Clinical trials: Genetic Mutation
Carcinoma, Non-Small-Cell Lung
Expanded Access Study of Afatinib in Treatment-naive or Chemotherapy Pre-treated Patients With Non-small Cell Lung Cancer (NSCLC)
To provide expanded access and to evaluate the safety, tolerability and efficacy of afatinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring Epidermal Growth Factor Receptor (EGFR)…
- Trial name
- NCT01931306
- Testing
- Afatinib
- Where
- 8 sites in South Korea
- Sponsor
- Boehringer Ingelheim
Colorectal Neoplasms, Advanced Metastatic Colorectal Cancer
Biomarker Analysis for Patients With Metastatic Colorectal Cancer (MCC)
Patients are being asked to participate in this study who have colorectal cancer that has come back after initial treatment. The investigators want to improve treatment in patients with this disease.
- Trial name
- NCT01909362
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- US Oncology Research
Advanced Solid Tumors
JAB-23E73 in Adult Participants With Advanced Solid Tumors With KRAS Alteration
This study is to evaluate the safety and tolerability of pan-KRAS inhibitor JAB-23E73 Monotherapy and in Combination with Other Agents in adult participants with advanced solid tumors
- Trial name
- NCT06973564
- Testing
- JAB-23E73, Cetuximab
- Where
- 4 sites in United States
- Volunteers
- 344 people
- Sponsor
- Jacobio Pharmaceuticals Co., Ltd.
Non-small Cell Lung Cancer (NSCLC)
A Study to Compare Setidegrasib (ASP3082) With Docetaxel, in People With Non-small Cell Lung Cancer With a KRAS G12D Mutation
Non-small cell lung cancer (NSCLC) is the most common type of lung cancer. The first treatment is usually chemotherapy, given with another treatment that targets specific proteins on cancer cells.
- Trial name
- NCT07566052
- Testing
- Setidegrasib, Docetaxel
- Where
- 23 sites in United States, China and 1 more
- Volunteers
- 356 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt…
- Trial name
- NCT07002398
- Testing
- VG801
- Where
- China
- Volunteers
- 18 people
- Sponsor
- VeonGen Therapeutics GmbH
Alzheimer Disease
Tau Positron Emission Tomography (PET) Longitudinal Substudy Associated With: Study of Crenezumab Versus Placebo in Preclinical Presenilin1 (PSEN1) E280A Mutation Carriers in the Treatment of Autosomal-Dominant Alzheimer's Disease
This substudy will evaluate the effect of crenezumab on the longitudinal tau burden in a subgroup of preclinical Presenilin1 (PSEN1) E280A mutation carriers and non-carriers, who were enrolled in study NCT01998841 (GN28352).
- Trial name
- NCT03977584
- Testing
- Crenezumab, [^18F]GTP1
- Where
- Colombia
- Volunteers
- 114 people
- Sponsor
- Hoffmann-La Roche
Plasma EGFR Mutation-positive Lung Cancer
Efficiency of Icotinib in Plasma ctDNA EGFR Mutation-positive Patients Diagnosed With Lung Cancer
this single arm，open-label，multi-center study is aimed to evaluate the efficiency of icotinib in plasma ctDNA EGFR mutation-positive patients diagnosed with lung cancer
- Trial name
- CHALLENGE (NCT03346811)
- Testing
- Icotinib
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Acute Myeloid Leukemia (AML)
Ivosidenib (TIBSOVO®) Combined With Azacitidine According to Current SmPC
The goal of this non-interventional study is to evaluate quality of life (QoL) in adult patients with newly diagnosed IDH1 R132-mutated AML who are not eligible to receive standard induction chemotherapy and who are treated with…
- Trial name
- CONFIDHENCE (NCT06181734)
- Testing
- Ivosidenib
- Where
- Germany
- Volunteers
- 8 people
- Sponsor
- iOMEDICO AG
OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy
Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA.
- Conditions
- OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
- Trial name
- PYC-001-CL-102 (NCT06970106)
- Testing
- PYC-001
- Where
- 3 sites in Australia, New Zealand
- Volunteers
- 24 people
- Sponsor
- PYC Therapeutics
PIK3CA Mutation, Solid Tumor, Adult
First-in-Human Study of RLY-5836 in Advanced Breast Cancer and Other Solid Tumors
This is a Phase 1, first-in-human, open-label study designed to evaluate the maximum tolerated dose (MTD), recommended phase 2 dose (RP2D), safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor…
- Conditions
- PIK3CA Mutation, Solid Tumor, Adult, HER2-negative Breast Cancer, Breast Cancer
- Trial name
- NCT05759949
- Testing
- RLY-5836, Fulvestrant, Palbociclib
- Where
- 7 sites in United States
- Volunteers
- 41 people
- Sponsor
- Relay Therapeutics, Inc.
