EUCTR2008-003915-11-DE进行中（未招募）不适用
Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I
niversity of Utah0 个研究点目标入组 36 人开始时间: 2009年6月25日最近更新:
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 36
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
- •Laboratory documentation of SMN mutation or deletion consistent with genetic diagnosis of SMA
- •Clinical diagnosis of SMA type I
- •Age between 2 weeks and 12 months
- •Written informed consent of parent/guardian
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •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 subjects for this age range
排除标准
- •Any clinical or laboratory evidence of hepatic or pancreatic insufficiency.
- •Laboratory results drawn within 14 days prior to start of study drug demonstrating:
- •- Liver transaminases (AST, ALT), lipase, amylase: >1.5 x ULN,
- •- White Blood Cell Count: < 3 x 10^9/l
- •- Neutropenia: <1 x 10^9/l
- •- Platelet: <100 x 10^9/l
- •- Hematocrit: <30 %, persisting over a 30-day period
- •Serious illness requiring systemic treatment and/or hospitalization within two weeks prior to study entry.
- •Use of medications or supplements within 30 days of study enrollment that
- •- interfere with VPA or carnitine metabolism
- •- that increase the potential risks of VPA or carnitine
- •- that are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders, including riluzole, valproic acid, hydroxyurea, oral use of albuterol, sodiumphenylbutyrate, butyrate derivatives, creatinine, growth hormone, anabolic steroids, probenecid, oral or parenteral use of corticosteroids at entry, or
- •agents anticipated to increase or decrease muscle strength or agents with presumed histone deacetylase (HDAC) inhibition
- •Infants who have participated in a treatment trial for SMA within 30 days of study entry or who will become enrollees in any other treatment trial during the course
- •of this study.
- •Unwillingness to travel for study assessments.
- •Coexisting medical conditions that contradict use of VPA/carnitine or travel to and from study site.
- •Requirement of ventilator support for more than 12 hours per day.
研究者
相似试验
已完成
1 期
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type ISpinal Muscular Atrophy Type INCT00661453University of Utah40
终止
1 期
Phase I/II Trial of Valproic Acid and Karenitecin for MelanomaMalignant MelanomaNCT00358319H. Lee Moffitt Cancer Center and Research Institute42
已完成
2 期
PhII 5-Azacytidine Plus Valproic Acid and Eventually Atra in Intermediate II and High Risk MDSMyelodysplastic SyndromesNCT00439673Gruppo Italiano Malattie EMatologiche dell'Adulto62
招募中
1 期
Randomized phasE 2 study of Valproic acid in combination with bevacizumab and Oxaliplatin/fLUoropyrimidine regimens in patients with ras-mutated metastaTIc cOlorectal caNcerpatients with ras-mutated metastaTIc colorectal cancerMedDRA version: 20.0Level: LLTClassification code 10007446Term: Carcinoma of rectumSystem Organ Class: 100000004864EUCTR2018-001414-15-ITISTITUTO NAZIONALE TUMORI - IRCCS FONDAZIONE PASCALE200
进行中（未招募）
不适用
Preclinical and clinical study of valproic acid plus cisplatin and cetuximab in recurrent and/or metastatic squamous cell carcinoma of head and neckRecurrent and/or metastatic squamous cell carcinoma of head and neckMedDRA version: 17.0Level: PTClassification code 10067821Term: Head and neck cancerSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)EUCTR2014-001523-69-ITIstituto Nazionale Tumori di Napoli - Fondazione G. Pascale39