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Gene Editing's lasting impact: a 10-Year safety watch

NCT ID NCT04628871

Knowledge-focused Sponsor: Sangamo Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 13, 2026 · Last updated Aug 14, 2026 · Updated 1 time

Summary

This study checks on people who previously received gene editing for hemophilia B or mucopolysaccharidosis (MPS) I or II. No new treatment is given; instead, participants are monitored for up to 10 years to see if any new health problems or worsening of existing conditions appear. The goal is to understand the long-term safety of these one-time gene therapies.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
No new treatment is given; this is a long-term follow-up of people who previously received gene editing (SB-318, SB-913, or SB-FIX).
What this could lead to
If this follow-up shows the gene editing remains safe over many years, it could support the use of these one-time treatments for hemophilia B and certain metabolic disorders.
What could go wrong
This is a small, observational follow-up, not a new treatment test. It cannot prove the gene editing works, and long-term safety issues may still emerge.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

13 people

The number who actually took part.

Started

Nov 2020

Finished

Jun 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

All subjects who have received SB-318 in Study Protocol SB-318-1502, SB-913 in Study Protocol SB-913-1602 or SB-FIX in Study Protocol SB-FIX-1501 and who have consented to participate in this Long Term Follow-up study.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Subjects who received SB-318 under Study Protocol SB-318-1502, SB-913 under Study Protocol SB-913-1602, or SB-FIX under Study Protocol SB-FIX-1501 2. Subjects who have provided consent to participate in the LTFU study. Exclusion Criteria: 1. Unable to comply with study visit schedule or study visit procedures. 2. Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for participation in the study.

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Conditions

The condition(s) this trial relates to.

hemophilia B Mucopolysaccharidosis I mucopolysaccharidosis type 1 mucopolysaccharidosis type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemophilia B Mucopolysaccharidosis I Mucopolysaccharidosis ii

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • New York University Grossman School of Medicine

    New York, New York, 10016, United States

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • University of North Carolina

    Chapel Hill, North Carolina, 27514, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.