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

Phase 4
Completed
Conditions
Spinobulbar Muscular Atrophy
Kennedy's Disease
Registration Number
NCT00851461
Lead Sponsor
Ramathibodi Hospital
Brief Summary

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

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
Male
Target Recruitment
10
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 & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Primary Outcome Measures
NameTimeMethod
muscle powerevery 3 months
Secondary Outcome Measures
NameTimeMethod
electrophysiologic study (Nerve conduction and Electromyogram)every 3 months

Trial Locations

Locations (1)

Medical Genetics and Molecular Medicine Unit,, Department of Medicine, Ramathibodi Hospital

🇹🇭

Bangkok, Thailand

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