Clinical Trials

Clinical trial information for Kennedy's Disease

The best candidate for future trials:

• 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.

Trial Results

BVS857 clinical trial results are in.

The results of Novartis Pharmaceuticals drug BVS857 study involving patients with Spinal and Bulbar Muscular Atrophy has been published. For more information, click here. 

• Dr. Fischbeck discusses the current trial for BVS857 in the March 01, 2014 Chat.
• Excerpts from the chat can be found in the Living with Kennedy's Disease blog
• The Clinical Trial announcement can be found at the NIH Clinical Trial webpage 

NIH Releases Results from Exercise Trial 

We are contacting you to let you know the results of the exercise trial in SBMA here at the NIH.  The trial did not show a statistically significant change in the primary outcome measure, the AMAT, overall.  However, a subgroup analysis done after the study did indicate that low-functioning men with SBMA may respond better to functional exercise. We also found that functional exercise is safe and well tolerated.  

Please click HERE for the results and supplemental charts.  

We want to thank you for your time and commitment that you gave to us during this trial.  We certainly could not do this without you.

Thank you,

The National Institute of Health had a two year clinical trial on the potential benefits of exercise.  A summary of the trial follows:

June 10, 2011 - Clinical Research Study on Kennedy’s Disease

Effect of Functional Exercise in Patients 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.

B. Objective:
-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

C. Eligibility:
-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.

D. Design:
-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:

INCLUSION 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.
7. Male.
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

EXCLUSION CRITERIA:

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.

Contact(s):

Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793
Electronic Mail:

The National Institute of Health finished a two year clinical trial in 2008.  A summary of the results of the study follows:

A trial of dutasteride in spinal and bulbar muscular atrophy

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

Abstract

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

Efficacy and safety of leuprorelin in patients with spinal and bulbar muscular atrophy (JASMITT study): a multicentre, randomised, double-blind, placebo-controlled 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.