Infusion of activated NK cells for treating sarcomas in young patients

Multicenter, Open-label, Phase I/II Clinical Trial of Infusion of Activated NK Cells for the Treatment of Children, Adolescents and Young Adults With Sarcomas

This study is testing whether infusing special immune cells from matched donors can help young patients with hard-to-treat sarcomas feel better when other treatments haven't worked.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: N/A to 30 Years
• Sex: All
• Sponsor: Instituto de Investigación Hospital Universitario La Paz (Academic / other)
• Drugs / interventions: chemotherapy, immunotherapy, cyclophosphamide, fludarabine
• Locations: 1 site (Madrid)
• Trial ID: NCT05952310 on ClinicalTrials.gov

What this trial studies

This exploratory therapeutic study aims to evaluate the safety and efficacy of infusing activated Natural Killer (NK) cells from haploidentical donors in pediatric, adolescent, and young adult patients with refractory malignant sarcoma. The study is open-label and non-randomized, focusing on patients who have not responded to conventional treatments. Participants will undergo lymphoablative chemotherapy followed by localized irradiation before receiving NK cell infusions. The trial will take place at multiple hospitals in Spain, targeting a small cohort of 10 patients.

Who should consider this trial

Good fit: Ideal candidates are patients aged 0 to 30 years with malignant sarcoma who have residual disease after conventional treatment.

Not a fit: Patients with sarcoma who have not undergone prior conventional treatment or those with severe organ dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve outcomes for young patients with refractory sarcoma, potentially increasing survival rates.

How similar studies have performed: While NK cell therapy is a developing field, this specific approach in treating refractory sarcoma is novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

1. Patients aged between 0 and 30 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 voluntarily signed informed consent prior to the first study intervention.
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 the time of 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 partner with vasectomy) during their participation in the study and within 30 days of the last visit.
9. Presence of a compatible haploidentical donor (father or mother or sibling).

Exclusion Criteria:

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. Contraindications, interactions, precautions for use and dose reductions indicated in the corresponding data sheets must be considered.
5. Subjects who have been administered other investigational drugs in the 90 days prior to inclusion.

Where this trial is running

Madrid

• Hospital Universitario La Paz — Madrid, Spain (Recruiting)

Study contacts

• Study coordinator: Antonio Pérez Martínez, PI

Conditions Malignant Sarcoma