Clinical trials: Genetic Mutation
Clonal Cytopenia of Undetermined Significance (CCUS), Low-Risk Myelodysplastic Syndrome (LR-MDS)
Safety and Tolerability of REGN17235 in Adult Participants With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 Mutation
This study will test a study drug called REGN17235 (the "study drug") to see if it can help treat Clonal Cytopenia of Undetermined Significance (CCUS) and Low-Risk Myelodysplastic Syndrome (LR-MDS) with a specific genetic…
- Trial name
- NCT07753148
- Testing
- REGN17235
- Where
- 2 sites in Belgium
- Volunteers
- 52 people
- Sponsor
- Regeneron Pharmaceuticals
Cystic Fibrosis
A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes
The study evaluates the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in participants with CF who are 6 years of age and older with F/MF genotypes.
- Trial name
- NCT04545515
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 34 sites in Australia, Canada and 8 more
- Volunteers
- 120 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Arrhythmogenic Right Ventricular Cardiomyopathy
Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2…
- Trial name
- RIDGE (NCT06311708)
- Where
- 21 sites in United States, France and 4 more
- Volunteers
- 200 people
- Sponsor
- Tenaya Therapeutics
Advanced Solid Tumors, Metastatic Solid Tumors
Study of RAS(ON) Inhibitors in Combination With Ivonescimab in Patients With Solid Tumors
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary antitumor activity of RAS(ON) inhibitors in combination with ivonescimab in adults with advanced or metastatic solid tumors…
- Conditions
- Advanced Solid Tumors, Metastatic Solid Tumors, Non-small Cell Lung Cancer (NSCLC), NSCLC
- Trial name
- NCT07397338
- Testing
- Daraxonrasib, Elironrasib, Zoldonrasib
- Where
- 19 sites in United States
- Volunteers
- 370 people
- Sponsor
- Revolution Medicines, Inc.
Chronic Myeloid Leukemia
Homoharringtonine (Omacetaxine Mepesuccinate) in Treating Patients With Chronic Myeloid Leukemia (CML) With the T315I BCR-ABL Gene Mutation
To evaluate the safety and efficacy of subcutaneous administration of omacetaxine mepesuccinate (HHT) in achieving a clinical response in CML patients in chronic, accelerated, or blast phase who have failed prior imatinib therapy…
- Trial name
- NCT00375219
- Testing
- Omacetaxine mepesuccinate
- Where
- 32 sites in United States, Canada and 8 more
- Volunteers
- 103 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Non-small Cell Lung Cancer
A Confirmatory Clinical Study in NSCLC Patients With MET Exon 14 Mutation (KUNPENG-2)
The goal of this clinical trial is to test if PLB1001 works well and safely in Non-small cell lung cancer patients with MET exon 14 mutation.
- Trial name
- KUNPENG-2 (NCT05989542)
- Testing
- PLB1001
- Where
- China
- Volunteers
- 136 people
- Sponsor
- Beijing Pearl Biotechnology Limited Liability Company
Non-Small-Cell Lung Carcinoma
A Study of Nivolumab + Chemotherapy or Nivolumab + Ipilimumab Versus Chemotherapy in Non-Small Cell Lung Cancer (NSCLC) Participants With Epidermal Growth Factor Receptor (EGFR) Mutation Who Failed 1L or 2L EGFR Tyrosine Kinase Inhibitor (TKI) Therapy
The main purpose of this study is to determine whether nivolumab + chemotherapy is effective as compared to chemotherapy in the treatment of patients with EGFR mutation, NSCLC who failed first line (1L) or second-line (2L) EGFR…
- Trial name
- CheckMate722 (NCT02864251)
- Testing
- Nivolumab, Ipilimumab, Pemetrexed
- Where
- 109 sites in United States, Canada and 8 more
- Volunteers
- 367 people
- Sponsor
- Bristol-Myers Squibb
HER2 Gene Mutation
IAH0968 in Combination With GC for the Treatment of HER2-Positive Unresectable Advanced/Metastatic Malignant Tumors
The study aims to evaluate the efficacy and safety of IAH0968 in combination with gemcitabine and cisplatin for the treatment of HER2-positive unresectable advanced/metastatic malignant tumors and cholangiocarcinoma.
- Trial name
- NCT05991518
- Testing
- Injection of IAH0968+Gemcitabine+Cisplatin, Gemcitabine+Cisplatin
- Where
- China
- Volunteers
- 136 people
- Sponsor
- SUNHO（China）BioPharmaceutical CO., Ltd.
Solid Tumor Cancer, PDAC
IN10018 in Combination With RNK08954 for the Treatment of KRASG12D Mutation-Positive Locally Advanced or Metastatic Solid Tumors
This is a multicenter, open-label, Phase Ib/II clinical study. The study includes Phase Ib-Dose Exploration Stage and Phase II - Efficacy Exploration and Determination Stage.
