Clinical trials: Gene Therapy
Esophageal Cancer
Cologne Esophageal Response Prediction Study (CERP-Study)
RATIONALE: The prognosis of patients with advanced esophageal cancer may be improved by preoperative chemoradiation. But only those patients have a benefit from this additional therapy, whose tumor shows a response after…
- Trial name
- CERP (NCT00953511)
- Testing
- ERCC1 pathways analysis
- Where
- Germany
- Volunteers
- 80 people
- Sponsor
- Elfriede Bollschweiler
Urinary Bladder Neoplasms
A Study of Stimulator of Interferon Genes (STING) Agonist E7766 in Non-muscle Invasive Bladder Cancer (NMIBC) Including Participants Unresponsive to Bacillus Calmette-Guerin (BCG) Therapy, INPUT-102
This is an open label, multicenter, phase 1/1b study to assess safety/tolerability and preliminary clinical activity of E7766 as a single agent administered intravesically in participants with NMIBC.
- Trial name
- NCT04109092
- Testing
- E7766
- Where
- 9 sites in United States
- Sponsor
- Eisai Inc.
Endocrine/Neuroendocrine, Non-Small Cell Lung Cancer
Non-Viral TCR Gene Therapy
Background: A person s white blood cells can be modified in a lab to recognize certain changes in their tumor. Many of these cells are collected from the person, modified, then given back to the person.
- Conditions
- Endocrine/Neuroendocrine, Non-Small Cell Lung Cancer, Breast Cancer, Gastrointestinal/Genitourinary Cancers
- Trial name
- NCT04102436
- Testing
- Fludarabine, Cyclophosphamide, Aldesleukin
- Where
- United States
- Sponsor
- National Cancer Institute (NCI)
NSCLC (Non-small Cell Lung Cancer), Neoadjuvant Immunotherapy
A Prospective, Single-arm, Phase II Clinical Study of Tislelizumab Combined With Anlotinib and Platinum-based Doublet Perioperative Therapy for Resectable Stage II-IIIB Driver Gene-negative NSCLC
The goal of this clinical trial is to learn if tislelizumab in combination with anlotinib and platinum-based doublet chemotherapy works to treat for resectable stage II-IIIB driver gene-negative NSCLC.
- Conditions
- NSCLC (Non-small Cell Lung Cancer), Neoadjuvant Immunotherapy, Adjuvant Immunotherapy
- Trial name
- NCT06977828
- Testing
- tislelizumab in combination with anlotinib and platinum-based doublet chemotherapy
- Where
- China
- Volunteers
- 40 people
- Sponsor
- The First Affiliated Hospital with Nanjing 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.
Retinitis Pigmentosa (RP)
Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection)
This is a Phase 1/2, multi-center, randomized, sham-controlled, dose-escalation study evaluating ZM-02 in patients with advanced retinitis pigmentosa (RP).
- Trial name
- PRISM (NCT07282457)
- Testing
- ZM-02 (low dose), ZM-02 (high dose), ZM-02 (selected dose)
- Volunteers
- 21 people
- Sponsor
- Zhongmou Therapeutics
Multiple Myeloma, Triple Negative Breast Cancer
Systemic Anti-Cancer Therapy Dose Modifications for Individuals With Duffy Null Phenotype
This study is comprised of a main study, an observational study, and optional survey studies. The main study is being done to see whether using Duffy null specific treatment dosing guidelines can reduce or delay dose…
- Conditions
- Multiple Myeloma, Triple Negative Breast Cancer, Duffy Blood Group, Chemokine Receptor Gene Mutation, Duffy Blood Group, Chemokine Receptor Gene C.-67T>C
- Trial name
- NCT07341867
- Testing
- Dexamethasone, Bortezomib, LENALIDOMIDE
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Andrew Hantel, MD
Bladder Cancer, Recurrent Bladder Cancer
A Study of Oral Erdafitinib in People With Recurrent Non-Invasive Bladder Cancer
The purpose of this study is to assess the effectiveness of erdafitinib in people with non-muscle invasive bladder cancer (NMIBC) that has come back after standard treatment, such as Bacillus Calmette-Guerin (BCG) or chemotherapy…
- Conditions
- Bladder Cancer, Recurrent Bladder Cancer, FGFR3 Gene Mutation
- Trial name
- NCT04917809
- Testing
- Erdafitinib
- Where
- 7 sites in United States
- Volunteers
- 20 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Hemophilia A, Hemophilia B
Liver Biopsy Following Gene Therapy For Hemophilia
This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have…
- Trial name
- NCT05932914
- Testing
- Liver Biopsy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- St. Jude Children's Research Hospital
Parkinson Disease, Parkinson's Disease
A Long-Term Follow-up Study of the Severe Parkinson's Disease Patients Administered the IPS101A Gene Therapy Product.
