Clinical trials: Gene Mutations
Non-Small Cell Lung Cancer (NSCLC)
Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Relapsed or Refractory Advanced Non-Small Cell Lung Cancer With an FGFR Alteration
This is an open-label, single arm study to study the safety, efficacy and tolerability of Pemigatinib when used on participants with squamous or nonsquamous NSCLC with a documented FGFR1-3 mutations or fusions/rearrangement who…
- Trial name
- FIGHT-210 (NCT05253807)
- Testing
- Pemigatinib
- Where
- 36 sites in United States, France and 3 more
- Volunteers
- 8 people
- Sponsor
- Incyte Corporation
Advanced Solid Tumors, Selected Solid Tumors
Safety, Pharmacokinetics and Pharmacodynamics of BKM120 Plus MEK162 in Selected Advanced Solid Tumor Patients
This is an open label, dose finding, phase Ib clinical trial to determine the maximum tolerated dose (MTD) and/or the recommended phase II dose (RP2D) of the orally administered phosphatidylinositol 3'-kinase (PI3K) inhibitor…
- Trial name
- NCT01363232
- Testing
- BKM120 + MEK162
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 89 people
- Sponsor
- Array Biopharma, now a wholly owned subsidiary of Pfizer
Advanced Cancers (Breast, Ovarian, mCRPC, Pancreatic Ductal Adenocarcinoma (PDAC), Brain Mets) With Specific Mutations (Homologous Recombination Deficiency)
Study to Assess Safety, Tolerability and Activity of DSB2455 in Participants With Advanced Malignancies
Open label, multi-centre, Phase Ia/b adaptive design study with an initial 2-stage Dose Escalation Phase followed by a Dose Expansion Phase.
- Trial name
- NCT06458712
- Testing
- DSB2455
- Where
- 21 sites in United States, France and 3 more
- Volunteers
- 180 people
- Sponsor
- Duke Street Bio Ltd
Advanced Solid Tumor, Esophageal Cancer
A Study of PRT3789 in Combination With Pembrolizumab in Patients With Advanced or Metastatic Solid Tumors With a SMARCA4 Mutation
This is a Phase 2 an open-label, multi-center study to determine the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and anti-tumor activity of PRT3789 in combination with pembrolizumab in patients with…
- Conditions
- Advanced Solid Tumor, Esophageal Cancer, Metastatic Solid Tumor, Non-small Cell Lung Cancers
- Trial name
- NCT06682806
- Testing
- PRT3789, pembrolizumab
- Where
- 9 sites in United States, Spain
- Volunteers
- 6 people
- Sponsor
- Prelude Therapeutics Incorporated
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.
Chronic Granulomatous Disease, Granulomatous Disease, Chronic
A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with…
- Trial name
- NCT06559176
- Testing
- PM359
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 12 people
- Sponsor
- Prime Medicine, Inc.
NSCLC (Advanced Non-small Cell Lung Cancer)
GFH375 Monotherapy and Combination Therapy as First-Line Treatment for Advanced KRAS G12D-Mutant Non-Small Cell Lung Cancer
This study is a multicenter, open-label, randomized clinical trial aimed at exploring the efficacy and safety of three treatment regimens for treatment-naive advanced NSCLC patients with KRAS G12D mutation: GFH375 monotherapy…
- Trial name
- NCT07554859
- Testing
- ArmA:GFH375, Arm B:GFH375, Arm C:GFH375
- Where
- 2 sites in China
- Volunteers
- 90 people
- Sponsor
- Genfleet Therapeutics (Shanghai) Inc.
Colorectal Cancer
A Study of Avastin (Bevacizumab) in Combination With mFOLFOX6 in Treatment-Naïve Patients With Metastatic Colorectal Cancer With or Without K-RAS Mutations, and Comparison to Cetuximab
This randomized, open-label study will evaluate the safety and efficacy of Avastin (Bevacizumab) added to standard mFOLFOX6 chemotherapy in treatment-naïve patients with Stage IV metastatic colorectal cancer.
- Trial name
- NCT01338558
- Testing
- bevacizumab [Avastin], cetuximab, mFOLFOX6
- Sponsor
- Hoffmann-La Roche
Autosomal Dominant Retinitis Pigmentosa, Eye Diseases
A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene
This study evaluates the safety, tolerability and efficacy of QR-1123 injection in the eye (intravitreal; IVT) injections (one eye/unilateral) in subjects receiving a single dose or repeat doses.
- Conditions
- Autosomal Dominant Retinitis Pigmentosa, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies
- Trial name
- AURORA (NCT04123626)
- Testing
- QR-1123
- Where
- 5 sites in United States
- Volunteers
- 11 people
- Sponsor
- ProQR Therapeutics
Retinitis Pigmentosa, Usher Syndrome Type 2
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with…
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05176717
- Testing
- QR-421a
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Laboratoires Thea
AXIN1 Gene Mutation, APC Gene Mutation
A Study of REC-4881 in Participants With Cancers Which Have an AXIN1 or APC Mutation
This is a multi-center, open-label study to investigate the safety, efficacy and pharmacokinetics of REC-4881 (12 mg PO daily doses) for the treatment of participants with unresectable locally advanced or metastatic solid tumors…
- Conditions
- AXIN1 Gene Mutation, APC Gene Mutation, Solid Tumor
- Trial name
- NCT06005974
- Testing
- REC-4881
- Where
- 13 sites in United States
- Volunteers
- 18 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Solid Tumor
QUILT-3.036: AMG 337 in Subjects With Advanced or Metastatic Solid Tumors
This is a phase 2 study of AMG 337 in subjects with advanced or metastatic solid tumors that overexpress MET or harbor METex14del mutations resulting in MET exon 14 skipping.
