Clinical trials: Gene Therapy
Duchenne Muscular Dystrophy
AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.
- Trial name
- NCT04240314
- Testing
- scAAV9.U7.ACCA
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Megan Waldrop
Refractory B-Cell Non-Hodgkin Lymphoma, Refractory B-Cell Small Lymphocytic Lymphoma
CD19.CAR Allogeneic NKT for Patients With Relapsed or Refractory B-Cell Malignancies (ANCHOR)
This study is for patients who have lymphoma or leukemia that has come back or has not gone away after treatment. Because there is no standard treatment for this cancer, patients are being asked to volunteer for a gene transfer…
- Conditions
- Refractory B-Cell Non-Hodgkin Lymphoma, Refractory B-Cell Small Lymphocytic Lymphoma, Relapsed Adult ALL, Relapsed CLL
- Trial name
- NCT03774654
- Testing
- CD19.CAR-aNKT cells
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Baylor College of Medicine
Pancreatic Cancer
Vaccine Therapy Combined With Adjuvant Chemoradiotherapy in Treating Patients With Resected Stage I or Stage II Adenocarcinoma (Cancer) of the Pancreas
RATIONALE: Vaccines made from gene-modified pancreatic cancer cells may make the body build an immune response to kill tumor cells.
- Trial name
- NCT00084383
- Testing
- GVAX pancreatic cancer vaccine
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Achromatopsia
Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia…
- Trial name
- NCT03278873
- Testing
- Prior exposure to AAV-CNGA3 or AAV-CNGB3
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 34 people
- Sponsor
- MeiraGTx UK II Ltd
Metastatic Cancer, Metastatic Melanoma
CAR T Cell Receptor Immunotherapy Targeting VEGFR2 for Patients With Metastatic Cancer
Background: The National Cancer Institute (NCI) Surgery Branch has developed an experimental therapy for treating patients metastatic cancer that involves taking white blood cells from the patient, growing them in the laboratory…
- Conditions
- Metastatic Cancer, Metastatic Melanoma, Renal Cancer
- Trial name
- NCT01218867
- Testing
- Anti-VEGFR2 CAR CD8 plus PBL, Cyclophosphamide, Aldesleukin
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Cancer Institute (NCI)
Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation
A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy
This was a clinical study for adult participants who were recently diagnosed with acute myeloid leukemia or AML. AML is a type of cancer. It is when bone marrow makes white blood cells that are not normal.
- Trial name
- NCT02752035
- Testing
- gilteritinib, azacitidine
- Where
- 111 sites in United States, Australia and 11 more
- Volunteers
- 183 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP)…
- Trial name
- BMN 270-301 (NCT03370913)
- Testing
- valoctocogene roxaparvovec
- Where
- 49 sites in United States, Australia and 11 more
- Volunteers
- 144 people
- Sponsor
- BioMarin Pharmaceutical
VHL Gene Mutation, VHL
MK-3795 (PT2385) for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma (MK-3795-003)
The primary objective of this study is to assess the overall response rate (ORR) of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC) tumors in VHL participants treated with MK-3795.
- Conditions
- VHL Gene Mutation, VHL, VHL Syndrome, VHL Gene Inactivation
- Trial name
- NCT03108066
- Testing
- MK-3795
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Multiple Myeloma
Pomalidomide in Gene Expression Profiling (GEP)-Defined High-risk Multiple Myeloma
This is a phase II study, open-label, single institution trial of pomalidomide in GEP-defined, high-risk relapsing/refractory multiple myeloma. Prior therapy must have included lenalidomide.
