Clinical trial • Phase IV • Haematology
VALOCTOCOGENE ROXAPARVOVEC for Hemophilia A
Phase IV trial of VALOCTOCOGENE ROXAPARVOVEC for Hemophilia A. open-label, none/not specified-controlled. 184 participants.
Overview
- Trial Therapeutic Area
- Haematology
- Trial Disease
- Hemophilia A
- Trial Stage
- Phase IV
- Drug Modality
- Gene therapy
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 16-02-2024
- First CTIS Authorization Date
- 18-06-2024
Trial design
open-label, none/not specified-controlled Phase IV trial across 8 sites in Belgium, France, Germany and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 184
- Trial Duration For Participant
- 4532
Eligibility
Recruits 184 Participants must be capable of giving signed informed consent as described in Appendix 10.1.3 of the protocol; no vulnerable populations selected in the trial metadata..
- Vulnerable Population
- Participants must be capable of giving signed informed consent as described in Appendix 10.1.3 of the protocol; no vulnerable populations selected in the trial metadata.
Inclusion criteria
- {"criterion_text":"- Participants must have completed the End of Study Visit in their primary treatment study (Study 270-201, 270-203, 270-205, 270-301, 270-302, or 270-303) or be currently enrolled in one of these studies and have completed a minimum of 24 months of post-infusion follow-up in that study at the time of closure by the Sponsor. Participants may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.\n- Participants must be capable of giving signed informed consent as described in Appendix 10.1.3 of the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol."}
Exclusion criteria
- {"criterion_text":"- Participants who do not directly enroll in 270-401 at the time of the study completion visit in their primary treatment study should enroll in 270-401 within 4 months of the date of that study completion visit. If a participant wishes to enroll in 270-401 after 4 months, critical data (in particular FVIII activity, bleeding episodes, FVIII use, AEs, SAEs, and the use of hemophilia medications) must be available from the extended duration between studies, and, in the opinion of the Investigator or Medical Monitor, any missing data would not impact or interfere with evaluation and interpretation of the study.\n- Participants must be overtly healthy and not have any condition that, in the opinion of the Investigator or Medical Monitor, would prevent the participant from fully complying with the requirements of the study and/or would impact or interfere with evaluation and interpretation of the study data (including, if applicable, advanced HIV disease).\n- Where applicable, per country regulation, the participant must not currently be committed to an institution by virtue of an order issued either by judicial or administrative authorities."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Occurrence of adverse drug reactions, serious adverse events, and events of special interest, including: a) Hepatotoxicity; b) Thromboembolic events; c) Development of FVIII inhibitors; d)\tTransmission to third parties; e) Integration with theoretical risk of tumorigenesis","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds)","definition_or_measurement_approach":"Measured as change in annualized bleeding rate (ABR) covering treated bleeds and all bleeds"}
- {"endpoint_text":"- FVIII activity measured over time (CSA and OSA)","definition_or_measurement_approach":"FVIII activity measured over time using Chromogenic Substrate Assay (CSA) and One-Stage Assay (OSA)"}
- {"endpoint_text":"- Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate)","definition_or_measurement_approach":"Measured as annualized utilization of FVIII and annualized FVIII infusion rate"}
- {"endpoint_text":"- Changes in Haemo-QoL-A","definition_or_measurement_approach":"Measured using the Haemo-QoL-A patient-reported questionnaire"}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 184
- Recruitment Window Months
- 148
- Consent Approach
- Participants must provide signed informed consent as described in Appendix 10.1.3 of the protocol; country-specific ICF documents are provided (subject information and informed consent forms listed per country). No pediatric/assent process specified in the public record.
Methods
- Recruitment from participants who completed the End of Study Visit in their primary BMN 270 treatment studies (270-201, 270-203, 270-205, 270-301, 270-302, or 270-303) or who are currently enrolled in one of these studies and meet the follow-up criteria.
