Study of rare enzyme disorders in monoclonal gammopathy or myeloma
Part of Blood & lymphatic, Brain & nervous system, Cancer, Genetic & congenital, Heart & circulation, Hormones & metabolism, Immune system & allergy clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are an adult (18 or older).
- You have an enlarged spleen (felt at least 1 cm below your ribs) or your spleen was removed for a reason not related to liver problems.
- Or you have low platelet counts (thrombocytopenia) that were there before you started treatment for your blood disorder.
- You are willing and able to give consent to join the study.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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