ETUVETIDIGENE AUTOTEMCEL for Wiskott-Aldrich syndrome

Inclusion criteria

• {"criterion_text":"- The patient has been treated with Telethon003\n- The patient or, if applicable, the patient’s parent(s)/legal guardian(s), are able and willing to provide informed consent.\n- Willingness to attend the study visit schedule and be compliant with the protocol."}

Exclusion criteria

• {"criterion_text":"- Participation in an ongoing clinical study with an investigational drug (other to gene therapy study or early access, object for this study) prior to enrolment in this study or willingness to participate in another interventional clinical trial. If in deviation to this exclusion criteria, a subject took or takes another investigational drug or other available therapy (e.g. hematopoietic stem cell transplant) he will be followed anyway to collect safety events."}

Primary endpoints

• {"endpoint_text":"- To characterize the long-term safety of the gene therapy treatment with Telethon003 as measured by: recording of adverse event (AE) and serious adverse event (SAE) related to gene therapy including: − insertional mutagenesis and oncogenesis (blood and solid malignancies); − transgene immunogenicity; − development of replication-competent lentiviruses (RCL);","definition_or_measurement_approach":"Recording of adverse events (AE) and serious adverse events (SAE) related to gene therapy including insertional mutagenesis and oncogenesis (blood and solid malignancies), transgene immunogenicity, and development of replication-competent lentiviruses (RCL)."}
• {"endpoint_text":"- To evaluate the overall survival (OS) as primary efficacy endpoint up to 15 years of follow-up post treatment with Telethon003","definition_or_measurement_approach":"Overall survival (OS) measured up to 15 years of follow-up post treatment with Telethon003."}

Secondary endpoints

• {"endpoint_text":"- Event free survival","definition_or_measurement_approach":""}
• {"endpoint_text":"- Annualized rate of severe infections compared with 1 year prior to gene therapy;","definition_or_measurement_approach":"Annualized rate compared with the rate in the 1 year prior to gene therapy."}
• {"endpoint_text":"- Annualized rate of moderate and severe bleeding episodes compared with 1 year prior to gene therapy","definition_or_measurement_approach":"Annualized rate compared with the rate in the 1 year prior to gene therapy."}
• {"endpoint_text":"- Eczema","definition_or_measurement_approach":""}
• {"endpoint_text":"- Autoimmunity","definition_or_measurement_approach":""}
• {"endpoint_text":"- Hematologic disorders (i.e. neutropenia and/or other cytopenias);","definition_or_measurement_approach":""}
• {"endpoint_text":"- Malignancies","definition_or_measurement_approach":""}
• {"endpoint_text":"- Use of IgRT","definition_or_measurement_approach":""}
• {"endpoint_text":"- New or exacerbations of pre-existing neurological disorders","definition_or_measurement_approach":""}
• {"endpoint_text":"- Variation in height and weight (for paediatric patients)","definition_or_measurement_approach":"Change in height and weight for paediatric patients over time."}
• {"endpoint_text":"- Karnofsky/Lansky performance scales","definition_or_measurement_approach":""}
• {"endpoint_text":"- Platelet count and MPV","definition_or_measurement_approach":""}
• {"endpoint_text":"- Vector Copy Number (VCN)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Insertion site analysis (ISA)","definition_or_measurement_approach":""}
• {"endpoint_text":"- PedsQL (Pediatric Quality of Life Inventory) health-related quality of life scales","definition_or_measurement_approach":""}
• {"endpoint_text":"- EQ-5D, and SF-10 or SF-36 health-related quality of life questionnaires","definition_or_measurement_approach":""}

Italy

Earliest CTIS Part Ii Submission Date

11-03-2025

Latest Decision Or Authorization Date

19-06-2025

Processing Time Days

100

Number Of Sites

1

Number Of Participants

38

Primary sponsor

Full Name

Fondazione Telethon Ets

Organisation Type

Patient organisation/association

Country Of Registered Address

Italy

Contract research organisations

Name

IQVIA Limited

Responsibilities

sponsorDuties codes: 1,10,5,6,7; contact eu_clinical_trials_information@iqvia.com

Name

4S4p Consulenze S.r.l.

Responsibilities

sponsorDuties include 15 (Pharmacovigilance), 8; contact marco.anelli@4s4p.eu

Third parties

• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"sponsorDuties codes: 1,10,5,6,7","organisation_type":"Pharmaceutical company"}
• {"country":"Italy","full_name":"4S4p Consulenze S.r.l.","duties_or_roles":"15 (Pharmacovigilance), 8","organisation_type":"Non-Pharmaceutical company"}