ACTRN12609000849291暂停1 期
A multi-centre, open-label study of the safety, tolerability and pharmacodynamics of intravenously administered Cyclic Pyranopterin Monophosphate (cPMP) (precursor Z) in patients with molybdenum CoFactor deficiency type A
PMP(Vic) Pty Ltd0 个研究点目标入组 10 人开始时间: 2009年9月30日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 暂停
- 发起方
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomised trial
- 主要目的
- Treatment
- 盲法
- Open (masking not used)
入排标准
- 年龄范围
- Weeks 至 6 Weeks（—）
- 性别
- All
入选标准
- •Neonate or infant, less then 6 weeks at the time of diagnosis, age less than 8 weeks at start of treatment with the study medication. It is important to diagnose the condition and initiate treatment as soon after birth as possible.
- •-Documented diagnosis of molybdenum cofactor deficiency (MoCD) Type A based on the absence of cPMP and the presence of sulfite and s-sulfocysteine in the urine, absence of urothione in the urine and genetic analysis showing a mutation in the MOCS1 gene
- •- A parent or legal guardian voluntarily provided written informed consent to participate in the study and comply with study procedures.
- •- Approval of the study protocol by the local Human Research Ethics Committee (HREC) / Independent Review Board (IRB) and government or regulatory authorities (if applicable).
排除标准
- •- MoCD Type B (MOCS2 mutation) or Type C (gephyrin gene mutation)
- •- Sulfite oxidase deficiency
- •- Patients older than 6 weeks at the time of diagnosis
研究者
相似试验
进行中（未招募）
4 期
Cephalexin Extended Release and Clavulanate Potassium Tablets in Patients with Upper Respiratory Tract Infection and Uncomplicated Skin & Soft Tissue Infection.CTRI/2022/07/044190Sun Pharma Laboratories Ltd
进行中（未招募）
1 期
A Study of Trastuzumab-MCC-DM1 (T-DM1) in Combination With Docetaxel in Patients With Advanced Breast Cancer.EUCTR2009-010000-28-GBF. Hoffmann-La Roche Ltd98
进行中（未招募）
1 期
An open-label, multi-center study of the safety and tolerability of the combination of Trastuzumab-MCC-DM1 (T-DM1) with docetaxel, and potentially pertuzumab, for treatment for patients with advanced breast cancer.Treatment in patients with advanced, HER2-positive breast cancerMedDRA version: 9.1Level: LLTClassification code 10065430Term: HER-2 positive breast cancerMedDRA version: 9.1Level: LLTClassification code 10027475Term: Metastatic breast cancerEUCTR2009-010000-28-FRF. Hoffmann-La Roche Ltd98
进行中（未招募）
不适用
A Multicenter, Open-label Study of the Safety, Efficacy, and Pharmacokinetics of the Human Anti-TNF Monoclonal Antibody Adalimumab in Children With Polyarticular Juvenile Rheumatoid ArthritisMedDRA version: 17.1Level: PTClassification code 10039073Term: Rheumatoid arthritisSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disordersJuvenile Rheumatoid ArthritisEUCTR2014-004558-33-Outside-EU/EEAAbbott Japan Co., Ltd.25
进行中（未招募）
不适用
An Open-label, Multicentre Study of the Safety of Twice Daily Oxycodone Hydrochloride Controlled-release Tablets in Opioid Experienced Children from Ages 6 to 16 Years Old, Inclusive, with Moderate to Severe Malignant and/or Nonmalignant Pain Requiring Opioid Analgesics.Opioid experienced paediatric patients with moderate to severe malignant and/or nonmalignant pain requiring opioid therapy.MedDRA version: 12.1Level: LLTClassification code 10002182Term: AnalgesiaEUCTR2010-020471-23-FIPurdue Pharma L.P.100