Clinical trials: Genetic Mutation
Solid Tumor, Adult, NSCLC
Adagrasib in Combination With Nab-Sirolimus in Patients With Advanced Solid Tumors and Non-Small Cell Lung Cancer With a KRAS G12C Mutation (KRYSTAL -19)
This study will evaluate the safety, MTD and/or RP2D, PK, and clinical activity of the combination of adagrasib with nab-sirolimus in patients with advanced solid tumors/NSCLC with a KRAS G12C mutation.
- Conditions
- Solid Tumor, Adult, NSCLC, Advanced Cancer, Metastatic Cancer
- Trial name
- NCT05840510
- Testing
- Adagrasib, nab-Sirolimus
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Mirati Therapeutics Inc.
Essential Thrombocythemia (ET)
Exploratory Multi-centre Trial In Patients With ET Treated With XAGRID®
This study is hypothesis-generating to explore the impact of JAK2 (V617F) mutation status on the treatment response to anagrelide hydrochloride
- Trial name
- EMIX (NCT01352585)
- Testing
- Anagrelide hydrochloride
- Where
- 10 sites in Italy
- Volunteers
- 47 people
- Sponsor
- Shire
Farber Disease, Farber's Disease
Farber Disease Natural History Study
The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber…
- Conditions
- Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency
- Trial name
- NCT03233841
- Where
- 15 sites in United States, Argentina and 7 more
- Volunteers
- 45 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Non-Squamous Non-Small Cell Lung Cancer
An Observational Study of Tarceva (Erlotinib) in First Line in Patients With Advanced EGFR Mutation-Positive Non-Small Cell Lung Cancer (GERTAC)
This observational study will evaluate the efficacy and safety of Tarceva (erlotinib) in first-line therapy in patients with locally advanced or metastatic EGFR mutation-positive non-small cell lung cancer.
- Trial name
- NCT01790217
- Where
- Germany
- Volunteers
- 52 people
- Sponsor
- Hoffmann-La Roche
FLT3-ITD Mutation, FLT3/TKD Mutation
Individual Patient Compassionate Use of Crenolanib
Compassionate use of crenolanib for patients with serious life-threatening illness that have exhausted all available therapies used to treat the disease, with no other viable therapy options, who is not eligible for clinical…
- Conditions
- FLT3-ITD Mutation, FLT3/TKD Mutation, PDGFR-Alpha D842V, PDGFRA Gene Amplification
- Trial name
- NCT03620318
- Testing
- Crenolanib besylate
- Where
- Italy
- Sponsor
- Arog Pharmaceuticals, Inc.
Acute Myeloid Leukemia
A Clinical Study of SKLB1028 Capsule in the Treatment of Recurrence/Refractory AML Patients
Patients will receive oral SKLB1028 for 28 days as a course of treatment, and then to evaluate the side effects,tolerability and best dose for treating relapsed or refractory acute myeloid leukemia With FLT3 Mutations.
- Trial name
- NCT04015024
- Testing
- SKLB1028 150mg bid, SKLB1028 200mg bid, SKLB1028 300mg qd
- Where
- China
- Volunteers
- 30 people
- Sponsor
- CSPC ZhongQi Pharmaceutical Technology Co., Ltd.
Parkinson's Disease
A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation
Primary Objectives: Part 1: To determine the safety and tolerability of 4, 8, and 15 milligrams of GZ/SAR402671 (venglustat) administered orally for 4 weeks, as compared to placebo in participants with early-stage Parkinson's…
- Trial name
- MOVES-PD (NCT02906020)
- Testing
- venglustat GZ/SAR402671
- Where
- 52 sites in United States, Austria and 14 more
- Volunteers
- 273 people
- Sponsor
- Genzyme, a Sanofi Company
X-Linked Hypophosphataemia
A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH
This study aims to provide Health-related Quality of Life (HRQoL) data from children and adolescents with growing skeletons in the United Kingdom (UK), including those treated with burosumab or alternative XLH treatment, as part…
- Trial name
- NCT04819490
- Where
- 6 sites in United Kingdom
- Volunteers
- 32 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
NSCLC, EGFR Activating Mutation
TY-9591 in the Patients With EGFR Mutations in Advanced NSCLC With Brain Metastases
This study is to evaluate the efficacy and safety of TY-9591 in first-line treatment of patients with EGFR-sensitive mutation-positive non-small cell lung cancer with brain metastases compared to Osimertinib.
- Conditions
- NSCLC, EGFR Activating Mutation, Brain Metastases
- Trial name
- NCT05948813
- Testing
- TY-9591, Osimertinib
- Where
- China
- Volunteers
- 420 people
- Sponsor
- TYK Medicines, Inc
PIK3CA-Related Overgrowth Spectrum (PROS), Lymphatic Malformations
A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study)
This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, zovegalisib (RLY-2608), in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations…
- Conditions
- PIK3CA-Related Overgrowth Spectrum (PROS), Lymphatic Malformations, Vascular Malformations, PIK3CA Mutation
- Trial name
- NCT06789913
- Testing
- RLY-2608
- Where
- 40 sites in United States, Australia and 7 more
- Volunteers
- 277 people
- Sponsor
- Relay Therapeutics, Inc.
