Clinical trial of infusion of activated NK cells for the treatment of children, adolescents and young adults with sarcomas.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Hospital Universitario La Paz, Hospital Universitario La Paz

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

18 Oct 2022 → ongoing

Decision date (initial)

2024-04-18

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-513431-25-00

EudraCT number

2016-003578-42

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

To evaluate the safety of immunotherapy with haploidentical allogeneic NK cells together with IL-2 after lymphoablative chemotherapy and/or low-dose radiotherapy in pediatric, adolescent and young adult patients with refractory sarcoma.

Secondary objectives 3

1. To analyze the incidence of episodes of febrile neutropenia, bacteremia, infections (viral, fungal), hematologic recovery and hospital admission associated with treatment with NK + IL-2 cells.
2. To evaluate the 5-year rate of disease progression after treatment with NK + IL-2 cells.
3. Determine the expression of NK cell inhibitory/activator ligands in both solid tumor and serum samples from pediatric sarcoma patients. To correlate these results with their disease progression to identify new prognostic biomarkers.

Conditions and MedDRA coding

Sarcomas

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Patients aged 0-40 years diagnosed with malignant sarcoma, who at the end of conventional treatment still have detectable residual disease (based on imaging techniques) or in case of stable disease or minimal disease when there is an absence of clinical benefit from chemotherapy (poor tolerance due to adverse effects related to chemotherapy).
2. Lansky/Karnofsky index > 60%.
3. Mild-moderate (<4) organ functional impairment (hepatic, renal, respiratory), according to National Cancer Institute criteria (NCI CTCAE v5.0).
4. Left ventricular ejection fraction >39%.
5. Adult subjects who had voluntarily signed the informed consent prior to the first intervention of the study.
6. Minor subjects whose representative/legal guardian voluntarily signed the informed consent prior to the first intervention of the study.
7. In the case of mature minors (12 - 17 years of age), in addition to the consent signed by the legal guardian, the minor's assent will be obtained.
8. Women of childbearing capacity must have a negative pregnancy test at inclusion and must agree to use highly effective contraceptive methods (diaphragms plus spermicide or male condom plus spermicide, oral contraceptive combined with a second method of contraceptive implant, injectable contraceptive, permanent intrauterine device, sexual abstinence or vasectomized partner) during their participation in the study and within 30 days of the last visit.
9. Presence of a compatible haploidentical donor (parent or sibling).

Exclusion criteria 5

1. Patients with a history of poor therapeutic compliance.
2. Patients who after a psycho-social evaluation are censored as unsuitable for the procedure.  Socio-familial situation that makes proper participation in the study impossible.  Patients with emotional or psychological problems secondary to the disease, such as post-traumatic stress disorder, phobias, delirium, psychosis, requiring specialist support.  Evaluation of the involvement of family members in the patient's health.  Impossibility to understand information about the trial.
3. Severe functional organ impairment (hepatic, renal, respiratory) (4), according to the criteria of the National Cancer Institute (NCI CTCAE 5.0).
4. The contraindications, interactions, precautions for use and dose reductions indicated in the corresponding data sheets should be considered.
5. Subjects who have been administered other investigational drugs in the 90 days prior to inclusion.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Toxicity will be classified according to the common toxicity criteria established by the National Cancer Institute (CTC NCI, version 5.0).

Secondary endpoints 3

1. Incidence of episodes of febrile neutropenia, bacteraemia, infections (viral, fungal), haematological recovery and hospital admission associated with treatment.
2. Five-year disease progression rate after treatment with NK cells + IL-2. This will be determined by imaging techniques.
3. Expression levels of NK cell inhibitory/activating ligands in solid tumour samples and patient serum. These shall be determined by immunohistochemistry and PCR techniques.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Hospital Universitario La Paz

Sponsor organisation

Hospital Universitario La Paz

Address

Paseo De La Castellana 261

City

Madrid

Postcode

28046

Country

Spain

Scientific contact point

Organisation

Hospital Universitario La Paz

Contact name

Antonio Pérez Martínez

Public contact point

Organisation

Hospital Universitario La Paz

Contact name

Antonio Pérez Martínez

Hospital Universitario La Paz

Sponsor organisation

Hospital Universitario La Paz

Address

Paseo De La Castellana 261

City

Madrid

Postcode

28046

Country

Spain

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Application history

1 submissions — initial application plus substantial / non-substantial modifications