← Back to Search

Gene Therapy

Engineered Stem Cell Transplant for Fabry Disease

Phase 1

Waitlist Available

Research Sponsored by University Health Network, Toronto

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

Study Summary

This trial is to test a new treatment for Fabry disease using the patient's own stem cells. The stem cells are modified to produce the missing enzyme, then transplanted back into the patient. The goal is to see if this is safe and effective.

Who is the study for?

This trial is for male patients aged 18-50 with Fabry Disease, specifically the Classic FD Type I phenotype. They must have been on enzyme replacement therapy (ERT), have adequate organ function, and be willing to follow study procedures and use contraception post-treatment. Exclusions include uncontrolled conditions like hypertension or psychiatric disorders, severe heart or kidney disease, immune deficiencies, prior transplants, active infections including HIV/HBV/HCV/HTLV or syphilis.Check my eligibility

What is being tested?

The trial tests a new treatment where patients' own stem cells are modified with a lentivirus to carry the alpha-gal A gene and then returned to their bodies. The goal is to see if this can safely increase alpha-gal A enzyme levels in adult males with Fabry disease.See study design

What are the potential side effects?

Potential side effects may include reactions related to stem cell transplantation such as infection risks due to weakened immunity, possible infusion-related reactions from the modified cells, and toxicity from Melphalan used before transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment

Secondary outcome measures

Alpha-gal A enzyme activity levels

Gb3 levels

lyso-Gb3 analogue (+16)

+7 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment ArmExperimental Treatment1 Intervention

Patients will receive Health Canada approved transduced autologous CD34+ cell product.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Benzalkonium

FDA approved

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

University Health Network, TorontoLead Sponsor

1,471 Previous Clinical Trials

484,965 Total Patients Enrolled

1 Trials studying Fabry Disease

250 Patients Enrolled for Fabry Disease

Ozmosis Research Inc.Industry Sponsor

20 Previous Clinical Trials

5,010 Total Patients Enrolled

Media Library

Fabry Disease Research Study Groups: Treatment Arm

Fabry Disease Clinical Trial 2023: Lentivirus Alpha-gal A transduced stem cells Highlights & Side Effects. Trial Name: NCT02800070 — Phase 1

Lentivirus Alpha-gal A transduced stem cells (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02800070 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is there an age restriction on participants for this investigation?

"This clinical trial is open to individuals between 18 and 50 years old. Separately, there are 5 studies for those below the age of consent as well as 17 programmes available for participants over 65."

Answered by AI

To what extent do Lentivirus Alpha-gal A modified stem cells endanger individuals?

"Our assessment of lentivirus alpha-gal A transduced stem cells' safety is a 1, as this trial is currently in phase 1 and there exists only scant information on efficacy and security."

Answered by AI

Is it possible to be a volunteer for this research study?

"Eligibility requirements for this trial include a diagnosis of fabry disease and being between the ages of 18-50. To keep results consistent, only 5 participants are accepted to participate in the study."

Answered by AI

Is recruitment for this research initiative still open?

"According to clinicaltrials.gov records, this trial is not presently recruiting patients -- the posting was first published on July 1st 2016 and last updated April 20th 2020. Nevertheless, there are 22 other trials open for enrollment at present."

Answered by AI

Recent research and studies

Nature CommunicationsJournal

Lentivirus-mediated Gene Therapy for Fabry Disease

Khan, Aneal, Dwayne L. Barber, Ju Huang, C. Anthony Rupar, Jack W. Rip, Christiane Auray-Blais, Michel Boutin, et al.. 2021. “Lentivirus-mediated Gene Therapy for Fabry Disease”. Nature Communications. Springer Science and Business Media LLC. doi:10.1038/s41467-021-21371-5.

clinicaltrials.govStudy

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

2016. "Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02800070.