EUCTR2011-000860-90-GR进行中（未招募）1 期
A three-arm, randomized, double-blind, placebo-controlled study of the efficacy and safety of two trough-ranges of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC) who have refractory partial-onset seizures
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 345
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Male or female between the ages of 2 and 65 years (except in Europe where the minimum age will be 1 at the request of the EMA).
- •2. Clinically definite diagnosis of TSC per modified Gomez criteria
- •3. Diagnosis of partial-onset epilepsy according to the classification of the International League Against Epilepsy (1989) and revised in 2009.
- •4. Uncontrolled partial-onset seizures; must meet the following:
- •a. At least 16 reported quantifiable partial-onset seizures over the Baseline period with no continuous 21-day seizure-free period between Visit 1 (Screening Visit) and Visit 2 (Randomization visit), as per data captured in daily seizure diaries.
- •b. Prior history of failure to control partial-onset seizures despite having been treated with two or more sequential regimens of single or combined antiepileptic drugs.
- •c. Prior or concurrent use of vagal nerve stimulator (VNS) is allowed. If the patient is using VNS, device stimulator parameters must remain constant throughout the study.
- •d. Prior epilepsy surgery is allowed if performed at least 12 months before study entry.
- •5. Must be receiving one, two, or three AEDs at a stable dose for at least 4 weeks at the start of the 8-week prospective Baseline phase, remain on the same regimen throughout the Baseline phase, and intend to continue the same regimen throughout the 18-week double blind Core phase (rescue medications are permitted).
- •6. If female of child bearing potential, documentation of negative pregnancy test at time of informed consent and must use highly effective contraception during the study and for 8 weeks after stopping treatment
- •7. Sexually active males must use a condom during intercourse while taking study drug, and for 8 weeks after stopping study treatment
- •8. Hepatic, renal and blood laboratory values within the following range at both screening and randomization:
- •a. AST and ALT levels < 2.5 x ULN
- •b. serum bilirubin <1.5 × ULN (this limit does not apply to patients with an elevated indirect bilirubin, if they have Gilbert’s Syndrome)
- •c. serum creatinine < 1.5 x ULN
- •d. hemoglobin = 9 g/dL
- •e. platelets = 80,000/mm3
- •f. absolute neutrophil count = 1,000/mm3
- •9. Written informed consent. Subjects or their legal guardians must have the ability to comprehend the informed consent form and be willing to provide informed consent.
- •10. Patient or caregiver must be able to reliably record seizures and keep a daily diary and recall adverse events.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 120
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 205
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 20
排除标准
- •1. Patients with seizures secondary to metabolic, toxic, infectious or psychogenic disorder or drug abuse or current seizures related to an acute medical illness
- •2. Presence of only non-motor partial seizures
- •3. Patients with TSC who have SEGA in need of immediate surgical intervention
- •4. Patients who suffer from active infantile spasms
- •5. Within 52 weeks prior to study entry, an episode of status epilepticus as defined in the protocol
- •6. Patients with history of seizure clusters (where individual seizures cannot be accurately counted
- •according to the judgment of the investigator) occurring within 26 weeks prior to study entry
- •7. Patients who require rescue medication during the Baseline phase for more than 6 days
- •8. Patients with non-TSC related progressive encephalopathy
- •9. Patients who weigh less than 12 kg
- •10. Patients with coexisting malignancies within the 3 years prior to randomization, except for adequately treated carcinoma of the cervix or basal or squamous cell carcinomas of the skin.
- •11. Patients with any severe and/or uncontrolled medical conditions at randomization such as:
- •a. Symptomatic congestive heart failure of New York Heart Association Class III or IV, history of left ventricular ejection fraction (LVEF) <50%, QTc interval >460ms, congenital QT syndrome, unstable angina pectoris, myocardial infarction within 6 months of study entry, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease
- •b. Significant symptomatic deterioration of lung function
- •c. Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of everolimus (e.g., ulcerative disease, malabsorption syndrome or small bowel resection)
- •d. liver disease such as cirrhosis, decompensated liver disease, and chronic hepatitis
- •e. Uncontrolled diabetes as defined by fasting serum glucose > 1.5 × ULN
- •f. Active skin, mucosa, ocular or GI disorders of Grade > 1.
- •g. Active (acute or chronic) or uncontrolled severe infections
- •h. A known history of HIV seropositivity or other active viral infections
- •12. Patients with an active, bleeding diathesis
- •13. Patient with uncontrolled hyperlipidemia: fasting serum cholesterol > 300 mg/dL OR >7.75 mmol/L AND fasting triglycerides > 2.5 x ULN
- •14. Patients who have had a major surgery or significant traumatic injury within 4 weeks of study entry.
- •15. Patients with a prior history of organ transplant
- •16. Patients receiving more than 3 antiepileptic drugs at any time in the baseline phase or at randomization or who change the dose of the AEDs during 4 weeks before screening or during the baseline period
- •17. Patients being treated with felbamate, unless treatment has been continuous for = 1 years
- •18. Patients currently receiving anticancer therapies or who have received anticancer therapies within 4 weeks of study entry (including chemotherapy, radiation therapy, antibody based therapy, etc.)
- •19. Prior treatment with any investigational drug within the preceding 4 weeks prior to study entry.
- •20. Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent at study entry. Topical or inhaled corticosteroids are allowed.
- •21. Patients who have received prior treatment with a systemic mTOR inhibitor (sirolimus, temsirolimus, everolimus) within 24 months of study entry. Patients who have received prior treatment with a topical mTOR inhibitor (siroli
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