The Efficacy of Minocycline in the Treatment of Angelman Syndrome

This prospective single arm cohort study is to be conducted at the University of South Florida. The study will examine the effect minocycline (MC) has on the traits of Angelman Syndrome.

Minocycline HCl is an FDA approved antimicrobial medication in the tetracycline family of drugs. Of all the tetracyclines, MC is the most lipid soluble and most active. Unlike other antibiotics in this family, MC possesses the unique characteristic of being able to cross the blood brain barrier. The study dosage has been used in other trial treatments of other neurologic disorders with positive outcomes. Studies of long-term administration of MC at the study dose have been shown to be safe and well tolerated. This dosage has already been approved by the FDA for use in the treatment of bacterial infections of multiple organ systems, and acne vulgaris.

It is important to note that minocycline is not approved to treat Angelman syndrome or to be used in children younger than 8 years old. The study protocol has been reviewed by several physicians and scientists and been deemed safe to proceed. As with any medication the potential for side effects exist. The side effects range from serious to mild and include allergic reactions to upset stomach. In order to minimize this risk, the medical staff will perform a thorough medical history and physical examination before the prescription is issued to ensure no allergy to this medication, penicillin or another tetracycline exists. Discoloration of the teeth is potential adverse effect that exists when taking high doses of MC over long periods of time. The tooth discoloration is permanent and the parent or guardian of the participants will be made aware of this potential side effect prior to enrollment in the study. In other studies using MC, the most common complaint was gastrointestinal upset.

Recruitment & Prescreening - We anticipate some of the study participants will live far from the study site. In order to reduce screen failures (travel to the site only to find out your child doesn't meet the study criteria) the following process is required. To give parents the opportunity to consider the study and have time to consult with their doctor(s), we will begin recruitment approximately one week after the study has been published on this website. Parents will be asked to submit their information electronically by clicking the link at the bottom of this webpage or by accessing weeberlab.com/clinical_trials.html. In the event they do not have internet access, they may call the study coordinator for assistance. Once you have indicated your interest in participation, a packet of information will be sent to you via email (or postal service if you prefer). The packet will include an informed consent document, a release of medical information and a form for your primary care doctor to complete and return directly to us. All of this information will be reviewed by the medical staff to determine your child's eligibility. Out of the first 50 eligible participants, 24 will be randomly selected by the Clinical and Investigational Science Institute (CTSI) at USF.

Study Procedure - Those selected will be required to travel to the study site a total of 3 times for 2 days each at their own expense (some assistance may be available through the Foundation for Angelman Syndrome Therapeutics, visit www.cureangelman.org). It is important that you seriously consider your ability to complete the study. The number of participants we are allowed to enroll is strictly governed and funding is limited. It is imperative data is collected from each of the 24 participants to ensure the best possible results. We do not know if your child will benefit from receiving this medication, which is why we are doing this study.