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

Mahidol University

Status and phase: Completed; Phase 4

Conditions: Kennedy's Disease; Spinobulbar Muscular Atrophy

Treatments: Procedure: Electrophysiologic study; Drug: Goserelin; Procedure: tissue biopsy

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00851461

ID11-50-17

Details and patient eligibility

About

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

Enrollment: 10 patients

Sex: Male

Ages: 20+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)