EUCTR2021-003222-76-IT进行中（未招募）1 期
A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED, FIXEDDOSE, MULTICENTER STUDY TO EXAMINE THE EFFICACY AND SAFETY OF ZX008 IN SUBJECTS WITH CDKL5 DEFICIENCY DISORDER FOLLOWED BY AN OPEN-LABEL EXTENSION - NA
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 100
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. Subject has a confirmed pathogenic or likely pathogenic mutation in the CDKL5 gene and a clinical diagnosis of CDD with epilepsy onset in the first year of life, plus motor and developmental delays.
- •2. Subject is male or female, aged 1 to 35 years, inclusive, as of the day of the Screening Visit. Subjects aged 1 to < 2 years will ONLY be permitted to enroll in the trial AFTER the DSMB has determined that it is appropriate to do so based on a planned unblinded interim safety review to be conducted after approximately 40 subjects aged = 2 to 35 years have completed Visit 6.
- •3. Subject must have failed to achieve seizure control despite previous or current use of 2 or more AETs.
- •4. Subject is currently receiving at least 1 concomitant antiseizure treatment: antiseizure medication (ASM), vagus nerve stimulation (VNS), responsive neurostimulation (RNS), or ketogenic diet (KD). During the trial, rescue medications or interventions for rescue treatment of seizures will not be counted towards the total number of antiseizure treatments established at Baseline.
- •5. All medications or interventions for epilepsy (including VNS, RNS, and KD) must be stable prior to screening and are expected to remain stable throughout the study. In order to establish stability at BL, duration of treatment with medications or interventions for epilepsy prior to the Screening visit must be as follows: VNS and RNS: = 6 months duration; ASMs or KD: = 4 weeks duration.
- •6. At the Screening Visit, parent/caregiver reports that subject has = 4 countable motor seizures (CMS) per week. CMS include distinct seizures of the generalized tonic-clonic [GTC], bilateral clonic, bilateral tonic, atonic (drop), bilateral tonic/atonic (drop), or focal to bilateral tonicclonic type lasting approximately 3 seconds or longer, to distinguish from short-clustered seizures, spasms, or jerks.
- •7. Subject (and/or subject's parent[s]/legal guardian[s]) has provided written informed consent (and assent if applicable).
- •8. Subject (and/or subject's parent/caregiver) is willing and able to comply with study requirements (including diary completion, visit schedule, and study drug accountability).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 20
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Subject has a known hypersensitivity to fenfluramine or any of the excipients in the study drug.
- •2. Subject has a diagnosis of pulmonary arterial hypertension.
- •3. Subject has a clinically significant medical condition, including chronic obstructive pulmonary disease, interstitial lung disease, or portal hypertension, or has had clinically relevant symptoms or a clinically significant illness currently or in the 4 weeks prior to the Screening Visit, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.
- •4. Subject has current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke, severe ventricular arrhythmias, or clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, patent ductus arteriosus, and patent foramen ovale with reversal of shunt. (Note: Patent foramen ovale or a bicuspid aortic valve are not considered exclusionary.)
- •5. Subject has current eating disorder that suggests anorexia nervosa or bulimia.
- •6. Subject has a current or past history of glaucoma.
- •7. Subject is taking > 4 concomitant ASMs. Rescue medications are not included in the count.
- •8. Subject is receiving concomitant treatment with cannabidiol (CBD) other than Epidiolex/Epidyolex or is being actively treated with tetrahydrocannabinol (THC) or any marijuana product for any condition. Disallowed medications are subject to wash-out requirements.
- •09. Subject has moderate to severe hepatic impairment, assessed based on the Child-Pugh system.
- •10. Subject has moderate to severe renal impairment (estimated glomerular filtration rate < 50 mL/min/1.73 m2 calculated with the Isotope Dilution Mass Spectrometry [IDMS] Traceable Schwartz equation for children and the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation for adults, using actual body weight).
- •11. Subject is receiving concomitant therapy with any of the following: centrally-acting anorectic agents; monoamine-oxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; other centrally-acting noradrenergic agonists, including
- •atomoxetine; or cyproheptadine. (Note: Short-term requirements for prohibited medications will be handled on a per case basis by the Medical Monitor.)
- •12. Subject has participated in another interventional clinical trial within 30 days of the Screening Visit or is currently receiving an investigational product.
- •13. Female subjects of childbearing potential must not be pregnant or breastfeeding. Female subjects of childbearing potential must have a negative urine or serum pregnancy test at Screening. Subjects of childbearing or child-fathering potential must be willing to use an approved method of highly effective contraception, which includes
- •abstinence, while participating in this study and for 90 days after the last dose of study drug.
- •14. Subject is known to be human immunodeficiency virus positive.
- •15. Subject is known to have active viral hepatitis B or C.
- •16. Subject is institutionalized in a facility that does not provide skilled epilepsy care.
- •17. Subject has previously been treated with Fintepla® (fenfluramine) prior to the Screening Visit.
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