NCT05039866

Brief Summary

Long-term follow-up of subjects who received ST-920 in a previous trial (ST-920-201) and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 5 years following ST-920 infusion.

Trial Health

82
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
48

participants targeted

Target at P25-P50 for all trials

Timeline
156mo left

Started Aug 2021

Longer than P75 for all trials

Geographic Reach
5 countries

13 active sites

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress27%
Aug 2021Mar 2039

Study Start

First participant enrolled

August 16, 2021

Completed
4 days until next milestone

First Submitted

Initial submission to the registry

August 20, 2021

Completed
21 days until next milestone

First Posted

Study publicly available on registry

September 10, 2021

Completed
7.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2029

Expected
10 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2039

Last Updated

May 25, 2025

Status Verified

May 1, 2025

Enrollment Period

7.5 years

First QC Date

August 20, 2021

Last Update Submit

May 21, 2025

Conditions

Keywords

Fabry DiseaseGene TherapyST-920RareGeneticDNASangamoLong Term

Outcome Measures

Primary Outcomes (1)

  • To evaluate long-term safety of ST-920

    To evaluate long-term safety of ST-920 by assessment of incidence and severity of adverse events (AEs)

    4 years

Study Arms (1)

Subjects who received ST-920

Subjects who received ST-920 in a separate parent trial

Biological: ST-920

Interventions

ST-920BIOLOGICAL

No study drug is administered in this study. Subjects who received ST-920 in a separate parent trial will be evaluated in this trial for long-term safety.

Subjects who received ST-920

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

All subjects who have received ST-920 in separate parent trial and who have consented to participate in this Long Term Follow-up study.

You may qualify if:

  • Subjects who received ST-920 therapy in a separate parent trial
  • Subjects who have consented to participate in this LTFU study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

University of California, Irvine

Irvine, California, 92697, United States

Location

University of South Florida

Tampa, Florida, 33620, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

Location

University of Iowa Hospital and Clinics

Iowa City, Iowa, 52242, United States

Location

University of Minnesota Medical Center

Minneapolis, Minnesota, 55455, United States

Location

Mt. Sinai Hospital

New York, New York, 10029, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)

Fairfax, Virginia, 22030, United States

Location

The Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia

Location

M.A.G.I.C. Clinic Ltd.

Calgary, Alberta, T2E 7Z4, Canada

Location

University Hospital of Würzburg

Würzburg, Germany

Location

Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

Royal Free Hospital

London, United Kingdom

Location

Biospecimen

Retention: SAMPLES WITH DNA

plasma, serum

MeSH Terms

Conditions

Fabry DiseaseFabry Disease, Cardiac Variant

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCerebral Small Vessel DiseasesCerebrovascular DisordersVascular DiseasesCardiovascular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Study Officials

  • Medical Monitor

    Sangamo Therapeutics, Inc.

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 20, 2021

First Posted

September 10, 2021

Study Start

August 16, 2021

Primary Completion (Estimated)

March 1, 2029

Study Completion (Estimated)

March 1, 2039

Last Updated

May 25, 2025

Record last verified: 2025-05

Locations