Testing a new drug for two rare brain diseases
Treatments studied
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 12 years or older.
- You have a confirmed genetic diagnosis of Niemann-Pick type C (NPC) or GM2 gangliosidosis.
- For NPC patients not in the earlier study: you have been taking a full dose of Miglustat for at least 12 months and your disease has stayed the same or gotten worse (not improved) in the last 3 months.
- You are willing to switch from Miglustat to the new drug if you have NPC.
- You can attend all study visits and follow the plan.
- You or your guardian can sign the consent form.
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 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.
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 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 early-stage study tests an IV gene therapy that delivers a working copy of an enzyme gene to help the body make beta-galactosidase in GM1 gangliosidosis. It may help slow or improve disease symptoms and looks closely at safety and how well the treatment works.
This Phase 3 trial tests whether N-acetyl-leucine can improve symptoms in people with Niemann-Pick disease type C (NPC). If you qualify, you will be asked to take study medicine and complete symptom and movement tests over time.
This study measures biochemical (blood/biologic) markers and may use clinical evaluation and MRI to better understand Niemann-Pick disease type C. It could help improve how the condition is tracked and understood over time.
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