Clinical trials: Gene Mutation
Primary Ciliary Dyskinesia
Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects
This is the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability data for future clinical studies.
- Trial name
- NCT05737485
- Testing
- RCT1100
- Where
- 4 sites in United States, Australia and 2 more
- Volunteers
- 9 people
- Sponsor
- ReCode Therapeutics
Non Small Cell Lung Cancer
A Study to Assess the Efficacy of WSD0922-FU in Patients With C797S+ Advanced Non-small Cell Lung Cancer
This is a Phase II, Open Label, Multicenter, Single Arm Study of WSD0922-FU for Patients with Locally Advanced or Metastatic Non-Small Cell Lung Cancer whose Disease has Progressed with First-Line Osimertinib Treatment and whose…
- Trial name
- NCT06868485
- Testing
- WSD0922-FU Tablets, Dose level A, WSD0922-FU Tablets, Dose level B
- Where
- 18 sites in United States, China and 1 more
- Volunteers
- 40 people
- Sponsor
- Wayshine Biopharm, Inc.
Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA)
Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)
This study is an early-stage clinical trial (Phase 1b/2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare genetic eye disease that leads to…
- Trial name
- RDH12 (NCT07681778)
- Testing
- OPGx-RDH12
- Where
- 4 sites in United States
- Volunteers
- 10 people
- Sponsor
- Opus Genetics, Inc
Advanced Solid Malignancy, Safety and Tolerability
Safety, Tolerability & Potential Anti-cancer Activity of Increasing Doses of AZD5363 in Different Treatment Schedules
This study is designed to investigate the safety and tolerability of a new drug, AZD5363, in patients with advanced cancer - and to identify a dose and schedule that can be used in the future.
- Conditions
- Advanced Solid Malignancy, Safety and Tolerability, Pharmacokinetics, Pharmacodynamics
- Trial name
- NCT01226316
- Testing
- AZD5363
- Where
- 34 sites in United States, Canada and 8 more
- Volunteers
- 285 people
- Sponsor
- AstraZeneca
Ovarian Cancer, Breast Cancer
Prospective Multicentre Phase-IV Clinical Trial of Olaparib in Indian Patients With Ovarian and Metastatic Breast Cancer
A Prospective, Multicentre, Phase-IV Clinical Trial of Olaparib in Indian Patients with Platinum Sensitive Relapsed Ovarian Cancer who are in Complete or Partial Response Following Platinum based Chemotherapy and Metastatic…
- Trial name
- SOLI (NCT04330040)
- Testing
- Olaparib
- Where
- 16 sites in India
- Volunteers
- 202 people
- Sponsor
- AstraZeneca
Ovarian Cancer, Fallopian Tube Cancer
A Study of Oral Rucaparib in Patients With a Solid Tumor (Phase I) or With gBRCA Mutation Ovarian Cancer (Phase II)
Part 1 (Completed Enrollment) - The purpose of the first part of the study was to evaluate the safety of different doses and dosing regimens of oral rucaparib administered daily to patients with solid tumors.
- Conditions
- Ovarian Cancer, Fallopian Tube Cancer, Peritoneal Cancer, Advanced Solid Tumor With Evidence of Germline or Somatic BRCA
- Trial name
- NCT01482715
- Testing
- Rucaparib
- Where
- 16 sites in United States, Canada and 3 more
- Volunteers
- 136 people
- Sponsor
- pharmaand GmbH
Non-small Cell Lung Cancer
Phase 1/2 Study of the Safety and Efficacy of Rociletinib in Combination With Trametinib in Patients With mEGFR-positive Advanced or Metastatic Non-small Cell Lung Cancer
The purpose of this study is to evaluate the safety and anti-tumor effect of rociletinib when administered in combination with trametinib.
- Trial name
- NCT02580708
- Testing
- Rociletinib, Trametinib
- Where
- 4 sites in United States
- Volunteers
- 7 people
- Sponsor
- Clovis Oncology, Inc.
