NCT00851461

Brief Summary

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

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Apr 2008

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2008

Completed
11 months until next milestone

First Submitted

Initial submission to the registry

February 25, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 26, 2009

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2011

Completed
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2013

Completed
Last Updated

May 21, 2015

Status Verified

February 1, 2009

Enrollment Period

3.6 years

First QC Date

February 25, 2009

Last Update Submit

May 20, 2015

Conditions

Spinobulbar Muscular AtrophyKennedy's Disease

Keywords

Spinobulbar muscular atrophyKennedy's diseaseGoserelinElectrophysiologic studyInclusion body

Outcome Measures

Primary Outcomes (1)

  • muscle power

    every 3 months

Secondary Outcomes (1)

  • electrophysiologic study (Nerve conduction and Electromyogram)

    every 3 months

Interventions

GoserelinDRUG

Goserelin 10.8 mg SC every 3 months

Also known as: Zoladex LA
Electrophysiologic studyPROCEDURE

Nerve conduction study Electromyogram

Also known as: NCV, EMG
tissue biopsyPROCEDURE

skin and muscle biopsy before and after treatment every year

Eligibility Criteria

Age20 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

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

Bangkok, Bangkok, 10400, Thailand

Location

MeSH Terms

Conditions

Bulbo-Spinal Atrophy, X-Linked

Interventions

Goserelin

Condition Hierarchy (Ancestors)

Muscular Atrophy, SpinalSpinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesMotor Neuron DiseaseNeuromuscular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Gonadotropin-Releasing HormonePituitary Hormone-Releasing HormonesHypothalamic HormonesPeptide HormonesHormonesHormones, Hormone Substitutes, and Hormone AntagonistsNeuropeptidesPeptidesAmino Acids, Peptides, and ProteinsOligopeptidesNerve Tissue ProteinsProteins

Study Officials

  • Thanyachau Sura, M.D., MRCP

    Ramathibodi Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

February 25, 2009

First Posted

February 26, 2009

Study Start

April 1, 2008

Primary Completion

November 1, 2011

Study Completion

November 1, 2013

Last Updated

May 21, 2015

Record last verified: 2009-02

Locations

TH(1)