Clinical trials: Gene Mutations
Glioma, Recurrence
Vorasidenib Expanded Access Program
This is an expanded access program to provide vorasidenib for treatment of patients 12 years or older with IDH1- or IDH2-mutated glioma.
- Conditions
- Glioma, Recurrence, Disease Attributes, Pathologic Processes
- Trial name
- NCT05592743
- Testing
- Vorasidenib
- Sponsor
- Servier
Non-Small-Cell Lung Cancer (NSCLC), Epidermal Growth Factor Receptor Mutations
A Dose Escalation Study of ASP8273 in Subjects With Non-Small-Cell Lung Cancer (NSCLC) Who Have Epidermal Growth Factor Receptor (EGFR) Mutations
The purpose of this study is to assess the safety and tolerability of ASP8273 and to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D).
- Trial name
- NCT02113813
- Testing
- naquotinib, midazolam
- Where
- 12 sites in United States
- Volunteers
- 133 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Amyotrophic Lateral Sclerosis
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for people with a certain type of amyotrophic lateral sclerosis, also known as…
- Trial name
- NCT07223723
- Testing
- Tofersen
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Biogen
Advanced Solid Tumor, Non-Small Cell Lung Cancer
A First-in-Human Trial of BLU-924 (SAR449336) in Advanced Solid Tumors Harboring KRAS Mutations
A first in human study to evaluate the safety, tolerability, pharmacokinetics, and antitumor activity of BLU-924 / SAR449336, a pan-KRAS inhibitor, in participants with advanced Pancreatic Cancer, Non-Small Cell Lung Cancer, or…
- Conditions
- Advanced Solid Tumor, Non-Small Cell Lung Cancer, Colorectal Neoplasms, Pancreatic Ductal Adenocarcinoma
- Trial name
- NCT07629960
- Testing
- BLU-924
- Where
- 16 sites in United States, Spain
- Volunteers
- 265 people
- Sponsor
- Blueprint Medicines Corporation
Solid Tumor
Phase 1/2 Study of Derazantinib (ARQ 087) in Adult Subjects With Advanced Solid Tumors With FGFR Genetic Alterations
This was an open-label, Phase 1/2, dose escalation and signal finding study of derazantinib administered to patients with advanced solid tumors (Part 1; Dose Escalation/Food-effect Cohorts) or with advanced solid tumors with FGFR…
- Trial name
- NCT01752920
- Testing
- Derazantinib low dose range, Derazantinib middle dose range, Derazantinib high dose range
- Where
- 12 sites in United States, Italy
- Volunteers
- 119 people
- Sponsor
- Basilea Pharmaceutica
DMD, Duchenne Muscular Dystrophy
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.
- Conditions
- DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
- Trial name
- EXPLORE44OLE (NCT06244082)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 39 people
- Sponsor
- Avidity Biosciences, Inc.
Carcinoma, Pancreatic Ductal, Colorectal Neoplasms
A Study to Learn About the Study Medicine PF-07985045 When Given Alone or With Other Anti-cancer Therapies in People With Advanced Solid Tumors That Have a Change in a Gene.
The purpose of this study is to learn about the safety and effects of the study medicine when given alone or together with other anti-cancer therapies. Anti-cancer therapy is a type of treatment to stop the growth of cancer.
- Conditions
- Carcinoma, Pancreatic Ductal, Colorectal Neoplasms, Carcinoma, Non-Small-Cell Lung
- Trial name
- NCT06704724
- Testing
- PF-07985045, Gemcitabine, Nab-paclitaxel
- Where
- 13 sites in United States, Puerto Rico
- Volunteers
- 30 people
- Sponsor
- Pfizer
Metastatic Solid Tumors, Advanced Non-squamous Non-small-cell Lung Cancer
A First-in-human Study of BGB-53038, a Pan-KRAS Inhibitor, Alone or in Combinations in Participants With Advanced or Metastatic Solid Tumors With KRAS Mutations or Amplification
This is a first-in-human (FIH), open-label, multicenter, dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary antitumor activity of BGB-53038 as…
- Conditions
- Metastatic Solid Tumors, Advanced Non-squamous Non-small-cell Lung Cancer, Advanced Colorectal Cancer, Advanced Pancreatic Ductal Adenocarcinoma
- Trial name
- NCT06585488
- Testing
- BGB-53038, Tislelizumab, Cetuximab
- Where
- 17 sites in United States, Australia and 3 more
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- BeOne Medicines
HIV Infections, Human Immunodeficiency Virus
SP01A: The Study of an Oral Entry Inhibitor in Treatment-Experienced HIV Patients
This is a 28-day, multi-center, placebo-controlled study designed to look at the dose response, efficacy, and safety of SP01A, given as a pill to be swallowed, in the treatment of HIV-infected subjects.
- Trial name
- NCT00299897
- Testing
- SP01A
- Where
- 5 sites in United States
- Volunteers
- 60 people
- Sponsor
- Samaritan Pharmaceuticals, Inc
Solid Tumor
A Study to Evaluate Rucaparib in Participants With Solid Tumors and With Deleterious Mutations in HRR Genes
A Phase 2, open-label, single-arm trial to evaluate the response of rucaparib in participants with various solid tumors and with deleterious mutations in Homologous Recombination Repair (HRR) genes.
