ADX-629 for Sjogren-Larsson Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests if ADX-629, a medicine taken by mouth, is safe and effective for people with Sjögren-Larsson syndrome (SLS). SLS is a rare genetic disorder that causes harmful fats to build up in the body. ADX-629 helps remove these harmful fats, which may reduce symptoms in the skin, brain, and eyes.
Will I have to stop taking my current medications?
The trial requires participants to stop using all topical creams 7 days before the study and any experimental drugs 1 month before the study. If you are on immunosuppressive therapy or certain systemic or topical medications, you must be willing to stop them 2 weeks before and during the study.
Who Is on the Research Team?
William B Rizzo, MD
Principal Investigator
University of Nebraska
Are You a Good Fit for This Trial?
Adults aged 18-50 with Sjogren-Larsson Syndrome (SLS), weighing at least 35 kg, able to swallow pills and follow the study plan. Participants must have ichthyosis, spasticity, a genetic confirmation of SLS, and agree to contraception if sexually active. Excluded are those in other studies, with certain health conditions or treatments that could affect results or safety.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive oral ADX-629 treatment for 12 weeks to assess safety and biochemical efficacy
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ADX-629
Find a Clinic Near You
Who Is Running the Clinical Trial?
William Rizzo, MD
Lead Sponsor
University of Nebraska
Lead Sponsor