Diffuse Astrocytoma, Anaplastic Astrocytoma
Study of Efficacy and Safety of Dabrafenib in Combination With Trametinib in Pediatric Patients With BRAF V600 Mutation Positive LGG or Relapsed or Refractory HGG Tumors
The purpose of this study was to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma (LGG) or relapsed or refractory high…
- Conditions
- Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood
- Trial name
- NCT02684058
- Testing
- Dabrafenib, trametinib, Carboplatin
- Where
- 57 sites in United States, Argentina and 18 more
- Volunteers
- 151 people
- Sponsor
- Novartis Pharmaceuticals
Melanoma
A Study Comparing Trametinib and Dabrafenib Combination Therapy to Dabrafenib Monotherapy in Subjects With BRAF-mutant Melanoma
This was a two-arm, double-blinded, randomized, Phase III study comparing dabrafenib and trametinib combination therapy to dabrafenib administered with a placebo (dabrafenib monotherapy).
- Trial name
- NCT01584648
- Testing
- Dabrafenib, Trametinib
- Where
- 121 sites in United States, Argentina and 12 more
- Volunteers
- 423 people
- Sponsor
- Novartis Pharmaceuticals
Non-Small Cell Lung Cancer
Combination Study of RMC-4630 and Sotorasib for NSCLC Subjects With KRASG12C Mutation After Failure of Prior Standard Therapies
The purpose of this study is to evaluate the antitumor effects of sotorasib and RMC-4630 in subjects with KRASG12C mutant NSCLC
- Trial name
- NCT05054725
- Testing
- RMC-4630, Sotorasib
- Where
- 60 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Revolution Medicines, Inc.
Advanced Solid Tumor, Advanced Malignant Neoplasm
The Evaluation of PC14586 in Patients With Advanced Solid Tumors Harboring a TP53 Y220C Mutation (PYNNACLE)
The Phase 2 monotherapy portion of this study is currently enrolling and will evaluate the efficacy and safety of PC14586 (INN rezatapopt) in participants with locally advanced or metastatic solid tumors harboring a TP53 Y220C…
- Conditions
- Advanced Solid Tumor, Advanced Malignant Neoplasm, Metastatic Cancer, Metastatic Solid Tumor
- Trial name
- NCT04585750
- Testing
- rezatapopt, pembrolizumab
- Where
- 76 sites in United States, Australia and 7 more
- Volunteers
- 300 people
- Sponsor
- PMV Pharmaceuticals, Inc
Non-Small Cell Lung Cancer
Study of Intravenous Telisotuzumab Vedotin in Combination Osimertinib or Standard of Care Chemotherapy to Assess Change in Disease Activity in Adult Participants With Non-Small Cell Lung Cancer That Has a Mutation in the Epidermal Growth Factor Receptor Gene and That Overexpresses the c-Met Protein
Cancer is a condition where cells in a specific part of body grow and reproduce uncontrollably. Non-small cell lung cancer (NSCLC) is a solid tumor, a disease in which cancer cells form in the tissues of the lung.
- Trial name
- NCT06093503
- Testing
- Telisotuzumab Vedotin, Osimertinib, Cisplatin
- Sponsor
- AbbVie
Non-Small Cell Lung Cancer With EGFR Mutation
A Phase 2 Clinical Study of Combination Therapy With ABSK043 and Firmonertinib
This is an open-label phase 2 study to evaluate the safety, tolerability and preliminary anti-tumour activity of ABSK043 in combination with Firmonertinib in patients with Epidermal Growth Factor Receptor-mutated (EGFRm+) locally…
- Trial name
- NCT06668103
- Testing
- ABSK043 in combination with Firmonertinib
- Where
- 5 sites in China
- Volunteers
- 54 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
Retinitis Pigmentosa
A Study in Subjects With Retinitis Pigmentosa
An open-label Phase II clinical trial, 8 (eight) subjects with retinitis pigmentosa due to rhodopsin mutations (including P23H) will be identified and treated with serial intravitreal injections of ADX-2191 in the worse seeing…
- Trial name
- NCT05392179
- Testing
- ADX-2191
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Aldeyra Therapeutics, Inc.
