Clinical trials: Gene Therapy, active
Diabetic Macular Edema
Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME)
Phase 2 open label, randomized, active controlled, dose-ranging trial in adults with Center Involved - Diabetic Macular Edema (CI - DME)
- Trial name
- ELAAVATE (NCT06942520)
- Testing
- RGX-314 Dose 1, RGX-314 Dose 2, Aflibercept (2.0 mg)
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Sierra Eye Associates
Heart Failure With Reduced Ejection Fraction
Study Assessing Left Ventricular Administration of a Genetic Medicine Directing Organ Regeneration in Heart Failure
This clinical trial investigates the safety and preliminary effectiveness of YAP101, a gene therapy designed to improve heart function in adults with ischemic heart failure and reduced ejection fraction (HFrEF).
- Trial name
- SALVADOR-HF (NCT06831825)
- Testing
- YAP101 (AAV9-Sav-shRNA)
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Medley Therapeutics
SMARD1, CMT2S
Gene Therapy for IGHMBP2-Related Diseases
Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.
- Trial name
- NCT05152823
- Testing
- Gene Therapy
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Megan Waldrop
High Grade Glioma, MGMT-Unmethylated Glioblastoma
DB107-RRV, DB107-FC, and Radiation Therapy With or Without Temozolomide (TMZ) for High Grade Glioma
This is a multicenter, open-label study of DB107-RRV (formerly Toca 511) and DB107-FC (formerly Toca FC) when administered following surgical resection in newly diagnosed High Grade Glioma (HGG) patients.
- Conditions
- High Grade Glioma, MGMT-Unmethylated Glioblastoma, MGMT-Methylated Glioblastoma
- Trial name
- NCT06504381
- Testing
- DB107-RRV, DB107-FC, Radiation Therapy (RT)
- Where
- 5 sites in United States
- Volunteers
- 70 people
- Sponsor
- University of California, San Francisco
Inherited Retinal Diseases
Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy
The goal of this clinical trial is to evaluate the safety and efficacy of PUMCH-E101 injection in subjects with RDH12 retinopathy.
- Trial name
- NCT06749639
- Testing
- PUMCH-E101 Injection（Low dose）, PUMCH-E101 Injection（High dose）
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Peking Union Medical College Hospital
Refractory Hypercholesterolemia, Familial Hypercholesterolemia
Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT006 in patients with refractory Hypercholesterolemia diagnosed by gene testing…
- Trial name
- NCT06293729
- Testing
- NGGT006
- Volunteers
- 9 people
- Sponsor
- Suzhou Municipal Hospital
Huntington Disease
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.
- Trial name
- NCT06826612
- Testing
- SPK-10001
- Where
- 5 sites in United States
- Volunteers
- 53 people
- Sponsor
- Hoffmann-La Roche
Cancer, Cancer Gene Mutation
INTERogating Cancer for Etiology, Prevention and Therapy Navigation
This study is being done to identify markers and causes of cancer by analyzing patient's DNA (i.e., genetic material), RNA, plasma, tissues, or other samples that could be informative for patients with cancer.
- Conditions
- Cancer, Cancer Gene Mutation, PAN Gene Mutation, Hematopoietic and Lymphoid System Neoplasm
- Trial name
- INTERCEPTioN (NCT06008392)
- Testing
- Pan-genomic Testing
- Where
- 5 sites in United States
- Volunteers
- 500 people
- Sponsor
- Mayo Clinic
Brain Tumor Adult
Novel Gamma-Delta (γδ)T Cell Therapy for Treatment of Patients With Newly Diagnosed Glioblastoma
This study is being conducted to find out if the safety and tolerability of an experimental cell therapy is safe to administer to patients with a newly diagnosed glioblastoma multiforme (GBM) in combination with temozolomide…
- Trial name
- DRI (NCT04165941)
- Testing
- DRI cell therapy
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Retinitis Pigmentosa (RP), Usher Syndrome Type 2
Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
The purpose of this Phase 2 Open-label study is to evaluate the long-term safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in participants with Retinitis Pigmentosa due to mutations in exon 13…
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- CAPELLA (NCT07796646)
- Testing
- No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen
- Volunteers
- 81 people
- Sponsor
- Laboratoires Thea
Nephrotic Syndrome
INSIGHT (Insight Into Nephrotic Syndrome)
INSIGHT is a longitudinal study of childhood nephrotic syndrome to determine genetic, serologic and environmental factors contributing to nephrotic syndrome and disease progression.
