Clinical Trials

Are you or a loved one living with symptoms of Adrenomyeloneuropathy (AMN)?

If so, you or your loved one may be eligible to participate in a clinical research study to determine whether Lorenzo’s Oil (a 4:1 mixture of glyceryl trioleate and glyceryl trierucate) can help reduce symptoms of AMN. In addition to the Lorenzo’s oil portion of the study, the research will measure the benefits that an individualized physical therapy program can have on individuals with AMN.

Upon enrolling for the AMN study, participants will receive a comprehensive physical evaluation. The AMN study staff will then design a customized physical therapy program tailored to each patient’s individual needs. In addition, AMN study staff will work to identify a qualified physical therapist in the patient’s home community who can support the individual’s program and track their progress.

Studies have shown physical therapy to be a key component to the health and long-term prognosis of patients with AMN; therefore, the trial will benefit all trial participants who maintain the program.

The Lorenzo’s Oil part of the AMN study is placebo-controlled, that is, half of the patients receive Lorenzo's Oil, while the other half receive a placebo (soybean oil).

The direct medical benefits of Lorenzo’s Oil are to be determined; however, there are no significant medical risks associated with this study.

Patients are required to visit the study facilities in Baltimore for a series of tests at the beginning of the study, and yearly thereafter, for four years. For each visit, customary travel expenses, cost of hospital or hotel stay and all tests associated with the study will be covered, with few exceptions.

During these four visits (each requiring an overnight stay, with patients arriving on the evening before the tests and leaving by 5:00 PM the following day), studies performed will include:

· general medical examination and nutritional evaluation
· neurological examination
· analysis of adrenal function
· MRI studies of the nervous system
· analysis of muscle strength, spasticity, sensation, balance and gait

Common Symptoms of AMN

AMN is a rare disease that is often misdiagnosed as multiple sclerosis (MS). Some of the symptoms that can be present with AMN are:

· Difficulty in walking/change in walking pattern
· Gradual, progressive weakness and stiffness of the legs
· Loss of the ability to coordinate muscle movement
· Excessive muscle tone
· Visual defects
· Difficulty in articulating words
· Seizures
· Adrenal insufficiency affecting control of blood pressure, heart rate, and sexual development and reproduction
· Sexual dysfunction/impotence
· Behavioral changes
· Bladder dysfunction
· Mild peripheral neuropathy
· Weight loss
· Nausea

To learn more about the AMN clinical research study at the Kennedy Krieger Institute, call 1-443-923-2772 or email Kim Hollandsworth at hollandsworth@kennedykrieger.org.

To learn more about clinical research studies, also known as clinical trials, visit www.clinicaltrials.gov, a Web site of the U.S. National Institutes of Health. For more information about AMN, visit www.ulf.org or www.myelin.org.

Principal Study Investigator: Hugo W. Moser, MD
Research Project Number (RPN#): 03-09-29-06