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

Primary Purpose

Spinobulbar Muscular Atrophy, Kennedy's Disease

Status
Completed
Phase
Phase 4
Locations
Thailand
Study Type
Interventional
Intervention
Goserelin
Electrophysiologic study
tissue biopsy
Sponsored by
Ramathibodi Hospital
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinobulbar Muscular Atrophy focused on measuring Spinobulbar muscular atrophy, Kennedy's disease, Goserelin, Electrophysiologic study, Inclusion body

Eligibility Criteria

20 Years - undefined (Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

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

Outcomes

Primary Outcome Measures

muscle power

Secondary Outcome Measures

electrophysiologic study (Nerve conduction and Electromyogram)

Full Information

First Posted
February 25, 2009
Last Updated
May 20, 2015
Sponsor
Ramathibodi Hospital
Collaborators
AstraZeneca
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1. Study Identification

Unique Protocol Identification Number
NCT00851461
Brief Title
Effect of Goserelin (Zoladex®) in Spinal and Bulbar Muscular Atrophy
Official Title
Effect of Goserelin (Zoladex®) in Spinal and Bulbar Muscular Atrophy in Thai Patients
Study Type
Interventional

2. Study Status

Record Verification Date
February 2009
Overall Recruitment Status
Completed
Study Start Date
April 2008 (undefined)
Primary Completion Date
November 2011 (Actual)
Study Completion Date
November 2013 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Ramathibodi Hospital
Collaborators
AstraZeneca

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This is a therapeutic trial study to demonstrate whether Goserelin, a LHRH agonist has benefit in SBMA Objective: To study effects of Goserelin to clinical course of patients with spinal and bulbar muscular atrophy in Thailand To demonstrate physiological and pathological changes in treated patients with Goserelin. To assess tolerability and adverse effect of Goserelin therapy

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinobulbar Muscular Atrophy, Kennedy's Disease
Keywords
Spinobulbar muscular atrophy, Kennedy's disease, Goserelin, Electrophysiologic study, Inclusion body

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Goserelin
Other Intervention Name(s)
Zoladex LA
Intervention Description
Goserelin 10.8 mg SC every 3 months
Intervention Type
Procedure
Intervention Name(s)
Electrophysiologic study
Other Intervention Name(s)
NCV, EMG
Intervention Description
Nerve conduction study Electromyogram
Intervention Type
Procedure
Intervention Name(s)
tissue biopsy
Intervention Description
skin and muscle biopsy before and after treatment every year
Primary Outcome Measure Information:
Title
muscle power
Time Frame
every 3 months
Secondary Outcome Measure Information:
Title
electrophysiologic study (Nerve conduction and Electromyogram)
Time Frame
every 3 months

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Provision of informed consent Males, age over 20 years, undesired fertility 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 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: Have weakness caused by other etiologies and have liver or kidney disease. Have currently prostatic cancer Want to have a child Participation in a clinical study during the last 30 days. Females and children age < 20 years old
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Thanyachau Sura, M.D., MRCP
Organizational Affiliation
Ramathibodi Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Medical Genetics and Molecular Medicine Unit,, Department of Medicine, Ramathibodi Hospital
City
Bangkok
ZIP/Postal Code
10400
Country
Thailand

12. IPD Sharing Statement

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

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