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Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

NCT04418414 · Status: NOT_YET_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2024-02-20

No results posted yet for this study

Summary

This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A. The study will evaluate gene therapy by transplantation of autologous CD34+ hematopoietic stem cells transduced ex vivo with the CD68-ET3 lentiviral vector.

Conditions

Interventions

DRUG

Gene therapy

CD34+ hematopoietic stem cells transduced with CD68-ET3 lentiviral vector (encoding human factor VIII gene) is administered by IV infusion following conditioning regimen with busulfan and anti-thymocyte globulin.

OTHER

Biological

G-CSF and Plerixafor are administered by subcutaneous injection prior to apheresis.

Sponsors & Collaborators

  • Expression Therapeutics, LLC

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-09-01
Primary Completion
2029-08-31
Completion
2039-08-31
FDA Drug
Yes

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Entities

Diseases

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 NCT04418414 on ClinicalTrials.gov