EUCTR2015-001324-36-IT进行中（未招募）不适用
A Multicenter, Open-Label Proof-of-Concept Trial of Ganaxolone in Children with PCDH19 Female Pediatric Epilepsy
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 10
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
- Female
入选标准
- •1. Have parent or legal guardian available and willing to give written informed consent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures.
- •2. Female outpatients > 2 years of age and younger than 11 years of age at time of consent.
- •3. Have confirmed PCDH19 genetic mutation.
- •4. Have uncontrolled cluster seizures (approximately 10 or more seconds between seizures), lasting for one hour or more per episode, with inter-cluster episodes of not more than 6 weeks, AND/OR, non-clustered focal dyscognitive or focal convulsive seizures with a frequency =4 seizures per 28-day period during baseline.
- •5. Subjects should be on a stable regimen of AED medication, and generally in good health.
- •6. Parent or guardian is able and willing to maintain an accurate and complete daily written seizure calendar for the duration of the study.
- •7. Able and willing to take study medication with food, two or three times daily. Ganaxolone must be administered with food.
- •8. Subject must be approved to participate by Sponsor and Principal Investigator after review of medical history and baseline seizure calendars.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •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. Have had previous exposure to ganaxolone.
- •2. Known sensitivity or allergy to any component in the study drug, progesterone, or other related steroid compounds.
- •3. Have SCN1A genetic mutation.
- •4. Exposure to any investigational drug or device < 90 days prior to screening, or plans to participate in another drug or device trial at any time during the study.
- •5. Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system (CNS) disease deemed progressive, metabolic illness, or progressive degenerative disease.
- •6. Concurrent use of vigabatrin, tiagabine, ezogabine or finasteride is not permitted, nor use of moderate or severe inducers or inhibitors of CYP3A4/5/7. Individuals with prior use of vigabatrin must have had stable visual fields tested twice over the 12 months after the last dose of vigabatrin. A list of CYP3A4/5/7 inhibitors and inducers is included in the Appendix.
- •7. Have any medical condition that, in the investigator's judgment, is considered to be clinically significant and could potentially affect subject safety or study outcome, including but not limited to: clinically significant cardiac, renal, pulmonary, gastrointestinal, hematologic or hepatic conditions; or a condition that affects the absorption, distribution, metabolism or excretion of drugs.
- •8. Have active suicidal plan/intent, or have had active suicidal thoughts in the past 6 months or a suicide attempt in the past 3 years.
- •9. Have Alanine transferase (ALT; SGPT) or Aspartate transferase (AST; SGOT) levels > 3 times upper limits of normal (ULN), or total bilirubin >1.5 time ULN at the baseline visit.
- •10. Are currently following or planning to follow a ketogenic diet.
- •11. Is sexually active or pregnant.
- •12. Unwilling to forgo grapefruit and grapefruit juice from diet during the entire clinical trial.
研究者
相似试验
进行中（未招募）
1 期
An Open-label, One-arm, Proof of Concept Trial to Evaluate the Safety of ATX-MS-1467 (MSC2358825A) and its Effect on Immune Tolerance in Subjects with Relapsing Multiple SclerosisEUCTR2013-002916-28-LVMerck KGaA37
进行中（未招募）
不适用
A Multicentre, Multinational, Open-Label Trial (including Double-Blind, Placebo-Controlled Cross-Over Extension Part) to Evaluate the Time to Significant Pain Relief with Fentanyl TAIFUN® in the Treatment of Breakthrough Pain in Patients with Cancer on Maintenance Opioid Therapy for Persistent Pain. - FIND 2: Fentanyl Inhalation in Neoplastic DiseasePatients with cancer having episodes of breakthrough painMedDRA version: 7.1Level: cancClassification code 10058019EUCTR2005-002544-25-FIAB Pharma Ltd62
招募中
3 期
A Multicenter, Open-Label, Extension Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults with Growth Hormone DeficiencyJPRN-jRCT2041210156Beckert Michael24
招募中
1 期
An extension study to check the safety of once weekly lonapegsomatropin when given for a long time in adults with growth hormone deficiency.EUCTR2021-004313-39-ITAscendis Pharma Endocrinology Division A/S240
招募中
1 期
An extension study to check the safety of once weekly lonapegsomatropin when given for a long time in adults with growth hormone deficiency.Adult Growth Hormone Deficiency (AGHD)MedDRA version: 20.0Level: PTClassification code 10056438Term: Growth hormone deficiencySystem Organ Class: 10014698 - Endocrine disordersEUCTR2021-004313-39-SKAscendis Pharma Endocrinology Division A/S240