Effect of Goserelin (Zoladex®) in Spinal and Bulbar Muscular Atrophy

Effect of Goserelin (Zoladex®) in Spinal and Bulbar Muscular Atrophy in Thai Patients

This is a therapeutic trial study to demonstrate whether Goserelin, a LHRH agonist has benefit in SBMA

Objective:

1. To study effects of Goserelin to clinical course of patients with spinal and bulbar muscular atrophy in Thailand
2. To demonstrate physiological and pathological changes in treated patients with Goserelin.
3. To assess tolerability and adverse effect of Goserelin therapy

Study Overview

• Status: Completed
• Conditions: Spinobulbar Muscular Atrophy; Kennedy's Disease
• Intervention / Treatment: Drug: Goserelin; Procedure: Electrophysiologic study; Procedure: tissue biopsy

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 4

Contacts and Locations

Study Locations

• Thailand
  • Bangkok, Thailand, 10400
    • Medical Genetics and Molecular Medicine Unit,, Department of Medicine, Ramathibodi Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

1. Provision of informed consent
2. Males, age over 20 years, undesired fertility

3. Have positive family history of muscle weakness with these clinical signs:

   • bulbar involvement (difficulty in breathing, swallowing, talking)
   • muscle atrophy with or without fasciculation
   • decrease or normal deep tendon reflex
   • normal Babinski response
   • no sensory impairment
   • mild tremor (either postural or intention)
   • gynecomastia
   • decrease libido and infertility
4. Subjects have a confirmed diagnosis to SBMA by both NCV test and DNA study demonstrated the number of CAG repeated more than 35.

Exclusion Criteria:

1. Have weakness caused by other etiologies and have liver or kidney disease.
2. Have currently prostatic cancer
3. Want to have a child
4. Participation in a clinical study during the last 30 days.
5. Females and children age < 20 years old

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: SINGLE_GROUP
• Masking: NONE

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
muscle power	every 3 months

Secondary Outcome Measures

Outcome Measure	Time Frame
electrophysiologic study (Nerve conduction and Electromyogram)	every 3 months

Collaborators and Investigators

• Sponsor: Ramathibodi Hospital
• Collaborators: AstraZeneca
• Investigators: Principal Investigator: Thanyachau Sura, M.D., MRCP, Ramathibodi Hospital

Study record dates

Study Major Dates

• Study Start: April 1, 2008
• Primary Completion (ACTUAL): November 1, 2011
• Study Completion (ACTUAL): November 1, 2013

Study Registration Dates

• First Submitted: February 25, 2009
• First Submitted That Met QC Criteria: February 25, 2009
• First Posted (ESTIMATE): February 26, 2009

Study Record Updates

• Last Update Posted (ESTIMATE): May 21, 2015
• Last Update Submitted That Met QC Criteria: May 20, 2015
• Last Verified: February 1, 2009

More Information

Terms related to this study

• Keywords: Goserelin; Kennedy's disease; Spinobulbar muscular atrophy; Electrophysiologic study; Inclusion body
• Additional Relevant MeSH Terms: 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; Heredodegenerative Disorders, Nervous System; Motor Neuron Disease; Muscular Atrophy, Spinal; Muscular Atrophy; Atrophy; Bulbo-Spinal Atrophy, X-Linked; Antineoplastic Agents; Antineoplastic Agents, Hormonal; Goserelin
• Other Study ID Numbers: ID11-50-17