spinocerebellar ataxia
Conditions
Interventions
Sponsors
The First People’s Hospital of Yichang, Yichang, China
Eligibility
Sex/Gender
Male
Age
18 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1. Male or female subjects aged 18 to 75 years; 2. Diagnosis or suspected diagnosis of the following specific hereditary ataxia: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10; i. The subjects should have genotype diagnosis determined by CLIA certified laboratory or equivalent National Laboratory (examination results can be provided, the sponsor should be consulted and the sponsor should confirm the equivalence of the laboratory); 3. Able to walk 8 meters without help (walking sticks and other appliances are allowed); 4. The total score of SARA was 3 or more; 5. The gait score is more than 1 points; 6. At baseline, based on the evaluation of medical history, physical examination, laboratory results and ECG, the investigator determined that the condition was stable. The subject must have sufficient physical strength and is expected to complete the test according to the design; 7. Have at least 6 years or equivalent educational experience, and be fluent in Chinese enough to complete the necessary scale filling tasks and understand the contents of the informed consent form; 8. The subjects must have sufficient hearing, visual acuity and language expression ability to carry out SARA assessment and other neuropsychiatric tests and interviews specified in the protocol; Adequate vision is defined as meeting the following two items: A. binocular vision >= 20 / 200 under the condition of best traditional correction (wearing conventional glasses or contact lenses); And, B. according to the judgment of PI, the subject can receive Sara evaluation without being significantly affected by visual impairment; 9. The subject must be able to understand and agree to abide by the specified administration protocol and procedures; Regularly participate in planned clinic visits; And reliably communicate with researchers on adverse events and drug combination; 10. Fertile women a. all sexually active and fertile women (WOCBP) must agree to use two contraceptive methods throughout the study period (i.e., from 30 days before baseline to 30 days after the last administration of the study drug): i. a barrier method (for example, uterine cap plus sperm killing agent, condom killing gel, intrauterine device, cervical cap); ii. And another way. Another method includes oral contraceptives or another barrier method; b. The serum pregnancy test must be negative at the screening visit of WOCBP, and the urine pregnancy test must be negative within 72 hours of administration. c. Sexually active men with WOCBP partners must agree to use both contraceptive methods throughout the study period (from the beginning of the first treatment to 90 days after the last administration of the study drug); 11. Voluntarily participate in the test and sign the informed consent form by himself or his legal guardian.
Exclusion criteria
Exclusion criteria: 1.Between enrollment and baseline, except for a hereditary ataxia specified in the inclusion criteria, Any other medical condition that can significantly explain or significantly cause the subject's symptoms of ataxia (e.g., alcoholism, vitamin deficiency, multiple sclerosis, vascular disease, tumor, paraneoplastic disease, head injury, idiopathic delayed ataxia, multisystem atrophy), Or other medical conditions that may interfere with the assessment of ataxia symptoms (e.g., stroke, arthritis); 2.MMSE score < 24; 3.According to the judgment of the investigator, the subject's obvious spastic state or dystonia can affect the ability of SARA tool to evaluate the severity of potential ataxia; 4.The subject may not have started physiotherapy or occupational therapy within 1 month before screening, and it is expected that such therapy will not be started during the treatment period. For subjects who are receiving occupational or physical therapy, if the treatment intensity starts from 2 months before screening and remains unchanged throughout the treatment period, they can continue to receive such treatment; 5.During screening or baseline examination, if there are changes in medical status or disease status that may affect the accuracy of SARA, subjects should be excluded; 6.Had or was suffering from malignant tumor within 5 years before screening; Have a history of chronic infection, serious infection in recent 2 months or opportunistic infection in recent 6 months; Current active hepatitis or history of hepatitis C; HIV infected persons; A history of lymphoproliferative diseases such as lymphoma; 7.Multiple sclerosis or other central nervous demyelinating diseases; 8.Have or are suffering from congestive heart failure; Serious, progressive and uncontrolled diseases of liver, kidney, blood, gastrointestinal tract, endocrine, heart, lung, nerve and brain; 9.Protein purified derivative (PPD) positive skin test or (and) CT showed signs of pulmonary infection or close contacts with TB patients; 10.Those who participated in other drug observation within 4 weeks; 11.Patients who are allergic to rapamycin and its derivatives or any component of this drug, pregnant women and lactating women; 12.Patients who participate in another clinical trial that may affect the purpose of this study at the same time or within 1 month; 13.The researchers believe that other reasons are not suitable for clinical trials.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| SARA;Beck Depression Inventory;MMSE;SF-36; | — |
Countries
China
Contacts
Public ContactZhao Hailong
The First People’s Hospital of Yichang, Yichang, China
Outcome results
None listed