RecruitingNCT05047354
Rare condition
Smith-Lemli-Opitz syndrome
1
studies recruiting now
as of 7 Sept 2026
20
studies registered in total
as of 7 Sept 2026
1
countries with a recruiting site
as of 7 Sept 2026
17 Sept 2021
most recent study posted
among recruiting studies
Recruiting trials
Showing the 1 most recently updated recruiting study, as recorded 7 Sept 2026. Live status on each study page.
Search all Smith-Lemli-Opitz syndrome studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Patient organisations
We do not yet list a dedicated organisation for this condition. The directories below are the best route.
About Smith-Lemli-Opitz syndrome
RareTrial does not yet hold a plain-language description of this condition. The most reliable starting point is Orphanet’s expert page, which lists specialist centres, registries and patient organisations, and the gene involved (DHCR7).
Treatments being studied
3 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 2Simvastatin (Flolipid)
Phase 1/2Cholesterol (Cholesterol component of alf)
Phase 1/2Cholic Acid (Cholbam)
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).