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

Ramathibodi Hospital10 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Spinobulbar Muscular Atrophy Kennedy's Disease

Interventions

GoserelinDRUG

Goserelin 10.8 mg SC every 3 months

Electrophysiologic studyPROCEDURE

Nerve conduction study Electromyogram

tissue biopsyPROCEDURE

skin and muscle biopsy before and after treatment every year

Eligibility

Sex: MALEMin age: 20 Years

Medical Language ↔ Plain English

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

Locations (1)

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

Bangkok, Bangkok, Thailand

Outcomes

Primary Outcomes

muscle power

Time frame: every 3 months

Secondary Outcomes

electrophysiologic study (Nerve conduction and Electromyogram)

Time frame: every 3 months

Data from ClinicalTrials.gov

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