Biochemical and Phenotypical Aspects of Smith-Lemli-Opitz Syndrome and Related Disorders of Cholesterol Metabolism

Recruiting Observational Study
Smith Lemli Opitz Syndrome CHILD Syndrome Lathosterolosis Desmosterolosis
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Healthy Volunteers Welcome You do not need to have the condition being studied to take part. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
1 Day – 100
Sex
Any
Study type
Observational
Participants needed
250 (estimated)
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · NIH
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About This Trial
Background: Smith-Lemli-Opitz Syndrome (SLOS) is a genetic disorder. It can cause birth defects and developmental delays. There is no cure for SLOS or other inherited diseases related to cholesterol production or storage. The data gained in this study may help researchers find ways to measure how well future treatments work. Objective: To learn more about SLOS and related disorders and how thes…
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA: Males or females of any age with any one of the following: * Clinical, biochemical, or genetic diagnosis of Smith-Lemli-Opitz Syndrome OR * Clinical, biochemical, or genetic diagnosis of desmosterolosis, lathosterolosis, CHILD syndrome, X-linked dominant chondrodysplasia type…
Contacts

Derek M Alexander

(301) 827-0387

derek.alexander@nih.gov

CONTACT

Forbes D Porter, M.D.

(301) 435-4432

fdporter@mail.nih.gov

CONTACT