Clinical trials: Gene Mutations
NSCLC
A Study of Poziotinib in Previously Treated Participants With Locally Advanced or Metastatic NSCLC Harboring HER2 Exon 20 Mutations
The primary purpose of this study is to compare the progression-free survival (PFS) in participants with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring human epidermal growth factor receptor 2 (HER2)…
- Trial name
- PINNACLE (NCT05378763)
- Testing
- Poziotinib, Docetaxel
- Where
- United States
- Volunteers
- 268 people
- Sponsor
- Spectrum Pharmaceuticals, Inc
Lung Cancer
Gefitinib Combine Radiotherapy as Therapy for Patients With NSCLC Harbouring Sensitive Mutations of EGFR
To explore the survival benefit of the gefitinib combined with radiotherapy as adjuvant therapy for completely resected patients with Pathological stage IIIA-N2 NSCLC harbouring sensitive mutations of EGFR.
- Trial name
- NCT03381430
- Testing
- Gefitinib
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Acute Myeloid Leukemia
Study of Crenolanib Combined With Chemotherapy in FLT3-mutated Acute Myeloid Leukemia Patients
This is an open label, two-arm, Phase I-II trial, non-randomized. Arm 1: crenolanib with standard chemotherapy (Idarubicin/Cytarabine, MEC;Mitoxantrone/Etoposide/Cytarabine, FLAG-Ida: Fludarabine/Cytarabine/G-CSF/Idarubicin) Arm…
- Trial name
- NCT02400281
- Testing
- Crenolanib besylate, Idarubicin, Cytarabine
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Arog Pharmaceuticals, Inc.
Amyotrophic Lateral Sclerosis (ALS), Mutation in the Superoxide Dismutase-1 (SOD1) Gene
First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1)…
- Trial name
- GOALS (NCT06351592)
- Testing
- ALN-SOD, Diluent
- Where
- 22 sites in Australia, Belgium and 7 more
- Volunteers
- 54 people
- Sponsor
- Regeneron Pharmaceuticals
Breast Cancer Metastatic, BRCA 1 Gene Mutation
A Phase II Study to Assess the Efficacy and Safety of SC10914 in the Metastatic Breast Cancer Patients
This open label, multi-centre phase II study will assess the efficacy and safety of single agent SC10914 in metastatic breast cancer patients with gBRCA 1/2 mutations.
- Conditions
- Breast Cancer Metastatic, BRCA 1 Gene Mutation, BRCA 2 Gene Mutation
- Trial name
- NCT04556292
- Testing
- SC10914
- Where
- China
- Volunteers
- 78 people
- Sponsor
- Jiangxi Qingfeng Pharmaceutical Co. Ltd.
Pompe Disease
Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Pompe disease (also known as glycogen storage disease type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Trial name
- NCT00113035
- Where
- 5 sites in United States
- Volunteers
- 60 people
- Sponsor
- Genzyme, a Sanofi Company
Acute Myeloid Leukemia (AML), Acute Promyelocytic Leukemia (APL)
Accuracy Testing of the Chromosomal Aberration and Gene Mutation Markers of the AMLProfiler
This clinical study will demonstrate the accuracy of the chromosomal aberration and gene mutation markers of the AMLProfiler molecular diagnostic assay and generate clinical performance data to support a Pre-Market Approval (PMA)…
- Conditions
- Acute Myeloid Leukemia (AML), Acute Promyelocytic Leukemia (APL), Refractory Anemia With Excess of Blasts (RAEB)
- Trial name
- NCT01463410
- Where
- 5 sites in United States, Germany and 2 more
- Volunteers
- 264 people
- Sponsor
- Skyline Diagnostics BV
Advanced Solid Cancer, Pancreatic Ductal Adenocarcinoma (PDAC)
Phase 1 Study of NW-301V TCR-T Cells in Advanced Solid Tumors With KRAS G12V Mutation
This is a single-arm, multicenter, open-label, Phase 1a/1b dose-escalation and dose-expansion study designed to evaluate the safety, preliminary antitumor activity, and pharmacokinetic/pharmacodynamic (PK/PD) characteristics of…
- Conditions
- Advanced Solid Cancer, Pancreatic Ductal Adenocarcinoma (PDAC), Colorectal Adenocarcinoma, Lung Adenocarcinoma
- Trial name
- NCT07875920
- Testing
- Fludarabine, Cyclophosphamide, NW-301V
- Where
- 2 sites in China
- Volunteers
- 66 people
- Sponsor
- Neowise Biotechnology
Sickle Cell Disease (SCD)
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis…
- Trial name
- STAND (NCT03814746)
- Testing
- Crizanlizumab (SEG101)
- Where
- 58 sites in United States, Belgium and 19 more
- Volunteers
- 253 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Solid Tumors, Colorectal Cancer (CRC)
A Study to Investigate the Safety and Efficacy of KQB368 as Monotherapy in Participants With Advanced Solid Malignancies
The goal of this study is to learn if KQB368 works to treat advanced solid malignancies in adults. The study will also learn about the safety of KQB368.
