Study of oral nizubaglustat for rare genetic disorders
Treatments studied
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new oral medicine, nizubaglustat (AZ-3102), for children and adults (4+) with late-infantile or juvenile Niemann-Pick type C, GM1, or GM2 gangliosidosis. It aims to see if the drug is safe and helps slow disease progression.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be 4 years old or older
- Have a confirmed diagnosis of late-infantile or juvenile Niemann-Pick type C disease, or GM1 or GM2 gangliosidosis (Tay-Sachs, Sandhoff, or GM2AB variant)
- Have the late-infantile or juvenile form of the disease
- Not have certain other serious health problems (detailed info in the full subprotocol)
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study tests an oral medication called nizubaglustat for people with Niemann-Pick type C disease who cannot take or are not satisfied with the current treatment miglustat. It aims to see if the drug can help with symptoms like coordination and movement problems.
This trial is for people with Niemann-Pick type C or GM2 gangliosidosis who are already on or have been in a related study. It tests a new drug called Nizubaglustat to see if it is safe and works better than current treatments.
This trial tests a new medicine called GC1130A for children with Sanfilippo syndrome type A (MPS IIIA). The goal is to see if it is safe and if it might help with symptoms.
This early-stage trial tests JR-446, a new treatment for MPS IIIB, a rare genetic disorder that affects the brain and body. The treatment is delivered directly into the fluid around the spinal cord and aims to slow or improve symptoms in young children.
This trial tests an experimental drug called nizubaglustat (AZ-3102) for people with Gaucher disease type 3. It aims to see if the drug can improve symptoms like anemia, low platelets, and enlarged spleen, which are common in this condition.
This study follows people with rare genetic conditions (GM1, GM2, sialidosis, or galactosialidosis) that affect how the body breaks down certain substances in cells. Researchers track how these diseases progress to better understand them and potentially help future patients.
Hear when a new GM1 Gangliosidosis trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.