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

Kennedy's Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00851461
• Other study ID #: ID11-50-17
• Status: Completed
• Phase: Phase 4
• First received: February 25, 2009
• Last updated: May 20, 2015
• Start date: April 2008
• Est. completion date: November 2013

Study information

• Verified date: February 2009
• Source: Ramathibodi Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: Thailand: Ethical Committee
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: November 2013
• Est. primary completion date: November 2011
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 20 Years and older

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Atrophy
• Bulbo-Spinal Atrophy, X-Linked
• Kennedy's Disease
• Muscular Atrophy
• Muscular Disorders, Atrophic
• Spinobulbar Muscular Atrophy

Intervention

Drug:
• Goserelin
Goserelin 10.8 mg SC every 3 months

Procedure:
• Electrophysiologic study
Nerve conduction study Electromyogram
• tissue biopsy
skin and muscle biopsy before and after treatment every year

Locations

Country	Name	City	State
Thailand	Medical Genetics and Molecular Medicine Unit,, Department of Medicine, Ramathibodi Hospital	Bangkok

Sponsors (2)

Lead Sponsor	Collaborator
Ramathibodi Hospital	AstraZeneca

Country where clinical trial is conducted

Thailand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	muscle power		every 3 months	Yes
Secondary	electrophysiologic study (Nerve conduction and Electromyogram)		every 3 months	Yes