- Trial name
- NCT01177735
- Testing
- Pomalidomide
- Where
- United States
- Volunteers
- 71 people
- Sponsor
- University of Arkansas
Chronic Myelomonocytic Leukemia, Chronic Myeloid Leukemia
Therapy of HES, PV, Atypical Chronic Myelocytic Leukemia (CML) or Chronic Myelomonocytic Leukemia (CMML), and Mastocytosis With Imatinib Mesylate
The goal of this clinical research study is to see if Gleevec, known as imatinib mesylate (STI571), can improve the disease condition in patients with hypereosinophilic syndrome, polycythemia vera, atypical CML or CMML with…
- Conditions
- Chronic Myelomonocytic Leukemia, Chronic Myeloid Leukemia, Polycythemia Vera, Hypereosinophilic Syndrome
- Trial name
- NCT00038675
- Testing
- Imatinib Mesylate (Gleevec)
- Where
- United States
- Volunteers
- 125 people
- Sponsor
- M.D. Anderson Cancer Center
Non Small Cell Lung Cancer, EGFR Gene Mutation
Neo-DIANA: Neoadjuvant Treatment for EGFR Mutated Patients
This is an open-label, non-randomised, phase II, multi-centre clinical trial 26 patients will be enrolled in this trial to evaluate the major pathologic response in patients with neoadjuvant treatment with Carboplatin Pemetrexed…
- Trial name
- NCT04512430
- Testing
- Atezolizumab, Bevacizumab, Carboplatin
- Where
- 18 sites in Spain
- Volunteers
- 4 people
- Sponsor
- Fundación GECP
Hemophilia A, Gene Therapy
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe…
- Conditions
- Hemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder
- Trial name
- GENEr8-AAV5+ (NCT03520712)
- Testing
- Valoctocogene Roxaparvovec
- Where
- 9 sites in South Africa, South Korea and 2 more
- Volunteers
- 3 people
- Sponsor
- BioMarin Pharmaceutical
Prostate Adenocarcinoma, Stage I Prostate Cancer
Androgen Deprivation Therapy and Vorinostat Followed by Radical Prostatectomy in Treating Patients With Localized Prostate Cancer
This phase II trial studies how well androgen deprivation therapy and vorinostat followed by radical prostatectomy works in treating patients with prostate cancer that has not spread to other parts of the body.
- Conditions
- Prostate Adenocarcinoma, Stage I Prostate Cancer, Stage IIA Prostate Cancer, Stage IIB Prostate Cancer
- Trial name
- TARGET (NCT00589472)
- Testing
- Bicalutamide, Goserelin Acetate, Laboratory Biomarker Analysis
- Where
- 15 sites in United States
- Volunteers
- 19 people
- Sponsor
- National Cancer Institute (NCI)
Spinal Muscular Atrophy
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
- Trial name
- STRONG (NCT03381729)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 11 sites in United States
- Volunteers
- 32 people
- Sponsor
- Novartis Gene Therapies
Rhinosinusitis Chronic, Microbiome
Impact of Symbiofilm On Nasal Microbiota
Healsea® chronic is CE marked medical device indicated in adults for the treatment of nasal symptoms of chronic rhinosinusitis. This is an hypertonic seawater-based nasal spray supplemented with natural Symbiofilm® extract (0.2%)…
- Trial name
- ISONAM (NCT05015530)
- Testing
- Healsea® Chronic, hypertonic seawater-based nasal spray supplemented with natural Symbiofilm® extract
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Lallemand Pharma AG
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
Pancreatic Cancer
Adjuvant GVAX Vaccine Therapy in Patients With Pancreatic Cancer
This is an open label, phase II trial study of adjuvant GVAX pancreas vaccine in patients with pancreatic cancer.
- Trial name
- NCT00389610
- Testing
- GVAX pancreas vaccine
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Skin Cancer, Melanoma
Phase II Study of Metastatic Melanoma With Lymphodepleting Conditioning and Anti-gp100:154-162 TCR Gene Engineered Lymphocytes
Background: Human peripheral blood lymphocytes have been engineered to express a T-cell receptor (TCR) that recognizes a blood type, human leukocyte antigen (HLA-A*0201) derived from the gp100 protein.
- Trial name
- NCT00509496
- Testing
- fludarabine phosphate, cyclophosphamide, aldesleukin
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- National Cancer Institute (NCI)
HIV Infection, Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma
Gene Therapy in Treating Patients With Human Immunodeficiency Virus-Related Lymphoma Receiving Stem Cell Transplant
This phase I/II trial studies the side effects and best dose of gene therapy in treating patients with human immunodeficiency virus (HIV)-related lymphoma that did not respond to therapy or came back after an original response…
- Conditions
- HIV Infection, Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma, Plasmablastic Lymphoma, Recurrent Adult Hodgkin Lymphoma
- Trial name
- NCT02797470
- Testing
- Autologous Hematopoietic Stem Cell Transplantation, Carmustine, Cytarabine
- Where
- 4 sites in United States
- Volunteers
- 11 people
- Sponsor
- AIDS Malignancy Consortium
Hemophilia A
Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
SIG-001-121 is a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adults with severe or moderately severe haemophilia A…
- Trial name
- NCT04541628
- Testing
- SIG-001
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Eli Lilly and Company