Periodic Paralysis
Conditions
Interventions
Control group:to give placebo, 30mg / time, 3 times / day
treatment group:to give Idebenone, 30mg / time, 3 times / day
Sponsors
Xuanwu Hospital, Capital University of Medical Sciences
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Genetically definite, clinically definite or clinically probable HypoKPP or HyperKPP as outlined in the protocol; 2. Male and female participants, age 18 and older who are able to comply with the study conditions; 3. Participants who have distinct regular episodes of weakness with an average frequency of > or = to 1 a week and < or = to 3 a day either on or off treatment, whichever is higher; 4. Normal thyroid-stimulating hormone (TSH) level and thyroxine(T4); 5. The patient or the patients legal representative have provided singed written informed consent.
Exclusion criteria
Exclusion criteria: 1.Evidence for Andersen-Tawil syndrome (any one of the following 3 criteria): (1) Prolonged QT interval or complex ventricular ectopy between attacks (2) Distinctive physical features (2 of the following 5); Low set ears; Short stature; HyperKPPo-/micrognathia; Clinodactyly; HyperKPPo-/HyperKPPertelorism; (3) KIR 2.1 gene mutation. 2. The patient has permanent weakness; 3. Allergy to Idebenone or intolerance to side effects; 3. Coincidental renal, hepatic, active thyroid disease, restrictive or obstructive lung disease, other neuromuscular disease, or heart disease; 4. Use of any of the following medications for reasons other than treatment of periodic paralysis: diuretics, antiarrhythmics, corticosteroids, beta-blockers, calcium channel blockers, antiepileptics, magnesium; 5. History of life-threatening episodes of respiratory muscle weakness or cardiac arrhythmias during attacks; 6. Pregnancy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Attack Rate; | — |
Secondary
| Measure | Time frame |
|---|---|
| Severity-weighted Attack Rate;Attack Duration;Endpoint of Acute Worsening; | — |
Countries
China
Contacts
Public ContactDa Yuwei
Xuanwu Hospital, Capital University of Medical Sciences
Outcome results
None listed