Protocol Details

Phase II Clinical Trial to Examine the Efficacy and Safety of Dutasteride in Patients with Kennedy's Disease (Spinal and Bulbar Muscular Atrophy)

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number	06-N-0113
Sponsoring Institute	National Institute of Neurological Disorders and Stroke (NINDS)
Recruitment Detail	Type: Completed Study; data analyses ongoing Gender: Male Min Age: 18 Max Age: N/A
Referral Letter Required	No
Population Exclusion(s)	Female; Children
Special Instructions	Currently Not Provided
Keywords	Motor Neuron; Androgen Receptor; Polyglutamine; X-Linked; Ligand Dependency
Recruitment Keyword(s)	Spinal and Bulbar Muscalr Atrophy; SBMA; Kennedy Disease
Condition(s)	Kennedy's Disease; Spinal and Bulbar Muscular Atrophy
Investigational Drug(s)	None
Investigational Device(s)	None
Intervention(s)	Drug: Placebo Drug: Dutasteride
Supporting Site	National Institute of Neurological Disorders and Stroke

This study will determine if the drug dutasteride can improve weakness, mobility, functioning, nerve function, and quality of life in patients with spinal and bulbar muscular atrophy (SBMA). Patients with this inherited disease have an abnormal androgen receptor protein. The male hormones testosterone and dihydrotestosterone (DHT) bind to this abnormal receptor, causing damage to nerve cells that innervate muscle and leading to weakness. Dutasteride decreases DHT production. Lowering DHT levels may decrease the harmful effects of DHT to the nerves and improve strength in people with SBMA.

Males 18 years of age and older with SBMA who have neurological symptoms and can walk 100 feet (with or without assistive devices) may be eligible for this study. Candidates are screened with a blood test and a review of their medical records and genetic studies.

Participants undergo the following procedures:

-Blood and urine tests, history and physical examination, assessment of muscle strength

-Quality-of-life questionnaire

-Tests to assess functional abilities, such walking up steps, keeping the head up while lying down, and other measures

-Nerve conduction study and motor unit number estimation to assess nerve damage. A probe placed on the skin delivers small electrical impulses and wires taped to the skin record the impulses.

-Quantitative muscle testing to measure strength. The subject pushes and pulls levers attached to a gauge. Strength is recorded by a computer.

-Medication. Participants are divided into two groups. One group is given the study drug, dutasteride; the other receives a placebo (sugar pill). All participants take their assigned medication once a day for 24 months.

-Follow-up evaluations. Every 6 months for 2 years, participants return to NIH to repeat the tests described above to determine the effects of the dutasteride. Nerve and quantitative muscle testing is not done at the 6- and 18-month visits.

-In addition to their follow-up appointments here at the NIH every 6 months, participants will also have blood tests and a physical examination performed after 3, 9, 15 and 21 months of treatment by the patient's local physician.

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Eligibility

INCLUSION CIRTERIA:

Genetically confirmed SBMA.

Neurological symptoms of SBMA.

Ability to ambulate 100 feet with or without the use of assistive devices.

Willingness to participate in all aspects of trial design and follow-up.

Male sex.

EXCLUSION CRITERIA:

Age less than 18 years.

Female sex.

A history of hypersensitivity to dutasteride or 5 alpha-reductase inhibitors.

Patients who are taking potent cytochrome P450 3A4 (CYP3A4) inhibitors for over 4 weeks.

Patients with any pre-existing liver disease.

Alkaline phosphatase, gamma glutamyl transferase, or direct bilirubin greater than 1.5 times the upper limit of normal.

Alanine aminotransferase or aspartate aminotransferase greater than 1.5 times upper limit of normal in subjects with normal creatine kinase levels.

Creatinine greater than 1.5 times the upper limit of normal.

Platelet count, white blood cell count or hemoglobin below the lower limit of normal.

Other clinically significant medical disease that, in the judgment of the investigators, would expose the patient to undue risk of harm or prevent the patient from completing the study.

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Citations:

Rhodes LE, Freeman BK, Auh S, Kokkinis AD, La Pean A, Chen C, Lehky TJ, Shrader JA, Levy EW, Harris-Love M, Di Prospero NA, Fischbeck KH. Clinical features of spinal and bulbar muscular atrophy. Brain. 2009 Dec;132(Pt 12):3242-51.

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Contacts:

Principal Investigator	Referral Contact	For more information:
Kenneth H. Fischbeck, M.D. National Institute of Neurological Disorders and Stroke (NINDS) National Institutes of Health Building 35 Room 2A1000 35 Convent Drive Bethesda, Maryland 20892 (301) 435-9318 kf@ninds.nih.gov	Angela Kokkinis, R.N. National Institute of Neurological Disorders and Stroke (NINDS) National Institutes of Health Building 10 Room 5S-219 10 Center Drive Bethesda, Maryland 20892 (301) 451-8146 akokkinis@mail.cc.nih.gov	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 prpl@mail.cc.nih.gov

Clinical Trials Number:

NCT00303446

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