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Study to Assess Dose, Efficacy and Safety of Setrusumab in Participants With Osteogenesis Imperfecta
The primary objectives of the study are to identify a setrusumab dosing strategy in participants with OI and to evaluate the effect of setrusumab vs placebo on reduction in fracture rate.
1至6岁APDS患儿
This is a 2-part, prospective, open-label, single arm, 多中心研究评价安全性, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Viaskin花生对4-7岁花生过敏儿童的安全性和有效性研究
The primary purpose of this study is to assess the efficacy and safety of daily DBV712 250 micrograms (mcg) to induce desensitization to peanut in peanut-allergic children 4-7 years of age over a 12-month treatment period.
DMCRN-02-001:评估DM1的儿科终点
The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and childhood myotonic dystrophy type 1, 并为这种疾病开发生物标志物.
评价EryDex治疗A-T患者的疗效
这是一个国际性的, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes), 每28天静脉输注1次, 非共济失调性毛细血管扩张症(Ataxia telangexasia, A-T)患者的神经学症状.
SGT-003基因治疗杜氏肌营养不良症的研究
This is a multicenter, open-label, 研究安全性的非随机研究, tolerability, 单次静脉输注SGT-003对杜氏肌营养不良患者的疗效. 本研究将有2个队列,按顺序给药. Cohort 1 will include participants 4 to <6 years of age, inclusive. 只有在给药和监测队列1中的一部分参与者后,才会开放队列2. Cohort 2 will include participants 6 to <8 years of age, inclusive. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
奥贝德西韦治疗儿童和青少年COVID-19的研究
The goal of this clinical study is to learn more about the safety and tolerability of obeldesivir (ODV) in children and adolescents with coronavirus disease 2019 (COVID-19).
主要目的是评估血浆药代动力学(PK)。, ODV在小儿COVID-19患者中的安全性和耐受性.
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. 本研究不使用任何研究药物. Pre-infusion baseline will be defined as the timepoint just prior to infusion of delandistrogene moxeparvovec from a previous clinical study. Each participant will be followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a previous clinical study. The duration of participation in this study is dependent on the length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec from a previous clinical study.
TPN & 微生物组:生物老化和认知
该研究尚未在ClinicalTrials上注册.Gov,这是目前显示详细资格标准的先决条件.
- 如果您需要临床试验方面的帮助.注册肿瘤学研究,请联系 Jonsson综合癌症中心的法规遵从办公室.
- 如果您需要临床试验方面的帮助.非肿瘤学研究的注册,请联系 监管事务办公室.
臂式OCT-A用于婴儿和儿童的非侵入性眼部成像
该研究尚未在ClinicalTrials上注册.Gov,这是目前显示详细资格标准的先决条件.
- 如果您需要临床试验方面的帮助.注册肿瘤学研究,请联系 Jonsson综合癌症中心的法规遵从办公室.
- 如果您需要临床试验方面的帮助.非肿瘤学研究的注册,请联系 监管事务办公室.
Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
This is a Phase 3 study to assess the efficacy and safety of twice-weekly subcutaneous (SC) doses of pegcetacoplan compared to placebo in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN) on the basis of a reduction in proteinuria.
A Study of DB-OTO, 基于腺相关病毒(AAV)的基因治疗, 因Otoferlin突变导致听力损失的儿童/婴儿
Regeneron正在进行一项名为DB-OTO的研究性新药的研究. DB-OTO is a gene therapy that is being developed to treat children who have hearing loss due to changes in the otoferlin gene.
本研究的目的是:
- 了解DB-OTO的安全性
- 确定DB-OTO的耐受性(不会引起持续不适)
- 评估DB-OTO的疗效(DB-OTO的效果如何)
EDG-5506治疗儿童杜氏肌营养不良症的研究
猞猁的研究分为两部分, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, 安慰剂对照的A部分, 然后是开标签的B部分.
原发性免疫调节障碍(PIRD):临床表现的纵向研究, Treatment and Outcomes
该研究尚未在ClinicalTrials上注册.Gov,这是目前显示详细资格标准的先决条件.
- 如果您需要临床试验方面的帮助.注册肿瘤学研究,请联系 Jonsson综合癌症中心的法规遵从办公室.
- 如果您需要临床试验方面的帮助.非肿瘤学研究的注册,请联系 监管事务办公室.
Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036)
This study aims to compare the efficacy of vericiguat versus placebo on change in n-terminal pro-brain natriuretic peptide (NTproBNP) from baseline to Week 16. 主要假设是Vericiguat在第16周降低NT-proBNP方面优于安慰剂.
Study to Evaluate Sotatercept (MK-7962) in Children With Pulmonary Arterial Hypertension (PAH) (MK-7962-008)
该研究的主要目的是评估安全性和耐受性, and pharmacokinetics (PK) of sotatercept over 24 weeks of treatment in children ≥1 to <18 years of age with PAH World Health Organization (WHO) Group 1 on standard of care (SoC). 没有正式的假设.
人猴痘病毒特可维林的研究
A5418 is a randomized, placebo-controlled, double-blind study to establish the efficacy of tecovirimat for the treatment of people with laboratory-confirmed or presumptive HMPXV disease.
Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.
4 - 11岁APDS患儿
This is a 2-part, prospective, open-label, single arm, 多中心研究评价安全性, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 4 to 11 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. 这是一项随机、双盲、安慰剂对照的两部分研究. 参与者将参加大约128周的研究. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.