Exploratory clinical study of DF-521 in ALS (2)

JRCT ID: jRCTs031180059

Registered date:20/12/2018

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Amyotrophic Lateral Sclerosis
• Date of first enrollment: 16/10/2017
• Target sample size: 10
• Study type: Interventional
• Intervention(s): A single treatment cycle consisted of 6 weeks of DF-521 administration period followed by 2 weeks observation period. DF-521 is administered every other day for 6 weeks(18 times in total) in the administration periods

Outcome(s)

• Primary Outcome: The change in ALSFRS-R score
• Secondary Outcome: Changes of FVC, grip strength(left/right), pinch strength( left/right), and time to death or a specified state of disease progression

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: <= 75age old
• Gender: Both
• Include criteria: (1)Patients who are categorized as either definite or probable ALS according to the revised El Escorial criteria (2)Patients at Grade 3 in Japan ALS severity classification(3)Patients scoring >=1 points on each single ALSFRS-R item(4)Patients with normal respiratory function(%FVC>=60%)(5)Patients with ALS that occurred within 3 years at the time of giving written informed consent(6)Patients aged 20 to 75 years at the time of giving written informed consent(7)Patients who provided written informed consent to participate in the present study
• Exclude criteria: (1)Patients with reduced respiratory function and complaints of dyspnea.Patients scoring=<3 points on each single ALSFRS-R item(10.Dyspnea11.Orthopnea12.Respiratory insufficiency) (2)Patients with complications that may substantially influence evaluation of drug efficacy, such as Parkinson's disease schizophrenia, and dementia etc.(3)Patients who underwent a spine surgery after ALS onset or who are scheduled for a spine surgery during this study (cervical spondylosis, slipped disk etc.) (4)Patients with the syndromes which may be ALS mimic syndromes such as cervical spondylosis, multifocal motor neuropathy (5)Patients with hemorrhage (those with thrombocytopenic purpura, bleeding tendency due to angiopathy, hemophilia and other coagulopathy, operation, digestive tract ulcer, hemorrhage of urinary tract, suspected hemoptysis etc.) (6)Patients with hemorrhagic tendency (those with visceral tumor, gastrointestinal diverticulitis, colitis, subacute bacterial endocarditis, etc.) (7)Patients who had just undergone an operation (8)Patients with serious liver dysfunction (9)Patients with plateletcount<100,000 /microliter (10)Patients who are taking drugs which are fibrinolytic agents( t-PA and UK) or anticoagulant agents or anti-platelet agents (11)Patients with severe hypertension whose systolic blood pressure>=180 mmHg cannot be lowered>=150 mmHg by lowering blood pressure agents (12)Patients undergoing cancer treatment (13)Patients with a history of hypersensitivity to Edaravone if they are going to use Edaravone (14)Patients with a history of hypersensitivity to Batroxobin (15)Patients with severe illness(16)Male patients who do not consent to using reliable contraception methods during the studied period (17)Premenopausal women (18)Patients who has participated in another clinical trial twelve weeks prior to inclusion in this study(19)Patients with renal dysfunction with creatinine clearance of 50mL per min or below(20)Patients who are not eligible due to physician's judgment

Related Information

• Primary Sponsor: Yoshino Hiide
• Secondary Sponsor: Tobishi Pharmaceutical Co.,Ltd.
• Secondary ID(s): JapicCTI-173708