptable 2024-11-14
|
2025-02-28 |
| 13 | NON SUBSTANTIAL MODIFICATION | NSM-5 | 2025-07-02 | Netherlands | Acceptable 2024-11-14
|
2025-07-02 |
| 14 | SUBSTANTIAL MODIFICATION | SM-5 | 2025-07-07 | Acceptable | 2025-08-08 | |
| 15 | SUBSTANTIAL MODIFICATION | SM-6 | 2025-07-07 | Acceptable | 2025-09-09 | |
| 16 | SUBSTANTIAL MODIFICATION | SM-7 | 2025-07-07 | Netherlands | Acceptable | 2025-08-18 |
| 17 | SUBSTANTIAL MODIFICATION | SM-8 | 2025-08-07 | Acceptable | 2025-10-23 | |
| 18 | SUBSTANTIAL MODIFICATION | SM-9 | 2025-11-12 | Acceptable | 2025-12-01 | |
| 19 | NON SUBSTANTIAL MODIFICATION | NSM-6 | 2026-02-16 | Netherlands | Acceptable | 2026-02-16 |