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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	6 Years - 30 Years
Gender	All

More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. 6-30 years old TSC patients (by Gomez criteria) 2. more than 8 seizures occurred in the 4-week baseline time,with no continued seizure-free time of more than 10 days a month. 3. more than two antiepileptic drugs (AED) had been administered but fail to control the situation; maintaining with 1 or more than 1 AEDS for over 2 months and intending to continue with the drugs. 4. patients who had been treated with rapamycin should have been stopped for more than 3 months. 5. vagus nerve stimulation (VNS) is allowed as a previous or current therapy and would maintain until the end of the trial.

Exclusion Criteria:

1. Subependymal Giant Cell Astrocytoma and requires immediate surgery； 2. a history of intracranial surgery within 6 months; 3. epilepsy caused by improper use of drugs; 4. patients treated with aspirin had severe or intolerant side effects, including gastrointestinal ulcer, bleeding, aspirin allergy, and other conditions; 5. psychogenic seizures; 6. severe renal dysfunction and infection. 7. pregnant women and lactating women. 8. not regular follow-up. 9. other: because when children and adolescents suffering from influenza or chickenpox, using aspirin may cause a rare life-threatening Reye syndrome (characterized with persistent vomiting）, should temporary withdrawal, medication needs to consult a physician before using again.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT03356769
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 2
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Peking Union Medical College Hospital
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Qing Liu, MD PhD
Principal Investigator Affiliation	Peking Union Medical College Hospital
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other, Industry
Overall Status	Recruiting
Countries	China
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Tuberous Sclerosis Complex, Aspirin, Epilepsy, Cognitive Decline, Skin Lesions
Study Website:	View Trial Website

Additional Details

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.

Arms & Interventions

Arms

Experimental: experimental：asprin & AEDS

Aspirin 5mg/kg，maximum 300mg; once a day plus AEDS

Placebo Comparator: control: placebo & AEDS

placebo 5mg/kg，maximum 300mg; once a day plus AEDS

Interventions

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

Contact a Trial Team

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International Sites

Beijing, Beijing, China

Status

Recruiting

Address

Department of Neurology, Peking Union Medical College Hospital

Beijing, Beijing, 100005

Site Contact

Qing Liu, MD, PhD

[email protected]

86-10-13366305331

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