Clinical trials: Gene
Hemophilia B, Hemophilia A
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
- Trial name
- NCT03587116
- Testing
- Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 212 people
- Sponsor
- Pfizer
Hemophilia B
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.
- Trial name
- NCT02618915
- Testing
- DTX101
- Where
- 9 sites in United States, Bulgaria and 1 more
- Volunteers
- 6 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Diabetes Mellitus, Type 2, Endothelial Dysfunction
Assessment of Dapagliflozin on Vascular Health in Patients With Type 2 Diabetes
Patients with Type 2 Diabetes Mellitus (T2DM) have changes in blood vessel health that can lead to a higher chance of developing heart attacks or strokes.
- Trial name
- SFRNDM2 (NCT05139914)
- Testing
- Dapagliflozin
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Boston University
Lymphoma, Mantle Cell, Mantle Cell Lymphoma
EPOCH-R Chemotherapy Plus Bortezomib to Treat Mantle Cell Lymphoma
This study will evaluate the effectiveness of etoposide, prednisone, vincristine, cyclophosphamide, doxorubicin-rituximab (EPOCH-R) chemotherapy plus bortezomib for treating mantle cell lymphoma, a cancer of white blood cells…
- Trial name
- NCT00114738
- Testing
- Rituximab (R), EPOCH, Bortezomib (B)
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- National Cancer Institute (NCI)
Actinic Keratosis
Gene Expression in Renal Transplant Patients With Field Actinic Keratosis Undergoing Metvix® Photodynamic Therapy (PDT)
The aim of this study is to determine possible molecular changes on large scale gene expression profiling after treatment with Metvix photodynamic therapy (PDT) of actinic keratoses (AK) and cancerised field in renal transplant…
- Trial name
- NCT01000636
- Testing
- Metvix PDT
- Where
- United Kingdom
- Volunteers
- 9 people
- Sponsor
- Galderma R&D
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
Prostate Cancer
Randomized Trial of Suicide Gene Therapy and Prostate Cancer
This is a randomized, controlled trial that will test the hypothesis that replication-competent adenovirus-mediated suicide gene therapy in combination with 80 Gy intensity modulated radiotherapy (IRMT)will improve freedom from…
- Trial name
- ReCAP (NCT00583492)
- Testing
- Ad5-yCD/mutTKSR39rep-ADP, IMRT
- Where
- 2 sites in United States
- Volunteers
- 44 people
- Sponsor
- Henry Ford Health System
NSCLC (Non-small Cell Lung Cancer)
A Study Of Crizotinib Versus Chemotherapy In Previously Untreated ALK Positive East Asian Non-Small Cell Lung Cancer Patients
This is a Phase III, Randomized, Open-label, Efficacy and Safety Study of Crizotinib single agent versus Chemotherapy Regimens (Pemetrexed/Cisplatin or Pemetrexed/Carboplatin) in First-Line ALK (Anaplastic Lymphoma Kinase)…
- Trial name
- NCT01639001
- Testing
- Crizotinib, Pemetrexed/Cisplatin, Pemetrexed/Carboplatin
- Where
- 47 sites in China, Hong Kong and 3 more
- Volunteers
- 207 people
- Sponsor
- Pfizer
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
Alcohol Use Disorder
Naltrexone for Individuals of East Asian Descent
This study will elucidate the pharmacogenetic effects of the Asn40Asp SNP of the OPRM1 gene on biobehavioral and neural markers of response to naltrexone in individuals of East Asian descent, an ethnic group most likely to…
- Trial name
- NCT02026011
- Testing
- Naltrexone
- Where
- United States
- Volunteers
- 87 people
- Sponsor
- University of California, Los Angeles
Neoplasms Malignant
Studying An Investigational Drug Crizotinib (PF-02341066) In Non Non-Small Cell Lung Cancer Tumors That Are Positive For Anaplastic Lymphoma Kinase (ALK)
This is a Phase 1 trial evaluating the safety and efficacy of crizotinib in patients with tumors except non-small cell lung cancer that are positive for ALK.
- Trial name
- NCT01121588
- Testing
- Crizotinib
- Where
- 21 sites in United States, China and 5 more
- Volunteers
- 44 people
- Sponsor
- Pfizer
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
Oncology, MET Gene Amplification
Sym015 (Anti-MET) in Patients With Advanced Solid Tumor Malignancies
This is the first study to test Sym015 in humans. The primary purpose of this study is to see if Sym015 is safe and effective for patients with advanced solid tumor malignancies without available therapeutic options.
- Conditions
- Oncology, MET Gene Amplification, NSCLC, MET Gene Mutation
- Trial name
- NCT02648724
- Testing
- Sym015
- Where
- 27 sites in United States, Denmark and 4 more
- Volunteers
- 57 people
- Sponsor
- Symphogen A/S
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