Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - First administration to humans
Status
Ended
Participants planned
9
Countries
2
Sites
6
Haemophilia B
To assess the long-term safety (6-10 years after dosing, inclusive) of a systemic administration of CSL220, an AAV vector containing a codon-optimized human coagulation hFIX gene, to adult subjects with severe or moderately severe haemophilia B.
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
- 8 Jan 2021 → 27 Mar 2026
- Decision date (initial)
- 2024-06-17
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- CSL Behring LLC
External identifiers
- EU CT number
- 2024-512603-39-00
- EudraCT number
- 2020-000739-28
- ClinicalTrials.gov
- NCT05360706
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety
To assess the long-term safety (6-10 years after dosing, inclusive) of a systemic administration of CSL220, an AAV vector containing a codon-optimized human coagulation hFIX gene, to adult subjects with severe or moderately severe haemophilia B.
Secondary objectives 1
- To assess the long-term efficacy of a systemic administration of CSL220, an AAV vector containing a codon-optimised human coagulation Factor IX (hFIX) gene, to adult subjects with severe or moderately severe haemophilia B.
Conditions and MedDRA coding
Haemophilia 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, extension study enrolling subjects who have successfully completed all assessments in
Study CT-AMT-060-01 (Years 1-5). Assessment phase will begin at Visit 36 (the first clinical visit in this extension study, approximately 5.5 years after the initial dosing visit Study CT-AMT-060-01) and go to Visit 45 (10-years post-dosing in Study CT-AMT-060-01).
|
Not Applicable | None | CSL220: Long-term safety and efficacy follow up for subjects who have successfully completed all assessments in Study CT-AMT-060-01 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Subjects with congenital haemophilia B who completed Study CSL220_1001
- Able to provide informed consent following receipt of verbal and written information about the trial
Exclusion criteria 1
- Enrolled subjects will have already been assessed based on the exclusion criteria for Study CSL220_1001
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 6
- The primary endpoint is to demonstrate the long-term safety (6-10 years) after dosing of CSL220.
- Primary safety endpoints include the following: - AEs possibly or probably related to previous CSL220 administration
- Neutralising FIX antibodies (FIX inhibitors)
- ALT/aspartate aminotransferase (AST) levels
- Liver pathology (assessed by ultrasound)
- Alpha-fetoprotein (AFP)
Secondary endpoints 7
- The secondary endpoints will focus on the long-term efficacy (6-10 years) after dosing of CSL220 on FIX activity, overall FIX utilisation, bleeding events, any procedures, and QoL.
- Secondary efficacy endpoints include the following: - Endogenous FIX activity
- Utilisation of FIX-replacement therapy
- Annualized bleeding rate; including the following: o All bleeds (treated and untreated) o Spontaneous bleeds o Traumatic bleeds o Joint bleeds
- Procedures (including major and minor surgeries)
- 36-Item Short Form Survey (SF-36) and EQ-5D-5L Quality of Life (QoL) scores
- Hemophilia Joint Health Score (HJHS)
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
- 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 6
| Organisation | City, country | Duties |
|---|---|---|
| ProtaGene CGT GmbH ORG-100041450
|
Heidelberg, Germany | 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 |
| MEDPACE LABORATORIES ORG-100042942
|
Leuven, Belgium | Laboratory analysis |
| Inseption Group LLC ORG-100041732
|
Lansdale, United States | Other |
| Unilabs A/S ORG-100032351
|
Copenhagen Oe, Denmark | Laboratory analysis |
Locations
2 EU/EEA countries · 6 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Ended | 3 | 2 |
| Netherlands | Ended | 6 | 4 |
| Rest of world | — | 0 | — |
Investigational sites
Universitaetsklinikum Frankfurt AöR
ZIM-Med II / Institut für Transfusionsmedizin, Theodor-Stern-Kai 7, 60590, Frankfurt Am Main
Vivantes Netzwerk fuer Gesundheit GmbH
Klinik für Innere Medizin – Angiologie und Hämostaseologie, 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, Wytemaweg 80, 3015 CN, 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 |
|---|---|---|---|---|---|
| Germany | 2021-01-26 | 2026-02-24 | 2021-07-06 | 2021-10-26 | |
| Netherlands | 2021-01-08 | 2026-03-26 | 2021-03-19 | 2021-11-11 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 21 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2024-512603-39_CSL Behring_redacted | 5.0 |
| Protocol (for publication) | D4_Patient facing document_Bleed diary_DE | 3.0 |
| Protocol (for publication) | D4_Patient facing document_bleed diary_en | 3.0 |
| Protocol (for publication) | D4_Patient facing document_FIX diary_DE | 2.0 |
| Protocol (for publication) | D4_Patient facing document_FIX diary_en | 2.0 |
| Protocol (for publication) | D4_Patient facing document_questionnaire EQ-5D_DE_deu_Blank | 1 |
| Protocol (for publication) | D4_Patient facing document_questionnaire EQ-5D_NL_dut_Blank | 1 |
| Protocol (for publication) | D4_Patient facing document_questionnaire SF-36_DE_deu_Redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_questionnaire SF-36_NL_dut | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Germany_CSL_blank | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_NL_CSL | N/A |
| Subject information and informed consent form (for publication) | L1_ICF_LTSFU ICF_Germany_DE_CSL | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF_Main ICF_Germany_DE_CSL | 3.0 |
| Subject information and informed consent form (for publication) | L1_ICF_Pregnant partner ICF_Germany_DE_CSL | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_LTFU_CSL_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_CSL_redacted | 3.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Partner_CSL_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Patient_CSL_redacted | 2.0 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_Hemgenix_CSL Behring | N/A |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_DE-GER_2024-512603-39-00_for publication | 5.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_NL-DUT_2024-512603-39-00_for publication | 5.0 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-05-16 | Netherlands | Acceptable 2024-06-12
|
2024-06-12 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-02-28 | Netherlands | Acceptable 2025-05-12
|
2025-05-15 |