The purpose of this study is to evaluate the long-term safety of IPS101A and to assess the durability of efficacy in subjects who received IPS101A.
- Trial name
- NCT07629115
- Where
- South Korea
- Volunteers
- 6 people
- Sponsor
- Innopeutics Corporation
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.
Hyperlipidemia
Studies of Apolipoprotein Genotyping on the Drug Treatment of Hyperlipidemic Patients
Cardiovascular-related diseases have been the majorities of the leading ten causes of death in Taiwan. Atherosclerotic cardiovascular diseases are multifactorial. Some non-modifiable risk factors (e.g.
- Trial name
- NCT00451464
- Where
- Taiwan
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Non-small Cell Lung Cancer Metastatic
Low-dose Gemcitabine Combined With Nivolumab for Second-line and Above Line Treatment of NSCLC
In recent years, immunotherapy research has made great progress, especially the immunocheckpoint inhibitors represented by anti-pd-1 antibody have shown good efficacy in the treatment of malignant tumors, and some patients can…
- Trial name
- NCT04331626
- Testing
- Gemcitabine Injectable Product
- Volunteers
- 50 people
- Sponsor
- Henan Cancer Hospital
Multiple Myeloma
2015-09: a Phase II Randomized, Open-label Study of Anti-signaling Lymphocytic Activation Molecule Monoclonal Antibody During Maintenance Therapy
This study will compare the effectiveness and safety of maintenance therapy with continuous bortezomib, lenalidomide, and dexamethasone (VRD) compared to maintenance therapy that alternates VRD with Elotuzumab, lenalidomide, and…
- Trial name
- NCT03000634
- Testing
- Elotuzumab, Bortezomib, Lenalidomide
- Sponsor
- University of Arkansas
Neovascular Age-Related Macular Degeneration (nAMD) Wet AMD
Ixoberogene Soroparvovec (Ixo-vec) Contralateral Dosing Study in Participants With Neovascular Age-related Macular Degeneration
The purpose of this study is to evaluate safety, effectiveness and durability of a gene therapy called Ixo-vec (Ixoberogene soroparvovec) when administered to the contralateral (second) eye of adult participants (≥ 50 years of…
- Trial name
- NCT07630649
- Testing
- Ixo-vec
- Where
- 12 sites in United States
- Volunteers
- 15 people
- Sponsor
- Adverum Biotechnologies, Inc.
Parkinson's Disease
Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease
The objective of this study is to evaluate the long-term safety of AAV-GAD delivered bilaterally to the subthalamic nuclei (STN) in participants with Parkinson's disease.
- Trial name
- NCT05894343
- Testing
- AAV-GAD
- Where
- 6 sites in United States
- Volunteers
- 13 people
- Sponsor
- MeiraGTx, LLC
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Dry Age-related Macular Degeneration
Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59
Age related macular degeneration (AMD) is the leading cause of vision loss in individuals over age 60. AMD is classified as wet and dry.
- Trial name
- NCT03144999
- Testing
- AAVCAGsCD59
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Janssen Research & Development, LLC
Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA)
Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)
This study is an early-stage clinical trial (Phase 1b/2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare genetic eye disease that leads to…
- Trial name
- RDH12 (NCT07681778)
- Testing
- OPGx-RDH12
- Where
- 4 sites in United States
- Volunteers
- 10 people
- Sponsor
- Opus Genetics, Inc
X-Linked Chronic Granulomatous Disease
Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)
X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder, which affects boys. It is caused by an error in a gene that makes part of the immune system.
- Trial name
- CGD (NCT01855685)
- Testing
- X vivo gene therapy
- Where
- 2 sites in United Kingdom
- Volunteers
- 3 people
- Sponsor
- Genethon
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