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.
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
Systemic Lupus Erythematosus (SLE), Cutaneous Lupus Erythematosus (CLE)
A Long-Term Extension (LTE) Study Of Enpatoran In Cutaneous Manifestations Of Lupus With Or Without Systemic Disease [Elowen-LTE]
The purpose of this study is to evaluate the long-term safety and efficacy of orally administered enpatoran in participants with cutaneous manifestations of lupus erythematosus who have completed the Elowen-1 (MS504908_0001)…
- Trial name
- NCT07840287
- Testing
- Enpatoran
- Where
- 2 sites in United States, Germany
- Volunteers
- 404 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Rett Syndrome
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
- Trial name
- NCT05898620
- Testing
- NGN-401
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 33 people
- Sponsor
- Neurogene Inc.
Acute Lymphoblastic Leukemia, Acute Myelogenous Leukemia
Safety Study of Gene Modified Donor T-cells Following Partially Mismatched Stem Cell Transplant
This study will evaluate patients with blood cell cancers who are going to have an allogeneic (donor) blood stem cell transplant from a partially matched relative.
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myelogenous Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT01744223
- Testing
- BPX-501 dose 1, Rimiducid, BPX-501 dose 2
- Where
- 8 sites in United States
- Volunteers
- 36 people
- Sponsor
- Bellicum Pharmaceuticals
Prediabetes, Diabetes Type 1
An Exploratory Clinical Trial to Generate Whole Blood Samples for Analysing Genetic Polymorphisms
G (guanine nucleotide binding) proteins associating with G protein-coupled receptors (GPCR) are key players in the pathogenesis of obesity and diabetes and are targets of pharmacotherapeutic inter-ventions.
- Conditions
- Prediabetes, Diabetes Type 1, Diabetes Type 2
- Trial name
- NCT02057497
- Volunteers
- healthy volunteers welcome
- Sponsor
- Profil Institut für Stoffwechselforschung GmbH
glp1 Agonist, Gene Expression
Ellacor® Micro-Coring Technology® in Fitzpatrick Skin Types I-VI, Including Patients Receiving Glucagon-Like Peptide-1 Receptor Agonists
The main purpose of the study is to collect information about how the device functions in a variety of treatment areas and skin types and to find out what changes, if any, occur in the skin treated with the study device compared…
- Conditions
- glp1 Agonist, Gene Expression, Skin Laxity
- Trial name
- NCT07224880
- Testing
- ellacor
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Cytrellis Biosystems, Inc.
Peripheral Arterial Disease, Peripheral Vascular Disease
Safety Study of MultiGeneAngio in Patients With Chronic Critical Limb Ischemia
The purpose of this study is to evaluate the safety and activity of two doses of MultiGeneAngio, a cell therapy product produced from the patient's own cells, as potential treatment for patients with chronic critical limb…
- Conditions
- Peripheral Arterial Disease, Peripheral Vascular Disease, Critical Limb Ischemia
- Trial name
- NCT00956332
- Testing
- MultiGeneAngio
- Where
- 7 sites in Israel
- Volunteers
- 28 people
- Sponsor
- MultiGene Vascular Systems Ltd.
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.
Healthy
The Use of Aging Biomarkers in Saliva, Based on DNA Methylation Status in Elovl2 to Predict Biological Age
There is a big change in our understanding of aging in recent years. Recent researches has revealed clock-like patterns of epigenetic change across in humans. The aging involves a timed epigenetic reprogramming.
- Trial name
- NCT03734419
- Volunteers
- healthy volunteers welcome
- Sponsor
- HKGepitherapeutics
Asthma
A Methodology Study To Look At Genes In Lung Cells And Tissue From Asthmatic Patients
The primary goal of this study is to identify a gene or panel of genes which are differentially expressed in lung cells and tissue from asthmatic patients following administration of Ipratropium bromide and eiTher nebulised…
- Trial name
- NCT00331292
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- GlaxoSmithKline
Head and Neck Cancer
Gene Therapy in Treating Patients With Recurrent Head and Neck Cancer
RATIONALE: Inserting the gene for p53 into a person's tumor may improve the body's ability to fight cancer or make the cancer more sensitive to chemotherapy.
- Trial name
- NCT00003257
- Testing
- Ad5CMV-p53 gene
- Where
- 10 sites in United States
- Volunteers
- 39 people
- Sponsor
- Aventis Pharmaceuticals
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
Retinitis Pigmentosa
A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
The purpose of this first-in-human study is to explore the maximum tolerated dose (MTD) of CPK850 as determined by the single ascending dose ranging portion of the study.
- Trial name
- NCT03374657
- Testing
- CPK850
- Where
- Sweden
- Volunteers
- 12 people
- Sponsor
- Novartis Pharmaceuticals
Solid Tumor, Adult, Intrahepatic Cholangiocarcinoma
A Study to Evaluate KIN-3248 in Participants With Advanced Tumors Harboring FGFR2 and//or FGFR3 Gene Alterations
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of KIN-3248, an oral small molecule FGFR inhibitor, in adults with advanced tumors harboring FGFR2 and/or FGFR3…
- Conditions
- Solid Tumor, Adult, Intrahepatic Cholangiocarcinoma, Urothelial Carcinoma
- Trial name
- NCT05242822
- Testing
- KIN-3248
- Where
- 21 sites in United States, China and 4 more
- Volunteers
- 54 people
- Sponsor
- Kinnate Biopharma
Vestibular Schwannoma
Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma
This trial will evaluate the safety and tolerability of a single unilateral administration of one of three dose levels of AAVAnc80-antiVEGF and will evaluate the Akouos delivery device to safely achieve the intended product…
- Trial name
- NCT06517888
- Testing
- AAVAnc80-antiVEGF via Akouos Delivery Device
- Where
- 4 sites in United States
- Volunteers
- 27 people
- Sponsor
- Akouos, Inc.
Solid Tumors Harboring NTRK Fusion
Expanded Access to Provide Selitrectinib for the Treatment of Cancers With a NTRK Gene Fusion
Selitrectinib expanded access is for minor and adult patients with cancer having a change in a particular gene (NTRK1, NTRK2, or NTRK3 gene fusion).
- Trial name
- NCT03206931
- Testing
- Selitrectinib (BAY2731954)
- Sponsor
- Bayer