- Data collection and participant contact via phone calls, mobile nursing (MN)/remote visits (where available and if participant consents), and/or site visits conducted quarterly.
Geography
- Total Number Of Sites
- 8
- Total Number Of Participants
- 16
Belgium
- Earliest CTIS Part Ii Submission Date
- 01-03-2024
- Latest Decision Or Authorization Date
- 15-07-2025
- Processing Time Days
- 502
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- UZ Leuven
- Department Name
- Cardiovascular Diseases
- Contact Person Name
- Quentin Van Thillo
- Contact Person Email
- quentin.vanthillo@uzleuven.be
France
- Earliest CTIS Part Ii Submission Date
- 09-04-2024
- Latest Decision Or Authorization Date
- 28-04-2025
- Processing Time Days
- 384
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Institut Cœur Poumon - Hemostase Clinique
- Contact Person Name
- Antoine Rauch
- Contact Person Email
- antoine.rauch@chu-lille.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Hôpital de La Timone - Hématologie, immunologie et oncologie pédiatrique
- Contact Person Name
- Hervé Chambost
- Contact Person Email
- herve.chambost@hp-hm.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 16-04-2024
- Latest Decision Or Authorization Date
- 25-04-2025
- Processing Time Days
- 374
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Vivantes Netzwerk fuer Gesundheit GmbH
- Department Name
- Vascular Medicines Center
- Contact Person Name
- Robert Klamroth
- Contact Person Email
- klinische.forschung@vivantes.de
- Site Name
- Universitaetsklinikum Bonn AöR
- Department Name
- Institute of Experimental Hematology and Transfusion Medicine
- Contact Person Name
- Johannes Oldenburg
- Contact Person Email
- studienzentrale-szb@ukbonn.de
Italy
- Earliest CTIS Part Ii Submission Date
- 07-05-2024
- Latest Decision Or Authorization Date
- 23-04-2025
- Processing Time Days
- 351
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- Complex Structure of Medicine Hemostasis and Thrombosis
- Contact Person Name
- Flora Peyvandi
- Contact Person Email
- flora.peyvandi@unimi.it
Spain
- Earliest CTIS Part Ii Submission Date
- 27-05-2024
- Latest Decision Or Authorization Date
- 17-10-2025
- Processing Time Days
- 508
- Number Of Sites
- 2
- Number Of Participants
- 5
Sites
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Hematology
- Contact Person Name
- Ramiro José Núñez Vázquez
- Contact Person Email
- ramirojosenv@gmail.com
- Site Name
- Complexo Hospitalario Universitario A Coruna
- Department Name
- Hematology
- Contact Person Name
- Marta Fernandez Docampo
- Contact Person Email
- Marta.Fernandez.Docampo@sergas.es
Sponsor
Primary sponsor
- Full Name
- Biomarin Pharmaceutical Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Psi Cro AG
- Responsibilities
- sponsorDuties codes: 1, 11, 12, 2, 5, 7, 8
- Name
- Fortrea Inc.
- Responsibilities
- sponsorDuties codes: 15 (Mobile Nursing Service)
Third parties
- {"country":"Germany","full_name":"ProtaGene CGT GmbH","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Psi Cro AG","duties_or_roles":"sponsorDuties codes: 1, 11, 12, 2, 5, 7, 8","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services S.a.r.l.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Centogene GmbH","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"sponsorDuties codes: 15 (Mobile Nursing Service)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Precision For Medicine Inc.","duties_or_roles":"sponsorDuties codes: 15 (Long term storage biorepository)","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- ROCTAVIAN 2 × 1013 vector genomes/mL solution for infusion
- Active Substance
- VALOCTOCOGENE ROXAPARVOVEC
- Modality
- Gene therapy
- Routes Of Administration
- INTRAVENOUS USE
- Route
- INTRAVENOUS USE
- Authorisation Status
- Authorised (EU marketing authorisation EU/1/22/1668/001)
- Orphan Designation
- Yes
- Maximum Dose
- 60000000000000 DF dosage form
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