Clinical trials: Gene Mutation
NSCLC (Non-small Cell Lung Cancer)
A Phase Ⅱ Study Assessing the Safety and Efficacy of PLB1004 in EGFR ex20ins Mutation Patients With Advanced and Metastatic Non-small Cell Lung Cancer(NSCLC)
It is a phase Ⅱ,open-label, single-line, Multiple cohorts, Multicenter study assessing the Safety and Efficacy of PLB1004 in EGFR ex20ins mutation patients with Advanced and Metastatic Non-small Cell Lung Cancer(NSCLC).
- Trial name
- KANNON (NCT06015503)
- Testing
- PLB1004
- Where
- China
- Volunteers
- 139 people
- Sponsor
- Avistone Biotechnology Co., Ltd.
Malignant Melanoma, Neoplasms
A Pharmacokinetics Study to Investigate the Effect of Ketoconazole on Vemurafenib in Patients With BRAFV600 Mutation-Positive Metastatic Melanoma
This open-label, multi-center, three-period, one sequence study will investigate the effect of ketoconazole on the pharmacokinetics of vemurafenib in patients with unresectable BRAFV600-mutation positive metastatic melanoma or…
- Trial name
- NCT01765556
- Testing
- ketoconazole, vemurafenib
- Sponsor
- Hoffmann-La Roche
Multiple Sclerosis
Managed Access Program (MAP) for Patients Diagnosed With Secondary Progressive Multiple Sclerosis With Active Disease
The purpose of this MAP Cohort Treatment Plan is to allow access to siponimod for eligible patients diagnosed with Secondary Progressive Multiple Sclerosis with active disease.
- Trial name
- NCT04540861
- Testing
- Siponimod
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein.
- Trial name
- NCT00264888
- Testing
- PTC124
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- PTC Therapeutics
Thalassemia
iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations
This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
- Trial name
- NCT03728322
- Testing
- iHSCs treatment group
- Volunteers
- 12 people
- Sponsor
- Allife Medical Science and Technology Co., Ltd.
Pancreatic Cancer
Trial of DN022150 Versus Chemotherapy in Previously Treated Advanced Pancreatic Cancer With KRAS G12D Mutation
This study plans to enroll participants with previously treated advanced pancreatic cancer and harbor centrally confirmed KRAS G12D mutation.
- Trial name
- NCT07689539
- Testing
- DN022150, Chemotherapy
- Volunteers
- 360 people
- Sponsor
- Jiangxi Kvvit Pharmaceutical Co., Ltd.
Multiple Myeloma
Elranatamab Expanded Access Protocol in Adults With Relapsed/Refractory Multiple Myeloma
Elranatamab is a bispecific antibody: binding of elranatamab to CD3- expressing T-cell and BCMA- expressing multiple myeloma cells causes targeted T-cell mediated cytotoxicity.
- Trial name
- MAGNETISMM17 (NCT05462639)
- Testing
- Elranatamab (PF-06863135)
- Where
- 36 sites in United States, Canada
- Sponsor
- Pfizer
Metastatic Colorectal Cancer, Metastatic Pancreatic Ductal Adenocarcinoma
A Study of ERAS-007 in Patients With Advanced Gastrointestinal Malignancies
To evaluate the safety and tolerability of escalating doses of ERAS-007 in combination with other cancer therapies in study participants with advanced GI malignancies.
- Trial name
- HERKULES-3 (NCT05039177)
- Testing
- ERAS-007, Encorafenib, Cetuximab
- Where
- 14 sites in United States
- Volunteers
- 101 people
- Sponsor
- Erasca, Inc.
Oncology, MEK Mutation
A Study to Investigate the Safety and Efficacy of NST-628 Oral Tablets in Subjects With Solid Tumors
This is a two-part Phase 1, open label, multi-center, single arm, non-randomized, multiple dose, safety, pharmacokinetic (PK) and preliminary efficacy study of single agent NST-628 in adult patients with MAPK pathway…
- Conditions
- Oncology, MEK Mutation, RAF Gene Mutation, Ras (KRAS or NRAS) Gene Mutation
- Trial name
- NST-628 (NCT06326411)
- Testing
- NST-628
- Where
- 23 sites in United States, Australia
- Volunteers
- 230 people
- Sponsor
- Nested Therapeutics, Inc
Solid Tumor
A Study to Evaluate Rucaparib in Participants With Solid Tumors and With Deleterious Mutations in HRR Genes
A Phase 2, open-label, single-arm trial to evaluate the response of rucaparib in participants with various solid tumors and with deleterious mutations in Homologous Recombination Repair (HRR) genes.
