Clinical trials: Gene
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
Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm
Testing the Addition of an Anti-cancer Drug, Copanlisib, to the Usual Immunotherapy (Nivolumab With or Without Ipilimumab) in Patients With Advanced Solid Cancers That Have Changes in the Following Genes: PIK3CA and PTEN
This phase I/II trial studies the side effects and best dose of copanlisib when given together with nivolumab and ipilimumab and to see how well they work in treating patients with solid cancers that may have spread from where it…
- Conditions
- Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm, Unresectable Malignant Solid Neoplasm
- Trial name
- NCT04317105
- Testing
- Biopsy Procedure, Biospecimen Collection, Computed Tomography
- Where
- 6 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- National Cancer Institute (NCI)
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.
Oxidative Stress, Endothelial Dysfunction
Genes, Fibrinolysis and Endothelial Dysfunction- Dialysis Aim 3
The purpose of the study is to see how two classes of blood pressure medications,angiotensin-converting enzyme inhibitors (Ace inhibitors) and angiotensin receptor blockers (ARBs), differ in their long term effects on certain…
- Trial name
- NCT00878969
- Testing
- valsartan (ARB), ramipril (ACE inhibitor)
- Where
- United States
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
Metastatic Squamous Neck Cancer With Occult Primary Squamous Cell Carcinoma
SF1126 in Recurrent or Progressive SCCHN and Mutations in PIK3CA Gene and/or PI-3 Kinase Pathway Genes
The purpose of this study is to test the good and bad effects of an experimental drug called SF1126. This drug is being tested in patients whose cancer has not been controlled by available standard therapies and who have certain…
- Trial name
- NCT02644122
- Testing
- SF1126
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Ezra Cohen
ATM Gene Mutation, ATR Gene Mutation
Lung-MAP: Talazoparib in Treating Patients With HRRD Positive Recurrent Stage IV Squamous Cell Lung Cancer
This phase II trial studies how well talazoparib works in treating patients with homologous recombination repair deficiency (HRRD) positive stage IV squamous cell lung cancer that has come back after previous treatment.
- Conditions
- ATM Gene Mutation, ATR Gene Mutation, BARD1 Gene Mutation, BRCA1 Gene Mutation
- Trial name
- NCT03377556
- Testing
- Laboratory Biomarker Analysis, Pharmacological Study, Talazoparib
- Where
- 1,157 sites in United States, Canada
- Volunteers
- 51 people
- Sponsor
- SWOG Cancer Research Network
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
Dilated Cardiomyopathy, Lamin A/C Gene Mutation
A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
This is a randomized, double-blind, placebo-controlled study in patients with dilated cardiomyopathy (DCM) due to a mutation of the gene encoding the lamin A/C protein (LMNA).
- Trial name
- REALM-DCM (NCT03439514)
- Testing
- ARRY-371797 (PF-07265803)
- Where
- 107 sites in United States, Argentina and 8 more
- Volunteers
- 77 people
- Sponsor
- Pfizer
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
Gene Expression
Gene Expression Study Between Two Dermal Injectables Hydroxylapatite Semi-permanent Filler
To compare gene expression stimulated by a semi-permanent filler and a biostimulator via punch biopsy
- Trial name
- Sculptra (NCT05620043)
- Testing
- Sculptra, Radiesse Plus
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Galderma R&D
Clear Cell Sarcoma
QUILT-3.031: AMG 337 in Subjects With Advanced or Metastatic Clear Cell Sarcoma
This is a phase 2 study that will assess the efficacy of AMG 337 in subjects with advanced or metastatic clear cell sarcoma that contains the EWSR1-ATF1 gene fusion.
- Trial name
- NCT03132155
- Testing
- AMG 337
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- NantPharma, LLC
Lung Cancer, Non-small Cell Lung Cancer (NSCLC)
Phase I Trial of Afatinib (BIBW 2992) and Dasatinib in Non-small Cell Lung Cancer (NSCLC)
The purpose of this study is to: Find out if the study drugs Afatinib and Dasatinib can be safely given together to patients with lung cancer Learn how these two drugs work in cancer cells when they are combined Learn more about…
- Trial name
- NCT01999985
- Testing
- Dasatinib - 1A, Afatinib - 1A, Dasatinib - 1B
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
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
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT04927247
- Testing
- Inclacumab
- Where
- 59 sites in United States, Brazil and 12 more
- Volunteers
- 72 people
- Sponsor
- Pfizer
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
Allergic Rhinitis, Asthma
Development of Human Nasal Challenge Models With Microbial Constituents and Grass Pollen
The investigators propose the development of a range of nasal spray challenge models to study the way the nose can respond to different types of nasal challenge that elicit different forms of inflammation.
- Conditions
- Allergic Rhinitis, Asthma, Latent Tuberculosis
- Trial name
- NCT02090374
- Testing
- Poly ICLC, Poly I:C, R848
- Where
- United Kingdom
- Volunteers
- 93 people, healthy volunteers welcome
- Sponsor
- Imperial College London
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age)…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
- Trial name
- NCT04935879
- Testing
- Inclacumab
- Where
- 58 sites in United States, Brazil and 10 more
- Volunteers
- 241 people
- Sponsor
- Pfizer
BRCA1 Gene Mutation, BRCA2 Gene Mutation
Pembrolizumab in Treating Participants With Metastatic, Recurrent or Locally Advanced Cancer and Genomic Instability
This phase II trial studies how well pembrolizumab works in treating participants with cancer that has spread to other places in the body, has come back or has spread to nearby tissues or lymph nodes.
- Conditions
- BRCA1 Gene Mutation, BRCA2 Gene Mutation, Locally Advanced Solid Neoplasm, Metastatic Malignant Solid Neoplasm
- Trial name
- NCT03428802
- Testing
- Laboratory Biomarker Analysis, Pembrolizumab
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Rutgers, The State University of New Jersey