Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting
20 studies found for:    "gene therapy" OR "gene transfer" OR "virus delivery" | Severe Combined Immunodeficiency Syndrome
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Rank Status Study
1 Active, not recruiting Gene Therapy for X-linked Severe Combined Immunodeficiency
Condition: X-linked Severe Combined Immunodeficiency
Intervention: Other: Gene transfer
2 Completed Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)
Condition: Severe Combined Immunodeficiency
Intervention: Drug: Gene-Transduced Autologous CD34+ Stem Cells
3 Recruiting Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
Condition: Severe Combined Immunodeficiency Disease, X-linked
Interventions: Genetic: CL20-4i-EF/a-hyc-OPT;   Drug: Busulfan
4 Completed Gene Therapy for ADA-SCID
Condition: Severe Combined Immunodeficiency Syndrome
Intervention: Genetic: gene transduced PBL and/or gene transduced HSC
5 Recruiting Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Condition: Severe Combined Immunodeficiency
Intervention: Biological: Gene transfer
6 Active, not recruiting Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)
Condition: X-linked Severe Combined Immunodeficiency
Intervention: Genetic: Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre
7 Completed
Has Results
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Condition: Severe Combined Immunodeficiency
Intervention: Biological: ADA gene transfer
8 Suspended Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
Conditions: X-linked Severe Combined Immunodeficiency;   XSCID;   SCID-X1;   Gamma C-Deficient SCID
Interventions: Other: Gene-modified CD34+ Hematopoietic stem cells;   Drug: Busulfan
9 Completed Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
Condition: Severe Combined Immunodeficiency
Interventions: Drug: ADA PBSC;   Drug: ADA Umbilical Cord Blood Cells;   Drug: Transduced Lymphocytes
10 Completed Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency
Condition: Severe Combined Immunodeficiency Syndrome
Intervention: Drug: CD34+ cells transduced with ADA retrovir
11 Recruiting Lentiviral Gene Therapy for Adenosine Deaminase (ADA) Deficiency
Conditions: Adenosine Deaminase Deficiency;   Severe Combined Immunodeficiencies (SCID)
Intervention: Genetic: EF1αS-ADA lentiviral vector transduced patient Cd34+ cells
12 Active, not recruiting Autologous Transplant of EFS-ADA Modified Bone Marrow Cells for ADA-Deficient Severe Combined Immunodeficiency (SCID)
Condition: ADA-SCID
Intervention: Genetic: EFS-ADA transduced CD34+ cells from the bone marrow
13 Withdrawn Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents
Condition: ADA-SCID
Intervention:
14 Terminated
Has Results
Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders
Conditions: Severe Combined Immunodeficiency Disease;   Severe Primary Immunodeficiency Disorder;   Undefined T Cell Deficiency Disorder;   Wiskott-Aldrick Syndrome
Interventions: Biological: Campath -1H;   Drug: Fludarabine;   Biological: Anti-CD45;   Procedure: Stem cell infusion
15 Recruiting Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
Conditions: Severe Combined Immunodeficiency;   Malignancy, Hematologic;   Neuroblastoma;   Neoplasm;   Mucopolysaccharidosis I
Intervention: Procedure: Venipuncture
16 Completed Gene Therapy ADA Deficiency
Condition: Adenosine Deaminase Deficiency
Intervention: Biological: Intravenous infusion of transduced cells
17 Completed Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector
Conditions: Adenosine Deaminase Deficiency;   ADA-SCID
Intervention: Genetic: Lentiviral Gene Transfer
18 Recruiting Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders
Conditions: Lymphohistiocytosis, Hemophagocytic;   Common Variable Immunodeficiency;   Severe Combined Immunodeficiency
Intervention:
19 Recruiting Patients Treated for SCID (1968-2010)
Conditions: SCID;   ADA-SCID;   XSCID;   Leaky SCID;   Omenn Syndrome;   Reticular Dysgenesis
Intervention:
20 Recruiting Natural History Study of SCID Disorders
Conditions: SCID;   Leaky SCID;   Omenn Syndrome;   Reticular Dysgenesis;   ADA Deficiency;   XSCID
Intervention:

Study has passed its completion date and status has not been verified in more than two years.