Clinical trials: Mutations
Cystic Fibrosis
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for F508del.
- Trial name
- NCT03125395
- Testing
- LUM/IVA
- Where
- 20 sites in United States, Canada
- Volunteers
- 57 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Metastatic Non-Small Cell Lung Cancer
A Study of Gentuximab in Combination With Almonertinib in EGFR Mutation-positive Metastatic NSCLC
This is a prospective, multicenter, open label study to investigate the safety and efficacy of Gentuximab plus Almonertinib in metastatic NSCLC patients with EGFR mutation-positive.
- Trial name
- NCT05223595
- Testing
- Gentuximab
- Where
- China
- Volunteers
- 42 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Carcinoma, Non-Small-Cell Lung
Study of Capmatinib Efficacy in Comparison With Docetaxel in Previously Treated Participants With Non-small Cell Lung Cancer Harboring MET Exon 14 Skipping Mutation
The purpose of the study was to learn whether the study drug (capmatinib) helps to control lung cancer better compared to a single agent chemotherapy (docetaxel) and whether it is safe when given to patients suffering from a…
- Trial name
- GeoMETry-III (NCT04427072)
- Testing
- Capmatinib, Docetaxel
- Where
- 43 sites in Belgium, Brazil and 16 more
- Volunteers
- 22 people
- Sponsor
- Novartis Pharmaceuticals
Healthy Volunteers
A Study to Investigate the Effects of Acid Reducing Agents on Pharmacokinetics of PC14586 in Healthy Participants
This study will assess the effect of a Proton Pump Inhibitor (PPI) (rabeprazole) on the pharmacokinetics (PK) of PC14586 and the effect of an H2-receptor antagonist (famotidine) on the PK of PC14586
- Trial name
- NCT06054464
- Testing
- PC14586, Rabeprazole, Famotidine
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- PMV Pharmaceuticals, Inc
Retinitis Pigmentosa, Usher Syndrome Type 2
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with…
- Trial name
- HELIA (NCT05085964)
- Testing
- RNA antisense oligonucleotide for intravitreal injection
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Laboratoires Thea
Non Small Cell Lung Cancer
A Study of RC48-ADC in Subjects With Advanced Non-small Cell Lung Cancer
This study will evaluate the efficacy and safety of RC48-ADC for injection in subjects with advanced non-small cell lung cancer with HER2 overexpression or HER2 mutation.
- Trial name
- NCT04311034
- Testing
- RC48
- Where
- China
- Volunteers
- 37 people
- Sponsor
- RemeGen Co., Ltd.
Non-Squamous Non-Small Cell Lung Cancer
A Study of Onartuzumab in Combination With Erlotinib in Patients With MET-Positive Stage IIIB or IV Non-Small Cell Lung Cancer Carrying an Activating Epidermal Growth Factor Receptor (EGFR) Mutation
This multicenter, randomized, double-blind, placebo-controlled study will evaluate the safety and efficacy of onartuzumab in combination with erlotinib in patients with previously untreated, unresectable stage IIIB or IV…
- Trial name
- NCT01887886
- Testing
- erlotinib, onartuzumab
- Where
- 35 sites in United States, France and 6 more
- Volunteers
- 10 people
- Sponsor
- Hoffmann-La Roche
Advanced or Metastatic NSCLS With Exon 20 Insertion Mutation
REZILIENT3 (REsearching ZIpaLertinib In Egfr Non-small Cell Lung Cancer Tumors)
The purpose of this study is to evaluate the safety and efficacy of zipalertinib in combination with standard first-line platinum-based chemotherapy compared to chemotherapy alone, in patients with locally advanced or metastatic…
- Trial name
- REZILIENT3 (NCT05973773)
- Testing
- TAS6417
- Where
- 130 sites in United States, Belgium and 21 more
- Volunteers
- 285 people
- Sponsor
- Taiho Oncology, Inc.
