A long-term monitoring study of patients suffering from a malignant neoplasm of the central nervous system previously treated with hematopoietic stem cells

A follow-up study evaluating the long term safety of autologous CD34+-enriched hematopoietic progenitor cells genetically modified with a lentiviral vector encoding for the human interferon-a2 gene previously administered to patients with glioblastoma multiforme - TEM-LT

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-000700-38-IT

Enrollment

Registered

2021-06-07

Start date

2021-07-12

Completion date

Unknown

Last updated

2024-12-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Glioblastoma multiforme (GBM) MedDRA version: 20.0 Level: PT Classification code 10018337 Term: Glioblastoma multiforme System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 20.0 Level: PT Classification code 10018337 Term: Glioblastoma multiforme System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

Interventions

Product Name: NA Product Code: [NA] Pharmaceutical Form: Bath additive Current Sponsor code: 99999 Concentration unit: % (V/V) percent volume/volume Concentration type: equal Concentration number: 999

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Inclusion Criteria at Entry: • Patients who have received Temferon and completed 2 years follow-up in the TEM-GBM study. • Able and willing to provide written informed consent and comply with the study protocol and procedures. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 4 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1

Exclusion criteria

Exclusion criteria: There are no exclusion criteria for this study.

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective is to evaluate the long term mutagenic safety of Temferon as determined by the incidence of second malignancies and specifically hematopoietic malignancies.;Secondary Objective: The secondary objectives are: •To assess the long term mutagenic potential of Temferon as determined by the incidence of insertional mutagenic events in peripheral white blood cells; •To assess the general long-term safety of Temferon as determined by the incidence of drug reactions attributed to Temferon; •To assess the presence in the long term of transduced myeloid cells in the peripheral blood; •To assess disease progression and survival in patients who have received Temferon.;Primary end point(s): The primary endpoint is to assess the mutagenic potential of Temferon over 8 years 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.;Timepoint(s) of evaluation of this end point: Over 8 years following Temferon administration (the first two years of the monitoring are included in the TEM-GBM_001 study).	—

Measure

Time frame

Main Objective: The primary objective is to evaluate the long term mutagenic safety of Temferon as determined by the incidence of second malignancies and specifically hematopoietic malignancies.;Secondary Objective: The secondary objectives are: •To assess the long term mutagenic potential of Temferon as determined by the incidence of insertional mutagenic events in peripheral white blood cells; •To assess the general long-term safety of Temferon as determined by the incidence of drug reactions attributed to Temferon; •To assess the presence in the long term of transduced myeloid cells in the peripheral blood; •To assess disease progression and survival in patients who have received Temferon.;Primary end point(s): The primary endpoint is to assess the mutagenic potential of Temferon over 8 years 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.;Timepoint(s) of evaluation of this end point: Over 8 years following Temferon administration (the first two years of the monitoring are included in the TEM-GBM_001 study).

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Secondary

Measure	Time frame
Secondary end point(s): Safety • The incidence of increasingly expanding clone of peripheral white blood cells with evidence of vector genome insertion within or in close proximity to a putative cancer associated gene • Long-term tolerability and safety of Temferon up to 8 years following Temferon administration, evaluated by: o Routine clinical and laboratory surveillance; o Development or exacerbation of non-GBM related neurologic disorders attributed to Temferon exposure; o Development or exacerbation of hematologic disorders attributed to Temferon exposure; o Development or exacerbation of rheumatologic disorders attributed to Temferon exposure; o Development or exacerbation of autoimmune manifestations attributed to Temferon exposure; o Development of infections that are attributed to Temferon exposure; Efficacy • Identify the persistence or progressive exhaustion of transduced myeloid cells in peripheral blood (PB) between Year 2 and Year 8 as determined by VCN>0.01. • Determine the proportions of patients achieving complete response (CR), partial response (PR), stable disease (SD), and progressive disease (PD) annually between Year 2 and Year 8 visits using standard iRANO criteria. • Overall Survival (OS) up to 8 years, will be calculated from the first day following Temferon administration.;Timepoint(s) of evaluation of this end point: Over 8 years following Temferon administration.	—

Measure

Time frame

Secondary end point(s): Safety • The incidence of increasingly expanding clone of peripheral white blood cells with evidence of vector genome insertion within or in close proximity to a putative cancer associated gene • Long-term tolerability and safety of Temferon up to 8 years following Temferon administration, evaluated by: o Routine clinical and laboratory surveillance; o Development or exacerbation of non-GBM related neurologic disorders attributed to Temferon exposure; o Development or exacerbation of hematologic disorders attributed to Temferon exposure; o Development or exacerbation of rheumatologic disorders attributed to Temferon exposure; o Development or exacerbation of autoimmune manifestations attributed to Temferon exposure; o Development of infections that are attributed to Temferon exposure; Efficacy • Identify the persistence or progressive exhaustion of transduced myeloid cells in peripheral blood (PB) between Year 2 and Year 8 as determined by VCN>0.01. • Determine the proportions of patients achieving complete response (CR), partial response (PR), stable disease (SD), and progressive disease (PD) annually between Year 2 and Year 8 visits using standard iRANO criteria. • Overall Survival (OS) up to 8 years, will be calculated from the first day following Temferon administration.;Timepoint(s) of evaluation of this end point: Over 8 years following Temferon administration.

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Countries

Italy

Contacts

Public ContactSportello Informazioni Sperimentazi

Genenta Science

info-trial@genenta.com0226434621

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026