spinal muscular atrophy
Conditions
Interventions
experimental group: salbutamol sulfate tablets
control group:placebo
Sponsors
The Children's Hospital, Zhejiang University School of Medicine
Eligibility
Sex/Gender
All
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: Participants should meet all the following selection criteria to be included in this study: (1) Males and females between 2 and 17 years old (including boundary values); (2) The diagnosis of 5q SMA is clear: genetic testing verifies that there are homozygous deletions or heterozygous mutations that cause loss of SMN1 gene function; (3) There are clinical symptoms attributable to type II or type III SMA: proximal generalized muscle weakness, hypotonia, muscle atrophy and/or tongue muscle atrophy, tremor, dyspnea, scoliosis, poor digestion and other symptoms. (4) The subjects legal guardian has understood and complied with the research process, and can communicate well with the researcher. The subject voluntarily participates in the trial and signs an informed consent form (children aged >= 8 years old and able to express their wishes independently should also sign the informed consent form).
Exclusion criteria
Exclusion criteria: Those who have any of the following cannot be included in this study: (1) Those who cannot comply with the research requirements; (2) Those who has used or concomitantly used drugs that can interfere with the efficacy of salbutamol sulfate; (3) Those suffering from cardiovascular diseases that the researchers believe are of clinical significance; (4) Poor blood pressure control (sitting systolic blood pressure >= 160 mmHg or = 100 mmHg or <= 60 mmHg); (5) Those who are allergic to two or more drugs or foods, or those who are known to be allergic to test drugs (including the ingredients of their prescriptions); (6) Those who have currently or previously received albuterol/SMN protein-enhancing disease-modifying drug therapy or who have participated in drug clinical trials within 3 months before screening; (7) Concomitant diseases, physical conditions, or treatments for concomitant diseases may interfere with the results of the test, or in the eyes of the investigator, the risk of patients participating in this study is greater than the beneficiaries; (8) Other patients who have not been clearly specified but the investigator considers not suitable to participate in this clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Motor function assessment; | — |
Secondary
| Measure | Time frame |
|---|---|
| Lung function;Nutritional assessment; | — |
Countries
China
Contacts
Public ContactMao Shanshan
The Children's Hospital, Zhejiang University School of Medicine
Outcome results
None listed