An Open Label, Single Arm, Phase I/II Clinical Study of Autologous CD4+ T-Cells Edited Ex-Vivo at the CD40LG Locus by CRISPR/Cas9 and IDLV-based vector in Patients with X-linked Hyper IgM Syndrome Type 1 (HIGM1)

Status

Not yet recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2025-524635-39-00

Enrollment

Registered

2026-05-20

Start date

Unknown

Completion date

Unknown

Last updated

2026-05-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

X-linked immunodeficiency with hyper-IgM type 1 (HIGM1)

Brief summary

Proportion of patients experiencing dose-limiting toxicities (DLTs) after each drug product (DP) dose

Detailed description

Occurrence of: - Adverse events (AEs) - Serious adverse events (SAEs)Adverse events of special interest (AESIs) - DP-related AEs - Immune reconstitution inflammatory Syndrome (IRIS) (number, grading and proportion of patients experiencing IRIS) evaluated at 28 days after each dose, 6 months, 1 year and 2 years after treatment, Proportion of patients receiving the boost DP dose, Overall survival at 6 months, 1 year and 2 years after treatment, Incidence rate, duration and proportion of days with moderate-severe infection in the 2 years preceding and in the 2 years after treatment, evaluated in consecutive 6- month time windows (6 months, 1 year, 1.5 years and 2 years)

Interventions

DRUGRabipur Polvere e solvente per soluzione iniettabile in siringa preriempita. Vaccino rabico (inattivato).

DRUGTICOVAC 0

DRUG25 ml per uso pediatrico Sospensione iniettabile in una siringa preriempita Vaccino (virus intero

DRUGinattivato) contro l'encefalite da zecca

DRUGAUTOLOGOUS PERIPHERAL BLOOD-DERIVED CD4+ T-CELLS CRISPR-EDITED AT THE CD40LG LOCUS

DRUGInfanrix hexa

DRUGPowder and suspension for suspension for injection. Diphtheria (D)

DRUGtetanus (T)

DRUGpertussis (acellular

DRUGcomponent) (Pa)

DRUGhepatitis B (rDNA) (HBV)

DRUGpoliomyelitis (inactivated) (IPV) and Haemophilus influenzae type b (Hib) conjugate vaccine (adsorbed).

DRUG5 ml Sospensione iniettabile in una siringa preriempita Vaccino (virus intero

DRUGinattivato) contro l’encefalite da zecca

Eligibility

Sex/Gender

Male

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Proportion of patients experiencing dose-limiting toxicities (DLTs) after each drug product (DP) dose	—

Secondary

Measure	Time frame
Occurrence of: - Adverse events (AEs) - Serious adverse events (SAEs)Adverse events of special interest (AESIs) - DP-related AEs - Immune reconstitution inflammatory Syndrome (IRIS) (number, grading and proportion of patients experiencing IRIS) evaluated at 28 days after each dose, 6 months, 1 year and 2 years after treatment, Proportion of patients receiving the boost DP dose, Overall survival at 6 months, 1 year and 2 years after treatment, Incidence rate, duration and proportion of days with moderate-severe infection in the 2 years preceding and in the 2 years after treatment, evaluated in consecutive 6- month time windows (6 months, 1 year, 1.5 years and 2 years)	—

Measure

Time frame

—

Outcome results

None listed

Source: EU CTIS · Data processed: May 21, 2026