Clinical trial • Phase IV • Oncology | Neurology

Allogeneic peripheral blood-derived haematopoietic stem cells, TCR alpha/beta+ CD19+ depleted for Neuroblastoma | Relapsed or refractory high-risk neuroblastoma

Phase IV trial of Allogeneic peripheral blood-derived haematopoietic stem cells, TCR alpha/beta+ CD19+ depleted for Neuroblastoma | Relapsed or refractory…

Overview

Trial Therapeutic Area
Oncology | Neurology
Trial Disease
Neuroblastoma | Relapsed or refractory high-risk neuroblastoma
Trial Stage
Phase IV
Drug Modality
Cell therapy | Monoclonal antibody | Other antibody | Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
20-12-2024
First CTIS Authorization Date
31-01-2025

Trial design

Phase IV trial across 1 site in Italy.

Target Sample Size
19

Eligibility

Recruits 19 paediatric patients.

Vulnerable Population
Children/paediatric patients are included (isVulnerablePopulationSelected = true). Informed parental or legal guardian consent is required. Subject information and informed consent forms are provided for parents/guardians, prepubertal child, postpubertal child and adults (documents listed: L1_SIS and ICF parents-guardian; L1_SIS and ICF prepuberal child; L1_SIS and ICF postpuberal child; L1_SIS and ICF adults). Assent from children is handled via age-appropriate child ICFs (prepuberal and postpuberal) and parental/legal guardian provides consent.

Inclusion criteria

  • {"criterion_text":"- High-risk neuroblastoma (stage 4 with age > 12 months at diagnosis, or stage 2, 3, 4s with MYCN amplification) in local, metastatic or combined recurrence after first-line therapy protocol\n- Stabilization of disease (at or above STABLE DISEASE according to INRC criteria) achieved after salvage chemotherapy according to the center’s clinical practice\n- Availability of a suitable HLA haploidentical familial donor to perform HSC donation from peripheral blood after stimulation with GCSF (+/-Plerixafor)\n- Informed parental or legal guardian consent"}

Exclusion criteria

  • {"criterion_text":"- Disease progressing after salvage therapy.\n- Severe organ dysfunction, defined as grade 4 or higher dysfunctions according to international reference tables (CTCAE v 5.0).\n- Life expectancy of less than 3 months."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Overall Survival (OS)","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"- Engraftment and donor chimerism analysis","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Immune reconstitution (+30, +60, +90, +120, +180, +360)","definition_or_measurement_approach":"Measured at +30, +60, +90, +120, +180 and +360 days"}
  • {"endpoint_text":"- Infectious events","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Transplant-related mortality (TRM)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Progression-free Survival (PFS)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Early and late transplant-related toxicities","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Immunotherapy (antui-GD2 mAb)–related toxicities","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Donor-derived NK-cells infusion–related toxicities","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Disease response (INRC criteria)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Acute and chronic GvHD","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Antitumor activity of donor-derived NK-cells (graft-versus-tumor effect)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Relevance of the donor NK cell selection criteria","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
19
Recruitment Window Months
59
Consent Approach
Informed consent is provided by parents or legal guardians for paediatric participants. Age-appropriate ICFs and assent forms exist (documents for parents/guardian, prepubertal child, postpubertal child, adults). Specific languages of the consent documents are not specified in the available records.

Geography

Total Number Of Sites
1
Total Number Of Participants
19

Italy

Earliest CTIS Part Ii Submission Date
20-12-2024
Latest Decision Or Authorization Date
31-01-2025
Processing Time Days
42
Number Of Sites
1
Number Of Participants
19

Sites

Site Name
IRCCS Istituto Giannina Gaslini
Department Name
U.O.S.D. Centro trapianto di midollo osseo
Principal Investigator Name
Stefano Giardino
Principal Investigator Email
stefanogiardino@gaslini.org
Contact Person Name
Stefano Giardino
Contact Person Email
stefanogiardino@gaslini.org
Number Of Participants
19

Sponsor

Primary sponsor

Full Name
IRCCS Istituto Giannina Gaslini
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Italy

Investigational products

Investigational Product Name
Aplo-TCSE TCRaß+CD19+ depleto
Active Substance
Allogeneic peripheral blood-derived haematopoietic stem cells, TCR alpha/beta+ CD19+ depleted
Modality
Cell therapy
Routes Of Administration
Intravenous
Route
Intravenous
Authorisation Status
Not authorised (prodAuthStatus: 1)
Maximum Dose
10000 IU/Kg
Investigational Product Name
Allogeneic peripheral blood-derived NK cells CD3- CD56+
Active Substance
Allogeneic peripheral blood-derived NK cells CD3- CD56+
Modality
Cell therapy
Routes Of Administration
Intravenous
Route
Intravenous
Authorisation Status
Not authorised (prodAuthStatus: 1)
Maximum Dose
8000 IU/Kg
Combination Treatment
Yes

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