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Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease

NCT02800070 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2024-04-17

No results posted yet for this study

Summary

This is a first-in-human study for the treatment of Fabry disease. Eligible patients will have an autologous stem cell transplantation using CD34+ cells that are transduced with the lentivirus vector containing the human alpha-gal A gene. The researchers of this study would like to see if the re-introduction of transduced cells will help increase the levels of alpha-gal A enzyme levels and to determine the safety and toxicity of autologous stem cell transplantation using CD34+ cells transduced with lentivirus vector containing the alpha-gal A gene. This study's objective is to determine the safety and toxicity of lentivirus alpha-gal A transduced CD34+ cells in adult males with Fabry disease.

Conditions

Interventions

BIOLOGICAL

Lentivirus Alpha-gal A transduced stem cells

Sponsors & Collaborators

  • Ozmosis Research Inc.

    collaborator INDUSTRY

  • University Health Network, Toronto

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
50 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-07-31
Primary Completion
2024-04-30
Completion
2024-04-30

Countries

  • Canada

Study Locations

More Related Trials

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 NCT02800070 on ClinicalTrials.gov