Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - Other
Status
Ongoing, recruiting
Participants planned
5
Countries
1
Sites
2
Glioblastoma multiforme (GBM)
The primary objective is to evaluate the long term mutagenic safety of Temferon.
Key facts
- Sponsor
- Genenta Science S.p.A.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04]
- Trial duration
- 27 Oct 2022 → ongoing
- Decision date (initial)
- 2024-08-26
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- Genenta Science S.p.A.
External identifiers
- EU CT number
- 2024-516211-25-00
- EudraCT number
- 2021-000700-38
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
The primary objective is to evaluate the long term mutagenic safety of Temferon.
Secondary objectives 3
- To assess the long term mutagenic potential of Temferon
- To assess the general long-term safety of Temferon as determined by the incidence of drug reactions attributed to Temferon
- To assess disease progression and survival in patients who have received Temferon
Conditions and MedDRA coding
Glioblastoma multiforme (GBM)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10018337 | Glioblastoma multiforme | 100000004864 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Patients who have received Temferon and completed the follow-up in the TEM-GBM_001 study.
- Able and willing to provide written informed consent and comply with the study protocol and procedures.
Exclusion criteria 1
- There are no exclusion criteria for this study
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- 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.
Secondary endpoints 5
- Safety: the incidence of increasingly expanding clone of peripheral white blood cells.
- 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.
- Efficacy: identify myeloid cells in peripheral blood (PB)
- Efficacy: determine the proportions of patients achieving complete response (CR), partial response (PR), stable disease (SD), and progressive disease (PD) using standard iRANO criteria.
- Efficacy: Overall Survival (OS) will be calculated from the first day following Temferon administration.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD6845240 · Product
- Active substance
- Autologous CD34 Haematopoietic Stem and Progenitor Cells Transduced with a Lentiviral Vector Encoding the Interferon ALPHA-2 Gene
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS USE
- Authorisation status
- Not Authorised
- MA holder
- GENENTA SCIENCE S.R.L.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/23/2794
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Genenta Science S.p.A.
- Sponsor organisation
- Genenta Science S.p.A.
- Address
- Via Olgettina 58
- City
- Milan
- Postcode
- 20132
- Country
- Italy
Scientific contact point
- Organisation
- Genenta Science S.p.A.
- Contact name
- Genenta Science
Public contact point
- Organisation
- Genenta Science S.p.A.
- Contact name
- Genenta Science
Third parties 8
| Organisation | City, country | Duties |
|---|---|---|
| Arithmos S.r.l. ORG-100047544
|
Verona, Italy | Code 8, Code 9 |
| Alira Health S.r.l. ORG-100049885
|
Verona, Italy | On site monitoring, Code 10, Code 11, Data management, E-data capture |
| IRCCS Foundation Istituto Neurologico Carlo Besta ORG-100006637
|
Milan, Italy | Other |
| IRCCS Foundation Istituto Neurologico Carlo Besta ORG-100006637
|
Milan, Italy | Other |
| San Raffaele Hospital ORG-100029986
|
Milan, Italy | Other |
| San Raffaele Hospital ORG-100029986
|
Milan, Italy | Other |
| IRCCS Foundation Istituto Neurologico Carlo Besta ORG-100006637
|
Milan, Italy | Laboratory analysis |
| San Raffaele Hospital ORG-100029986
|
Milan, Italy | Laboratory analysis |
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Italy | Ongoing, recruiting | 5 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Italy | 2022-10-27 | 2022-10-28 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 5 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-516211-25-00_Transitioning 2021-000700-38_Redacted | 1.1 |
| Recruitment arrangements (for publication) | K1_ Recruitment arrangements_File Note | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main_Redacted | 2.1 |
| Subject information and informed consent form (for publication) | L2_Other subject information Biological material_Redacted | 2.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis IT 2024-516211-25-00_Transitioning 2021-000700-38_Redacted | 1.1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-08-01 | Italy | Acceptable 2024-08-20
|
2024-08-26 |