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Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

NCT05432310 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2026-04-07

No results posted yet for this study

Summary

The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.

Conditions

  • Adenosine Deaminase Severe Combined Immune Deficiency

Interventions

COMBINATION_PRODUCT

A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme

Autologous transplantation of EFS-ADA lentiviral vector transduced, mPB CD34+ cells by central venous infusion, following reduced intensity conditioning with busulfan

Sponsors & Collaborators

Principal Investigators

  • Satiro De Oliveira, MD · Assistant Professor

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Month
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-01-04
Primary Completion
2027-11-14
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05432310 on ClinicalTrials.gov