Duchene Muscular Dystrophy
Conditions
Interventions
Low dose group :primidone 25mg/d
High dose group :primidone 50mg/d
Placebo group:oral placebo
Sponsors
The first hospital of Yichang
Eligibility
Sex/Gender
Male
Age
7 Years to 13 Years
Inclusion criteria
Inclusion criteria: 1.Age between 7~13 years old. 2.Informed consent has been signed. 3.Participants should have a confirmed diagnosis of DMD by genetic or muscle biopsy and have a confirmed elevation of serum creatine kinase 15 to 30 times or more above the upper limit of normal (ULN) and show phenotypic signs of DMD. 4.Ability to comply with scheduled visits, medication schedules and laboratory-related tests. 5.Able to receive oral medication in tablets. 6.Participants should be able to complete the relevant items in the baseline test.
Exclusion criteria
Exclusion criteria: 1.Participation in another interventional clinical trial or exposure to another experimental drug within the first 6 months of participation in the project, except for observational cohort studies or non-interventional studies. 2.Participants received a stable dose of continuous corticosteroid therapy for 12 weeks or longer prior to initiation of dosing. 3.In the judgment of the investigator, participants had significant abnormal laboratory tests at the time of screening that could affect safety. 4.Participants with a medical history or current medical condition that, in the judgment of the investigator, may affect safety, including but not limited to: creatinine clearance <50 ml/min, porphyria and allergy to phenobarbital, history of depression, mental illness or brain dysfunction, asthma or other respiratory conditions that may aggravate dyspnea or airway obstruction, or cardiac disease. 5.Participants received treatment including prescription/over-the-counter medications that were known to be cytochrome P3A4 (CYP3A4) enzyme inhibitors or inducers 14 days prior to the first dose. 6.Coagulation disorders. 7.Albuminuria showed ++ and above. 8.Any injury that could affect functional testing, such as a fracture of an upper or lower extremity. 9.Subjects with a history of major surgery within 12 weeks prior to the start of study treatment or who are scheduled to have major surgery (e.g., scoliosis) during the study period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Creatine kinase;Plasma concentration of primidone; | — |
Secondary
| Measure | Time frame |
|---|---|
| 6 minutes walking test;North Star Ambulatory Assessment;Receptor interacting protein kinase 1;Receptor interacting protein kinase 3;IL-6;IL-8;AST;ALT;Lactate dehydrogenase;TNF-a;Human mixed lineage kinase domain like protein; | — |
Countries
China
Contacts
Public ContactYunhong Zha
The First Hospital of Yichang
Outcome results
None listed