New hope for rare childhood brain disease

NCT ID NCT06488924

Summary

This early-stage study is testing a new drug called JR-446 in children under 18 with a rare, serious genetic disorder called MPS IIIB (Sanfilippo syndrome type B). The main goals are to check if the drug is safe and to see how it affects the body. Researchers will enroll about 10 participants to gather initial data on safety and potential effects.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Hiroshima University Hospital

    RECRUITING

    Hiroshima, Japan

    Contact Phone: •••-•••-••••

  • National Center for Child Health and Development

    RECRUITING

    Tokyo, Japan

    Contact Phone: •••-•••-••••

  • University of the Ryukyus Hospital

    RECRUITING

    Okinawa, Japan

    Contact Phone: •••-•••-••••

Conditions

Explore the condition pages connected to this study.