NCT05367960进行中（未招募）3 期
An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome
Tetra Discovery Partners17 个研究点 分布在 1 个国家目标入组 314 人开始时间: 2022年11月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 314
- 试验地点
- 17
- 主要终点
- Safety and tolerability of BPN14770
研究概览
简要总结
This is a 4-year, open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in adult males) and Study BPN14770-CNS-204 (in adolescent males).
详细描述
This is a 4-year open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in adult males) and Study BPN14770-CNS-204 (in adolescent males). The primary objective of this OLE is to assess the long-term safety and tolerability of BPN14770 in these subjects with fragile X syndrome (FXS) who were treated in one of those parent clinical trials.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 9 Years 至 45 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subject has completed the Week 13 visit, whether on treatment or discontinued from treatment, from one of two parent clinical trials with
- •Study 301 Subjects who discontinued study treatment early due to AEs deemed related to study treatment by the investigator in one of the parent studies will NOT be eligible, regardless of whether the Week 13 visit was completed.
- •Subjects with a history of seizure disorder who are currently receiving treatment with antiepileptics must have remained seizure-free during the parent study. Any subject experiencing a seizure during the parent study is not eligible to continue into this long-term safety study.
- •Subject must be willing to practice barrier methods of contraception while on study, if sexually active. Abstinence is also considered a reasonable form of birth control in this study population.
- •Subject has a parent, legal authorized guardian, or consistent caregiver.
- •Subject and caregiver are able to attend the clinic regularly and reliably.
- •If subject is his own legal guardian, he is able to understand and sign an informed consent form to participate in the study.
- •For subjects who are not their own legal guardian, subject's parent/legally authorized guardian is able to understand and sign an informed consent form for their child to participate in the study.
- •If subject is not his own legal guardian, subject must provide assent for participation in the study if he has the cognitive ability to do so.
排除标准
- •1. History of, or current cardiovascular, renal, hepatic, respiratory, gastrointestinal, psychiatric, neurologic, cerebrovascular, or other systemic disease that would place the subject at risk or potentially interfere with the interpretation of the safety, tolerability, or efficacy of the study treatment. Common conditions such as mild hypertension, etc. are allowed per the principal investigator's (PI) judgement as long as they are stable and controlled by medical therapy that is constant for at least 4 weeks before randomization.
- •2. Renal impairment, defined as serum creatinine >1.25×ULN per the latest available laboratory results from the parent study.
- •3. Cirrhosis, unstable chronic liver disease or acute liver disease. Hepatic impairment, defined as ALT or AST elevation > 2 × ULN per the latest available laboratory results from the parent study. Subjects with stable chronic hepatitis B on oral anti-viral therapy and subjects cured of chronic hepatitis C are allowed. Subjects with Gilbert syndrome are allowed.
- •4. Clinically significant abnormalities, in the investigator's judgement, in safety laboratory tests, vital signs, or ECG, as measured at the final visit of the parent study.
- •5. Substance abuse documented during the parent study.
- •6. Significant hearing or visual impairment that may affect the subject's ability to complete the test procedures.
- •7. Concurrent major psychiatric condition (eg, major depressive disorder, schizophrenia, or bipolar disorder) as confirmed by the investigator. Subjects with additional diagnosis of autism spectrum disorder or anxiety disorder will be allowed.
- •8. Subject has active diseases that would interfere with participation, such as acquired immunodeficiency disorder, hepatitis C, hepatitis B, or tuberculosis.
- •9. Subject has participated in another clinical trial within the 30 days preceding screening OTHER THAN one of the two studies required per Inclusion Criteria 1.
研究组 & 干预措施
Study Drug (BPN14770)
Other
25mg BID Study Drug BPN14770 (Adults) or 15mg BID Study Drug BPN14770 (Adolescents with body weight <43 kg)
干预措施: Zatolmilast/ BPN14770 (Drug)
结局指标
主要结局
Safety and tolerability of BPN14770
时间窗: 24 months
Long-term safety and tolerability of BPN14770 in these subjects with fragile X syndrome (FXS) who were treated in one of those parent clinical trials. Safety/tolerability endpoint: Treatment Emergent Adverse Events (TEAEs), which will be coded using the Medical Dictionary for Regulatory Activities. Subject incidence of each system organ class and unique preferred term will be tabulated, including all TEAEs, at least possibly related TEAEs, TEAEs resulting discontinuation of study treatment, TEAEs by intensity, and treatment-emergent SAEs. The safety analysis will be descriptive in nature.
次要结局
- Numerical rating scale scores (NRS) scores(24 Months)
- National Institute of Health (NIH)- Toolbox Cognitive Battery (TCB)(24 Months)
- Vineland-3 Adaptive Behavior Scale (Vineland-3)(24 Months)
- Caregiver Global Impression of Improvement (CaGI-I) assessments:(24 Months)
- Clinical Global Impression Severity (CGI-S) assessments:(24 Months)
- Verbal Knowledge Test from the Stanford-Binet (ed 5) IQ assessment(24 Months)
- Aberrant Behavior Checklist (ABC) scores(24 Months)
- Anxiety, Depression, and Mood Scale (ADAMS) scores(24 Months)
研究者
研究点 (17)
Loading locations...
相似试验
进行中（未招募）
不适用
Open label Extension Study Following Double-blind, Randomized, Placebo-controlled, Multicentre Study to Assess Efficacy and Safety of Adjunctive Zonisamide in Myoclonic Seizures associated with Idiopathic Generalized Epilepsymyoclonic seizures in idiopathic generalised epilepsy (IGE).MedDRA version: 9.1Level: LLTClassification code 10054859Term: Myoclonic epilepsyEUCTR2007-006696-36-HUEisai Limited110
进行中（未招募）
不适用
Open Label Extension Study Following Double-blind, Randomized, Placebo-controlled, Multicenter Study to Assess Efficacy and Safety of Adjunctive Zonisamide in Primary Generalized Tonic-Clonic SeizuresPrimary generalised tonic-clonic seizures (PGTCS) in idiopathic generalised epilepsy (IGE)MedDRA version: 9.1Level: HLTClassification code 10018101Term: Generalised tonic-clonic seizuresEUCTR2007-006695-10-HUEisai Limited154
已完成
4 期
A Second Open-Label Extension of a Double-Blind, Parallel, Phase IV Study to Assess the Efficacy and Safety of Adjunctive Lovaza® (Formerly Known as Omacor®) Therapy in Hypertriglyceridemic Subjects Treated With Antara™HypertriglyceridemiaNCT00891293GlaxoSmithKline93
已完成
2 期
Open-Label Extension Study of Patients Previously Enrolled in Study CIN-107-124HypertensionNCT05459688AstraZeneca175
已完成
4 期
Study Evaluating the Combination of Etanercept and Methotrexate in Rheumatoid Arthritis SubjectsRheumatoid ArthritisNCT00252668Wyeth is now a wholly owned subsidiary of Pfizer300