EUCTR2020-002396-35-IT进行中（未招募）1 期
A Phase 3 Study of Adjunctive XEN496 in Pediatric Subjects with KCNQ2Developmental and Epileptic Encephalopathy - EPIK
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
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. Parent(s) or guardian(s) fully comprehends the nature and risks of
- •the study and is able and willing to give informed consent in writing,
- •prior to the subject entering the study, in accordance with local legal
- •requirements.
- •2. Male or female subjects aged from 1 month (44 weeks postmenstrual
- •age) to less than 6 years, with a body weight of =3.0 kg, at screening
- •(Visit 1).
- •3. Documented evidence of a genetic test result from an appropriately
- •accredited laboratory, consistent with a diagnosis of KCNQ2-DEE
- •(pathogenic, likely pathogenic, variant of unknown significance, or
- •inconclusive but unlikely to support an alternate diagnosis).
- •4. Seizure onset within 2 weeks after birth and EEG and documented
- •clinical history consistent with KCNQ2-DEE.
- •5. Magnetic resonance imaging has been performed and is without
- •evidence of structural abnormalities, including but not limited to,
- •hypoxia, hypoxia-ischemia, ischemia (arterial or venous), stroke,
- •sinovenous thrombosis, intracranial hemorrhage, or focal or global brain
- •malformation.
- •6. A sufficient number of focal tonic or other countable motor seizures in
- •the month (28 days) prior to screening, documented by caregiver report
- •or investigator medical notes.
- •7. Subjects can be taking 1 and no more than the maximum required
- •number concomitant ASMs. All doses must be stable for the required
- •duration prior to screening and expected to be maintained throughout
- •the duration of the study (until the end of the treatment period).
- •8. VNS is allowed and will not be counted as a concomitant ASM. The
- •VNS device must be implanted for at least 6 months before before
- •screening, and the device settings must be stable for the required
- •duration prior to screening and throughout the duration of the study.
- •Use of the VNS device magnet is allowed.
- •9. Ketogenic diet is allowed and will not be counted as a concomitant
- •ASM. The subject must be on a stable dietary regimen that produces
- •ketosis for the required period of time prior to screening, and expected
- •to be maintained throughout the study.
- •10. In the opinion of the investigator, the caregiver is able and willing to
- •maintain an accurate and complete daily diary to monitor seizures,
- •diaper count (when required) and administration of study drug and
- •concomitant medications.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Presence of a pathogenic or likely pathogenic variant in an additional
- •gene associated with other epilepsy syndromes.
- •2. Presence of a known gain-of-function variant in the KCNQ2 gene, or
- •clinical characteristics consistent with previously reported pathogenic
- •gain-of-function variants in the KCNQ2 gene, such as subjects with
- •infantile spasms without a history of neonatal-onset seizures.
- •3. Seizures secondary to infection, neoplasia, demyelinating disease,
- •degenerative neurological disease, or CNS disease deemed progressive,
- •metabolic illness, or progressive degenerative disease.
- •4. Confirmed diagnosis of infantile spasms within the past month prior to
- •5. History or presence of any significant medical or surgical condition or
- •uncontrolled medical illness at screening including, but not limited to,
- •cardiovascular, gastrointestinal, hematologic, hepatic, ocular,
- •pulmonary, renal, or urogenital systems, or other conditions that would
- •not justify the subject's participation in the study, as determined by the
- •investigator's risk benefit assessment.
- •6. QT interval corrected for heart rate by Fridericia's formula (QTcF) of
- •>440 msec. In addition, subjects with a history of arrhythmia, prolonged
- •QT, heart disease or subjects taking medications known to increase the
- •QT interval.
- •7. History of hyperbilirubinemia, which lasts longer than 1 week will
- •require exclusion of hepatic disease before entering the study.
- •8. History of bilirubin-induced neurological dysfunction.
- •9. Current disturbance of micturition or known urinary obstructions or
- •history of bladder or urinary dysfunction including abnormal post-void
- •residual bladder ultrasound, vesicoureteral reflux, urinary retention, or
- •required urinary catheterization in the preceding 6 months.
- •10. Subjects who are known to have a terminal illness.
- •11. Any clinically significant laboratory abnormalities or clinically
- •significant abnormalities on pre-study physical examination, vital signs,
- •or ECG that in the judgment of the investigator indicates a medical
- •problem that would preclude study participation.
- •12. Subjects who are planned to begin to follow a ketogenic or other
- •specialized dietary therapy during the study.
- •13. Caregiver history of chronic noncompliance with their child's
- •prescribed drug regimens that has not been corrected.
- •14. Exposure to any other investigational drug or device within 5 halflives or 30 days prior to screening (Visit 1), whichever is longer or plans to participate in another drug or device trial at any time during the study.
- •15. Concurrent enrollment in any other type of medical research judged
- •by the investigator not to be scientifically or medically compatible with this study.
- •16. Subjects using felbamate who have presented with clinically
- •significant abnormalities and/or hepatic dysfunction during felbamate
- •treatment, and subjects who have taken felbamate for less than 6 months prior to screening
- •17. Subjects who are currently taking adrenocorticotropic hormone.
- •18. Subjects who did not tolerate ezogabine when taken previously.
研究者
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