SCA3/MJD
Conditions
Interventions
Test group:DL-3-n-butylphthalide
Control group:Placebo
Sponsors
Xiangya Hospital of Central South University
Eligibility
Sex/Gender
All
Age
10 Years to 60 Years
Inclusion criteria
Inclusion criteria: 1. Gene test found that ATXN3 gene CAG repeat amplification mutations in SCA3 / MJD patients; 2. Aged 18-60 years old; 3. Gait sub-score of SARA score ranged 2-5 points (including the boundary value); patients who can complete the 8MW test; 4. Willing to participate in the various stages of clinical trials, compliance is good; 5. At the research stage, women of childbearing age agreed to adopt two feasible methods to implement family planning; 6. selected a month before taking no formaldehyde, Chinese medicine ; 7. understanding and sign informed consent, willing to cooperate with the researchers to complete the relevant treatment, examination, follow-up.
Exclusion criteria
Exclusion criteria: 1) Health check does not meet the subject's criteria; 2) Pregnant women or lactating women,or women who are scheduled to be pregnant; 3) Had fever in the past three days; 4) Had participated in other drug clinical trials in the past three months; 5) Personal history or family history of jaundice by drug; 6) Complicated with malignant tumors or ongoing anti-tumor therapy; 7) Suffering from severe hypertension can not control: systolic blood pressure = 200mmHg (26.6kPa) or diastolic blood pressure = 110mmHg (14.6kPa); 8) Diseases with hemorrhagic predisposition; leukocytes and / or thrombocytopenia; 9) Hepatitis B virus infection; liver dysfunction (transaminase over 1.5 times the upper limit of normal), renal dysfunction (Cre> 2.0mg/dl or 177umol/L), heart dysfunction or other important organ dysfunction; 10) Suspected or indeed alcohol, drug abuse history; 11) Previous history of allergies to celery or DL-3-n-butylphthalide; 12) Suffering from other diseases and signs other than the symptoms and signs caused by SCA3 / MJD obvious symptoms of disease, is the application of prevention or treatment of drugs; 13) Researchers believe that there are other circumstances that are not appropriate for this clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| SARA score;SCAFI;MRS; | — |
Secondary
| Measure | Time frame |
|---|---|
| INAS score;Barthel score;UHDRS-IV;EQ-5D;PHQ-9;GAD-7;MoCA;9HPT;8MW;PATA; | — |
Countries
China
Contacts
Public ContactHong Jiang
Xiangya Hospital of Central South University
Outcome results
None listed