Clinical trials: Gene Therapy
Hodgkin Disease, Non-Hodgkins Lymphoma
Giving Gene Marked EBV Specific T-Cells to Patients Receiving a BMT for Relapsed EBV-Positive Hodgkin Disease
Subjects have a type of lymph gland cancer called Hodgkin or non-Hodgkin Lymphoma, which has come back or not gone away after treatment, including the best treatment we know for relapsed Lymphoma.
- Trial name
- ANGELA (NCT00058773)
- Testing
- Infusion of EBV Specific Cytotoxic T-Lymphocytes
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Baylor College of Medicine
Sickle Cell Disease, Pulmonary Disease
Cooperative Assessment of Late Effects for SCD Curative Therapies
Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States.
- Conditions
- Sickle Cell Disease, Pulmonary Disease, Renal Disease, Heart Disease
- Trial name
- COALESCE (NCT05153967)
- Where
- 5 sites in United States
- Volunteers
- 377 people
- Sponsor
- Vanderbilt University Medical Center
Oculocutaneous Albinism (OCA)
Safety and Efficacy of a Single Suprachoroidal Injection of JWK010 Gene Therapy in Subjects With Oculocutaneous Albinism Type 1 (OCA1)
Oculocutaneous albinism (OCA) is the most common type of albinism. People with OCA have little or no pigment (melanin) in their eyes, skin, and hair.
- Trial name
- NCT07313618
- Testing
- JWK010 gene therapy
- Where
- China
- Volunteers
- 18 people
- Sponsor
- West China Hospital
Neuroblastoma
Biological Therapy and Gene Therapy in Treating Children With Recurrent or Refractory Neuroblastoma
RATIONALE: Biological therapies use different ways to stimulate the immune system and stop cancer cell from growing. Inserting genetic material made in the laboratory into a person's blood cells may make the body build an immune…
- Trial name
- NCT00006480
- Testing
- aldesleukin, therapeutic autologous lymphocytes, chemotherapy
- Where
- 2 sites in United States
- Sponsor
- Fred Hutchinson Cancer Center
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
Gene Therapy, Hemophilia B
Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy
Gene therapy is a paradigm-shifting treatment for hemophilia B patients, particularly in resource-limited countries where factor availability remains low.
- Trial name
- NCT05044845
- Testing
- Interview
- Where
- 6 sites in United States, Nepal and 4 more
- Volunteers
- 150 people
- Sponsor
- St. Jude Children's Research Hospital
Dentatorubral-Pallidoluysian Atrophy
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide…
- Trial name
- NCT06706388
- Testing
- nL-ATN1-002
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- n-Lorem Foundation
Neuroblastoma
3RD GENERATION GD2 SPECIFIC CHIMERIC ANTIGEN RECEPTOR TRANSDUCED AUTOLOGOUS NATURAL KILLER T-CELLS FOR NEUROBLASTOMA
This research study is for patients that have a cancer called Neuroblastoma that has either come back after treatment or did not respond to the standard medicines used to treat it.
- Trial name
- GINAKIT (NCT02439788)
- Testing
- cyclophosphamide, Fludarabine, GINAKIT Cells
- Where
- United States
- Sponsor
- Baylor College of Medicine
Glucocorticoid Resistance, Gene
Association Between the Effect of Glucocorticoid Pulse Therapy on Neuromyelitis Optica (NMO) and Gene Polymorphism
Aims of this study are to observe the correlation between ABCB1、VDR、TBX21、NR3C1 and other gene polymorphisms and the effect of glucocorticoid shock therapy on neuromyelitis optica.
- Conditions
- Glucocorticoid Resistance, Gene, Neuromyelitis Optica
- Trial name
- NCT04601142
- Testing
- Methylprednisolone
- Where
- China
- Volunteers
- 350 people
- Sponsor
- Beijing Tongren Hospital
Duchene Muscular Dystrophy
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin.
- Trial name
- NCT07856329
- Testing
- GNT0004 (1x10e13 vg/kg), GNT0004 (3x10e13 vg/kg)
- Where
- 14 sites in Belgium, France and 2 more
- Volunteers
- 77 people
- Sponsor
- Genethon
Cancer
Immunotherapy Based on Tumor Associated Antigen-specific Immune Effector Cells
The primary objectives are to evaluate the safety and efficacy of infusion of autologous tumor associated antigen-specific engineered immune effector cells (EIE).
- Trial name
- NCT03535246
- Testing
- Engineered Immune Cells
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)
WAYFINDER is a Phase 1/2 study in Australia to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 6 to <84 months. The study follows an open-label, dose-escalation design.
- Trial name
- NCT06112275
- Testing
- ETX101
- Where
- Australia
- Volunteers
- 4 people
- Sponsor
- Encoded Therapeutics
Neuroblastoma
Gene Modified Allogeneic Neuroblastoma Cells For Treatment of Relapsed/Refractory Neuroblastoma
Neuroblastoma affects approximately 500 children a year in the United States. When the tumor occurs in infants, it is frequently localized and responds well to therapy.
- Trial name
- NCT00186862
- Testing
- Interleukin-2
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- St. Jude Children's Research Hospital
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)
Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
This is a historically controlled, non-randomized Phase I/II clinical trial to assess the safety and efficacy of autologous transplantation of CD34+ hematopoietic stem/progenitor cells (HSPCs), obtained from infants affected by…
- Trial name
- NCT01380990
- Testing
- Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT), Busulfan
- Where
- United Kingdom
- Volunteers
- 36 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Netherton Syndrome
Gene Therapy for Netherton Syndrome
Netherton Syndrome is a serious skin disorder caused by damage in a gene called SPINK5. This gene controls the formation of a protein called LEKTI, which important for skin barrier function.
- Trial name
- NCT01545323
- Testing
- One 20cm2/10cm2 autologous skin sheet graft
- Where
- 2 sites in United Kingdom
- Volunteers
- 5 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Severe Combined Immunodeficiency
Autologous Gene Therapy for Artemis-Deficient SCID
This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene.
- Trial name
- NCT03538899
- Testing
- AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University of California, San Francisco
Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE), Juvenile Dermatomyositis (JDM)
Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes
The Requesting Physician/Investigator contacts Lilly when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.
- Conditions
- Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE), Juvenile Dermatomyositis (JDM), Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI), Aicardi-Goutières Syndrome (AGS)
- Trial name
- NCT01724580
- Testing
- Baricitinib
- Where
- 3 sites in United States, United Kingdom
- Sponsor
- Eli Lilly and Company
Aromatic L-amino Acid Decarboxylase Deficiency
A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency
This trial includes dose-escalating part (phase 1) and dose confirming part, to prove the safety and efficacy of VGN-R09b to treat patients with severe AADC deficiency
- Trial name
- NCT06432140
- Testing
- VGN-R09b injection
- Where
- China
- Volunteers
- 13 people
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.