- Trial name
- INSIGHT (NCT01605266)
- Where
- 4 sites in Canada
- Volunteers
- 1,000 people
- Sponsor
- The Hospital for Sick Children
Recurrent High Grade Gliomas, Glioblastoma (GBM)
A Study of SRN-101 Gene Therapy in Adults With Recurrent High-Grade Glioma (ALERT-1)
ALERT-1 is a Phase 1/2 study evaluating the safety and preliminary anti-tumor activity of SRN-101, an investigational gene therapy, in adults with recurrent high-grade glioma. High-grade glioma is a type of brain cancer.
- Conditions
- Recurrent High Grade Gliomas, Glioblastoma (GBM), Astrocytoma, Oligodendroglioma
- Trial name
- ALERT-1 (NCT07902037)
- Testing
- SRN-101
- Volunteers
- 40 people
- Sponsor
- Siren Biotechnology
Hemophilia A
Lentiviral FVIII Gene Therapy
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene…
- Trial name
- NCT03217032
- Testing
- YUVA-GT-F801
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
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
Chronic Pelvic Pain Syndrome (CPPS), Radiation-Induced Disorder
Gene and Molecular Pathways of Ozone Treatment Response in Gynecological Tumor Patients With Chronic Pelvic Pain Secondary to Cancer Treatment
Gynecological cancers, including those affecting the ovaries, uterus, and cervix, represent a significant health burden for women.
- Conditions
- Chronic Pelvic Pain Syndrome (CPPS), Radiation-Induced Disorder, Radiotherapy Side Effects, Side Effect of Chemotherapy
- Trial name
- OzoGynEpigen (NCT06816095)
- Where
- Spain
- Volunteers
- 40 people
- Sponsor
- Bernardino Clavo, MD, PhD
CAR T Cell, CAR T Cell Therapy
Long-Term Follow-Up of Subjects Treated With Seattle Children's Therapeutics Gene Therapy Products
Subjects exposed to gene therapy products (GTPs) may be at risk for delayed or long-term adverse events. This is a long-term follow-up (LTFU) protocol designed to evaluate the long-term safety of Seattle Children's Therapeutics…
- Trial name
- LTFU-01 (NCT07424157)
- Testing
- Long term follow up in all patients who received an SCTx gene therapy product
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Seattle Children's Hospital
Parkinson's Disease (PD), Early Onset Parkinson Disease
AAV2-hAADC for Parkinson's Disease (PDCS-01)
This study is looking at whether a gene therapy called AAV2-hAADC is safe and may help people with Parkinson's Disease. AAV2-hAADC is intended to increase the levels of dopamine in your brain.
- Trial name
- PDCS-01 (NCT07267065)
- Testing
- AAV2-hAADC
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Russell Lonser
Hemophilia A
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.
- Trial name
- GENEr8-JPN (NCT06224907)
- Testing
- Valoctocogene roxaparvovec
- Where
- 4 sites in Japan
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Mesothelioma, Neoplasms
Anti-Mesothelin TNaive/SCM hYP218 (TNhYP218) CAR T Cells in Participants With Mesothelin-Expressing Solid Tumors Including Mesothelioma
Background: Mesothelioma is an aggressive cancer that grows in the linings of the body; this can include the membranes that line the heart, lungs, and internal organs.
- Conditions
- Mesothelioma, Neoplasms, Stomach Neoplasms, Pancreatic Neoplasms
- Trial name
- NCT06885697
- Testing
- cyclophosphamide, fludarabine, TNhYP218 CAR T Cells
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- National Cancer Institute (NCI)
ALL, Adult, AML, Adult
A Study of BN104 in the Treatment of Acute Leukemia
The Phase I/II trial is to learn the safety, pharmacokinetics, and preliminary efficacy of BN104 taken once daily or twice daily in patients with acute lymphocytic leukemia or acute myeloblastic leukemia.
- Conditions
- ALL, Adult, AML, Adult, Acute Leukaemia, KMT2A Rearrangement
- Trial name
- NCT06052813
- Testing
- BN104 monotherapy, BN104 monotherapy - rp2d
- Where
- China
- Volunteers
- 66 people
- Sponsor
- Institut de Recherches Internationales Servier (I.R.I.S.)