EUCTR2021-003441-38-IT进行中（未招募）1 期
A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy (TrustTSC) - Adjunctive GNX Treatment Compared with Placebo in Children and Adults with TSC-related Epilepsy
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 162
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Clinical or mutational diagnosis of TSC consistent with:
- •a. Molecular confirmation of a pathogenic mutation (TSC1 or TSC2) reviewed by the PI or designee.
- •b. Clinical diagnosis of definite TSC which includes 2 major features or 1 major feature with = 2 minor features documented by the PI or designee.
- •2. Male or female participants aged 1 through 65 years, inclusive.
- •3. Participant/parent or LAR willing to give written informed consent/assent, after being properly informedand prior to engaging in any study related procedures.
- •4. Assent for participants over 7 years of age should be obtained.
- •5. Failure to control seizures despite appropriate trial of 2 or more AEDs at therapeutic doses and for adequate duration of treatment per PI judgment.
- •6. Participants should be on a stable regimen of AEDs (including moderate or strong inducer or inhibitor anti-seizure medications at therapeutic doses for = 28 days prior to the screening visit, and without a foreseeable change in dosing for the duration of the study. (Note: Minor dose adjustment to address tolerability and safety events may be allowed on case-by-case basis)
- •7. A history of at least 8 countable seizures per month in the 2 months prior to screening with no more than 1 seizure free week in each month.
- •8. Have an average of at least 2 primary endpoint seizures per week in the 28 days following the screening visit. The primary endpoint seizure types are defined as the following:
- •a. focal motor seizures without impairment of consciousness or awareness
- •b. focal seizures with impairment of consciousness or awareness with motor features. focal seizures evolving to bilateral, tonic-clonic seizuresd. generalized motor seizures including tonic-clonic, bilateral tonic, bilateral clonic, or atonic/drop seizures.
- •Seizures that do not count towards the primary endpoint include:
- •a. Focal aware seizures without motor features
- •b. Focal and generalized nonmotor seizures (eg, absence or focal nonmotor seizures with or without impairment of awareness)
- •c. Infantile or epileptic spasms
- •d. Myoclonic seizures.
- •9. Participants with surgically implanted VNS will be allowed to enter the study provided that all of the following conditions are met:
- •a. The VNS has been in place for = 1 year prior to the screening visit.
- •b. The settings must have remained constant for 3 months prior to the screening visit and are expected to remain constant throughout the study.
- •c. The battery is expected to last for the duration of the study.
- •10. Parent/caregiver or the participant, as appropriate, is able and willing to maintain an accurate and complete daily seizure eDiary for the duration of the study.
- •11. Able and willing to take IP (suspension) as directed with food TID.
- •12. Sexually active WOCBP must be using a medically acceptable method of birth control and have a negative quantitative serum ß-HCG test collected at the initial screening visit. A medically acceptable method of birth control includes intrauterine devices in place for 1 month prior to the screening visit, surgical sterilization, or adequate double barrier methods 13. Male participants must agree to take all necessary measures to avoid causing pregnancy in their sexual partners during the study and for 30 days after the last dose of IP. Medically acceptable contraceptives include surgical sterilization (such as a vasectomy) and a condom used with a spermicidal gel or foam.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
排除标准
- •1. Previous exposure to GNX
- •2. Pregnant or breastfeeding
- •3. Participants who have been taking felbamate for less than 1 year prior to screening
- •4. Participants taking CBD preparations other than Epidiolex
- •5. A positive result on plasma drug screen for CBD or THC at Visit 1 (screening), with the exception of results that are fully explained by Epidiolex, which is being prescribed and managed by the investigator
- •6. Concurrent use of ACTH, prednisone or other glucocorticoid is not permitted, nor use of the strong inducers of CYP3A4, rifampin and St John's Wort. Participants on ACTH, prednisone, or other systemically (non-inhaled or topical) administered steroids should be off the product > 28 days prior to screening. Rifampin and St John's Wort must be discontinued at least 28 days before Visit 2, study drug initiation.
- •7. Changes in any chronic medications within the 4 weeks prior to the screening visit.
- •8. Participants who have epilepsy surgery planned during the study or who have undergone surgery for epilepsy within the 6 months prior to screening.
- •9. An active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (MRI). This includes tumor growth which in the opinion of the investigator could affect primary endpoint seizure control.
- •10. Any disease or condition (medical or surgical; other than TSC) at the screening visit that might compromise the hematologic, cardiovascular (including any cardiac conduction defect), pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the IP, or would place the participant at increased risk or interfere with the assessment of safety/efficacy.
- •11. Hepatic impairment sufficient to affect patient safety, or an AST/SGOT or ALT/SGPT > 3 × the ULN at screening or baseline visits and confirmed by a repeat test.
- •12. Biliary impairment sufficient to affect patient safety, or total bilirubin levels > 1.5 × ULN at screening or baseline visit and confirmed by a repeat test.
- •13. Renal impairment sufficient to affect patient safety, or eGFR < 30 mL/min, will be excluded from study entry or will be discontinued if the criterion is met post baseline.
- •14. Exposed to any other investigational drug or investigational device within 30 days or fewer than 5 half-lives prior to the screening visit.
- •15. Unwillingness to avoid excessive alcohol use throughout the study.
- •16. Have active suicidal plan/intent, active suicidal thoughts or a suicide attempt in the past 6 months.
- •17. Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.
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