Researchers at the National Institute of Neurological Disorders and Stroke (NINDS) are seeking people with progressive supranuclear palsy (PSP) for a study. The study will evaluate the safety and effectiveness of increasing doses of an experimental drug-recombinant-methionyl human glial cell line-derived neurotrophic factor (GDNF)-administered through an implanted pump and catheter system.
Eligible persons are those who have been diagnosed with mild to moderate PSP, are otherwise healthy and between the ages of 35 and 75, and who have a caregiver who can serve as their Durable Power of Attorney for Health Care. Persons not eligible for the study are those who have had a stroke or significant head trauma in the past two years, or have advanced PSP or clinically significant psychosis or hallucinations. Also ineligible are persons who are taking any antiparkinsonian medications that they or their doctors feel cannot be stopped, or those who have a known allergy to E. coli-derived products or a history of severe breathing difficulty after taking any medication, have an existing shunt or catheter system in their head, or have any other serious medical condition such as severe degenerative arthritis, cardiovascular disease, body weight less than 15 percent of ideal, or significant laboratory abnormalities that would interfere with assessment of the safety and effectiveness of the medication.
Participants in the study will have a catheter inserted directly into their brains. This is an ongoing study that will require participants to come to NIH every two to four weeks until the study concludes. Participation will last at least 2 years.
The study will take place at the National Institutes of Health (NIH) Clinical Center in Bethesda, MD. All study-related expenses will be paid by the NIH. This study is conducted under safety and testing standards of the Department of Health and Human Services.
To refer eligible patients, physicians should send a letter of referral and case summary to Dr. Thomas Chase, Chief, Experimental Therapeutics Branch, NINDS, NIH, Building 10, Room 5C106, 10 Center Drive MSC 1406, Bethesda, MD 20892-1406; fax: (301) 496-6609. Please call Amy Bridgeman with any questions at 301-496-4604 or 1-800-362-3479. Refer to study number: 00-N-0145.