A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
At a glance
Conditions
NCT07640984
Where you'd take part
This study runs at 5 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Children's Hospital Oakland Research Institution, Attn: 2900 Wing
Oakland, Californiastudy coordinator listed
Great Ormond Street Hospital for Children
London, United Kingdomstudy coordinator listed
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginiastudy coordinator listed
University Medical Center Hamburg-Eppendorf
Hamburg, Germanystudy coordinator listed
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvaniastudy coordinator listed
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- To establish the safety and tolerability of JR-446 in MPS IIIB patients following repeated dose administrationUp to 1 year (multiple visits)
Incidence and severity of treatment-emergent adverse events; Incidence and severity of infusion-associated reactions; Incidence of clinically significant changes/abnormalities in safety laboratory tests, vital signs, and electrocardiogram.