Spinal and Bulbar muscular atrophy
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 administratio
Sponsors
JASMITT Clinical Trial Office
Jichi med college, Chiba univ., Univ of Tokyo, Tokyo med & dental univ.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: (1) Male patients who present one or more of the following clinical symptoms attributed to lower motor neuron impairment, and were given a definitive diagnosis by a genetic testing or have not received any genetic testing but were clinically diagnosed with SBMA 1) Muscle weakness of extremities 2) Bulbar palsy (2) Patients whose screening test (genetic testing) results showed that they bear at least 38 of CAG repeats within the AR genes. (3) Patients who are thirty (>=30) to seventy (<70) years of age at the time of informed consent. (4) Patients who do not desire children. (5) Patients whose hepatic and renal functions meet the following criteria: 1) AST(GOT): <=4.0xUpper Limit of Institutional Reference Value 2) ALT(GPT): <=4.0xUpper Limit of Institutional Reference Value 3) Creatinine: <1.5xUpper Limit of Institutional Reference Value (6) Patients who can keep standing for six minutes without any assistance (Walking aids can be used.) (7) Patients who can undergo admission assessment when study drug administration is perfomed initially (Week 0), Week 48 (last administration) or upon withdrawal. (8) Patients who can visit hospital regularly as an outpatient. (9) Patients who gave written informed consent by themselves.
Exclusion criteria
Exclusion criteria: (1) Patients who received any of the following twelve months (48 weeks) before giving informed consent: LH-RH agonists, testosterone preparations, 5-alpha reductase inhibitors, anti-androgen preparations, anabolic steroids, progesterone preparations, or unapproved drugs. (2) Patients who have received LH-RH agonists if they will be enrolled in this trial as previously treated cases. Patients who have received LH-RH agonists over thirty six months (144 weeks) in total if they will be enrolled in this trial as untreated cases. (3) Patients who have received a surgical treatment (such as castration) that would cause a decrease in the serum testosterone level. (4) Patients who may not be able to undergo scrotal skin biopsy (e.g. those with scrotal skin disease, those who are susceptible to haemorrhage or allergic to xylocaine). (5) Patients who have no swallowing dysfunction by any of following evaluations. (Index for swallowing function, QOL evaluation for swallowing function, and evalution of swallowing function in videofluorography) (6) Patients who may not be able to undergo videofluorography (e.g. those with severe bulbar palsy causing recurrent aspiration pneumoniae, those with other neurological disorders accompanied by bulbar palsy or pseudobulbar palsy, or those who are allergic to barium sulfate). (7) Patients who are diagnosed with suspected depression by the Mini-International Neuropsychiatric Interview (M.I.N.I. Japanese version 5.0.0 Major Depression Episode) (8) Patients who developed serious complication (such as those corresponding to Grade 3 Episode) (9) Patients who have experienced angina pectoris or myocaridal infarction within one month before giving informed consent. (10) Patients who meet any of the following: Pulse of over 120 beats per minute, systolic blood pressure of over 180mmHg, diastolic blood pressure of over 100mmHg.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Evaluation of dysphagia (pharyngeal barium residue after initial swallowing) (Evaluation index: change between the start and the end of medication) | — |
Secondary
| Measure | Time frame |
|---|---|
| (1) Index for swallowing function (SDQ-J) (2) Videofluorographical parameters of dysphagia: pharyngeal barium residue after initial swallowing (average of three times of swallowing); pharyngeal barium residue after piecemeal deglutition; oral barium residue; swallowing times; and laryngeal penetration aspiration scale. (3) QOL evaluation (SWAL-QOL [Japanese version], ALSAQ-5 [Japanese version]) (4) Indexes for motor functions (ALSFRS-R [Japanese version], SBMAFRS, QMG score) (5) Ratio of 1C2-positive cells in the scrotal skin (6) Serum CK levels at hospitalization (7) Urinary 8-OHdG | — |
Countries
Japan
Contacts
Public ContactAtsushi Hashizume
Nagoya University Graduate School of Medicine
Outcome results
None listed