Clincosm LogoSign in Get a demo

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease

Sponsor
Sangamo Therapeutics (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04046224
Collaborator
(none)
48
Enrollment
13
Locations
1
Arm
54.3
Anticipated Duration (Months)
3.7
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
  • Biological: ST-920
 Phase 1/Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
48 participants
Allocation:
N/A
Intervention Model:
Sequential Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
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
Actual Study Start Date :
Jul 23, 2019
Anticipated Primary Completion Date :
Dec 1, 2023
Anticipated Study Completion Date :
Feb 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Sequential dose escalation

ST-920 is administered as a single infusion

Biological: ST-920
Single dose of investigational product ST-920

Outcome Measures

Primary Outcome Measures

  1. Incidence of treatment-emergent adverse events (TEAEs) [Up to 12 months after the ST-920 infusion]

    Incidence of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:

  • ≥ 18 years of age

  • Documented diagnosis of Fabry disease

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

  1. anhidrosis, iv) angiokeratoma
Exclusion Criteria:

  • Known to be unresponsive to ERT

  • Neutralizing antibodies to AAV2/6

  • Currently receiving migalastat (Galafold™)

  • eGFR ≤ 40 ml/min/1.73m2

  • New York Heart Association Class III or higher

  • Active infection with hepatitis A, B or C, HIV or TB

  • History of liver disease such as secondary steatosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, cholangitis or biliary disease within 6 months of informed consent; except for Gilbert's syndrome

  • Elevated circulating serum AFP

  • Recent or recurrent hypersensitivity response to ERT within previous 6 months

  • Current or history of systemic (IV or oral) immunomodulatory agents, or biologics or steroid use in the past 6 months (topical treatment and inhaled allowed).

  • Contraindication to use of corticosteroids

  • History of malignancy except for non-melanoma skin cancer

  • Recent history of alcohol or substance abuse

  • Participation in prior investigational interventional drug or medical device study within previous 3 months

  • Prior treatment with a gene therapy product

  • Known hypersensitivity to components of ST-920 formulation

  • Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study including but not limited to risk of COVID-19 infection

Contacts and Locations

Locations

Site City State Country Postal Code
1 University of California, Irvine Irvine California United States 92697
2 University of Florida Gainesville Florida United States 32611
3 Emory University School of Medicine Atlanta Georgia United States 30322
4 University of Iowa Hospital and Clinics Iowa City Iowa United States 52242
5 University of Minnesota Medical Center Minneapolis Minnesota United States 55455
6 NYU Langone Health Neurogenetics New York New York United States 10017
7 Mt. Sinai School of Medicine New York New York United States 10029
8 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
9 University of Pittsburgh Medical Center Pittsburgh Pennsylvania United States 15213
10 Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) Fairfax Virginia United States 22030
11 Queen Elizabeth Hospital Birmingham United Kingdom B15 2TH
12 Addenbrooke's Hospital Cambridge United Kingdom CB2 0QQ
13 Royal Free Hospital London United Kingdom

Sponsors and Collaborators

  • Sangamo Therapeutics

Investigators

  • Study Director: Medical Monitor, Sangamo Therapeutics, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Sangamo Therapeutics
ClinicalTrials.gov Identifier:
NCT04046224
Other Study ID Numbers:
  • ST-920-201
First Posted:
Aug 6, 2019
Last Update Posted:
Apr 19, 2022
Last Verified:
Apr 1, 2022
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Sangamo Therapeutics
Sangamo
Rare
Lysosomal Storage Disease
Gene Therapy
Additional relevant MeSH terms:
Fabry Disease

Study Results

No Results Posted as of Apr 19, 2022