Clinical trials: Genetic Mutation
Non-Small Cell Lung Cancer
Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322)
This is an Open-label, multicenter clinical study conducted in two Phases to establish the efficacy, safety, tolerability, and pharmacokinetics of the ataxia telangiectasia mutated and Rad3-related protein kinase (ATR) inhibitor…
- Trial name
- NCT05882734
- Testing
- M1774, Cemiplimab
- Where
- 54 sites in United States, Belgium and 6 more
- Volunteers
- 61 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
NSCLC
Phase 2 Study of Poziotinib in Participants With NSCLC Having EGFR or HER2 Exon 20 Insertion Mutation
This is a Phase 2, open-label, multi-center study to evaluate the efficacy and the safety/tolerability of poziotinib in seven participant cohorts for up to 603 previously treated and treatment-naïve NSCLC participant.
- Trial name
- NCT03318939
- Testing
- Poziotinib
- Where
- 63 sites in United States, Belgium and 6 more
- Volunteers
- 648 people
- Sponsor
- Spectrum Pharmaceuticals, Inc
Factor V Leiden, Prothrombin G20210A
Engagement Study for Participants With Factor V Leiden and Prothrombin G20210A Mutations
This study will enrol individuals who have, or may have, Factor V Leiden or Prothrombin G20210A mutations, which are genetic changes linked to an increased risk of blood clots.
- Trial name
- NCT07584265
- Where
- United Kingdom
- Volunteers
- 100 people
- Sponsor
- Richmond Research Institute
Chronic Myeloid Leukemia - Accelerated Phase
A Pivotal Study of HQP1351 in Patients of Chronic Myeloid Leukemia in Accelerated Phase With T315I Mutation
The purpose of this study is to evaluate the efficacy of HQP1351 in patients with chronic myeloid leukemia in accelerated phase (CML-AP) harboring T315I mutation.
- Trial name
- NCT03883100
- Testing
- HQP1351
- Where
- 9 sites in China
- Volunteers
- 23 people
- Sponsor
- Ascentage Pharma Group Inc.
Congenital Hearing Loss
a Study in Subjects With Otoferlin Mutation-related Hearing Loss Using RNA Base-eDiting Therapy(SOUND)
The purpose of the study is to determine whether HG205 as CRISPR/Cas13 RNA base-editing therapy is safe and effective for the treatment of hearing loss caused by p.Q829X mutation in OTOF gene.
- Trial name
- NCT06025032
- Testing
- HG205
- Where
- China
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
RAS Mutation, NRAS Gene Mutation
NAUTILUS: OKI-179 Plus Binimetinib in Patients with Advanced Solid Tumors in the RAS Pathway (Phase 1b) and NRAS-mutated Melanoma (Phase 2)
The NAUTILUS study is a Phase 1b/2, multi-center, open-label study in which patients with activating mutations in the RAS pathway (Phase 1b) and patients with NRAS-mutated Melanoma (Phase 2) will be treated with a combination of…
- Conditions
- RAS Mutation, NRAS Gene Mutation, Melanoma
- Trial name
- NCT05340621
- Testing
- OKI-179 + binimetinib
- Where
- 12 sites in United States
- Volunteers
- 36 people
- Sponsor
- OnKure, Inc.
Non-Small Cell Lung Cancer, EGFR Mutation
A Study to Evaluate ANS014004 in Combination With EGFR-TKI in Non-Small Cell Lung Cancer
Protocol Title A Study to Evaluate ANS014004 in Combination with EGFR-TKI in Patients with EGFR Mutation-Positive Locally Advanced or Metastatic Non-Small Cell Lung Cancer The main purpose of this research study is to Find a safe…
- Conditions
- Non-Small Cell Lung Cancer, EGFR Mutation, Metastatic Lung Cancer, MET Alteration
- Trial name
- NCT07285148
- Testing
- ANS014004 + PLB1004, ANS014004 + Osimertinib
- Where
- United States
- Volunteers
- 253 people
- Sponsor
- Beijing Pearl Biotechnology Limited Liability Company
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.
Cohort 1a and 1b: Glioma (Advanced Gliomas and Glioblastoma Multiforme), Cohort 2a and 2b: Hepatobiliary Tumors (Hepatocellular Carcinoma, Bile Duct Carcinoma, Intrahepatic Cholangiocarcinoma, Other Hepatobiliary Carcinomas)
A Study of FT-2102 in Patients With Advanced Solid Tumors and Gliomas With an IDH1 Mutation
This Phase 1/2 study will evaluate the safety, efficacy, PK, and PD of FT-2102 as a single agent and in combination with other anti-cancer drugs in patients with advanced solid tumors and gliomas.
- Conditions
- Cohort 1a and 1b: Glioma (Advanced Gliomas and Glioblastoma Multiforme), Cohort 2a and 2b: Hepatobiliary Tumors (Hepatocellular Carcinoma, Bile Duct Carcinoma, Intrahepatic Cholangiocarcinoma, Other Hepatobiliary Carcinomas), Cohort 3a and 3b: Chondrosarcoma, Cohort 4a and 4b: Intrahepatic Cholangiocarcinoma
- Trial name
- NCT03684811
- Testing
- FT-2102, Azacitidine, Nivolumab
- Where
- 26 sites in United States, Australia and 4 more
- Volunteers
- 93 people
- Sponsor
- Forma Therapeutics, Inc.