X-linked Hypophosphatemia
Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH
This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab…
- Trial name
- NCT03920072
- Testing
- Burosumab
- Where
- 10 sites in France, Ireland and 2 more
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Lymphoma, B-Cell, Small Lymphocytic Lymphoma
A Study of Nemtabrutinib (MK-1026) in Participants With Relapsed or Refractory Hematologic Malignancies (ARQ 531-101/MK-1026-001)
This study aims to evaluate the safety, tolerability, pharmacodynamic, and pharmacokinetic (PK) of nemtabrutinib (formerly ARQ 531) tablets in selected participants with relapsed or refractory hematologic malignancies.
- Conditions
- Lymphoma, B-Cell, Small Lymphocytic Lymphoma, Chronic Lymphocytic Leukemia, Waldenstrom Macroglobulinemia
- Trial name
- NCT03162536
- Testing
- Nemtabrutinib
- Where
- 10 sites in United States
- Volunteers
- 190 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Muscular Dystrophy, Duchenne
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will…
- Trial name
- MIS51ON (NCT03992430)
- Testing
- Eteplirsen
- Where
- 59 sites in United States, Colombia and 24 more
- Volunteers
- 160 people
- Sponsor
- Sarepta Therapeutics, Inc.
Cystic Fibrosis
Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
- Trial name
- NCT02971839
- Testing
- VX-561, IVA
- Where
- 14 sites in United States
- Volunteers
- 11 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Metastatic Non-small Cell Lung Cancer
Clinical Trial of the Efficacy and Safety of AC0010 in the Treatment of EGFR T790M Patients With Advanded NSCLC
The study is a single-arm, multi-center, open-label clinical trial. The study aims to expand the sample size based on the fixed dose recommended by the results of previous dose exploration studies in order to further evaluate the…
- Trial name
- NCT03300115
- Testing
- AC0010
- Where
- 25 sites in China
- Volunteers
- 222 people
- Sponsor
- Hangzhou ACEA Pharmaceutical Research Co., Ltd.
EGFR Positive Non-small Cell Lung Cancer, Adenocarcinoma
Icotinib as First-line and Maintenance Treatment in EGFR Mutated Patients With Lung Adenocarcinoma
This study is designed to compare the efficacy and safety of first-line icotinib treatment and first-line chemotherapy followed by maintenance treatment with icotinib.
- Trial name
- NCT01665417
- Testing
- Experimental, Chemotherapy
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Non-Small Cell Lung Cancer, NSCLC
First-In-Human Study of STX-721/PFL-721 in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer Harboring EGFR or HER2 Exon 20 Insertion Mutations
Study STX-721-101/PFL-721CI101 is an open label, Phase 1/2 study evaluating the safety, tolerability, pharmacokinetic (PK) exposure, and preliminary antitumor activity of STX-721/PFL-721 in participants with non-small cell lung…
- Conditions
- Non-Small Cell Lung Cancer, NSCLC, EGFR/HER2 Exon 20 Insertion Mutation
- Trial name
- NCT06043817
- Testing
- STX-721/PFL-721 (Escalated), STX-721/PFL-721 (3 dose levels), STX-721/PFL-721 (RP2D)
- Where
- 32 sites in United States, Australia and 6 more
- Volunteers
- 345 people
- Sponsor
- Pierre Fabre Medicament
Non-small Cell Lung Cancer
Phase III, Open-label Study of First-line Osimertinib With or Without Datopotamab Deruxtecan for EGFRm Locally Advanced or Metastatic Non-small Cell Lung Cancer
The purpose of this study is to evaluate efficacy and safety of osimertinib (tablet) in combination with Dato-DXd (i.v. infusion) compared with osimertinib (tablet) monotherapyas a first-line therapy in participants with locally…
- Trial name
- TROPION-Lung14 (NCT06350097)
- Testing
- Osimertinib, Datopotamab Deruxtecan
- Where
- 167 sites in United States, Australia and 17 more
- Volunteers
- 582 people
- Sponsor
- AstraZeneca
EGFR-mutant Non-small Cell Lung Cancer
Study of EGF816 in Combination With Selected Targeted Agents in EGFR-mutant NSCLC
The study purpose is to evaluate the safety, tolerability, and preliminary efficacy of the addition of INC280, trametinib, ribociclib, gefitinib, or LXH254 to EGF816 in adult patients with advanced Epidermal growth factor…
- Trial name
- NCT03333343
- Testing
- EGF816, trametinib, ribociclib
- Where
- 11 sites in Canada, Germany and 4 more
- Volunteers
- 105 people
- Sponsor
- Novartis Pharmaceuticals
Non-Small Cell Lung Cancer (NSCLC), Colorectal Cancer (CRC)
Dose Escalation and Dose Expansion Study of RMC-6291 Monotherapy in Subjects With Advanced KRASG12C Mutant Solid Tumors
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of escalating doses of RMC-6291 (KRAS G12C(ON) inhibitor) monotherapy in adult subjects with advanced solid tumors and to identify the…
- Conditions
- Non-Small Cell Lung Cancer (NSCLC), Colorectal Cancer (CRC), Pancreatic Ductal Adenocarcinoma, Advanced Solid Tumor
- Trial name
- NCT05462717
- Testing
- RMC-6291
- Where
- 64 sites in United States, Australia and 11 more
- Volunteers
- 222 people
- Sponsor
- Revolution Medicines, Inc.
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