Study Details & Procedures

How many visits are there?

The study requires 9 visits over the course of approximately 6 months. The second visit requires two overnight stays in the hospital, and for some patients a third night in a hotel close to the hospital. The 9th visit also requires one overnight stay in the hospital.

How long is the study?

The screening period is 2 to 4 weeks. The treatment period is 3 months. The follow-up period is 6 weeks.

How often is the study drug given?

Patients will have 4 doses of the study drug given about one month apart. The drug is given by a lumbar puncture (LP) or spinal tap, which is a needle put into the lower back. Before the spinal tap the patient will receive anesthesia.

Once the dosing of study drug starts at the second visit, patients will be required to come back to see the doctor every 2 weeks until the eighth visit. The ninth and last visit is 6 weeks after the eighth visit. If your child is screened for the study, the study staff will review these details with you.

What tests are done during the study?

There are a variety of tests performed during the study including blood draws, urine collections, psychological testing, ECGs (electrical recording of the heart), and video EEGs (electrical recording of brainwave activity). Patients will also be asked to wear a small device on the ankle or waist every night to measure sleep and another device on each ankle during the day for a couple of weeks at the beginning and end of the study to measure walking. All of these things will be explained in more detail if your child is screened for the study by the study site staff.

Enrolling Into the Study

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