Autologous CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector encoding the interferon alpha-2 gene for Glioblastoma multiforme

Inclusion criteria

• {"criterion_text":"- Patients who have received Temferon and completed the follow-up in the TEM-GBM_001 study."}
• {"criterion_text":"- Able and willing to provide written informed consent and comply with the study protocol and procedures."}

Exclusion criteria

• {"criterion_text":"- There are no exclusion criteria for this study"}

Primary endpoints

• {"endpoint_text":"- The primary endpoint is to assess the mutagenic potential of Temferon following administration (the first two years of the monitoring are included in the TEM-GBM_001 study), as evaluated by the incidence of hematopoietic malignancies or potentially life threatening, malignant solid or other hematological tumors.","definition_or_measurement_approach":"Assessment by incidence of hematopoietic malignancies or potentially life-threatening malignant solid or other hematological tumours following Temferon administration (first two years included in TEM-GBM_001)."}

Secondary endpoints

• {"endpoint_text":"- Safety: the incidence of increasingly expanding clone of peripheral white blood cells.","definition_or_measurement_approach":""}
• {"endpoint_text":"- Safety: Long-term tolerability and safety of Temferon following Temferon administration, evaluated by: - routine clinical and laboratory surveillance; - development or exacerbation of non-GBM related neurologic disorders attributed to Temferon exposure; - development or exacerbation of hematologic disorders, rheumatologic disorders, autoimmune manifestations attributed to Temferon exposure; - development of infections that are attributed to Temferon exposure.","definition_or_measurement_approach":"Evaluated by routine clinical and laboratory surveillance and monitoring for development/exacerbation of specified disorders and infections attributed to Temferon."}
• {"endpoint_text":"- Efficacy: identify myeloid cells in peripheral blood (PB)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Efficacy: determine the proportions of patients achieving complete response (CR), partial response (PR), stable disease (SD), and progressive disease (PD) using standard iRANO criteria.","definition_or_measurement_approach":"Responses determined using standard iRANO criteria."}
• {"endpoint_text":"- Efficacy: Overall Survival (OS) will be calculated from the first day following Temferon administration.","definition_or_measurement_approach":"Overall Survival calculated from first day after Temferon administration."}

Italy

Earliest CTIS Part Ii Submission Date

15-07-2024

Latest Decision Or Authorization Date

26-08-2024

Processing Time Days

42

Number Of Sites

2

Number Of Participants

5

Primary sponsor

Full Name

Genenta Science S.p.A.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Italy

Third parties

• {"country":"Italy","full_name":"Arithmos S.r.l.","duties_or_roles":"codes: 8, 9","organisation_type":"Pharmaceutical company"}
• {"country":"Italy","full_name":"Alira Health S.r.l.","duties_or_roles":"codes: 1, 10, 11, 6, 7","organisation_type":"Pharmaceutical company"}
• {"country":"Italy","full_name":"IRCCS Foundation Istituto Neurologico Carlo Besta","duties_or_roles":"Histopathology","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Italy","full_name":"IRCCS Foundation Istituto Neurologico Carlo Besta","duties_or_roles":"Diagnostic Imaging/Controls - X-Rays","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Italy","full_name":"San Raffaele Hospital","duties_or_roles":"Diagnostic Imaging/Controls - X-Rays","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Italy","full_name":"San Raffaele Hospital","duties_or_roles":"Vector Copy Number (VCN)","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Italy","full_name":"IRCCS Foundation Istituto Neurologico Carlo Besta","duties_or_roles":"code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Italy","full_name":"San Raffaele Hospital","duties_or_roles":"code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}