Phase I / II clinical trial, randomized, exploratory and prospective to evaluate the safety and efficacy of the transfusion of autologous TREG cells obtained from thymic tissue in the prevention of rejection in heart transplanted children

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

30 Jan 2025 → ongoing

Decision date (initial)

2025-01-30

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Sponsor- Rafael Correa · Instituto de Salud Carlos III

External identifiers

EU CT number

2024-519845-30-00

EudraCT number

2018-003574-28

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Prophylaxis, Efficacy

The main objective is to evaluate the feasibility, safety and efficacy of
the infusion of autologous thyTreg cells in the prevention of acute
rejection in children transplanted from the heart.

Secondary objectives 3

1. To evaluate the recovery of the population of circulating Treg and its phenotype throughout the follow-up, the immunological state of the patient, and the reduction of the immunological markers associated with rejection.
2. Determine the incidence of episodes of acute rejection in the 2 years following transplantation and determine the efficacy of treatment in the prevention of these episodes.
3. Assess the tolerability of the product under investigation

Conditions and MedDRA coding

To evaluate the efficacy in the prevention of the rejection of the transfusion of autologous thyTreg cells in children transplanted from the heart

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Patient under two years of age, who meets all the requirements necessary to undergo a heart transplant.
2. Patients without contraindication to immunosuppressive drugs.
3. Parents and / or guardians must be willing and able to understand the purpose and risks of the study and must sign the informed consent document.

Exclusion criteria 9

1. Patients with DiGeorge Syndrome, since their thymic function is affected. 2) HIV positive
2. HIV positive serology.
3. Active infection by EBV
4. Patients hyperimmunized with cytotoxic anti-HLA antibodies
5. Patients with a history of prior malignancy
6. Patients who have received induction therapy with Basiliximab or Thymoglobulin
7. Patients who have been previously timectomized or transplanted.
8. Patients who have been diagnosed with severe autoimmune disease (celiac disease, autoimmune hypothyroidism, autoimmune diabetes)
9. Patiens who have to receive a heart in asystolia

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Repopulation of Treg cells in the patient, determined as the increase of their blood values and the recovery of the ratio Treg / T-effector cells with respect to the values prior to transplantation

Secondary endpoints 4

1. Incidence of episodes of acute myocardial rejection (diagnosed by echocardiography and / or biopsy) that requires treatment in the 2 years post-transplant
2. Restoration of immunological homeostasis in terms of values of immune populations. The percentages and absolute values of T, B, NK and Treg lymphoid populations and of monocytes, dendritic cells and granulocytes during the post-transplant follow-up period (+7, +14, +21, +30, +60, +90, +120, +150, +180, +270, +365, +545, +730 days)
3. Survival of the patient
4. Security (according to criteria NCI-CTCAE V4.03)

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Sponsor organisation

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Address

Calle Del Doctor Esquerdo 46

City

Madrid

Postcode

28007

Country

Spain

Scientific contact point

Organisation

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Contact name

Maria del Carmen de la Cruz Arguedas

Public contact point

Organisation

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Contact name

Maria del Carmen de la Cruz Arguedas

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