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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)

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.

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.

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.

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.

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.

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.

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.

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.

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.

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)

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.

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.

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.

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.

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.

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.

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).