Clinical trial • Phase II/III • Haematology|Rare Disease
ETRANACOGENE DEZAPARVOVEC for Hemophilia B
Phase II/III trial of ETRANACOGENE DEZAPARVOVEC for Hemophilia B. open-label, none/not specified-controlled. 28 participants.
Overview
- Trial Therapeutic Area
- Haematology|Rare Disease
- Trial Disease
- Hemophilia B
- Trial Stage
- Phase II/III
- Drug Modality
- Gene therapy
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 27-10-2023
Trial design
open-label, none/not specified-controlled Phase II/III trial across 10 sites in Denmark, Sweden, Germany and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 28
- Trial Duration For Participant
- 3650
Eligibility
Recruits 28 No vulnerable population selected; participants are adult male subjects; consent requirement: "Provided written informed consent.".
- Vulnerable Population
- No vulnerable population selected; participants are adult male subjects; consent requirement: "Provided written informed consent."
Inclusion criteria
- {"criterion_text":"- 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.\n- Provided written informed consent.\n- Willing and able to adhere to all protocol requirements."}
Exclusion criteria
- {"criterion_text":"- There are no exclusion criteria in this study"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)","definition_or_measurement_approach":""}
- {"endpoint_text":"- Percentage of Participants with SAEs and AESIs","definition_or_measurement_approach":""}
- {"endpoint_text":"- Number of SAEs and AESIs","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Annualized Bleeding Rate (ABR)","definition_or_measurement_approach":""}
- {"endpoint_text":"- Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes","definition_or_measurement_approach":""}
- {"endpoint_text":"- Endogenous Factor IX (FIX) Activity","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change from Baseline by Visit in Endogenous FIX Activity","definition_or_measurement_approach":""}
- {"endpoint_text":"- Annualized Consumption of FIX Replacement Therapy","definition_or_measurement_approach":""}
- {"endpoint_text":"- Annualized Infusion Rate of FIX Replacement Therapy","definition_or_measurement_approach":""}
- {"endpoint_text":"- Number of Participants Remaining Free of Continuous FIX Prophylaxis","definition_or_measurement_approach":""}
- {"endpoint_text":"- Percentage of Participants Remaining Free of Continuous FIX Prophylaxis","definition_or_measurement_approach":""}
- {"endpoint_text":"- Number of New Target Joints","definition_or_measurement_approach":""}
- {"endpoint_text":"- Percentage Resolution of Pre-existing Target Joints","definition_or_measurement_approach":""}
- {"endpoint_text":"- EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change From Baseline in EQ-5D-5L Index Value","definition_or_measurement_approach":""}
- {"endpoint_text":"- EQ-5D VAS Score","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change From Baseline in EQ-5D VAS Score","definition_or_measurement_approach":""}
- {"endpoint_text":"- Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change From Baseline in Hem-A-QoL Total Score","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 28
- Recruitment Window Months
- 126
- Consent Approach
- Written informed consent provided by adult participants ("Provided written informed consent."). Subject information and informed consent documents (adult) available for participating countries in multiple languages including English, Danish, Dutch, French and German.
