Clinical trials: Gene Therapy
Parkinson's Disease
A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
The objective of this clinical trial was to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of…
- Trial name
- NCT05603312
- Testing
- AAV-GAD Low Dose, AAV-GAD High Dose
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- MeiraGTx, LLC
Metastatic Cancer, Metastatic Renal Cancer
MAGE-A3/12 Metastatic Cancer Treatment With Anti-MAGE-A3/12 TCR-Gene Engineered Lymphocytes
Background: MAGE-A3/12 is a type of protein commonly found on certain types of cancer cells, particularly in metastatic cancer. Researchers have developed a process to take lymphocytes (white blood cells) from cancer patients…
- Conditions
- Metastatic Cancer, Metastatic Renal Cancer, Metastatic Melanoma
- Trial name
- NCT01273181
- Testing
- PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood Lymphocytes, Aldesleukin, Cyclophosphamide
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- National Cancer Institute (NCI)
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Leber Congenital Amaurosis
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene. Funding Source - FDA OOPD
- Trial name
- NCT00749957
- Testing
- rAAV2-CB-hRPE65
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Beacon Therapeutics
Critical Limb Ischemia, Vascular Diseases
A Phase IIB Pilot Study of a Modified Dosage Regimen of AMG0001 in Subjects With Critical Limb Ischemia
The purpose of the study is to confirm the feasibility of study procedures and the tolerability of a new dose regimen of AMG0001 in subjects with Critical Limb Ischemia (CLI)
- Conditions
- Critical Limb Ischemia, Vascular Diseases, Peripheral Arterial Disease
- Trial name
- NCT02016755
- Testing
- HGF Plasmid
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- AnGes USA, Inc.
Hemophilia B
Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
The purpose of the study is to evaluate the safety, tolerability and effect on FIX antigen and activity levels of ascending doses of SB-FIX.
- Trial name
- NCT02695160
- Testing
- SB-FIX
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Sangamo Therapeutics
Hypercholesterolemia
A Study of Alirocumab in Participants With Autosomal Dominant Hypercholesterolemia (ADH) and Gain-of-Function Mutations (GOFm) of the Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Gene or Loss-of-Function Mutations (LOFm) of the Apolipoprotein (Apo) B Gene
The primary objective of the study is to assess the pharmacodynamic (PD) effect of alirocumab on serum low density lipoprotein cholesterol (LDL-C) during 14 weeks of subcutaneous (SC) administered alirocumab in patients with…
- Trial name
- NCT01604824
- Testing
- Alirocumab
- Where
- 4 sites in United States, France
- Volunteers
- 23 people
- Sponsor
- Regeneron Pharmaceuticals
Lysosomal Storage Diseases, Cystinosis
Stem Cell Gene Therapy for Cystinosis
This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis.
- Trial name
- NCT03897361
- Testing
- CTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of California, San Diego
Leber's Congenital Amaurosis
Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.
- Trial name
- NCT03140969
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 11 people
- Sponsor
- Laboratoires Thea
Neoplasms, Solid Tumors
My Pathway: A Study Evaluating Herceptin/Perjeta, Tarceva, Zelboraf/Cotellic, Erivedge, Alecensa, and Tecentriq Treatment Targeted Against Certain Molecular Alterations in Participants With Advanced Solid Tumors
This multicenter, non-randomized, open-label study will evaluate the efficacy and safety of six treatment regimens in participants with advanced solid tumors for whom therapies that will convey clinical benefit are not available…
- Conditions
- Neoplasms, Solid Tumors, Biliary Cancer, Salivary Cancer
- Trial name
- NCT02091141
- Testing
- Trastuzumab, Pertuzumab, Erlotinib
- Where
- 60 sites in United States
- Volunteers
- 673 people
- Sponsor
- Genentech, Inc.
