Currently, there is only one clinical trial for Kennedy's Disease going on.
The best candidates for future trials are:
- ASC-J9 - This drug looks very promising, but it could take at least two years before ready for trial. It has to pass FDA requirements and additional work on mouse models are required before consideration. Currently it is only available as an injection, but they are looking at developing an oral tablet.
- IGF-1 - This could be made available within the near future for a trial. The hormone injection in mouse models showed improved strength and longevity. Toxicology testing and other FDA approvals are already complete. This hormone is muscle specific. NIH needs to test this hormone on more mouse models.
Recent News of Clinical Importance
UNIVERSITY OF CALIFORNIA, DAVIS
Department of Physical Medicine and Rehabilitation
Dr. Jay Han,
Principal Investigator, Associate Professor
Co-Director, Muscular Dystrophy Association Neuromuscular Disease Clinic
VOLUNTEERS ARE NEEDED FOR A RESEARCH STUDY ON
Study Coordinator: Alina Nicorici (916) 734-0968
Before clinical trials can begin in subjects with Neuromuscular Disorders, the FDA requires the development of clinically meaningful outcome measures that clearly show that a potential therapy will represent a “life-altering event”. There are several clinically meaningful measures. The department of Physical Medicine and Rehabilitation at the University of California is trying to determine the relationship between the commonly-used measures for clinical trials of Neuromuscular Disorders.
Subjects with Neuromuscular Disorders are needed between the Ages of 12 and 75 years old. What will participants be asked to do?
- Spend approximately 3.5 hours on two occasions (1 year apart) in the Exercise Physiology Lab in the Lawrence J. Ellison Ambulatory Care Center located at 4860 “Y” St. Suite 1113, Sacramento, CA.
- Participants will be asked to fill out Socio-demographic and functional questionnaire
- Participants will be asked to complete Health-related quality of life assessment questionnaires
- Participants will be measured for Height, Weight, Limb Length Measurements and Waist Circumference
- A Body Composition Measurement will be taken
- Arm and Leg Functional Grading will be assessed
- Timed Motor Performance will be assessed
- Pulmonary Function Testing will be performed
- Mobility and Motor Function will be measured
Participants will receive a stipend of $40 in gas cards for each visit to offset travel expenses. If you have any questions or are interested in this study, please contact Study Coordinator: Alina Nicorici (916) 734-0968
Click here to read a copy of the consent form
The National Institute of Health has a two year clinical trial on the potential benefits of exercise. A summary of the trial follows:
For several months now we knew a clinical study was coming. This week NIH posted the following information on their website. (If you are interested in participating in this study, the contact information is near the bottom of this page)
Effect of Functional Exercise in Patients
Link to the article: http://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2011-N-0171.html
with Spinal and Bulbar Muscular Atrophy
- A. Summary: Background:
- -Spinal and bulbar muscular atrophy (SBMA) is an inherited disorder that affects men. People with SBMA often have weakness throughout the body, including the muscles they use for swallowing, breathing, and speaking. We do not know if exercise helps or harms people with SBMA.
-To see if a 12-week program of either strength exercise or stretching exercises will improve strength, function, or quality of life in people with SBMA
-Participants will be men 18 years of age or older who have genetic confirmation of SBMA.
-They must be able to walk at least 50 feet with or without an assistive device such as a cane or a walker and stand for 10 minutes without using an assistive device.
-They must have access to a computer with an Internet connection.
-At the first visit to NIH (2 days), participants will have a medical history taken and undergo a physical exam. They will also have blood tests and an EKG, and complete questionnaires about mood, health, and exercise. Tests of muscle strength, balance, and endurance will also be done.
-Participants who qualify for the study will receive instruction about either strengthening or stretching exercises. They will do these exercises at home one to three times a week for 12 weeks.
-They will wear a small activity monitor while they exercise and record their exercise in a diary.
-At the end of 12 weeks, participants will return to the NIH for 2 days. They will undergo the same tests as they had on the first visit.
-Participants will receive follow-up phone calls and e-mails during the study and for 4 weeks after the last visit.
