Duchenne Muscular Dystrophy, DMD
Conditions
Interventions
Control group:Placebo was administered during months 1 through 6, and yeast beta-glucan compensation was administered during months 7 through 12. The dose was 40 mg/kg/day, administered orally continu
Experimental group:Yeast ß-glucan was administered from 1 to 6 months and continued from 7 to 12 months. The dose was 40 mg/kg/day, administered orally continuously.
Sponsors
Chinese PLA General Hospital
Eligibility
Sex/Gender
Male
Age
5 Years to 8 Years
Inclusion criteria
Inclusion criteria: 1. Male pediatric patients with a confirmed diagnosis of Duchenne muscular dystrophy (DMD) based on clinical and genetic testing. 2. Aged 5 to 8 years. 3. Able to ambulate independently, with a 6-minute walk distance (6MWD) between 300 and 450 meters. 4. Able to cooperate with motor function assessments (including 10-meter walk time, time to stand from supine [TTSTAND], North Star Ambulatory Assessment [NSAA] score, etc.) and hematological, biochemical, and immunological tests. 5. Has been on a stable standard of care (e.g., glucocorticoids and routine rehabilitation management) for at least 3 months prior to enrolment, with no major changes to the treatment regimen planned in the near future. 6. Able to undergo a muscle biopsy under anesthesia without contraindications. (If the biopsy is required for mechanistic exploration, this must be clearly stated in the informed consent; if it is an optional substudy, a separate biopsy-specific consent should be obtained.) 7. The guardian agrees to the child’s participation and signs the informed consent form. (If the child has sufficient comprehension, they may sign the informed consent or an assent form as appropriate.)
Exclusion criteria
Exclusion criteria: 1. Patients with severe heart disease (such as clinically unstable heart failure, severe arrhythmia, etc.), liver function or renal dysfunction, who are judged by the investigator to be ineligible to participate; 2. Known allergic or previous severe allergic reaction to granule yeast ß-1,3/1,6-glucan or preparation excipients/packaging ingredients; 3. HIV positive or have active hepatitis B/C infection (evidence of HBV/HCV activity); or syphilis screening positive and without standardized evaluation/treatment (TPPA, etc., should be combined with clinical judgment); 4. Presence of malignant tumor or high suspicion of tumor (if abnormal liver function needs to be further excluded, enrollment can be delayed until the exclusion is clear); 5. Severe psychiatric/behavioral problems or significant cognitive impairment that may result in inability to cooperate with critical assessments; 6. Not suitable for muscle biopsy due to coagulopathy or other reasons (biopsy is not necessary); 7. Uncontrolled hypertension or other circulatory conditions with safety implications (determined by the investigator in combination with age/height percentile and clinical criteria for children); 8. Other serious systemic or organic diseases that may affect safety or outcome interpretation; 9. Prior to enrollment, patients had received gene or cell therapy for Duchenne muscular dystrophy or had received an investigational intervention that could affect the interpretation of the study outcome; 10. Fracture, claudication and other factors affecting the functional outcome score at admission; 11. Recent severe infection; 12. Other conditions deemed unsuitable for inclusion by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time to stand from supine, TTSTAND; | — |
Secondary
| Measure | Time frame |
|---|---|
| 6-minute walking distance,6WMD;10-meter walk time;North Star Ambulatory Assessment score;Electrocardiogram;Echocardiogram;Pulmonary function test; | — |
Countries
China
Contacts
Public ContactShiwen Wu
Chinese PLA General Hospital
Outcome results
None listed