跳至主要内容
临床试验/EUCTR2011-003212-22-GB
EUCTR2011-003212-22-GB进行中(未招募)1 期

Phase I study of ex-vivo lentiviral gene therapy for the inherited skin disease Netherton Syndrome - Gene Therapy for Netherton Syndrome

GOSH/ICH Joint Research & Development Office0 个研究点目标入组 5 人开始时间: 2013年3月8日最近更新:
适应症

试验速览

阶段
1 期
状态
进行中(未招募)
发起方
入组人数
5

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

入选标准

  • 1. Confirmed SPINK5 mutations in both alleles by direct DNA sequencing
  • 2. Absence of LEKTI protein expression in the skin by in situ immunostaining
  • 3. Patient informed consent, or parental/guardian consent in the case of minor
  • participant
  • Are the trial subjects under 18? yes
  • Number of subjects for this age range: 2
  • F.1.2 Adults (18-64 years) yes
  • F.1.2.1 Number of subjects for this age range 3
  • F.1.3 Elderly (>=65 years) no
  • F.1.3.1 Number of subjects for this age range 0

排除标准

  • 1. History of skin malignancy or evidence of current active malignant skin disease
  • 2. Pregnancy
  • 3. Hepatitis A, B, C or HIV positive
  • 4. Current antibiotic resistant bacterial colonisation

研究者

发起方
GOSH/ICH Joint Research & Development Office

相似试验

进行中(未招募)
1 期
Phase I/II ex vivo gene therapy clinical trial for RDEB using autologous skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector - EBGraftThe trial aims to treat the recessive dystrophic epidermolysis bullosa (RDEB) by grafting one to three subjects with RDEB with autologous COL7A1-modified skin equivalents, using SIN-RV encoding COL7A1 cDNA.MedDRA version: 20.0 Level: LLT Classification code 10074980 Term: Epidermolysis bullosa aquisita System Organ Class: 100000004858
EUCTR2016-002790-35-FRINSERM3
进行中(未招募)
1 期
Clinical study to assess the efficacy and safety of gene therapy for the treatment of Sickle Cell DiseaseMedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersSickle Cell Disease
EUCTR2019-000331-63-FRbluebird bio, Inc.35
进行中(未招募)
1 期
Clinical study to assess the efficacy and safety of gene therapy for the treatment of Sickle Cell DiseaseSickle Cell Disease
EUCTR2019-000331-63-NLbluebird bio, Inc.35
进行中(未招募)
1 期
Clinical study to assess the efficacy and safety of gene therapy for the treatment of Sickle Cell DiseaseMedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersSickle Cell Disease
EUCTR2019-000331-63-ITBLUEBIRD BIO, INC.35
进行中(未招募)
1 期
Clinical study to assess the efficacy and safety of gene therapy for the treatment of Sickle Cell DiseaseMedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersSickle Cell Disease
EUCTR2019-000331-63-GBbluebird bio, Inc.35