Clinical trials: Gene Therapy
Immunotherapy
Study Evaluating the Efficacy of a Double Immunotherapy Combined With Olaparib in Patients With Solid Cancers and Carriers of Homologous Recombination Repair Genes After Olaparib Treatment
The study propose to generate a clinical trial based on precision medicine to evaluate the use of immunotherapy in patients with altered homologous recombination repair genes and without progression after prior targeted therapy.
- Trial name
- GUIDE2REPAIR (NCT04169841)
- Testing
- olaparib, durvalumab, tremelimumab
- Where
- 8 sites in France
- Volunteers
- 270 people
- Sponsor
- Centre Georges Francois Leclerc
Lung Cancer, Gene Abnormality
iCAGES-guided Precision Therapy for Cancers in Contrast to Standard Care or IHC-guided Theray
This study prospectively evaluates whether the use of iCAGES (integrated CAncer GEnome Score) tool in guiding the treatment of advanced cancers is superior to current standard care or IHC-guided therapy in progress free survival…
- Conditions
- Lung Cancer, Gene Abnormality, Gene Product Sequence Variation, Cancer
- Trial name
- NCT03192501
- Testing
- inhibitors, ADC drugs such as tarceva, capmatinib, padcev et al for A/C group.
- Where
- China
- Volunteers
- 250 people
- Sponsor
- Second Affiliated Hospital of Guangzhou Medical University
Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
- Trial name
- NCT05693142
- Testing
- RGX-202
- Where
- 23 sites in United States, Canada
- Volunteers
- 65 people
- Sponsor
- REGENXBIO Inc.
Lung Cancer
Vaccine Therapy in Treating Patients With Stage IIIB, Stage IV, or Recurrent Non-Small Cell Lung Cancer
RATIONALE: Vaccines made from a gene-modified virus may help the body build an effective immune response to kill tumor cells. PURPOSE: This phase I trial is studying the side effects and best dose of vaccine therapy in treating…
- Trial name
- NCT00601094
- Testing
- autologous dendritic cell-adenovirus CCL21 vaccine
- Where
- 2 sites in United States
- Volunteers
- 17 people
- Sponsor
- Jonsson Comprehensive Cancer Center
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
Diabetes, Glucose Tolerance
Melatonin and Glucose Tolerance Among Individuals With a Variant of the MTNR1B Gene
The effect of 3 months of melatonin 4mg treatment on glucose tolerance in different variants of the melatonin receptor 1B gene will be evaluated.
- Trial name
- MELAG (NCT01705639)
- Testing
- Melatonin
- Where
- 2 sites in Finland
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- University of Helsinki
Ischemic Stroke
Safety and Preliminary Efficacy of NXL-001 in Patients With Ischemic Stroke
This is a single-arm, open-label, single center, dose-escalation exploratory clinical study to evaluate the safety and tolerability of a single intracerebral injection of NXL-001, a NeuroD1 base gene therapy, in patients with…
- Trial name
- NXL-001 (NCT06761183)
- Testing
- intracerebral stereotactic injection of NXL-001
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Beijing Tiantan Hospital
Severe Combined Immunodeficiency Due to ADA Deficiency
A Clinical Study to Enable Process Validation of Commercial Grade OTL-101
The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process.
- Trial name
- NCT04140539
- Testing
- OTL-101
- Where
- United States
- Sponsor
- University of California, Los Angeles
Autologous Stem Cell Transplantation, Allogeneic Stem Cell Transplantation
CIBMTR Research Database
The primary purpose of the Research Database is to have a comprehensive source of observational data that can be used to study HSC transplantation and cellular therapies.
- Conditions
- Autologous Stem Cell Transplantation, Allogeneic Stem Cell Transplantation, Solid Tumors, Blood Cancers
- Trial name
- NCT01166009
- Where
- 2 sites in United States
- Volunteers
- 99,999,999 people
- Sponsor
- Center for International Blood and Marrow Transplant Research
Gaucher Disease Type 1
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate…
- Trial name
- GD (NCT06818838)
- Testing
- LY-M001
- Where
- 3 sites in China
- Volunteers
- 12 people
- Sponsor
- Lingyi Biotech Co., Ltd.
Recessive Dystrophic Epidermolysis Bullosa
Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB.
Prospective open-label, uncontrolled clinical study to assess the safety and efficacy of autologous cultured epidermal grafts containing epidermal stem cells genetically modified with the aid of a gamma-retroviral vector carrying…
- Trial name
- HOLOGENE7 (NCT02984085)
- Testing
- Genetically corrected cultured epidermal autograft (ATMP)
- Where
- Austria
- Volunteers
- 3 people
- Sponsor
- Holostem s.r.l.
Non Small Cell Lung Cancer, EGFR Gene Mutation
Osimertinib and Etoposide as First-Line Treatment in Osimertinib-Resistant Advanced EGFR-Mutant NSCLC
Osimertinib, though a standard first-line treatment for EGFR-mutant advanced NSCLC, shows primary resistance in 10-30% of patients, leading to disease progression within 3-4 months.