- Trial name
- LODESTAR (NCT04171700)
- Testing
- Rucaparib
- Where
- 19 sites in United States
- Volunteers
- 83 people
- Sponsor
- pharmaand GmbH
Hereditary Angioedema
Efficacy and Safety of GNR-038 vs Berinert® in Patients With Hereditary Angioedema
It is a placebo-controlled randomized trial to evaluate the efficacy and safety of GNR-038 in comparison with Berinert® in patients with hereditary angioedema
- Trial name
- NCT04898309
- Testing
- GNR-038, 50 МЕ/ kg, GNR-038, 100 МЕ/ kg, Berinert®, 20 МЕ/ kg
- Where
- 5 sites in Russia
- Sponsor
- AO GENERIUM
Solid Tumor, Adult, Metastatic Solid Tumor
SHP2 Inhibitor BBP-398 in Combination With Sotorasib in Patients With Advanced Solid Tumors and a KRAS-G12C Mutation
This is a Phase 1 study of BBP-398, a SHP2 inhibitor, in combination with sotorasib, a KRAS-G12C inhibitor (KRAS-G12Ci), in patients with a KRAS-G12C mutation.
- Conditions
- Solid Tumor, Adult, Metastatic Solid Tumor, Metastatic NSCLC, Non Small Cell Lung Cancer
- Trial name
- Argonaut (NCT05480865)
- Testing
- BBP-398, sotorasib
- Where
- 25 sites in Australia, Denmark and 5 more
- Volunteers
- 28 people
- Sponsor
- Navire Pharma Inc., a BridgeBio company
Cardiomyopathy
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality…
- Trial name
- MyCLIMB (NCT05112237)
- Where
- 29 sites in United States, Canada and 2 more
- Volunteers
- 200 people
- Sponsor
- Tenaya Therapeutics
Acute Myeloid Leukemia With FLT3/ITD Mutation
A Study of XY0206 Versus Salvage Chemotherapy In Patients With Relapsed or Refractory AML With FLT3-ITD-Mutation (ALIVE)
The purpose of this study is to determine the clinical benefit of XY0206 therapy in participants with FLT3-ITD mutated AML who are refractory to or have relapsed after prior AML therapy as shown with overall survival (OS)…
- Trial name
- NCT05876832
- Testing
- XY0206, Salvage Chemotherapy
- Volunteers
- 312 people
- Sponsor
- Shijiazhuang Yiling Pharmaceutical Co. Ltd
Carcinoma, Non-Small-Cell Lung
A Study to Learn About Dacomitnib in Patients With Non-small Cell Lung Cancer Which Has Spread to the Brain.
The purpose of this study is to learn about dacomitinib for the possible treatment of lung cancer which has spread to other parts of the body.
- Trial name
- NCT06075615
- Testing
- dacomitnib
- Sponsor
- Pfizer
Leukocyte Adhesion Defect - Type I
A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
The primary purpose of the Phase I portion of the study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the…
- Trial name
- NCT03812263
- Testing
- RP-L201
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Non Small Cell Lung Cancer
AZD6244 in Combination With Docetaxel Versus Docetaxel Alone in KRAS Mutation Positive NSCLC Patients
The purpose of this study is to compare the efficacy of AZD6244 in combination with docetaxel versus docetaxel alone in patients with KRAS mutation positive locally advanced or metastatic non small cell lung cancer.
- Trial name
- NCT00890825
- Testing
- AZD6244, docetaxel
- Where
- 63 sites in United States, Belgium and 10 more
- Volunteers
- 88 people
- Sponsor
- AstraZeneca
Lymphatic Malformations
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.
- Trial name
- EPIK-L1 (NCT05948943)
- Testing
- Alpelisib
- Where
- 61 sites in United States, Argentina and 10 more
- Volunteers
- 232 people
- Sponsor
- Novartis Pharmaceuticals
Triple-Negative Breast Cancer (TNBC), Gastric Cancer (GC)
A Study of MDSY-1240, a STAT3 Degrader, for the Treatment of Hematologic Malignancies and Advanced Solid Tumors
This clinical trial is evaluating a drug called MDSY-2140 in participants with hematologic malignancies and advanced solid tumors.
- Conditions
- Triple-Negative Breast Cancer (TNBC), Gastric Cancer (GC), Gastric Cancer Adenocarcinoma Metastatic, Ovarian Cancer Metastatic
- Trial name
- NCT07899632
- Testing
- MDSY-1240
- Volunteers
- 75 people
- Sponsor
- Medsyn Biopharma, LLC
Alport Syndrome
A Study of ELX-02 in Patients With Alport Syndrome
This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation.
- Trial name
- NCT05448755
- Testing
- ELX-02
- Where
- 4 sites in Australia, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
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