Geography
- Total Number Of Sites
- 10
- Total Number Of Participants
- 28
Denmark
- Earliest CTIS Part Ii Submission Date
- 02-02-2024
- Latest Decision Or Authorization Date
- 18-11-2024
- Processing Time Days
- 290
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Rigshospitalet
- Department Name
- Department of Hematology
- Contact Person Name
- Peter Kampmann
- Contact Person Email
- peter.kampmann@regionh.dk
Sweden
- Earliest CTIS Part Ii Submission Date
- 23-05-2024
- Latest Decision Or Authorization Date
- 18-11-2024
- Processing Time Days
- 179
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Region Skane Skanes Universitetssjukhus
- Department Name
- Hematologi, Onkologi och Strålningsfysik
- Contact Person Name
- Jan Astermark
- Contact Person Email
- Jan.Astermark@med.lu.se
Germany
- Earliest CTIS Part Ii Submission Date
- 17-05-2024
- Latest Decision Or Authorization Date
- 09-09-2025
- Processing Time Days
- 480
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Vivantes Netzwerk fuer Gesundheit GmbH
- Department Name
- Klinikum für Angiologie/Hömostaseologie
- Contact Person Name
- Robert Klamroth
- Contact Person Email
- klinische.forschung@vivantes.de
Netherlands
- Earliest CTIS Part Ii Submission Date
- 09-02-2024
- Latest Decision Or Authorization Date
- 18-08-2025
- Processing Time Days
- 556
- Number Of Sites
- 4
- Number Of Participants
- 12
Sites
- Site Name
- Universitair Medisch Centrum Utrecht
- Department Name
- Van Creveldkliniek
- Contact Person Name
- Paul van der Valk
- Contact Person Email
- p.r.vandervalk@umcutrecht.nl
- Site Name
- Academisch Medisch Centrum
- Department Name
- Internal and Vascular Medicine & Haemophilia
- Contact Person Name
- Michiel Coppens
- Contact Person Email
- m.coppens@amsterdamumc.nl
- Site Name
- Universitair Medisch Centrum Groningen
- Department Name
- Haematology
- Contact Person Name
- Karina Meijer
- Contact Person Email
- k.meijer@umcg.nl
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- Haematology
- Contact Person Name
- Frank Leebeek
- Contact Person Email
- f.leebeek@erasmusmc.nl
Ireland
- Earliest CTIS Part Ii Submission Date
- 12-06-2024
- Latest Decision Or Authorization Date
- 23-10-2025
- Processing Time Days
- 498
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- St James's Hospital
- Department Name
- Haematology
- Contact Person Name
- Niamh O'Connell
- Contact Person Email
- research@stjames.ie
Belgium
- Earliest CTIS Part Ii Submission Date
- 05-02-2024
- Latest Decision Or Authorization Date
- 01-12-2025
- Processing Time Days
- 665
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Cliniques Universitaires Saint-Luc
- Department Name
- Hematology
- Contact Person Name
- Cedric Hermans
- Contact Person Email
- cedric.hermans@uclouvain.be
- Site Name
- UZ Leuven
- Department Name
- Vascular Medicine
- Contact Person Name
- Quentin Van Thillo
- Contact Person Email
- quentin.vanthillo@uzleuven.be
Sponsor
Primary sponsor
- Full Name
- CSL Behring LLC
- Organisation Type
- Laboratory/Research/Testing facility
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Block Clinical Inc.
- Responsibilities
- Patient travel and reimbursement
- Name
- Everest Clinical Research Corporation
- Responsibilities
- codes: 6,7 (as listed)
- Name
- Medpace Inc.
- Responsibilities
- Institutional Review Board (IRB)/Ethics Committee (EC) Management; Site budget and contract negotiation; Investigator Site Payments; other operational duties (codes listed)
- Name
- Inseption Group LLC
- Responsibilities
- eTMF management
- Name
- Precision For Medicine Inc.
- Responsibilities
- supporting laboratory/diagnostic duties (code: 4)
Third parties
- {"country":"United States","full_name":"Block Clinical Inc.","duties_or_roles":"Patient travel and reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Denmark","full_name":"Unilabs A/S","duties_or_roles":"code: 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Canada","full_name":"Everest Clinical Research Corporation","duties_or_roles":"code: 6; code: 7","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Medpace Inc.","duties_or_roles":"codes: 1,12,13,15 (Institutional Review Board (IRB)/Ethics Committee (EC) Management; Site budget and contract negotiation; Investigator Site Payments); code:5; code:8","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Precision For Medicine Inc.","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Inseption Group LLC","duties_or_roles":"eTMF management","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"code: 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Belgium","full_name":"MEDPACE LABORATORIES","duties_or_roles":"code: 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Germany","full_name":"ProtaGene CGT GmbH","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medpace Reference Laboratories LLC","duties_or_roles":"code: 4","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- Hemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion
- Active Substance
- ETRANACOGENE DEZAPARVOVEC
- Modality
- Gene therapy
- Routes Of Administration
- IV INFUSION
- Route
- IV INFUSION
- Authorisation Status
- Marketing authorisation referenced (EU/1/22/1715/001), product authorization status code: 2
- Orphan Designation
- Yes
- Maximum Dose
- 2 millilitre(s)/kilogram
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