Gene Editing's lasting impact: a 10-Year safety watch
NCT ID NCT04628871
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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New York University Grossman School of Medicine
New York, New York, 10016, United States
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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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.
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- Can a single gene shot free hemophilia b patients from regular infusions?
- Can a video call replace the clinic for hemophilia pain relief?
- Newborn screening study aims to catch rare diseases at birth
- Monthly shot could free kids with hemophilia from frequent infusions
- New shot aims to cut bleeding in hemophilia patients