New hope for kids with sanfilippo syndrome b?
NCT ID NCT07640984
First seen Jun 25, 2026 · Last updated Jul 08, 2026 · Updated 3 times
Summary
This early-stage trial tests a new drug called JR-446 in 12 children under 6 with MPS IIIB, a rare genetic disorder that damages the brain and body. The main goal is to see if the drug is safe and tolerable. Researchers will also measure changes in certain substances in the body that may show if the drug is working.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- JR-446 (given as an IV infusion)
- What this could lead to
- If successful, this could point toward a treatment that slows or stabilizes MPS IIIB in young children.
- What could go wrong
- This is a very early, small trial (12 participants) focused on safety, not proof of effectiveness. The drug may not work or could cause side effects.
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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Children's Hospital Oakland Research Institution, Attn: 2900 Wing
Oakland, California, 94609, United States
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Great Ormond Street Hospital for Children
London, WC1N 3JH, United Kingdom
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Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginia, 22030, United States
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UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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University Medical Center Hamburg-Eppendorf
Hamburg, 20246, Germany