- Conditions
- Advanced Solid Tumors, Colorectal Cancer (CRC), Non-Small Cell Lung Cancer, Uterine Cancer
- Trial name
- NCT07542704
- Testing
- KQB368
- Volunteers
- 48 people
- Sponsor
- Kumquat Biosciences Inc.
Relapsed/Refractory Leukemias, Acute Lymphoblastic Leukemia
A Study of Revumenib in Combination With Chemotherapy in Participants With R/R Acute Leukemia
The purpose of this study is to determine the safety and tolerability of revumenib when given in combination with 2 different chemotherapy regimens in participants with relapsed/refractory acute leukemias harboring KMT2A…
- Conditions
- Relapsed/Refractory Leukemias, Acute Lymphoblastic Leukemia, Acute Lymphocytic Leukemia, Mixed Phenotype Acute Leukemia
- Trial name
- NCT05326516
- Testing
- Revumenib, Chemotherapy Regimen 1, Chemotherapy Regimen 2
- Where
- 11 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Syndax Pharmaceuticals
Solid Tumor
Dose-Esc/Exp RMC4630 & Cobi in Relapsed/Refractory Solid Tumors & RMC4630& Osi in EGFR+ Locally Adv/Meta NSCLC
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles of RMC-4630 and cobimetinib in adult participants with relapsed/refractory solid tumors with specific…
- Trial name
- NCT03989115
- Testing
- RMC-4630, Cobimetinib, Drug: Osimertinib
- Where
- 24 sites in United States, South Korea
- Volunteers
- 113 people
- Sponsor
- Revolution Medicines, Inc.
Non-Squamous Non-Small Cell Lung Cancer
ASPET Study: An Observational Study of Tarceva (Erlotinib) in First Line in Patients With Advanced Non-Small Cell Lung Cancer Harbouring EGFR Activating Mutations
This observational study will evaluate the patterns of disease progression in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harbouring EGFR activating mutations receiving Tarceva (erlotinib) as…
- Trial name
- NCT01723878
- Where
- 43 sites in Spain
- Volunteers
- 144 people
- Sponsor
- Hoffmann-La Roche
Hypercholesterolemia
Evolocumab Compared to LDL-C Apheresis in Patients Receiving LDL-C Apheresis Prior to Study Enrollment
To evaluate the efficacy of subcutaneous (SC) evolocumab, compared to regularly scheduled low-density lipoprotein cholesterol (LDL-C) apheresis, on reducing the need for future apheresis.
- Trial name
- NCT02585895
- Testing
- Evolocumab, Low-density Lipoprotein Cholesterol (LDL-C) Apheresis
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 39 people
- Sponsor
- Amgen
Advanced or Metastatic NSCLC Harboring Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertion (ex20ins) Mutations
A Study of Zipalertinib in Patients With Advanced Non-Small Cell Lung Cancer With Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertions or Other Uncommon Mutation.
The purpose of this study is to evaluate the safety, efficacy and pharmacokinetics (PK) of zipalertinib in participants with locally advanced or metastatic Non-Small Cell Lung Cancer (NSCLC) harboring EGFR ex20ins mutations and…
- Trial name
- REZILIENT2 (NCT05967689)
- Testing
- TAS6417, CYP Cocktail, Transporter Cocktail
- Where
- 80 sites in United States, Australia and 10 more
- Volunteers
- 220 people
- Sponsor
- Taiho Oncology, Inc.
Ovarian Cancer
Prevalence of BRCA1 and BRCA2 Mutations in Ovarian Cancer Patients in the Gulf Region
A prospective, multi-centre, epidemiological observational study designed to evaluate the prevalence of BRCA1 and BRCA2 (BReast CAncer gene) mutations in current and newly diagnosed ovarian cancer patients across different…
- Trial name
- PREDICT (NCT03082976)
- Where
- United Arab Emirates
- Volunteers
- 108 people
- Sponsor
- AstraZeneca
Mastocytosis
Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis
This is a 12 weeks study aimed at assessing the safety and efficacy of 2 doses of AB1010 in patients suffering from indolent systemic mastocytosis with handicap.
- Trial name
- NCT00831974
- Testing
- masitinib (AB1010)
- Volunteers
- 23 people
- Sponsor
- AB Science
Inherited Retinal Dystrophy Due to RPE65 Mutations
Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Multi-site, non-randomized, observational study, for up to 15 years after subretinal AAV2-hRPE65v2 administration for each subject.
- Trial name
- NCT03602820
- Testing
- AAV2-hRPE65v2
- Volunteers
- 41 people
- Sponsor
- Genentech, Inc.
Mitochondrial Myopathies, Mitochondrial Pathology
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary…
- Conditions
- Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases
- Trial name
- NuPower (NCT05162768)
- Testing
- Elamipretide
- Where
- 32 sites in United States, Australia and 8 more
- Volunteers
- 102 people
- Sponsor
- Stealth BioTherapeutics Inc.
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