Advanced Malignant Solid Neoplasm, Cholangiocarcinoma
Infigratinib for the Treatment of Advanced or Metastatic Solid Tumors in Patients With FGFR Gene Mutations
This phase II trial studies how well infigratinib works in treating solid tumors that have spread to other places in the body (advanced or metastatic) in patients with FGFR gene mutations such as FGFR1-3 gene fusions or other…
- Conditions
- Advanced Malignant Solid Neoplasm, Cholangiocarcinoma, Metastatic Malignant Solid Neoplasm, Refractory Malignant Solid Neoplasm
- Trial name
- NCT04233567
- Testing
- Infigratinib
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Sameek Roychowdhury
Gaucher Disease, Type 1
A Gene Therapy Study in Patients With Gaucher Disease Type 1
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1.
- Trial name
- GALILEO-1 (NCT05324943)
- Testing
- FLT201
- Where
- 10 sites in United States, Brazil and 4 more
- Volunteers
- 10 people
- Sponsor
- Spur Therapeutics
Recurrent Acute Leukemia of Ambiguous Lineage, Recurrent Acute Lymphoblastic Leukemia
High Throughput Drug Sensitivity Assay and Genomics- Guided Treatment of Patients With Relapsed or Refractory Acute Leukemia
This pilot clinical trial studies the feasibility of choosing treatment based on a high throughput ex vivo drug sensitivity assay in combination with mutation analysis for patients with acute leukemia that has returned after a…
- Conditions
- Recurrent Acute Leukemia of Ambiguous Lineage, Recurrent Acute Lymphoblastic Leukemia, Recurrent Acute Myeloid Leukemia, Refractory Acute Lymphoblastic Leukemia
- Trial name
- NCT02551718
- Testing
- Chemosensitivity Assay, Cytology Specimen Collection Procedure, Gene Expression Analysis
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- University of Washington
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
Idiopathic Parkinson's Disease
Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease
The purpose of this study was to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease.
- Trial name
- NCT00985517
- Testing
- CERE-120: Adeno-Associated Virus Delivery of Neurturin
- Where
- 11 sites in United States
- Volunteers
- 57 people
- Sponsor
- Sangamo Therapeutics
Triple Negative Breast Cancer, Anthracycline-refractory TNBC
Docetaxel Chemotherapy and Pembrolizumab Plus Interleukin-12 Gene Therapy in Triple Negative Breast Cancer
The purpose of this research study is to test the safety and effectiveness of docetaxel chemotherapy and pembrolizumab plus adenoviral-mediated interleukin-12 (ADV/IL-12) gene therapy in patients with anthracycline-refractory…
- Trial name
- INTEGRAL (NCT04095689)
- Testing
- Docetaxel, Pembrolizumab, IL-12 gene therapy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- The Methodist Hospital Research Institute
Spinal Muscular Atrophy 1
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT02122952
- Testing
- AVXS-101
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novartis Gene Therapies
Mild Cognitive Impairment (MCI), Depression
Brain Aging and Treatment Response in Geriatric Depression
The proposed project will evaluate the role of neuroimaging biomarkers of brain aging (i.e., neurodegenerative and vascular brain changes) and mild cognitive impairment in the patterns of treatment response to memantine combined…
- Trial name
- NCT01902004
- Testing
- Escitalopram, Memantine
- Where
- United States
- Volunteers
- 115 people
- Sponsor
- University of California, Los Angeles
Myelodysplastic Syndrome
A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy.
Several previous studies (clinical and non-clinical) hypothesize that treatment with deferasirox causes a hematological improvement in transfused patients with low and intermediate-1 risk myelodysplastic syndrome.
- Trial name
- EXPHAR (NCT02663752)
- Testing
- Bone marrow aspirate, Deferasirox
- Where
- Belgium
- Volunteers
- 1 people
- Sponsor
- Novartis Pharmaceuticals
Erectile Dysfunction
Phase 2A Clinical Trial Evaluating the Potential Activity and Safety of hMaxi-K Gene for ED
To evaluate the safety and efficacy of a single intracavernous injection of hMaxi K (8000 µg and 16000 µg) or placebo upon penile rigidity or erection in males with erectile dysfunction longer than six months that is attributable…
- Trial name
- NCT02713789
- Testing
- hMaxi-K Single Treatment/ 2 escalating dose levels (8000 µg and 16000 µg injection)
- Where
- Kuwait
- Volunteers
- 26 people
- Sponsor
- Urovant Sciences GmbH