Breast Cancer, Cancer
Combination Study of a New Taxane and Capecitabine in Patients With Metastatic Breast Cancer
The goal of this phase I-II clinical research study is to find the highest safe dose of XRP9881 and capecitabine that can be given in combination in the treatment of metastatic breast cancer in patients who have been previously…
- Trial name
- NCT00327743
- Testing
- larotaxel, capecitabine
- Where
- 2 sites in United States, France
- Volunteers
- 34 people
- Sponsor
- Sanofi
NSCLC
A Study of TY-9591 With Chemotherapy as 1st Line Treatment in Patients With Mutated Epidermal Growth Factor Receptor NSCLC
This study was a parallel, randomized, multicenter phase II clinical trial to evaluate the efficacy and safety of TY-9591 combined with platinum-based chemotherapy as first-line treatment in patients with locally advanced or…
- Trial name
- NCT06672068
- Testing
- TY-9591 80mg QD+pemetrexed+Cisplatin/Carboplatin, TY-9591 160mg QD+pemetrexed+Cisplatin/Carboplatin
- Volunteers
- 60 people
- Sponsor
- TYK Medicines, Inc
Isocitrate Dehydrogenase Gene Mutation
A Study of HMPL-306 in Advanced Hematological Malignancies With mIDH
An open label single-arm clinical trial to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of HMPL-306 in subjects with advanced relapsed, refractory, or resistant hematological malignancies that harbor IDH…
- Trial name
- NCT04764474
- Testing
- HMPL-306
- Where
- 15 sites in United States, Spain
- Volunteers
- 46 people
- Sponsor
- Hutchmed
Sensorineural Hearing Loss, Bilateral
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Part A of this trial evaluated the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF (LY888891) and evaluated the Akouos Delivery Device…
- Trial name
- NCT05821959
- Testing
- AAVAnc80-hOTOF via Akouos Delivery Device, AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
- Where
- 9 sites in United States, Canada and 2 more
- Volunteers
- 48 people
- Sponsor
- Eli Lilly and Company
Stage IIIB Non-small Cell Lung Cancer, Stage IIIC Non-Small Cell Lung Cancer
Study of ONO-7475 in Combination With Osimertinib in EGFR Gene Mutation-positive Non-small Cell Lung Cancer
This study is to evaluate the tolerability and safety of ONO-7475 in combination with osimertinib in the first-line treatment of patients with EGFR-mutated, stage IIIB/IIIC/IV or recurrent non-small cell lung cancer (NSCLC)…
- Conditions
- Stage IIIB Non-small Cell Lung Cancer, Stage IIIC Non-Small Cell Lung Cancer, Stage IV Non-small Cell Lung Cancer, Recurrent Non-Small Cell Lung Cancer
- Trial name
- NCT06525246
- Testing
- ONO-7475, Osimertinib Mesylate
- Where
- 13 sites in Japan
- Volunteers
- 78 people
- Sponsor
- Ono Pharmaceutical Co., Ltd.
BRAF V600 Mutation, Unresectable Melanoma
Hepatic Impairment Study of Encorafenib in Combination With Binimetinib in BRAF Melanoma
Encorafenib in combination with binimetinib have been approved in USA, Europe, Australia, Japan and Switzerland for the treatment of adult patients with unresectable or metastatic melanoma with BRAF V600 mutation.
- Conditions
- BRAF V600 Mutation, Unresectable Melanoma, Metastatic Melanoma, Hepatic Impairment
- Trial name
- NCT04759846
- Testing
- Encorafenib + Binimetinib
- Where
- 7 sites in Czechia, Italy and 1 more
- Sponsor
- Pierre Fabre Medicament
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