- Conditions
- Non Small Cell Lung Cancer, EGFR Gene Mutation, Non Small Cell Lung Cancer Stage IIIB, Non-small Cell Lung Cancer Stage IV
- Trial name
- NCT06436144
- Testing
- Osimertinib
- Volunteers
- 93 people
- Sponsor
- Daping Hospital and the Research Institute of Surgery of the Third Military Medical University
Lymphoma, Non-Hodgkin, HIV Infections
Gene Therapy in HIV-Positive Patients With Non-Hodgkin's Lymphoma
The purpose of this study is to see if it is safe and effective to use gene therapy to treat non-Hodgkin's lymphoma (NHL) in HIV-positive patients. Stem cell transplantation is a procedure used to treat NHL.
- Trial name
- NCT00002221
- Testing
- Peripheral blood stem cells
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Ribozyome
Refractory Metastatic Colorectal Cancer, pMMR
RP2/RP3 in Combination With Atezolizumab and Bevacizumab for the Treatment of Patients With CRC
This is an open-label, Phase 2 clinical trial evaluating therapy with an oncolytic immunotherapy (RP2 or RP3) in combination with atezolizumab and bevacizumab in patients with advanced Microsatellite Stable and Mismatch Repair…
- Conditions
- Refractory Metastatic Colorectal Cancer, pMMR, MSS
- Trial name
- NCT05733611
- Testing
- RP2, RP3, atezolizumab
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Replimune, Inc.
Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2)
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)
- Conditions
- Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases
- Trial name
- PHYOX2 (NCT03847909)
- Testing
- DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
- Where
- 28 sites in United States, Australia and 13 more
- Volunteers
- 35 people
- Sponsor
- Novo Nordisk A/S
Arrhythmogenic Cardiomyopathy, PKP2-ACM
Gene Therapy for ACM Due to a PKP2 Pathogenic Variant
This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM
- Conditions
- Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
- Trial name
- NCT06109181
- Testing
- LX2020
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Lexeo Therapeutics
Prostate Cancer, Castration-resistant Prostate Cancer
Docetaxel Versus Abiraterone as First-line Treatment in mCRPC Patients With Intraductal Carcinoma of the Prostate
Metastatic castration-resistant Prostate cancer (mCRPC) is a very late stage of prostate cancer with poor prognosis. Although there are several treatment strategies available for mCRPC, these drugs are not always effective for…
- Conditions
- Prostate Cancer, Castration-resistant Prostate Cancer, Drug
- Trial name
- NCT03356444
- Testing
- Abiraterone Acetate, Docetaxel
- Volunteers
- 140 people
- Sponsor
- West China Hospital
Diabetes, Type I
A First-in-human Clinical Trial Using a Gene Therapy With Patient's Own Stem Cells to Treat Early Type 1 Diabetes
Purpose: The purpose of the trial is to assess the safety profile of the study treatment and to evaluate its efficacy in terms of improvement in key diabetes management parameters, including insulin requirements and β-cell…
- Trial name
- IMMUNOSTEM (NCT06938334)
- Testing
- Autologous CD34+ cell enriched population containing HSPCs transduced ex vivo using a LVV encoding the hPD-L1 DNA
- Where
- Italy
- Volunteers
- 15 people
- Sponsor
- Altheia Science
Lung Squamous Cell Carcinoma Stage IV, Nonsquamous Nonsmall Cell Neoplasm of Lung
Trial of Radiation and Gene Therapy Before Nivolumab for Metastatic Non-Small Cell Lung Carcinoma and Uveal Melanoma
This is a Phase II trial to determine the efficacy and safety of in situ gene therapy and stereotactic body radiation therapy (SBRT) used as a window of opportunity treatment before nivolumab in patients with metastatic squamous…
- Conditions
- Lung Squamous Cell Carcinoma Stage IV, Nonsquamous Nonsmall Cell Neoplasm of Lung, Metastatic Uveal Melanoma
- Trial name
- ENSIGN (NCT02831933)
- Testing
- ADV/HSV-tk, Valacyclovir, SBRT
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Eric Bernicker, MD
Colorectal Cancer
Panitumumab and RAS, Diagnostically-useful Gene Mutation for mCRC
The purpose of this study is to verify the efficacy of mFOLFOX6 + panitumumab combination therapy and mFOLFOX6 + bevacizumab combination therapy in first-line treatment of chemotherapy-naive patients with KRAS/NRAS wild-type…
- Trial name
- PARADIGM (NCT02394795)
- Testing
- oxaliplatin (OXA), levofolinate calcium (l-LV), 5-FU, panitumumab, oxaliplatin (OXA), levofolinate calcium (l-LV), 5-FU, bevacizumab
- Where
- 155 sites in Japan
- Volunteers
- 823 people
- Sponsor
- Takeda