NCT07697118 Aldesleukin(Assistance Publique Hopitaux De Paris) Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome Clinical Landscape Report 2026: Design, Endpoints, Sponsor and Readout Outlook
Turn a newly registered trial into a decision-ready clinical landscape. This report examines NCT07697118 using PatSnap Clinical Trials MCP for protocol design and endpoint evidence, with Drug & Asset MCP and Company & Deal Intelligence MCP used as the companion asset and sponsor enrichment workflow. Explore PatSnap MCP Servers to reproduce the research sequence inside an AI workflow.
MCP evidence snapshot: 3 August 2026. Trial records, recruitment status and projected dates can change after the snapshot and should be rechecked before operational decisions.
Why NCT07697118 is a hot trial to watch
Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome is being segmented by mechanism, treatment setting, geography and endpoint architecture. NCT07697118 is notable because it evaluates Aldesleukin(Assistance Publique Hopitaux De Paris) in a Phase 1/2 design sponsored by Assistance Publique des Hôpitaux de Paris SA. The main development question is whether the protocol can translate its rationale into a clinically interpretable and operationally credible readout.
Trial landscape snapshot
| Field | Indexed detail |
|---|---|
| Registration | NCT07697118 |
| Official title | Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like. (THERIPEX) |
| Phase / status | Phase 1/2 / Not yet recruiting |
| Intervention | Aldesleukin(Assistance Publique Hopitaux De Paris) |
| Sponsor | Assistance Publique des Hôpitaux de Paris SA |
| Geography | France |
| Enrollment | [object Object] |
| Primary endpoint | Frequency of clinical AEs and pathological variations of laboratory parameters |
| Endpoint time frame | Up to 24 months post-infusion |
| Primary completion / readout proxy | [object Object] |
Protocol design and endpoint interpretation
The purpose of this study is to evaluate the safety and efficacy of FOXP3-T4 (an autologous gene therapy) alone or in combination with low-dose IL-2 for the treatment of IPEX syndrome. The therapy involves the transplantation of autologous CD4+ T-cells transduced ex vivo with the LV-EF1a-FOXP3-LNGFR lentiviral vector. The study follows a staggered approach: the first two patients will receive FOXP3-T4 monotherapy. Subsequent patients will receive FOXP3-T4 followed if needed by low-dose IL-2 treatment consisting of a daily dose for 5 days, then weekly administrations for 3 months. The study aims to stabilize autoimmune manifestations and potentially cure the underlying disease, ultimately allowing for the discontinuation of ongoing immunosuppressive treatments.
Allocation is N/A, masking is None (Open Label), and the intervention model is Single Group Assignment. Planned enrollment of [object Object] participants across France shapes statistical precision, execution risk and external validity. Interpretation should account for baseline risk, prior therapy, assessment schedule, missing-data handling and clinical relevance—not only statistical significance.
- Frequency of clinical AEs and pathological variations of laboratory parameters (Up to 24 months post-infusion) — Number of clinical AEs
- Severity of clinical AEs and pathological variations of laboratory parameters (Up to 24 months post-infusion) — Grading will be performed according to the CTCAE (Common Terminology Criteria for Adverse Events) current version. Severity is rated on a scale of Grade 1 (Mild) to Grade 5 (Death).
- Incidence of clinically detectable lymphoproliferation and abnormal clonal dominance (Up to 24 months post-infusion) — This is measured via Vector Insertion Site Analysis (VISA)
- Incidence of clinically detectable lymphoproliferation and abnormal clonal dominance (Beyond 3 months to 24 months post-infusion) — This is measured by proliferation of LNGFR+ cells
Readout outlook and evidence gap
The current protocol points to [object Object] as the best available readout proxy. Because this is an active or newly posted study, a trial-specific result citation is not included until a normalized clinical-trial-result record becomes available. That gap is itself operationally important: teams should monitor first result indexing, conference abstracts, protocol amendments and changes to the primary-completion date.
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Asset and sponsor context
Drug & Asset MCP profile: Aldesleukin(Assistance Publique Hopitaux De Paris) is indexed as No normalized modality returned, with target No normalized target returned, mechanism No normalized mechanism returned, and global highest development status No normalized global status returned.
Company & Deal Intelligence MCP profile: Assistance Publique des Hôpitaux de Paris SA is resolved to a normalized organization record in France. The organization workflow adds identity, location, portfolio and partnering context where available.
The sponsor profile matters because scientific rationale alone does not determine development value. Manufacturing readiness, portfolio fit, geographic reach, partnering capacity and the ability to fund confirmatory development can determine whether a positive signal becomes a competitive asset.
White space around this program
- Sharper patient selection: prospective biomarker or phenotype definitions that identify who is most likely to benefit.
- Clinically interpretable endpoints: outcomes connecting activity with function, symptoms, survival or treatment burden.
- Comparator relevance: evidence against the most decision-relevant contemporary standard of care.
- Broader external validity: evidence across additional geographies, demographic groups and real-world settings.
- Operational differentiation: a development path that closes the readout gap without sacrificing safety or durability.
What to monitor next
Monitor recruitment, enrollment changes, protocol amendments, endpoint hierarchy, primary-completion timing, first result indexing, asset ownership and sponsor partnerships. A change in endpoint, population or ownership can alter the probability of success before a headline data release.
Bottom line
NCT07697118 provides a focused lens on Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome development. Its value will be determined by whether Aldesleukin(Assistance Publique Hopitaux De Paris) can convert the current Phase 1/2 design into evidence that is clinically meaningful, operationally credible and differentiated from competing programs.
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