Clinical trials
Acid Sphingomyelinase Deficiency clinical trials
Below are recruiting acid sphingomyelinase deficiency clinical trials, each written for real people, not researchers. We’re tracking 3 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05992532Recruiting
Study of rare enzyme disorders in monoclonal gammopathy or myeloma
This study looks for two rare inherited disorders—Gaucher disease and acid sphingomyelinase deficiency (ASMD)—in people who already have a blood condition like monoclonal gammopathy (abnormal protein) or multiple myeloma. If you have an enlarged spleen or low platelets, you may be eligible to help researchers understand if these rare diseases are more common in this group.
Vitoria-Gasteiz, AlavaAges 18–99 - NCT06192576Recruiting
Long-term safety of olipudase alfa in young children with ASMD
This study looks at the long-term safety and how well the body accepts olipudase alfa therapy in children under 2 years old who have a certain type of ASMD. It helps understand if the treatment works safely over time.
Chicago, IllinoisAges Up to 2 years - NCT05368038Enrolling by invitation
Newborn screening program for babies up to 4 weeks old
This trial tests a flexible newborn screening process that looks for certain conditions early in life. It may help by catching problems sooner, when treatment can be started earlier.
Brooklyn, New YorkAges Up to 4 weeks
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Common questions
- Are there clinical trials for acid sphingomyelinase deficiency?
- Yes. Clin2 currently lists 3 recruiting acid sphingomyelinase deficiency studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a acid sphingomyelinase deficiency trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a acid sphingomyelinase deficiency trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.