Spinal and Bulbar Muscular Atrophy (SBMA)
Conditions
Interventions
Intervention type:DRUG
Name of intervention:TAP-144-SR(3M)
Dose form / Japanese Medical Device Nomenclature:INJECTION
Route of administration / Site of application:SUBCUTANEOUS
Dose per administration
Sponsors
JASMITT Clinical Trial Office
Hokkaido University, Tohoku University, Jichi Medical College, Chiba University, University of Tokyo, Tokyo Medical and Dental University, NHO Sagamitara hospital, Niigata University, Hamamatsu Medical college, Kobe University, Tottori University, Nagasaki University, Kumamoto University
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1) Clinically and/or genetically confirmed SBMA male patients in Japan who have more than one symptoms including muscle weakness, muscle atrophy, bulbar palsy and hand tremor. 2) Patients whose repeat number of CAG in androgen receptor gene is more than 38 in screening gene analysis. 3) Patients who are age 30 to 70 year-old at the time of informed consent. 4) Patients who have no desire to father a child. 5) Patients whose liver function test and renal function test are within below limits. AST (GOT) smaller than 4.0 X upper limit of normal ALT (GPT) smaller than 4.0 X upper limit of normal Creatinine smaller than 1.5 X upper limit of normal 6) Patients who are capable of standing for 6 min with or without cane. 7) Patients who are capable of undergoing in hospital examinations at 0, 24th, 48th week and the time of quitting the trial. 8) Patients who are capable of ambulatory hospital visits. 9) Patients with written informed consent.
Exclusion criteria
Exclusion criteria: 1) Patients who have taken LH-RH agonists, testosterone drugs, 5-alpha-reductase inhibitors, anti-androgen drugs or estrogen drugs within 48 weeks prior to the informed consent. 2) Patients who have taken LH-RH agonists for more than 48 weeks prior to the informed consent. 3) Patients who have undergone operations (eg. orchiectomy), which reduce serum testosterone levels. 4) Patients who are unable to undergo skin biopsies. 5) Patients who are unable to undergo videofluorography. 6) Patients who are diagnosed being depressed by M.I.N.I. Japanese version 5.0.0 major depression episode. 7) Patients who have severe complications. 8) Patients who have suffered angina pectoris or myocardial infarction within one month prior to the informed consent. 9) Heart rate > 120 bpm, systolic BP > 180mmHg or diastolic BP > 100 mmHg. 10) Patients with past medical history of allergy to leuprorelin acetate, synthetic LH-RH or LH-RH delivatives. 11) Patients who have participated in other clinical trials within 12 weeks prior to the informed consent. 12) Patients who are not appropriate to participate to the trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Pharyngeal barium residue (%) in videofluorography | — |
Secondary
| Measure | Time frame |
|---|---|
| Frequency of anti-poly Q staining in Scrotal Skin Clinical scores (ALSFRS-R, QMG Score, 6-minute walk) QOL (ALSAQ-5) serum creatine kinase (CK) | — |
Countries
Japan
Contacts
Public ContactHaruhiko Banno
Nagoya University Graduate School of Medicine
Outcome results
None listed