Clinical trials: Gene Therapy
Breast Cancer
Gene Expression Following Short Term Exposure to Neoadjuvant Endocrine Therapy in Invasive Breast Cancer
The purpose of this pilot study is to see if taking anastrozole (Arimidex) for 10 days causes changes in breast cancer cells. Anastrozole (Arimidex) is a drug used in the treatment of a type of breast cancer that depends on…
- Trial name
- NCT00588003
- Testing
- anastrozole
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Coronary Artery Disease
Evaluation of the GTX™ 5126 DES Coronary Stent System in the Treatment of Patients With a Lesion in the Coronary Artery
The GTX™ Drug Eluting Coronary Stent System is intended for the treatment of patients with a lesion in the coronary artery.
- Trial name
- NCT00777842
- Testing
- GTX™ Drug Eluting Coronary Stent System
- Where
- Germany
- Volunteers
- 19 people
- Sponsor
- Cook Group Incorporated
Human Papillomavirus-Related Carcinoma, Human Papillomavirus Positive Oropharyngeal Carcinoma
HPV-16/18 E6/E7-Specific T Lymphocytes, Relapsed HPV-Associated Cancers, HESTIA
Subjects have a type of cancer that has been associated with an infection with a virus called human papilloma virus (HPV). The cancer has come back, has not gone away after standard treatment or the subject cannot receive…
- Conditions
- Human Papillomavirus-Related Carcinoma, Human Papillomavirus Positive Oropharyngeal Carcinoma, Human Papillomavirus Positive Cervical Carcinoma, Human Papillomavirus Positive Anal Carcinoma
- Trial name
- HESTIA (NCT02379520)
- Testing
- HPV Specific T Cells, Cytoxan, Fludarabine
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Baylor College of Medicine
X-Linked Myotubular Myopathy
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for this serious condition.
- Trial name
- ASPIRO (NCT03199469)
- Testing
- Resamirigene bilparvovec
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 27 people
- Sponsor
- Astellas Gene Therapies
Cancer, PD-L1 Gene Mutation
Clinical Efficacy of Tumour Treatment Vaccine (TTV) Combined With PD-1 in the Treatment of Relapsed Refractory Advanced Solid Tumours
This study is intended to investigate the clinical efficacy of TTV vaccine combined with PD-1/PD-L1 inhibitor in the treatment of relapsed and refractory advanced solid tumours from a clinical perspective.
- Trial name
- NCT06430502
- Testing
- Tumor Treatment Vaccine; PD-1/L1 inhibitors
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Wuxi People's Hospital
Phelan-McDermid Syndrome, SHANK3 Haploinsufficiency
RB001 Gene Therapy Study in Children With SHANK3-related Phelan-McDermid Syndrome (PMS)
This is an open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of RB001 administered via intracerebroventricular (ICV) injection in pediatric with SHANK3 related…
- Trial name
- NCT07014020
- Testing
- RB001
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Peking University First Hospital
Acute Myelogenous Leukemia, Myelodysplasia
Study of Vaccination With Autologous Acute Myeloblastic Leukemia Cells in Patients With Advanced Myelodysplasia or Acute Myelogenous Leukemia
The purpose of this study is to test the safety of a new investigational acute myeloblastic leukemia (AML) vaccine and see what effects (good and bad) it has on patients with advanced myelodysplasia or acute myelogenous leukemia.
- Trial name
- NCT00136422
- Testing
- autologous tumor cells
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Dana-Farber Cancer Institute
Migraine
Head-to-head Study of Erenumab Against Topiramate in Patients With Episodic and Chronic Migraine
This study used a single-cohort, 2-treatment arm, parallel-group randomized, double-blind, double-dummy design in adult patients with episodic migraine and chronic migraine, who had to be either naïve or not suitable for or could…
- Trial name
- HER-MES (NCT03828539)
- Testing
- Erenumab, Topiramate
- Where
- 79 sites in Germany
- Volunteers
- 777 people
- Sponsor
- Novartis Pharmaceuticals
OTC Deficiency
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
- Trial name
- NCT05345171
- Testing
- DTX301, Oral Corticosteroids, Sodium Acetate
- Where
- 16 sites in United States, Argentina and 8 more
- Volunteers
- 37 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Endocrine Tumors, Non-Small Cell Lung Cancer
Administration of Autologous T-Cells Genetically Engineered to Express T-Cell Receptors Reactive Against Neoantigens in People With Metastatic Cancer
Background: A person s tumor is studied for mutations. When cells are found that can attack the mutation in a person s tumor, the genes from those cells are studied to find the parts that make the attack possible.
