A study of plasminogen in subjects with spinal muscular atrophy
Not Applicable
Completed
- Conditions
- Spinal muscular atrophyNervous System Diseases
- Registration Number
- ISRCTN21188633
- Lead Sponsor
- Talengen Institute of Life Sciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 20
Inclusion Criteria
The subjects were diagnosed with type I SMA with SMN gene mutation or non-5q SMA with mutation in the gene encoding immunoglobulin-binding protein 2 (IGHMBP2), according to genetic tests and clinical symptoms
Exclusion Criteria
Patients receiving more than 16 hours of invasive ventilation per day
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Motor function scores measured using the CHOP INTEND scoring system at baseline and weeks 2, 6, 10, 22 and 46
- Secondary Outcome Measures
Name Time Method 1. Respiratory function measured by the value of blood oxygen saturation without Oxygen inhalation in pulse oximetry at baseline and weeks 2, 6, 10, 22 and 46<br>2. Anthropometric nutritional status measured using the proportion of high body weight, body fat, and growth parameters at baseline and weeks 10, 22 and 46<br>3. Adverse events assessed by routine blood test, blood biochemistry, coagulation function, hemolysis function, urine routine test, 12 lead ECG, physical examination, vital signs, etc measured using standard procedures at baseline, and weeks 22 and 46