Solid Tumor, Adult, Non-small Cell Lung Cancer
A Study to Evaluate KIN-2787 in Participants With BRAF and/or NRAS Mutation Positive Solid Tumors
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of KIN-2787 in adults with BRAF/NRAS-mutated advanced or metastatic solid tumors.
- Conditions
- Solid Tumor, Adult, Non-small Cell Lung Cancer, Melanoma
- Trial name
- NCT04913285
- Testing
- KIN-2787, KIN-2787 and binimetinib
- Where
- 48 sites in United States, Australia and 5 more
- Volunteers
- 400 people
- Sponsor
- Pierre Fabre Medicament
Carcinoma, Non-Small-Cell Lung
Afatinib Expanded Access Program
This is an open-label, multi-center, single-arm trial, designed to provide early access to afatinib and to provide additional information on the safety and efficacy of afatinib in advanced NSCLC patients who harbor an EGFR…
- Trial name
- NCT01649284
- Testing
- afatinib
- Where
- 66 sites in United States
- Sponsor
- Boehringer Ingelheim
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
Advanced Solid Tumors
A Study of INCB099280 in Combination With Adagrasib in Adults With Advanced Solid Tumors Harboring a KRASG12C Mutation
The purpose of this study is to evaluate the safety and tolerability of INCB099280 in combination with adagrasib and to establish the MTD or identify RDE(s) for the combination of INCB099280 and adagrasib.
- Trial name
- NCT06039384
- Testing
- INCB099280, adagrasib
- Where
- 14 sites in United States, Italy and 2 more
- Volunteers
- 6 people
- Sponsor
- Incyte Corporation
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
The primary objective of the study was to evaluate the efficacy of lumacaftor in combination with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation…
- Trial name
- TRANSPORT (NCT01807949)
- Testing
- Lumacaftor Plus Ivacaftor Combination, Ivacaftor
- Where
- 82 sites in United States, Australia and 8 more
- Volunteers
- 563 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Breast Cancer
Study to Assess the Efficacy and Safety of Alpelisib Plus Fulvestrant in Participants With HR-positive (HR+), HER2-negative, Advanced Breast Cancer After Treatment With a CDK4/6 Inhibitor and an Aromatase Inhibitor.
The purpose of this study is to complement Study CBYL719C2301 (SOLAR-1) and obtain more comprehensive data on the efficacy and safety of alpelisib (BYL719) in combination with fulvestrant compared with placebo plus fulvestrant in…
- Trial name
- EPIK-B5 (NCT05038735)
- Testing
- Alpelisib, Fulvestrant
- Where
- 68 sites in Belgium, Bulgaria and 15 more
- Volunteers
- 210 people
- Sponsor
- Novartis Pharmaceuticals
Non-Small Cell Lung Cancer
A Global Study to Assess the Effects of Osimertinib in Participants With EGFRm Stage IA2-IA3 NSCLC Following Complete Tumour Resection
This is a global study to assess the effects of osimertinib in participants with EGFRm stage IA2-IA3 non-small cell lung cancer following complete tumour resection.
- Trial name
- ADAURA2 (NCT05120349)
- Testing
- Osimertinib
- Where
- 139 sites in United States, Argentina and 18 more
- Volunteers
- 390 people
- Sponsor
- AstraZeneca
Retinitis Pigmentosa 11, Retinal Degeneration
MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)
A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy
- Conditions
- Retinitis Pigmentosa 11, Retinal Degeneration, Eye Diseases, Retinal Disease
- Trial name
- NCT06455826
- Testing
- VP-001
- Where
- 5 sites in United States
- Volunteers
- 6 people
- Sponsor
- PYC Therapeutics
Advanced Stage Solid Tumors
ATRi Transition Rollover Study
The main purpose of the study was to monitor the safety of participant receiving long-term treatment of Berzosertib as monotherapy.
- Trial name
- NCT03309150
- Testing
- Berzosertib
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Non-Small Cell Lung Cancer
A Study of IRESSA Treatment Beyond Progression in Addition to Chemotherapy Versus Chemotherapy Alone
The purpose of this study is to assess the efficacy and safety of gefitinib in patients who have progressed on first line gefitinib, comparing continuing gefitinib in addition to cisplatin plus pemetrexed combination chemotherapy…
- Trial name
- IMPRESS (NCT01544179)
- Testing
- Gefitinib, Pemetrexed, Cisplatin
- Where
- 61 sites in China, France and 9 more
- Volunteers
- 265 people
- Sponsor
- AstraZeneca
Low Grade Ovarian Serous Adenocarcinoma, Ovarian Cancer
A Study of Avutometinib (VS-6766) v. Avutometinib (VS-6766) + Defactinib in Recurrent Low-Grade Serous Ovarian Cancer With and Without a KRAS Mutation
This study will assess the safety and efficacy of avutometinib (VS-6766) monotherapy and in combination with defactinib in subjects with recurrent Low-Grade Serous Ovarian Cancer (LGSOC)
- Trial name
- RAMP 201 (NCT04625270)
- Testing
- avutometinib (VS-6766), avutometinib (VS-6766) and defactinib
- Where
- 47 sites in United States, Belgium and 5 more
- Volunteers
- 225 people
- Sponsor
- Verastem, Inc.
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