- Conditions
- Endocrine Tumors, Non-Small Cell Lung Cancer, Ovarian Cancer, Breast Cancer
- Trial name
- NCT03412877
- Testing
- Cyclophosphamide, Fludarabine, Aldesleukin
- Where
- United States
- Volunteers
- 285 people
- Sponsor
- National Cancer Institute (NCI)
Spinal Cord Injury, Chronic Spinal Cord Injury
First-in-Human Clinical Trial of STUP-001, an In Vivo Direct Cell Conversion Gene Therapy for AIS-A/B Chronic Spinal Cord Injury
This study aim to investigate the safety and exploratory efficacy of STUP-001 which is AAV-based spinal cord injury investigational product.
- Trial name
- STUP-001 (NCT06922890)
- Testing
- STUP-001
- Where
- South Korea
- Volunteers
- 9 people
- Sponsor
- Yonsei University
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing…
- Trial name
- NCT06241950
- Testing
- delandistrogene moxeparvovec, imlifidase
- Where
- Spain
- Volunteers
- 5 people
- Sponsor
- Sarepta Therapeutics, Inc.
Lymphoma, B-cell Lymphoma
CD19-specific T Cell Infusion in Patients With B-Lineage Lymphoid Malignancies
Sometimes researchers change the DNA (genetic material in cells) of donated T cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient…
- Trial name
- NCT00968760
- Testing
- Leukapheresis, Stem Cell Transplant, CD19-specific T Cell Infusion
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- M.D. Anderson Cancer Center
Non-small Cell Lung Cancer(NSCLC)
Erlotinib Monotherapy Versus Docetaxel and Cisplatin as Neoadjuvant Therapy in Patients of stageIIIA Lung ca
To compare clinical response (complete response and partial response) by RECIST) rates by RECIST between erlotinib monotherapy and docetaxel plus cisplatin chemotherapy
- Trial name
- Oncology (NCT02036359)
- Testing
- erlotinib, docetaxel
- Where
- Taiwan
- Volunteers
- 76 people
- Sponsor
- National Taiwan University Hospital
Ovarian Cancer, Peritoneal Cavity Cancer
Gene Therapy in Treating Patients With Advanced Recurrent or Persistent Ovarian Cancer or Primary Peritoneal Cancer
RATIONALE: Inserting the p53 gene into a person's cancer cells may improve the body's ability to fight cancer or make the cancer more sensitive to chemotherapy.
- Trial name
- NCT00003450
- Testing
- Ad5CMV-p53 gene
- Where
- United States
- Sponsor
- University of Texas Southwestern Medical Center
Type 2 Diabetes
R230C and C230C Variants of ABCA1 and Glyburide Response
The objective of the study is to know if the R230C and C230C variants of the ATP-binding cassette transporter A1 (ABCA1) gene are associated with a smaller glucose lowering effect compared to the wild type allele (R230R) in…
- Trial name
- NCT01456650
- Testing
- Glyburide
- Where
- Mexico
- Volunteers
- 85 people
- Sponsor
- Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
Hemophilia B
A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients
A non-randomized, open-label, dose-escalation, phase I/II study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
- Trial name
- NCT05641610
- Testing
- ZS801
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Chronic Hepatitis b
Peginterferon Alfa-2b Treatment in HBeAg(+) Chronic Hepatitis b Patients Based on Interferon Gene Mutation and Receptor Detection
The study is to observe the anti-HBV therapeutic effects of peginterferon alfa-2b in chronic hepatitis b patients with e antigen positive based on the detection of interferon gene mutation (IFNA2 p.Ala120Thr) and interferon…
- Trial name
- NCT02973646
- Testing
- Entecavir or Tenofovir, Peginterferon Alfa-2B
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Third Affiliated Hospital, Sun Yat-Sen University
Diabetic Retinopathy (DR), Center-Involved Diabetic Macular Edema (CI-DME)
RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME)
ABBV-RGX-314 is being developed as a novel, potential one-time gene therapy treatment for the treatment of Diabetic Retinopathy (DR) with and without Center-Involved Diabetic Macular Edema (CI-DME).
- Trial name
- ALTITUDE® (NCT04567550)
- Testing
- ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, ABBV-RGX-314 Dose 3
- Where
- 25 sites in United States
- Volunteers
- 139 people
- Sponsor
- AbbVie
HER-2 Gene Amplification
A Study of MM-111 in Combination With Multiple Treatments in Patients With HER2 Positive Cancer
This study is an open-label, dose-escalation study of MM-111 with five different combination treatments with the main goal of determining the safety of MM-111 with each combination.
- Trial name
- NCT01304784
- Testing
- Cisplatin, Capecitabine, Trastuzumab and MM-111, Lapatinib +/- Trastuzumab and MM-111, Paclitaxel, Trastuzumab and MM-111
- Where
- 15 sites in United States
- Volunteers
- 100 people
- Sponsor
- Merrimack Pharmaceuticals