EUCTR2019-002907-17-NL进行中（未招募）1 期
A MulticeNter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in PediaTric IndividUals With AngelmaN SyndromE (NEPTUNE) - NEPTUNE
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 95
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.Is male or female and 2 to 12 years old (inclusive) at the time of informed
- •2. Has a diagnosis of AS with molecular confirmation
- •3. Has a CGI-S-AS score of 3 or more.
- •4. Meets the following age-appropriate body weight criterion:
- •a) Subjects 2 to 3 years old must have a minimum body weight of 9 kg.
- •b) Subjects 4 years and older must be between 17 kg and 64 kg (inclusive).
- •5. Has a legally acceptable representative (LAR)/caregiver capable of providing written informed consent and able to attend all scheduled study visits, oversee the administration of study drug, and provide feedback regarding the subject’s symptoms and performance as described in the protocol.
- •6. Provides assent to the protocol (to the extent possible and in accordance with local institutional review board [IRB] and regulatory requirements). Subjects providing assent must do so at the same visit as LAR/caregiver written informed consent is provided.
- •7. Can swallow study drug capsules with water or ingest the contents of study drug capsules after sprinkling the contents of each capsule onto up to 1 teaspoon of low-fat semiliquid food.
- •8. If a subject is currently receiving a regimen of concomitant medications such as antiepileptic medication, gabapentin, clonidine, trazodone, melatonin, or a special diet regimen, that subject’s regimen is stable for at least 4 weeks before Day 1 (first day of study drug administration) and will be maintained throughout the duration of the study (in the judgment of the investigator).
- •9. If a subject is a sibling in a family with multiple children diagnosed with AS, then only one of the siblings may enroll in study. The eldest eligible subject should be enrolled (investigator discretion may be used to enroll a younger sibling instead).
- •10. Has LAR(s)/caregiver(s) who agree not to post any of the subject’s personal medical data or information related to the study on any website, message board(s), online support group(s), or social media site (eg, Facebook, Instagram, Twitter, etc.) until notified that the study is completed.
- •11. Female subjects who are of child-bearing potential (defined as having experienced their first menarche) must agree to use either a highly effective or acceptable form of birth control during the study and for 30 days following the last dose of the study. Highly effective contraceptive methods are as follows:
- •a. Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
- •Intravaginal
- •Transdermal
- •b. Progestogen-only hormonal contraception associated with inhibition of ovulation:
- •Injectable
- •Implantable
- •c. Intrauterine device
- •d. Intrauterine hormone-releasing system
- •e. Bilateral tubal occlusion
- •f. Vasectomized partner
- •g. Sexual abstinence
- •Acceptable birth control methods that result in a failure rate of more than 1% per year include:
- •a. Progestogen-only oral hormonal contraception, where inhibition of ovulation is not the primary mode of action.
- •b. Male or female condom with or without spermicide.
- •c. Cap, diaphragm, or sponge with spermicide.
- •d. A combination of male condom with either cap, diaphragm, or sponge with spermicide (double barrier methods) are also considered acceptable, but not highly effective, birth control methods.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 95
- •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 subj
排除标准
- •1. Has a circumstance or concomitant disease (eg, gastrointestinal, renal, hepatic, endocrine, respiratory, or cardiovascular system disease), condition, or any clinically significant finding at screening that could interfere with the conduct of the study or that would pose an unacceptable risk to the subject in the opinion of the investigator.
- •2. Has poorly controlled seizures defined as any of the following:
- •a. Weekly seizures of any frequency with a duration more than 3 minutes.
- •b. Weekly seizures occurring more than 3 times per week, each with a duration
- •of less than 3 minutes.
- •c. Investigator assessment.
- •3. Has any of the following laboratory abnormalities: total bilirubin >1.5 × upper limit of normal (ULN), unless known Gilbert’s syndrome; alanine aminotransferase or aspartate aminotransferase >2.5 × ULN; serum creatinine >1.2 × ULN; absolute neutrophil count <1.5 × 109/L; platelets <80 × 109/L;
- •hemoglobin <80 g/L; or thyroid-stimulating-hormone >1.25 × ULN or <0.8 × lower limit of English:
- •1. Has a circumstance or concomitant disease (eg, gastrointestinal, renal, hepatic, endocrine, respiratory, or cardiovascular system disease), condition, or any clinically significant finding at screening that could interfere with the conduct of the study or that would pose an unacceptable risk to the subject in the opinion of the investigator.
- •2. Has poorly controlled seizures defined as any of the following:
- •a. Weekly seizures of any frequency with a duration more than 3 minutes.
- •b. Weekly seizures occurring more than 3 times per week, each with a duration
- •of less than 3 minutes.
- •c. Investigator assessment.
- •3. Has any of the following laboratory abnormalities: total bilirubin >1.5 × upper limit of normal (ULN), unless known Gilbert’s syndrome; alanine aminotransferase or aspartate aminotransferase >2.5 × ULN; serum creatinine >1.2 × ULN; absolute neutrophil count <1.5 × 109/L; platelets <80 × 109/L;
- •hemoglobin <80 g/L; or thyroid-stimulating-hormone >1.25 × ULN or <0.8 × lower limit of normal. Retesting of clinical laboratory parameters may be allowed after consultation with the medical monitor or designee.
- •4. Use of benzodiazepines, zolpidem, zaleplon, zopiclone, eszopiclone, barbiturates, or ramelteon for sleep, or minocycline or levodopa within the 4 weeks prior to Day 1 or during the study. Benzodiazepines administered as needed for situational anxiety related to occasional procedures or events are permitted, and benzodiazepines are also permitted for seizure control.
- •5. Cannot tolerate wearing the actigraph for at least 7 consecutive days (including 2 consecutive weekend days) during the 28-day screening period of the study, after 3 attempts.
- •6. Is at risk of harming self and/or others (based on investigator assessment).
- •7. Has enrolled in any other interventional clinical study or used any investigational agent or device, or has participated in any investigational procedure, within the 30 days before screening or does so concurrently with this study.
- •8. Is allergic to OV101 or any excipients of study drug.
- •9. The subject or LAR/caregiver is unable to comply with study requirements (based on investigator assessment).
- •10. Is a family member of the investigator and/or study site staff.
研究者
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