NL-OMON49584已完成3 期
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
- 3 期
- 状态
- 已完成
- 入组人数
- 12
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
- 年龄范围
- 2 至 17（—）
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. 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.
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 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 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
相似试验
进行中（未招募）
1 期
A Study of Gantenerumab in Patients With Prodromal Alzheimer's DiseaseProdromal Alzheimer’s DiseaseMedDRA version: 17.0Level: LLTClassification code 10066571Term: Progression of Alzheimer's diseaseSystem Organ Class: 100000004852EUCTR2010-019895-66-CZF. Hoffmann-La Roche Ltd797
进行中（未招募）
1 期
A study investigating new drug PD-0332991 (PALBOCICLIB) for treatment of women with Hormone Receptor-Positive, Her2-Negative Metastatic Breast Cancer. Study has two groups: Allpatients will be treated with Fulvestrant (FASLODEX) and Goserelin (Zoladex). In addition, one group will receive PD-0332991 (PALBOCICLIB), the other group an inactive substance. Neither the patient northe doctor will know the treatment group. The distribution into the groups will be random.HORMONE RECEPTOR-POSITIVE, HER2-NEGATIVE METASTATIC BREAST CANCERMedDRA version: 20.0Level: LLTClassification code 10027475Term: Metastatic breast cancerSystem Organ Class: 100000020826EUCTR2013-002580-26-DEPfizer Inc417
已完成
2 期
Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Two Year Study to Evaluate the Effect of Subcutaneous RO4909832 on Cognition and Function in Prodromal Alzheimer's Disease with option for an additional two years of treatment10011305Alzheimer's diseasedementiaNL-OMON39809Roche Nederland B.V.48
进行中（未招募）
1 期
A study investigating new drug PD-0332991 (PALBOCICLIB) for treatment of women with Hormone Receptor-Positive, Her2-Negative Metastatic Breast Cancer. Study has two groups: Allpatients will be treated with Fulvestrant (FASLODEX) and Goserelin (Zoladex). In addition, one group will receive PD-0332991 (PALBOCICLIB), the other group an inactive substance. Neither the patient northe doctor will know the treatment group. The distribution into the groups will be random.HORMONE RECEPTOR-POSITIVE, HER2-NEGATIVE METASTATIC BREAST CANCERMedDRA version: 20.0Level: LLTClassification code 10027475Term: Metastatic breast cancerSystem Organ Class: 100000004864EUCTR2013-002580-26-PTPfizer Inc521
进行中（未招募）
1 期
A Study of Gantenerumab in Patients With Prodromal Alzheimer's DiseaseEUCTR2010-019895-66-SEF. Hoffmann-La Roche Ltd797