E. Sponsoring Institute:
- National Institute of Neurological Disorders and Stroke (NINDS)
- F. Recruitment Detail
- Type: Participants currently recruited/enrolled
- Gender: Male
- G. Eligibility Criteria:
1. Genetically confirmed SBMA.
2. Ambulatory and walk a distance of at least 50 feet with or without a walker.
3. Able to stand for 10 minutes without the use of any assistive devices.
4. Willing to travel to the NIH at the beginning and end of the study.
5. Willing to participate in telephone monitoring.
6. AMAT score of less than 41, but greater than 14.
8. Willing to participate in all aspects of trial design and follow-up.
9. Access to a computer with an internet connection
10. Able to do all of the exercises according to the standards of the study examiners at the beginning and end of the study
11. Willing to forgo starting an additional exercise plan for the 12 week duration of the study
12. Age greater than 18 years
1. Medical condition which would preclude exercise such as COPD, congestive heart failure, and cardiac arrhythmias.
2. Presence of an additional comorbid condition such as stroke, myopathy, or radiculopathy which also results in weakness.
3. Beginning a separate exercise program involving at least two weekly sessions of 20 minutes of exercise each within two months of the start of the trial.
- Patient Recruitment and Public Liaison Office
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
The National Institute of Health finished a two year clinical trial in 2008. A summary of the results of the study follows:
Lindsay Rhodes, Angela Kokkinis, Michelle White, Charlotte Watts, Sungyoung Auh, Neal Jeffries, Joseph Shrader, Tanya Lehky, Li Li, Jennifer Ryder, Ellen Levy, Beth Solomon, Alison La Pean, Alice Schindler, Cheunju Chen, Nicholas Di Prospero and Kenneth Fischbeck
Spinal and bulbar muscular atrophy (SBMA) is caused by polyglutamine expansion in the androgen receptor, which results in ligand-dependent toxicity. SBMA animal models have a neuromuscular deficit that is mitigated by anti-androgen treatment. We explored the efficacy and safety of the anti-androgen drug dutasteride in a two year double-blind, placebo-controlled clinical trial. Physical, neurophysiological, quality of life, and biochemical outcomes were assessed in 50 SBMA subjects randomized to receive dutasteride or placebo. There was no significant difference in the primary outcome measure, change in strength as indicated by quantitative muscle assessment (QMA). Of the secondary outcome measures, only quality of life as measured by change in the SF-36v2 physical component summary showed a significant benefit (p=0.004), and this was balanced by a negative effect on the SF-36v2 mental component summary. In the placebo group the QMA showed an average decrease in muscle strength of 2% per year, indicating that SBMA patients may need to be studied over a longer period to show a significant effect on disease progression. These observations provide a basis for future therapeutic trials in SBMA, and indicate counter-balancing effects of androgens in this disease.
In a conference call with Dr. Kenneth Fischbeck, Angela Kokkinis, Ke-lian Chen, Alice Schindler and George Harrison in June of 2008 is summarized below:
The dutasteride trial did not show a significant effect on the progression of muscle weakness. Those patients on a placebo lost, on average, 2% of their strength per year (two-year study). Patients on Dutasteride showed a slight increase in strength, but statistically the difference was not significant. Dr. Fischbeck surmised that the trial needed to be longer (at least three years) to better determine any benefits. A few patients on Dutasteride did show significant improvement, while others did not. Dr. Fischbeck felt that this could mean that the drug reacts differently in certain patients.
The full report is available for download in PDF: Dutasteride Trial
Katsuno M, Banno H, Suzuki K, Takeuchi Y, Kawashima M, Yabe I, Sasaki H, Aoki M, Morita M, Nakano I, Kanai K, Ito S, Ishikawa K, Mizusawa H, Yamamoto T, Tsuji S,Hasegawa K, Shimohata T, Nishizawa M, Miyajima H, Kanda F, Watanabe Y, Nakashima K, Tsujino A, Yamashita T, Uchino M, Fujimoto Y, Tanaka F, Sobue G; for the Japan SBMA Interventional Trial for TAP-144-SR (JASMITT) study group.
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya, Japan; Institute for Advanced Research, Nagoya University, Nagoya, Japan.
INTERPRETATION: 48 weeks of treatment with leuprorelin did not show significant effects on swallowing function in patients with spinal and bulbar muscular atrophy, although it was well tolerated. Disease duration might influence the efficacy of leuprorelin and thus further clinical trials with sensitive outcome measures should be done in subpopulations of patients.