Theripex

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Decision date (initial)

2026-06-24

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

The primary objective of this clinical trial is to assess the initial safety and efficacy of treatment with FOXP3-T4 alone or combined with low-dose IL-2 treatment, including the procedure and infusion with LV-FOXP3-LNGFR lentiviral vector gene-modified induced CD4+ Treg-cells in IPEX patients.

Secondary objectives 1

1. The secondary objective is to assess the efficacy of treatment with FOXP3-T4 alone or combined with low-dose IL-2 treatment.

Conditions and MedDRA coding

IPEX syndrome is a primary immunodeficiency caused by hemizygous mutations in the gene FOXP3, which encodes an essential transcription factor required to maintain immunological tolerance by thymus-derived regulatory T (Treg) cells.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. 1) Male patients only 2) Patients aged from 1-45 years of age. The first three patients will be aged between 10 – 45 years of age.
2. 3) Patient with IPEX syndrome caused by mutation of the FOXP3 gene
3. 4) Patients are eligible from the second line of treatment onward, even those under controlled disease
4. 5) Patient with recurrent IPEX symptoms, under immune suppressive medications
5. 6)Patient for whom HSCT is not feasible or when no suitable compatible donor is available 7)Patients who have had prior allogeneic blood stem cell transplantation (HSCT) with engraftment failure defined as no intake of donor cells
6. 8) Parental, guardian’s patient signed informed consent
7. 9) For patients of childbearing age: willing to use an effective method of contraception during the trial and for at least 12 months post-infusion
8. 10) Affiliation to a French or European social security scheme

Exclusion criteria 9

1. 1) Unwillingness to return for follow-up during the 2-year study and during the 15 years of long-term follow-up study
2. 3) Patient on AME (state medical aid) (unless exemption from affiliation)
3. 4) Diagnosis of a significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study
4. 4) Eligible for an HLA matched sibling or matched unrelated donor blood stem cell transplant and be willing to undergo transplant
5. 7) HIV-1 or 2 or HTLV1 infections
6. 8) Patients with severe IPEX clinical presentation needing a rapid allogeneic HSCT treatment within 3 months
7. 9) hypersensitivity to IL-2 or any component of the formulation
8. 6) Patients with uncontrolled or ongoing active infections
9. 2) Patient with short life expectancy

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint of the study is safety and efficacy up to 24 months following IV infusion of FOXP3-T4 alone or combined with low doses of IL-2 treatment.

Secondary endpoints 1

1. The efficacy endpoint is assessed until 24 months following the infusion of the FOXP3-T4 treatment alone or combined with low-dose of IL-2 treatment

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Emmanuelle Six

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Marina Cavazzana,

Locations

1 EU/EEA country · 7 investigational sites

By country

Investigational sites

Application history

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