CRC
The Efficacy of HLX208 (BRAF V600E Inhibitor) With Cetuximab for Metastatic Colorectal Cancer (mCRC) With BRAF V600E Mutation After First-line Treatment
An open, multicenter phase II clinical study to evaluate safety and efficacy of HLX208 (BRAF V600E inhibitor) combined with cetuximab for metastatic colorectal cancer (mCRC) with BRAF V600E Mutation after first-line treatment
- Trial name
- NCT04984369
- Testing
- HLX208, Cetuximab Injection [Erbitux]
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Shanghai Henlius Biotech
Non-Small Cell Lung Cancer
Assessing an Oral EGFR Inhibitor, Sunvozertinib in Patients Who Have Advanced Non-small Cell Lung Cancer With EGFR or HER2 Mutation (WU-KONG1)
This study will treat patients with advanced NSCLC with EGFR or HER2 mutation who have progressed following prior therapy. This is the first time this drug is tested in patients, and so it will help to understand what type of…
- Trial name
- NCT03974022
- Testing
- Sunvozertinib
- Where
- 124 sites in United States, Argentina and 11 more
- Volunteers
- 315 people
- Sponsor
- Dizal Pharmaceuticals
Cystic Fibrosis
Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation
The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD), of ivacaftor in children with cystic fibrosis (CF) who are 2 through 5 years of age and have a CF Transmembrane Conductance…
- Trial name
- NCT01705145
- Testing
- Ivacaftor
- Where
- 20 sites in United States, Canada and 1 more
- Volunteers
- 35 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Leber Congenital Amaurosis 10, Blindness
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (<8…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- BRIGHTEN (NCT04855045)
- Testing
- sepofarsen
- Where
- 9 sites in Belgium, Brazil and 5 more
- Volunteers
- 15 people
- Sponsor
- ProQR Therapeutics
Non-Small Cell Lung Cancer, EGFR Gene Mutation
D-0316 Versus Icotinib in Patients With Locally Advanced or Metastatic EGFR Sensitising Mutation Positive NSCLC
To assess the efficacy and safety of D-0316 versus Icotinib, a standard of care epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI), in patients with locally advanced or Metastatic Non Small Cell Lung Cancer…
- Trial name
- NCT04206072
- Testing
- D-0316 Capsule, Icotinib Hydrochloride Tablets
- Where
- 2 sites in China
- Volunteers
- 362 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Cancer
Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations
This was a 4-part (Part A, Part B, Part C and Part D), Phase I/IIa, multi-center, open label, study in pediatric subjects with refractory or recurrent tumors.
- Trial name
- NCT02124772
- Testing
- Trametinib, Dabrafenib
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 139 people
- Sponsor
- Novartis Pharmaceuticals
Achromatopsia
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene.
- Trial name
- NCT02599922
- Testing
- rAAV2tYF-PR1.7-hCNGB3
- Where
- 8 sites in United States
- Volunteers
- 32 people
- Sponsor
- Beacon Therapeutics
Non-Small Cell Lung Cancer
A Phase II Trial of LM103 for Adjuvant Treatment in Patients With Non-small Cell Lung Cancer
After receiving neoadjuvant treatment with PD-1 antibody and undergoing radical resection, a total 36 to 45 NSCLC patients who met the inclusion criteria, will be randomly assigned in a 1:1:1 ratio to the experimental group 1…
- Trial name
- NCT07444437
- Testing
- LM103 TILs Injection, PD-1 / PD-L1 monoclonal antibody
- Where
- 5 sites in China
- Volunteers
- 45 people
- Sponsor
- Suzhou BlueHorse Therapeutics Co., Ltd.
KRAS G12C Mutation, KRAS G12S Mutation
A Study to Investigate the Safety and Efficacy of KQB365 as Monotherapy and in Combination in Participants With Advanced Solid Malignancies
The goal of this clinical trial is to learn if KQB365 works to treat advanced solid tumor cancer in adults. It will also learn about the safety of KQB365.
- Conditions
- KRAS G12C Mutation, KRAS G12S Mutation, Solid Tumor Malignancies, CRC (Colorectal Cancer)
- Trial name
- NCT06720987
- Testing
- KQB365, Cetuximab, KQB198
- Where
- 11 sites in United States
- Volunteers
- 140 people
- Sponsor
- Kumquat Biosciences Inc.
Ovarian Cancer
China Ovarian Cancer BRCA Testing Study
This is a prospective, multi-center, observational study, the primary objective is to evaluate the prevalence of gBRCA/sBRCAm in newly diagnosed ovarian cancer patients and explore ovarian cancer treatment outcomes of different…
- Trial name
- CRONUS (NCT03203993)
- Where
- 19 sites in China, Hong Kong
- Volunteers
- 29 people
- Sponsor
- AstraZeneca
Hematologic Neoplasms
Phase 1/2 Study of Enasidenib (AG-221) in Adults With Advanced Hematologic Malignancies With an Isocitrate Dehydrogenase Isoform 2 (IDH2) Mutation
The primary objectives of Phase 1 Dose Escalation/Part 1 Expansion are: To assess the safety and tolerability of treatment with enasidenib administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle…
- Trial name
- NCT01915498
- Testing
- Enasidenib
- Where
- 24 sites in United States, France
- Volunteers
- 345 people
- Sponsor
- Celgene
Lung Cancer, Non-squamous, Non-small Cell
Beamion 44: A Study to Test How Well Zongertinib is Tolerated by People With Advanced Non-small Cell Lung Cancer With HER2 Mutations When Given in Combination With Chemotherapy With or Without Pembrolizumab
This study is open to adults with a type of lung cancer called HER2-mutant non-squamous non-small cell lung cancer (NSCLC) that is advanced or has spread.
- Trial name
- NCT07486817
- Testing
- Zongertinib, Cisplatin, Carboplatin
- Where
- 17 sites in Australia, China and 5 more
- Volunteers
- 60 people
- Sponsor
- Boehringer Ingelheim