Study drug 4D-310 for Fabry disease with heart problems
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This Phase 1/2 study tests how safe and potentially effective 4D-310 is for adults with Fabry disease who have heart involvement. It may help people who can’t tolerate standard treatments like enzyme replacement therapy (ERT) or migalastat, or who still have disease progression.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are age 18 to 65
- You have Fabry disease confirmed by a known GLA gene mutation
- You have Fabry disease with heart involvement
- You cannot take ERT, do not want ERT, or your disease has progressed despite ERT or migalastat (or you can’t tolerate them)
- If you take ERT now, your dose has been steady for at least 6 months
- You agree to use very effective birth control during 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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