- Trial name
- LODESTAR (NCT04171700)
- Testing
- Rucaparib
- Where
- 19 sites in United States
- Volunteers
- 83 people
- Sponsor
- pharmaand GmbH
Cystic Fibrosis
Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR…
- Trial name
- NCT01946412
- Testing
- Ivacaftor
- Where
- 16 sites in United States, Canada and 1 more
- Volunteers
- 33 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Mastocytosis
Masitinib in Patients With Mastocytosis With Handicap and Bearing the D816V Mutation
The objective is to compare efficacy and safety of AB1010 at 3 or 6 mg/kg/day in treatment of patients with mastocytosis with handicap and bearing activating point mutations in the phosphotransferase domain of c-Kit such as the…
- Trial name
- NCT01266369
- Testing
- masitinib
- Volunteers
- 21 people
- Sponsor
- AB Science
Wilson Disease, Wilson's Disease
A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)
The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therapy, in adults and adolescents with Wilson disease (WD).
- Conditions
- Wilson Disease, Wilson's Disease, Wilsons Disease
- Trial name
- PM577a (NCT07748403)
- Testing
- PM577a
- Where
- 4 sites in United States, New Zealand
- Volunteers
- 42 people
- Sponsor
- Prime Medicine, Inc.
Pancreatic Adenocarcinoma Metastatic, BRCA1 Mutation
SHARON: A Clinical Trial for Metastatic Cancer Using Chemotherapy and Patients' Own Stem Cells
The clinical trial is a phase 1, single-arm trial that will evaluate the safety of the investigational treatment on metastatic pancreatic cancer and metastatic breast cancer.
- Conditions
- Pancreatic Adenocarcinoma Metastatic, BRCA1 Mutation, BRCA2 Mutation, Pancreatic Acinar Cell Carcinoma
- Trial name
- SHARON (NCT04150042)
- Testing
- Melphalan, BCNU, Vitamin B12B
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- General Oncology, Inc.
Ovarian Neoplasms
Characterizing the Cross-sectional Approach to Investigate the Prevalence of Tissue BRCA1/2 Mutations in Newly Diagnosed Advanced Ovarian Cancer Patients
This is a multi-center, observational study in Japan. Patients with newly diagnosed FIGO stage III - IV advanced OC will be enrolled sequentially.
- Trial name
- CHRISTELLE (NCT04222465)
- Where
- 20 sites in Japan
- Volunteers
- 207 people
- Sponsor
- AstraZeneca
Advanced Solid Tumor, Metastatic Solid Tumor
Study of LP-184 in Patients With Advanced Solid Tumors
The primary objective of this study is to evaluate the safety, tolerability, MTD and RP2D of LP-184 in patients with advanced solid tumors who have relapsed from or are refractory to standard therapy or for whom no standard…
- Conditions
- Advanced Solid Tumor, Metastatic Solid Tumor, GBM, TNBC - Triple-Negative Breast Cancer
- Trial name
- NCT05933265
- Testing
- LP-184, Olaparib, Nivolumab & Ipilimumab
- Where
- 7 sites in United States
- Volunteers
- 64 people
- Sponsor
- Lantern Pharma Inc.
Advanced Solid Tumors With KRAS G12C Mutations, Solid Tumor, Adult
A Study Evaluating FMC-376 in Participants With KRAS G12C Mutated Solid Tumors
The goal of this clinical trial is to evaluate FMC-376 monotherapy and in combination with pembrolizumab in participants with advanced solid tumors with KRAS G12C mutations.
- Conditions
- Advanced Solid Tumors With KRAS G12C Mutations, Solid Tumor, Adult, Unresectable Solid Tumor, Metastatic Solid Tumor
- Trial name
- PROSPER (NCT06244771)
- Testing
- FMC-376
- Where
- 26 sites in United States, Australia and 1 more
- Volunteers
- 293 people
- Sponsor
- Frontier Medicines Corporation
Intrahepatic Cholangiocarcinoma (Icc), Advanced Cholangiocarcinoma
Efficacy and Safety of TT-00420 (Tinengotinib) Tablets Versus Chemotherapy in Patients With Advanced Intrahepatic Cholangiocarcinoma Harboring FGFR2 Fusions/Rearrangements or Mutations
This is an open-label, randomized, controlled, multicenter, phase III clinical study designed to evaluate the efficacy and safety of TT-00420 tablets as monotherapy versus chemotherapy in subjects with unresectable advanced or…
- Trial name
- NCT07328919
- Testing
- TT-00420 (tinengotinib), Oxaliplatin, fluorouracil, calcium folinate, irinotecan, capecitabine
- Where
- 20 sites in China
- Volunteers
- 138 people
- Sponsor
- TransThera Sciences (Nanjing), Inc.
Stargardt Disease-1
Study of STG-001 in Subjects With Stargardt Disease
This is an open-label, multicenter study in subjects with Stargardt disease, comparing 2 doses of STG-001 with regard to safety, pharmacokinetics and pharmacodynamics.
- Trial name
- NCT04489511
- Testing
- STG-001
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Stargazer Pharmaceuticals, Inc.
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations
The purpose of this study is: to evaluate the safety of oral QLT091001 to evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP due to RPE65 or LRAT mutations to evaluate…
- Trial name
- NCT01014052
- Testing
- QLT091001
- Where
- 7 sites in United States, Canada and 3 more
- Volunteers
- 32 people
- Sponsor
- QLT Inc.