Plain-English translation of NCT01668186 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
This is a long-term research study for people diagnosed with rare peroxisome disorders—genetic conditions affecting how cells break down certain fats and other substances. Researchers are tracking patients' health over time, collecting medical records and images, and gathering tissue samples to identify new biomarkers and test whether candidate drugs might help restore how peroxisomes work in the body.
Very little is known about how these rare disorders progress over a lifetime, and there are currently no proven treatments. By following patients carefully and studying their medical information and tissue samples, researchers hope to better understand the disease and identify which drugs might be effective.
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If you enroll, you would visit McGill University Health Centre once a year for appointments with specialists in genetics, nutrition, neurology, and eye health (including special imaging tests). Researchers will collect your past and current medical records, imaging scans, and small tissue samples, and enter them anonymously into a database. These samples will help researchers test whether certain drugs might restore peroxisome function. The study continues until completion, and you can choose whether or not to participate in the in-person visits.
AI-generated summary from trial data · Jun 7, 2026 · Not medical advice
Canada