A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease

Who is this study for? Patients with Fabry Disease
What treatments are being studied? ST-920
Status: Recruiting
Location: See all (18) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

This is the first in human treatment with ST-920, a recombinant AAV2/6 vector encoding the cDNA for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a single dose of ST-920 and followed for a period of 52 weeks.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: No
View:

• ≥ 18 years of age

• Documented diagnosis of Fabry disease

• One or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma

• Subject must be fully vaccinated (as per the Centers for Disease Control and Prevention (CDC) definition in the US and as per local guidelines in other countries) for COVID-19 at least one month prior to dosing

• Additional Inclusion Criteria:

• Renal Cohort:

• Screening eGFR value between 40-90 mL/min/1.73 m²

• Linear negative eGFR slope (estimated from at least 3 serum creatinine values within 18 months, including the value obtained during screening visit) of ≥ 2 mL/min/1.73m²/year

• Cardiac Cohort:

• • Left ventricular hypertrophy (LVH) in 2D echocardiography or CMR defined as an end diastolic septum and posterior wall thickness ≥12 mm with no other explanation for LVH, OR presentation with cardiac changes indicative of disease progression such as decreased global longitudinal strain on 2D strain echocardiography or low native T1 mapping on CMR

Locations
United States
California
University of California, Irvine
Recruiting
Irvine
Florida
University of Florida
Recruiting
Gainesville
Georgia
Emory University School of Medicine
Recruiting
Atlanta
Iowa
University of Iowa Hospital and Clinics
Recruiting
Iowa City
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Recruiting
Chicago
Minnesota
University of Minnesota Medical Center
Recruiting
Minneapolis
New York
Mt. Sinai School of Medicine
Recruiting
New York
NYU Langone Health Neurogenetics
Withdrawn
New York
Ohio
Cincinnati Children's Hospital Medical Center
Recruiting
Cincinnati
Pennsylvania
University of Pittsburgh Medical Center
Recruiting
Pittsburgh
Tennessee
Vanderbilt University Medical Center
Recruiting
Nashville
Virginia
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Recruiting
Fairfax
Other Locations
Australia
The Royal Melbourne Hospital
Recruiting
Parkville
Canada
M.A.G.I.C. Clinic Ltd.
Recruiting
Calgary
Italy
Azienda Ospedaliero-Universitaria Careggi
Recruiting
Florence
United Kingdom
Queen Elizabeth Hospital
Recruiting
Birmingham
Addenbrooke's Hospital
Recruiting
Cambridge
Royal Free Hospital
Recruiting
London
Contact Information
Primary
Patient Advocacy
clinicaltrials@sangamo.com
510-307-7266
Time Frame
Start Date: July 23, 2019
Estimated Completion Date: February 2024
Participants
Target number of participants: 48
Treatments
Experimental: Sequential dose escalation
ST-920 is administered as a single infusion:~Cohort 1: 0.5e13 vg/kg~Cohort 2: 1.0e13 vg/kg~Cohort 3: 3.0e13 vg/kg~Cohort 4: 5.0e13 vg/kg
Experimental: Expansion Cohorts
Anti Alpha-Gal A Antibody Positive Cohort~Anti Alpha-Gal A Antibody Negative Cohort~Female Cohort~Renal Cohort~Cardiac Cohort
Related Therapeutic Areas
Sponsors
Leads: Sangamo Therapeutics

This content was sourced from clinicaltrials.gov

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