NCT03356769

Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis Complex

A Placebo-controlled Study of Efficacy & Safety of Aspirin as an add-on Treatment in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Seizures

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 98 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 6 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

There had been much evidence in aspirin controlling tumorous conditions conducted by basic researches, especially through mammilian target of rapamycin (mTOR) pathway. The investigator observed efficacy of aspirin in the treatment of tuberous sclerosis complex (TSC) in one child who got Kawasaki disease and in the addition four TSC patients with epilepsy. The investigator intend to evaluate whether aspirin would be an effective add-on treatment in TSC patients with refractory seizures.

Detailed description

There is no optional treatment for patients with tuberous sclerosis complex (TSC) and refractory epilepsy.The investigator observed efficacy of aspirin in the treatment of in one child who got Kawasaki disease. Subsequent adjunctive aspirin therapy in four patients yielded a reducted frequency of seizure for 51.2-89.7%. The investigator intend to evaluate whether aspirin would be an effective add-on treatment in TSC patients with refractory seizures. Refractory epilepsy was defined as more than 8 times of epileptic events in 4 weeks at baseline, and had been given more than two antiepileptic drugs maintaining for more than 3 months.TSC patients aged 6-30 years' old would be recruited with refractory seizures and randomly assigned to two groups, aspirin and antiepileptic drugs(AEDS) group and placebo-AEDS group after written informed consent be obtained. Patients and their guardians would be instructed to record their own seizure diary on the epileptic events and report monthly.The primary outcome would be reduction of seizure frequency (measured by average seizure frequency and response rate). The secondary outcome would include seizure-free days, seizure-free rates, changes in EEG, changes of facial angiofibromas, and exposure-response relationship analysis.The study is designed as a placebo-controlled, randomized, blinded evaluation trial.

Conditions

• Tuberous Sclerosis Complex
• Aspirin
• Epilepsy
• Cognitive Decline
• Skin Lesions

Interventions

Type	Name	Description
DRUG	Aspirin	low-dose of aspirin, 5mg/Kg/d, once every day, 25mg per tablets
DRUG	AED	maintain the dosages and the drugs throughout the 3-month observation time
DRUG	Placebo	placebo, 5mg/Kg/d, once every day, 25mg per tablets

Timeline

• Start date: 2017-11-20
• Primary completion: 2021-11-20
• Completion: 2021-11-20
• First posted: 2017-11-29
• Last updated: 2020-06-09

Locations

1 site across 1 country: China