- Trial name
- NCT03147976
- Testing
- AMG 337
- Where
- United States
- Sponsor
- NantPharma, LLC
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
NSCLC
A Phase II Study of Fruquintinib in Combination With Gefitinib in Patients With Advanced Non-small Cell Lung Cancer
Fruquintinib in combination with Gefitinib as first-line therapy in patients with advanced non-squamous non-small-cell lung cancer harboring activating EGFR mutations : a single-arm, multicenter, phase II study
- Trial name
- NCT02976116
- Testing
- Fruquintinib, Gefitinib
- Where
- 2 sites in China
- Volunteers
- 50 people
- Sponsor
- Hutchison Medipharma Limited
Solid Tumors, Homologous Recombination Repair Gene Mutation
Basket Trial of IDX-1197, a PARP Inhibitor, in Patients With HRR Mutated Solid Tumors (VASTUS)
This study is a Phase 1b/2a basket trial to assess safety and efficacy of IDX-1197 in patients with HRR mutation. There are two parts to this study: Phase 1b, IDX-1197 dose-selection study to determine RP2D and Phase 2a…
- Conditions
- Solid Tumors, Homologous Recombination Repair Gene Mutation, Homologous Recombination Deficiency
- Trial name
- VASTUS (NCT04174716)
- Testing
- IDX-1197
- Where
- 19 sites in South Korea
- Volunteers
- 108 people
- Sponsor
- Idience Co., Ltd.
Non Hodgkin Lymphoma, Diffuse Large B Cell Lymphoma
Safety, PK/PD, and Clinical Activity of KT-413 in Adult Patients with Relapsed or Refractory B-cell NHL
This Phase 1a/1b study will evaluate the safety, tolerability and the pharmacokinetics/ pharmacodynamics (PK/ PD) of KT-413 in patients with R/R NHL.
- Conditions
- Non Hodgkin Lymphoma, Diffuse Large B Cell Lymphoma, DLBCL, MYD88 Gene Mutation
- Trial name
- NCT05233033
- Testing
- KT-413
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Kymera Therapeutics, Inc.
Solid Tumor, Tumors of Any Histology With Activating Trk (NTRK) Point or NTRK Fusion Mutations
Phase 1 Study of PLX7486 as Single Agent in Patients With Advanced Solid Tumors
The objective of this study is to determine the safety, pharmacokinetics, maximum tolerated dose/recommended Phase 2 dose, and efficacy of PLX7486.
- Conditions
- Solid Tumor, Tumors of Any Histology With Activating Trk (NTRK) Point or NTRK Fusion Mutations, Tenosynovial Giant Cell Tumor
- Trial name
- NCT01804530
- Testing
- PLX7486 TsOH
- Where
- 4 sites in United States
- Volunteers
- 59 people
- Sponsor
- Plexxikon
Non-small Cell Lung Cancer
Non-interventional, Prospective Study to Determine Prevalence of EGFR Mutations in Non-small Cell Lung Cancer
This prospective, multicountry, multicentre, non-interventional study plans to include patients who have undergone surgery for early-stage (IA to IIIB on the basis of pathologic criteria) non-squamous NSCLC up to 6 weeks prior to…
- Trial name
- EARLY-EGFR (NCT04742192)
- Where
- 33 sites in Argentina, Chile and 13 more
- Volunteers
- 601 people
- Sponsor
- AstraZeneca
Fallopian Tube, Primary Peritoneal Cancer
Study of ABT-767 in Subjects With Breast Cancer 1 and Breast Cancer 2 (BRCA 1 and BRCA 2) Mutations and Solid Tumors or High Grade Serous Ovarian, Fallopian Tube, or Primary Peritoneal Cancer
This is a Phase 1, dose escalation trial evaluating the tolerability, pharmacokinetics, and pharmacodynamics of ABT-767 in subjects with advanced Breast Cancer 1 or 2 gene (BRCA1 or BRCA2)-mutated solid tumors and high grade…
- Conditions
- Fallopian Tube, Primary Peritoneal Cancer, Solid Tumors (e.g. Breast, Ovarian, Prostate, or Pancreatic) and Ovarian
- Trial name
- NCT01339650
- Testing
- ABT-767
- Where
- 3 sites in Netherlands
- Volunteers
- 93 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
EGFR Gene Mutation, Non Small Cell Lung Cancer Stage IIIA
Icotinib as Consolidation Therapy After Chemoradiotherapy in EGFR-Mutant Stage IIIA-IIIB Non-small Cell Lung Cancer
The main purpose of this study is to evaluate the relapse free survival of patients who have EGFR-mutant stage IIIA-IIIB Non-small Cell Lung Cancer and receive Icotinib as consolidation therapy after synchronous or sequential…
- Conditions
- EGFR Gene Mutation, Non Small Cell Lung Cancer Stage IIIA, Non Small Cell Lung Cancer Stage IIIB
- Trial name
- NCT03396185
- Testing
- Icotinib
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.