Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
97 studies found for:    Open Studies | "gene therapy"
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Rank Status Study
21 Recruiting Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation
Conditions: Leber's Hereditary Optic Neuropathy;   G11778A Gene Mutation;   Human Mitochondrial ND4 Gene
Interventions: Drug: scAAV2-P1ND4v2 5.00x10e9 vg (Low),;   Drug: scAAV2-P1ND4v2 2.46X10e10 vg (Med);   Drug: scAAV2-P1ND4v2 1.0X10e11vg (High)
22 Recruiting Gene Therapy for WAS Follow-up
Condition: Wiskott-Aldrich Syndrome
Intervention: Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector
23 Recruiting MVX-ONCO-1 in Patients With Solid Tumor
Conditions: Solid Tumor;   Cancer
Intervention: Other: Treatment
24 Recruiting Retroviral Vector Mediated Globin Gene Transfer to Correct Sickle Cell Anemia or Thalassemia
Conditions: Sickle Cell Anemia;   Thalassemia
Intervention: Genetic: Gene Therapy
25 Recruiting Gene Therapy for Blindness Caused by Choroideremia
Condition: Choroideremia
Intervention: Drug: rAAV2.REP1
26 Recruiting Gene Therapy for B-Cell Non-Hodgkin Lymphoma Using CD19 CAR Gene Transduced T Lymphocytes
Condition: Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma
Interventions: Drug: Cyclophosphamide or Bendamustine;   Genetic: Dose Level -1;   Genetic: Dose Level 1;   Genetic: Dose Level 2;   Genetic: Dose Level 3
27 Recruiting Gene Therapy and Radioactive Iodine in Treating Patients With Locally Recurrent Prostate Cancer That Did Not Respond to External-Beam Radiation Therapy
Condition: Prostate Cancer
Interventions: Biological: Ad5-CMV-NIS;   Drug: liothyronine sodium;   Genetic: reverse transcriptase-polymerase chain reaction;   Other: laboratory biomarker analysis;   Radiation: iodine I 131
28 Recruiting AADC Gene Therapy for Parkinson's Disease
Condition: Parkinson's Disease
Intervention: Genetic: AAV2-hAADC
29 Recruiting A Phase I Study of Systemic Gene Therapy With SGT-94 in Patients With Solid Tumors
Condition: Neoplasm
Intervention: Genetic: SGT-94
30 Recruiting Stem Cell Gene Therapy for Sickle Cell Disease
Condition: Sickle Cell Disease
Intervention: Genetic: βAS3-FB vector transduced BM CD34+ cells
31 Recruiting Gene Therapy After Frontline Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma
Conditions: AIDS-related Diffuse Large Cell Lymphoma;   AIDS-related Diffuse Mixed Cell Lymphoma;   AIDS-related Diffuse Small Cleaved Cell Lymphoma;   AIDS-related Immunoblastic Large Cell Lymphoma;   AIDS-related Lymphoblastic Lymphoma;   AIDS-related Small Noncleaved Cell Lymphoma;   HIV Infection
Interventions: Drug: busulfan;   Biological: lentivirus vector rHIV7-shI-TAR-CCR5RZ-transduced hematopoietic progenitor cells;   Other: pharmacological study;   Other: laboratory biomarker analysis
32 Unknown  A Phase I Gene Therapy Study of FP253/Fludarabine for Prostate Cancer
Condition: Prostate Cancer
Intervention: Biological: Gene Directed Enzyme Prodrug Therapy, FP253/Fludarabine
33 Recruiting AAV1-CMV-Serca2a GENe Therapy Trial in Heart Failure
Conditions: Heart Failure Congestive;   Ischemic Cardiomyopathy;   Non-ischemic Cardiomyopathy
Interventions: Genetic: MYDICAR-single intracoronary infusion;   Genetic: Placebo; single intracoronary infusion
34 Recruiting Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: intracerebral administration of AAVrh.10cuARSA
35 Unknown  Safety and Efficacy Study of INGN 241 Gene Therapy in Patients With In Transit Melanoma
Conditions: Malignant Melanoma;   Neoplasm Metastasis
Intervention: Genetic: investigational drug INGN 241
36 Not yet recruiting Safety and Efficacy Study of Gene Therapy Drug (Neovasculgen) to Treat Secondary Raynaud's Phenomenon
Condition: Secondary Raynaud's Phenomenon
Intervention: Drug: Neovasculgen
37 Recruiting A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects
Condition: Beta-thalassemia Major
Intervention: Genetic: LentiGlobin® BB305 Drug Product
38 Recruiting A Study of a Retroviral Replicating Vector Administered to Subjects With Recurrent Malignant Glioma
Conditions: Glioblastoma;   Anaplastic Astrocytoma;   Anaplastic Oligodendroglioma;   Anaplastic Oligoastrocytoma
Interventions: Biological: Toca 511;   Drug: 5-FC
39 Recruiting Long-Term Follow-Up of Recipient of Gene Transfer Research
Conditions: Retroviridae Infections;   Cancer
Interventions: Other: Blood Tests;   Behavioral: Questionnaires
40 Recruiting Gene Therapy for Fanconi Anemia
Condition: Fanconi Anemia
Interventions: Procedure: Harvest Procedure;   Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;   Other: Laboratory Biomarker Analysis

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