New and Updated Clinical Gene Therapy Trials
-
Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
Feb 2, 2023 | 18:00 pmCondition: Hemophilia BIntervention: Drug: BBM-H901Sponsor: Institute of Hematology & Blood Diseases HospitalNot yet recruiting
Read more... -
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
Jan 20, 2023 | 18:00 pmCondition: Duchenne Muscular DystrophyIntervention: Genetic: RGX-202Sponsor: REGENXBIO Inc.Recruiting
Read more... -
A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)
Apr 29, 2022 | 18:00 pmCondition: Sickle Cell DiseaseIntervention: Biological: Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11aSponsors: David Williams; National Heart, Lung, and Blood Institute (NHLBI); California Institute for Regenerative Medicine (CIRM); bluebird bio; Blood and Marrow Transplant Clinical Trials NetworkRecruiting
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... -
A Study of AAV9 Gene Therapy in Participants With Canavan Disease
Aug 10, 2021 | 18:00 pmCondition: Canavan DiseaseIntervention: Biological: AAV9 BBP-812Sponsor: Aspa TherapeuticsRecruiting
Read more... -
RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)
Apr 6, 2021 | 18:00 pmConditions: Neovascular Age-related Macular Degeneration; Wet Macular Degeneration; Wet Age-related Macular DegenerationIntervention: Genetic: RGX-314Sponsor: REGENXBIO Inc.Recruiting
Read more... -
GDNF Gene Therapy for Multiple System Atrophy
Dec 22, 2020 | 18:00 pmCondition: Multiple System AtrophyInterventions: Biological: AAV2-GDNF gene therapy; Procedure: Sham (Placebo) SurgerySponsor: Brain Neurotherapy Bio, Inc.Recruiting
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; Procedure: Abdominal ultrasound; Drug: Rituximab; Drug: Sirolimus; Drug: Methylprednisolone; Drug: Prednisone; Diagnostic Test: Audiology assessment with ABR; Diagnostic Test: Bone density scan (DEXA); Diagnostic Test: Electrocardiogram (EKG); Diagnostic Test: Echocardiogram; Other: Electroencephalogram (EEG) awake and extended overnight; Diagnostic Test: Laboratory tests; Procedure: Lumbar puncture; Procedure: Brain MRI/MRS/fMRI; Behavioral: Neurocognitive testing; Other: Neurology exam; Behavioral: PICC or other Central 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 Study in Severe Hemophilia A Patients With Antibodies Against AAV5
May 11, 2018 | 18:00 pmConditions: Hemophilia A; Gene Therapy; Clotting Disorders; Blood DisorderIntervention: Biological: Valoctocogene RoxaparvovecSponsor: BioMarin PharmaceuticalRecruiting
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...