New and Updated Clinical Gene Therapy Trials
-
Autologous Mobilized Peripheral Blood CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Transduced With the Elongation Factor Alpha Short Promoter (EFS) - Adenosine Deaminase (ADA) Gene (EFS-ADA) Lentiviral Vector for Adenosine Deaminase Severe Combined Immune Deficiency (ADA SCID)
Jun 27, 2022 | 18:00 pmCondition: Adenosine Deaminase Severe Combined Immune DeficiencyIntervention: Combination Product: A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzymeSponsor: University of California, Los AngelesNot yet recruiting
Read more... -
Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD
Jun 7, 2022 | 18:00 pmConditions: AMD; nAMD; Wet Age-related Macular Degeneration; wAMD; WetAMD; CNVInterventions: Genetic: RGX-314 Dose 1; Genetic: RGX-314 Dose 2; Biological: Aflibercept (EYLEA®)Sponsor: Regenxbio Inc.Recruiting
Read more... -
4D-710 in Adult Patients With Cystic Fibrosis
Feb 21, 2022 | 18:00 pmCondition: Cystic Fibrosis LungIntervention: Biological: 4D-710Sponsor: 4D Molecular TherapeuticsRecruiting
Read more... -
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
Nov 16, 2021 | 18:00 pmConditions: Hereditary Angioedema; HAEInterventions: Genetic: Dose 1 of BMN 331; Genetic: Dose 2 of BMN 331; Genetic: Dose 3 of BMN 331; Genetic: Dose 4 of BMN 331; Genetic: Dose 5 of BMN 331Sponsor: BioMarin PharmaceuticalRecruiting
Read more... -
T Cell Receptor Gene Therapy Targeting KK-LC-1 for Gastric, Breast, Cervical, Lung and Other KK-LC-1 Positive Epithelial Cancers
Sep 5, 2021 | 18:00 pmCondition: Kita-kyushu Lung Cancer Antigen 1, HumanInterventions: Drug: IL-2 (Aldesleukin); Drug: Cyclophosphamide; Biological: KK-LC-1 TCR; Drug: FludarabineSponsor: National Cancer Institute (NCI)Recruiting
Read more... -
Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease
May 13, 2021 | 18:00 pmCondition: Wilson DiseaseInterventions: Genetic: UX701; Other: Placebo; Drug: oral corticosteroids; Drug: Placebo for oral corticosteroidsSponsor: Ultragenyx Pharmaceutical IncRecruiting
Read more... -
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
May 16, 2019 | 18:00 pmConditions: Lysosomal Diseases; Gangliosidosis; GM1Interventions: Biological: AAV9-GLB1; Drug: Rituximab; Drug: Sirolimus; Drug: Methylprednisolone; Drug: Prednisone; Diagnostic Test: Audiology assessmentwith ABR; Diagnostic Test: Bone density scan (DEXA; Diagnostic Test: Electrocardiogram (EKG); Diagnostic Test: Echocardiogram; Other: Electroencephalogram (EEG) awake andextended overnight; Diagnostic Test: Laboratory tests; Procedure: Lumbar puncture; Procedure: Brain MRI/MRS/fMRI; Behavioral: Neurocognitive testing; Other: Neurology exam; Behavioral: PICC line placement; Procedure: Skeletal survey; Procedure: Skin biopsy; Procedure: Speech and modified barium swallow study; Procedure: Ophthalmology examSponsors: National Human Genome Research Institute (NHGRI); Sio Gene TherapiesRecruiting
Read more... -
Gene Therapy in Treating Patients With Human Immunodeficiency Virus-Related Lymphoma Receiving Stem Cell Transplant
Jun 13, 2016 | 18:00 pmConditions: HIV Infection; Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma; Plasmablastic Lymphoma; Recurrent Adult Hodgkin Lymphoma; Recurrent Adult Non-Hodgkin Lymphoma; Recurrent Burkitt Lymphoma; Recurrent Follicular Lymphoma; Stage III Follicular Lymphoma; Stage III Mantle Cell Lymphoma; Stage IV Follicular Lymphoma; Stage IV Mantle Cell LymphomaInterventions: Procedure: Autologous Hematopoietic Stem Cell Transplantation; Drug: Carmustine; Drug: Cytarabine; Drug: Etoposide; Other: Laboratory Biomarker Analysis; Biological: Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive Hematopoietic Progenitor Cells; Drug: Melphalan; Procedure: Peripheral Blood Stem Cell TransplantationSponsors: AIDS Malignancy Consortium; National Cancer Institute (NCI); California Institute for Regenerative Medicine (CIRM)Recruiting
Read more... -
Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy
Feb 13, 2015 | 18:00 pmConditions: Giant Axonal Neuropathy; Gene TransferIntervention: Genetic: scAAv9/JeT-GANSponsor: National Institute of Neurological Disorders and Stroke (NINDS)Recruiting
Read more... -
Follow-Up Study of Subjects Previously Enrolled in Poxviral Vector Gene Transfer Studies
Mar 22, 2007 | 17:00 pmConditions: Prostate Cancer; Liver Cancer; Breast Cancer; Colon Cancer; Lung CancerIntervention: Sponsor: National Cancer Institute (NCI)Recruiting
Read more...
Search Clinical Gene Therapy Trials
Quick search: