Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease

OBJECTIVES:

I. Evaluate the effects of androgen suppression with leuprolide and androgen replacement with testosterone enanthate on muscle strength in men with Kennedy's disease or other motor neuron disease.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis; Spinal Muscular Atrophy; Spinobulbar Muscular Atrophy
• Intervention / Treatment: Drug: testosterone; Drug: leuprolide

Detailed Description

PROTOCOL OUTLINE:

All patients receive androgen suppression with leuprolide acetate injections every 4 weeks for 6 months, plus hormone replacement therapy with testosterone enanthate injections every week for 24 weeks.

• Study Type: Interventional
• Enrollment: 40
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Men aged 18 and over with motor neuron disease, i.e.:

• X-linked spinal and bulbar muscular atrophy (Kennedy's disease)
• Confirmed by androgen receptor, exon-1 mutation genotype
• Amyotrophic lateral sclerosis
• Spinal muscular atrophy

Significant muscle weakness on manual muscle testing

No prisoners

No mental disability

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment

Collaborators and Investigators

• Sponsor: National Center for Research Resources (NCRR)
• Collaborators: Ohio State University
• Investigators: Study Chair: Jerry R. Mendell, Ohio State University

Study record dates

Study Major Dates

• Study Start: October 1, 1992
• Primary Completion: December 7, 2022
• Study Completion: December 7, 2022

Study Registration Dates

• First Submitted: February 24, 2000
• First Submitted That Met QC Criteria: February 24, 2000
• First Posted (Estimate): February 25, 2000

Study Record Updates

• Last Update Posted (Estimate): June 24, 2005
• Last Update Submitted That Met QC Criteria: June 23, 2005
• Last Verified: December 1, 2001

More Information

Terms related to this study

• Keywords: neurologic and psychiatric disorders; rare disease; amyotrophic lateral sclerosis; motor neuron disease; spinal and bulbar muscular atrophy; spinal muscular atrophy; Kennedy's Syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Heredodegenerative Disorders, Nervous System; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal; Bulbo-Spinal Atrophy, X-Linked; Physiological Effects of Drugs; Antineoplastic Agents; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Reproductive Control Agents; Fertility Agents, Female; Fertility Agents; Androgens; Leuprolide; Testosterone
• Other Study ID Numbers: 199/11792; OSU-92H0325

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No