Non-Small Cell Lung Cancer
Sunvozertinib (DZD9008) in Pretreated Lung Cancer Patients with EGFR Exon20 Insertion Mutation (WU-KONG6)
This is a Phase II, single-arm, multi-center clinical study to evaluate the anti-tumor efficacy, safety, tolerability and pharmacokinetics of Sunvozertinib in participants with locally advanced or metastatic NSCLC carrying EGFR…
- Trial name
- NCT05712902
- Testing
- Sunvozertinib
- Where
- 33 sites in China
- Volunteers
- 104 people
- Sponsor
- Dizal Pharmaceuticals
Melanoma and Metastatic Colorectal Cancer
A Phase I Study of Oral LGX818 in Adult Patients With Advanced or Metastatic BRAF Mutant Melanoma
CLGX818X2101 is a first-time in-human, phase I study to establish the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of daily administered LGX818 (daily, twice daily and/or every-other-day), a RAF kinase…
- Trial name
- NCT01436656
- Testing
- LGX818
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 107 people
- Sponsor
- Pfizer
Cystic Fibrosis
A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for…
- Trial name
- NCT03559062
- Testing
- TEZ/IVA, IVA
- Where
- 27 sites in Australia, Belgium and 7 more
- Volunteers
- 67 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)
The purpose of this program is to provide elexacaftor(ELX, VX-445)/tezacaftor(TEZ)/ivacaftor(IVA) combination therapy to CF patients in critical need who are 12 years of age and older, heterozygous for F508del and a minimal…
- Trial name
- NCT04058210
- Testing
- ELX/TEZ/IVA, IVA
- Sponsor
- Vertex Pharmaceuticals Incorporated
NSCLC
Poziotinib in Patients With NSCLC Having EGFR or HER2 Exon 20 Insertion Mutation
This is a Phase 2, open-label, multicenter study to evaluate the efficacy and the safety/tolerability of poziotinib in two patient cohorts for up to 114 previously treated NSCLC patients with any systemic therapy (57 patients…
- Trial name
- NCT04044170
- Testing
- Poziotinib
- Where
- 26 sites in China, South Korea
- Volunteers
- 5 people
- Sponsor
- Hanmi Pharmaceutical Company Limited
Leukemia, Myeloid, Chronic, Myeloid Leukemia
Study of HQP1351 in Subjects With Refractory CML and Ph+ ALL
A multi-center, open-label, randomized, phase Ib study to evaluate the pharmacokinetics (PK) of HQP1351 and to determine the recommended phase 2 dose (RP2D) of HQP1351 in subjects with CML chronic phase (CP), accelerated phase…
- Conditions
- Leukemia, Myeloid, Chronic, Myeloid Leukemia, Chronic Myeloid Leukemia, Philadelphia Positive Acute Lymphoblastic Leukemia
- Trial name
- NCT04260022
- Testing
- Ascentage Pharma HQP1351 bioavailable inhibitor, Blinatumomab
- Where
- 9 sites in United States, Canada
- Volunteers
- 242 people
- Sponsor
- Ascentage Pharma Group Inc.
Non-Small Cell Lung Carcinoma
Study of Capmatinib in Indian Patients With MET Exon 14 Skipping Mutation Positive Advanced NSCLC.
The Drugs Controller General of India (DCGI) granted approval for Rahika® (capmatinib) film-coated tablets (150 mg and 200 mg) for the treatment of adult patients with advanced or metastatic non-small cell lung cancer (NSCLC)…
- Trial name
- NCT05110196
- Testing
- Capmatinib 150 mg, Capmatinib 200 mg
- Where
- 15 sites in India
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Carcinoma, Non-Small-Cell Lung
Study of Immunotherapy (Sasanlimab) in Combination With Targeted Therapies in People With Advanced Non-small Cell Lung Cancer (NSCLC) (Landscape 1011 Study)
Phase 1b/Phase 2 Umbrella Study; open-label, multi-center, parallel group study. Sasanlimab (a PD-1 antagonist monoclonal antibody) will be combined with a different targeted therapy in each sub-study.
- Trial name
- NCT04585815
- Testing
- Sasanlimab Prefilled syringe, Encorafenib, Binimetinib
- Where
- 55 sites in United States, Australia and 2 more
- Volunteers
- 34 people
- Sponsor
- Pfizer
Carcinoma, Non-Small-Cell Lung
A Study of FWD1509 in Adults With Non-Small Cell Lung Cancer
The purpose of this study is to characterize the safety and tolerability of FWD1509 MsOH in advanced NSCLC patients and establish the maximum tolerable dose (MTD), recommended phase 2 dose (RP2D) in advanced NSCLC patients.
- Trial name
- FWD1509 (NCT05068024)
- Testing
- FWD1509 MsOH
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Forward Pharmaceuticals Co., Ltd.
Breast Cancer, Ovarian Cancer
Study to Evaluate TNG348 Alone and With a PARP Inhibitor in Patients With BRCA 1/2 Mutant or HRD+ Solid Tumors
The goal of this interventional clinical trial is to learn about TNG348, a ubiquitin specific peptidase 1 (USP1) inhibitor, alone and in combination with olaparib in patients with BRCA 1/2 mutant or HRD+ solid tumors.
- Conditions
- Breast Cancer, Ovarian Cancer, Pancreas Cancer, Prostate Cancer
- Trial name
- NCT06065059
- Testing
- TNG348, Olaparib
- Where
- 7 sites in United States
- Volunteers
- 7 people
- Sponsor
- Tango Therapeutics, Inc.
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