- Trial name
- NCT07441174
- Testing
- IN10018 in combination with RNK08954
- Where
- 14 sites in China
- Volunteers
- 92 people
- Sponsor
- InxMed (Shanghai) Co., Ltd.
Mitochondrial Myopathies, Mitochondrial Pathology
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary…
- Conditions
- Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases
- Trial name
- NuPower (NCT05162768)
- Testing
- Elamipretide
- Where
- 32 sites in United States, Australia and 8 more
- Volunteers
- 102 people
- Sponsor
- Stealth BioTherapeutics Inc.
AMN, AMN Gene Mutation
Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments.
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- CYGNET (NCT05008874)
- Testing
- Natural History Observation
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 65 people
- Sponsor
- SwanBio Therapeutics, Inc.
Non Small Cell Lung Cancer, Pancreatic Cancer
Study of Covalent Menin Inhibitor BMF-219 in Adult Patients With KRAS Driven Non-Small Cell Lung Cancer, Pancreatic Cancer, and Colorectal Cancer
A Phase 1/1b dose finding study to determine the OBD(s) and RP2D(s) of BMF-219, a covalent menin inhibitor small molecule, in subjects with KRAS mutated unresectable, locally advanced, or metastatic NSCLC (Cohort 1), PDAC (Cohort…
- Conditions
- Non Small Cell Lung Cancer, Pancreatic Cancer, Colorectal Cancer, NSCLC
- Trial name
- NCT05631574
- Testing
- BMF-219
- Where
- 23 sites in United States, South Korea
- Volunteers
- 13 people
- Sponsor
- Biomea Fusion Inc.
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Carcinoma, Non-Small-Cell Lung
A Clinical Study of Bemcentinib With Standard of Care Chemoimmunotherapy in Untreated Advanced/Metastatic Non-small Cell Lung Cancer Patients With a Mutation in the STK11 Gene
The primary purpose of this study is to determine the safety and tolerability of the combination of bemcentinib with chemo-immunotherapy (CIT) to identify the recommended phase 2 dose (RP2D) when administered as first line (1L)…
- Trial name
- NCT05469178
- Testing
- Bemcentinib, Pembrolizumab, Pemetrexed
- Where
- 34 sites in United States, France and 5 more
- Volunteers
- 26 people
- Sponsor
- BerGenBio ASA
Non Small Cell Lung Cancer
DZD6008 Combination Therapy in Patients With Locally Advanced or Metastatic NSCLC With EGFR Mutation (TIAN-SHAN7)
This study is designed to evaluate safety and antitumor activity of DZD6008 combination therapy in patients with advanced Non-Small Cell Lung Cancer (NSCLC) with Epidermal Growth Factor Receptor (EGFR) mutations.
- Trial name
- NCT07070440
- Testing
- DZD6008, Pemetrexed, Carboplatin
- Where
- China
- Volunteers
- 180 people
- Sponsor
- Dizal Pharmaceuticals
Mastocytosis
Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis
This is a 12 weeks study aimed at assessing the safety and efficacy of 2 doses of AB1010 in patients suffering from indolent systemic mastocytosis with handicap.
- Trial name
- NCT00831974
- Testing
- masitinib (AB1010)
- Volunteers
- 23 people
- Sponsor
- AB Science
Non Small Cell Lung Carcinoma
SELINE: Second-Line Iressa Phase IV Study in NSCLC Patients
The purpose of this study is to evaluate ORR (Objective Response Rate) of gefitinib as a second-line therapy for NSCLC patients based on RECIST (Response Evaluation Criteria in Solid Tumors Group) and check up ORR difference by…
- Trial name
- SELINE (NCT00608868)
- Testing
- Gefitinib
- Where
- South Korea
- Volunteers
- 156 people
- Sponsor
- AstraZeneca
Malignant Melanoma, Brain Metastases
E6201 Plus Dabrafenib for the Treatment of Metastatic Melanoma Central Nervous System Metastases (CNS)
This is a Phase 1 study of E6201 plus dabrafenib for the treatment of CNS metastases in BRAF V600-mutated metastatic melanoma. A total of up to N=28-34 subjects with melanoma metastasized to the CNS will be included.
- Trial name
- NCT03332589
- Testing
- E6201, E6201 plus dabrafenib
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Spirita Oncology, LLC
Cystic Fibrosis
Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) effects of VX-661 alone and when coadministered with ivacaftor in participants with cystic fibrosis (CF) who are…
- Trial name
- NCT01531673
- Testing
- VX-661, Ivacaftor
- Where
- 36 sites in United States, Canada and 2 more
- Volunteers
- 194 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline compared to hypertonic saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the…
- Trial name
- NCT02709109
- Testing
- VX-371 + HS, Hypertonic saline, Orkambi
- Where
- 40 sites in United States, France and 2 more
- Volunteers
- 147 people
- Sponsor
- Parion Sciences
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