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Results for “Gene Transfer”

1,111 trials

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Showing 20 of 1,111 results

Early research (Phase 1)Study completedNCT02193191
What this trial is testing

Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease

Who this might be right for
Sickle Cell Disease
Memorial Sloan Kettering Cancer Center 25
Large-scale testing (Phase 3)Active Not RecruitingNCT04281485
What this trial is testing

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Pfizer 114
Testing effectiveness (Phase 2)Study completedNCT00074997
What this trial is testing

An Efficacy and Safety Study of Autologous Cluster of Differentiation 34 (CD34+) Hematopoietic Progenitor Cells Transduced With Placebo or an Anti- Human Immunodeficiency Virus Type 1 (HIV-1) Ribozyme (OZ1) in Participants With HIV-1 Infection

Who this might be right for
HIV-1
Janssen-Cilag Pty Ltd 1
Not applicableLooking for participantsNCT07261891
What this trial is testing

Ex Vivo Evaluation of JAK-inhibitor and Gene Therapeutical Approach in JAK-STAT Related Disorders

Who this might be right for
Primary Immunodeficiency Diseases (PID)
prof. dr. Rik Schrijvers 20
Early research (Phase 1)Temporarily pausedNCT00664196
What this trial is testing

Trial of Anti-PSMA Designer T Cells in Advanced Prostate Cancer After Non-Myeloablative Conditioning

Who this might be right for
Prostate Cancer
Roger Williams Medical Center 18
Not applicableStudy completedNCT04819490
What this trial is testing

A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH

Who this might be right for
X-Linked Hypophosphataemia
Kyowa Kirin Pharmaceutical Development Ltd 32
Not applicableLooking for participantsNCT00001823
What this trial is testing

Evaluation for NCI Surgery Branch Clinical Research Protocols

Who this might be right for
Synovial Cell CancerMelanomaColorectal Cancer+2 more
National Cancer Institute (NCI) 7,000
Testing effectiveness (Phase 2)Study completedNCT01671072
What this trial is testing

Efficacy and Safety Study of TissueGene-C to Degenerative Arthritis

Who this might be right for
Degenerative Arthritis
Kolon Life Science 54
Testing effectiveness (Phase 2)Study completedNCT04455230
What this trial is testing

A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

Who this might be right for
Fabry DiseaseLysosomal Storage Diseases
Spur Therapeutics 3
Large-scale testing (Phase 3)Active Not RecruitingNCT04794101
What this trial is testing

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Who this might be right for
X-Linked Retinitis Pigmentosa
Janssen Research & Development, LLC 97
Testing effectiveness (Phase 2)Study completedNCT03374657
What this trial is testing

A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa

Who this might be right for
Retinitis Pigmentosa
Novartis Pharmaceuticals 12
Not applicableStudy completedNCT04148001
What this trial is testing

Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

Who this might be right for
Homozygous Familial Hypercholesterolemia (HoFH)
REGENXBIO Inc. 4
Early research (Phase 1)Study completedNCT02022696
What this trial is testing

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

Who this might be right for
Adenosine Deaminase DeficiencyADA-SCID
National Human Genome Research Institute (NHGRI) 1
Early research (Phase 1)WithdrawnNCT03825783
What this trial is testing

A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

Who this might be right for
Leukocyte Adhesion Defect - Type I
Rocket Pharmaceuticals Inc.
Not applicableStudy completedNCT04272554
What this trial is testing

AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)

Who this might be right for
Hemophilia BBlood Coagulation Disorders, InheritedBlood Coagulation Disorder+1 more
Spur Therapeutics 37
Early research (Phase 1)Active Not RecruitingNCT03774654
What this trial is testing

CD19.CAR Allogeneic NKT for Patients With Relapsed or Refractory B-Cell Malignancies (ANCHOR)

Who this might be right for
Refractory B-Cell Non-Hodgkin LymphomaRefractory B-Cell Small Lymphocytic LymphomaRelapsed Adult ALL+2 more
Baylor College of Medicine 13
Not applicableActive Not RecruitingNCT03432520
What this trial is testing

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

Who this might be right for
Hemophilia A
Spark Therapeutics, Inc. 29
Early research (Phase 1)Study completedNCT00494195
What this trial is testing

Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)

Who this might be right for
Muscular Dystrophies
Nationwide Children's Hospital 6
Testing effectiveness (Phase 2)Looking for participantsNCT03538899
What this trial is testing

Autologous Gene Therapy for Artemis-Deficient SCID

Who this might be right for
Severe Combined Immunodeficiency
University of California, San Francisco 24
Testing effectiveness (Phase 2)Ended earlyNCT04541628
What this trial is testing

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

Who this might be right for
Hemophilia A
Eli Lilly and Company 3
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