Clinical trials: Gene Mutations
Oncology, MET Gene Amplification
Sym015 (Anti-MET) in Patients With Advanced Solid Tumor Malignancies
This is the first study to test Sym015 in humans. The primary purpose of this study is to see if Sym015 is safe and effective for patients with advanced solid tumor malignancies without available therapeutic options.
- Conditions
- Oncology, MET Gene Amplification, NSCLC, MET Gene Mutation
- Trial name
- NCT02648724
- Testing
- Sym015
- Where
- 27 sites in United States, Denmark and 4 more
- Volunteers
- 57 people
- Sponsor
- Symphogen A/S
Advanced Solid Tumors With MAPK Pathway Mutations
A Phase 1/2 Study of D3S-002 as Monotherapy or Combination Therapy in Adult Subjects With Advanced Solid Tumors With MAPK Pathway Mutations
This first-in-human (FIH) study aims to assess the safety, tolerability, pharmacokinetics, and recommended phase 2 dose (RP2D) of D3S-002 given orally daily for 21-day cycles in adult subjects with advanced solid tumors with…
- Trial name
- NCT05886920
- Testing
- D3S-002, D3S-001
- Where
- 18 sites in United States, Australia and 2 more
- Volunteers
- 67 people
- Sponsor
- D3 Bio (Wuxi) Co., Ltd
Carcinoma, Non-Small-Cell Lung
Real World Data on Gi(l)Otrif® Dose Adjustment
This is a non-interventional, multi-country, multi-site study based on existing data from medical records of patients treated with Gi(l)otrif® as part of the routine treatment according to the approved label.
- Trial name
- NCT02751879
- Where
- 29 sites in United States, Austria and 11 more
- Volunteers
- 228 people
- Sponsor
- Boehringer Ingelheim
Frontotemporal Dementia, FTD
A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)
The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN).
- Conditions
- Frontotemporal Dementia, FTD, FTD-GRN, Dementia, Frontotemporal
- Trial name
- ASPIRE-FTD (NCT06064890)
- Testing
- Intrathalamic AAV.PGRN administration, Intrathalamic AVB-101
- Where
- 19 sites in United States, Belgium and 7 more
- Volunteers
- 18 people
- Sponsor
- AviadoBio Ltd
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
- Trial name
- NCT06275620
- Testing
- AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen)
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Beacon Therapeutics
Chronic Myeloid Leukemia, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Study to Evaluate Tolerability, Safety, Pharmacokinetics and Preliminary Efficacy of PF-114 for Oral Administration in Adults With Ph+ Chronic Myeloid Leukemia, Which is Resistant to the 2-nd Generation Bcr-Abl Inhibitors or Has T315I Mutation in the BCR-ABL Gene
A multicenter, open label cohort Phase 1 dose finding study to evaluate tolerability, safety, pharmacokinetics and preliminary efficacy of PF-114 for oral administration in adult patients with Philadelphia chromosome positive…
- Trial name
- NCT02885766
- Testing
- PF-114
- Where
- 3 sites in Russia
- Volunteers
- 65 people
- Sponsor
- Fusion Pharma LLC
Cholangiocarcinoma, Chondrosarcoma
Study of LY3410738 Administered to Patients With Advanced Solid Tumors With IDH1 or IDH2 Mutations
This is an open-label, multicenter Phase 1 study to evaluate safety, tolerability and preliminary efficacy of oral LY3410738 in patients with isocitrate dehydrogenase 1 (IDH1) arginine 132 (R132)-mutant advanced solid tumors…
- Conditions
- Cholangiocarcinoma, Chondrosarcoma, Glioma, Any Solid Tumor
- Trial name
- NCT04521686
- Testing
- LY3410738, Gemcitabine, Cisplatin
- Where
- 33 sites in United States, Australia and 7 more
- Volunteers
- 200 people
- Sponsor
- Eli Lilly and Company
Non-small Cell Lung Cancer
DZD9008 In Combination With Bevacizumab in Locally Advanced or Metastatic NSCLC Patients With EGFR Mutation (WU-KONG29)
This study tests a combination therapy (i.e., DZD9008 plus bevacizumab) in patients with advanced NSCLC harboring EGFR mutations who have progressed on or after standard of care, which aims to understand whether the combination…
- Trial name
- NCT06276283
- Testing
- DZD9008 plus Bevacizumab
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Dizal Pharmaceuticals
Tumor, Solid, Gastrointestinal Cancer
Trial of Ulixertinib in Combination With Hydroxychloroquine in Patients With Advanced Gastrointestinal (GI) Malignancies
This is an open-label, prospective phase two basket trial assessing the efficacy of ulixertinib in combination with hydroxychloroquine in patients with advanced gastrointestinal malignancies.
- Trial name
- NCT05221320
- Testing
- Ulixertinib, Hydroxychloroquine
- Where
- 9 sites in United States
- Volunteers
- 47 people
- Sponsor
- BioMed Valley Discoveries, Inc
Carcinoma, Non-Small-Cell Lung
GIOTRIF in First Line Therapy of Advanced NSCLC With EGFR-mutations
This observational study will investigate the efficacy, safety, tolerability and symptom control of GIOTRIF (Afatinib) in daily routine first-line therapy in patients with locally advanced or metastatic NSCLC harboring…
- Trial name
- NCT02047903
- Testing
- Afatinib
- Where
- Germany
- Volunteers
- 161 people
- Sponsor
- Boehringer Ingelheim
RET-fusion Non Small Cell Lung Cancer, Lung Neoplasm
A Study of Pralsetinib Versus Standard of Care for First-Line Treatment of Advanced Non-Small Cell Lung Cancer (NSCLC)
This is an international, randomized, open-label, Phase 3 study designed to evaluate whether the potent and selective RET inhibitor, pralsetinib, improves outcomes when compared to a platinum chemotherapy-based regimen chosen by…
- Conditions
- RET-fusion Non Small Cell Lung Cancer, Lung Neoplasm, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms
- Trial name
- AcceleRET-Lung (NCT04222972)
- Testing
- Pralsetinib, Carboplatin, Cisplatin
- Where
- 74 sites in Argentina, Australia and 20 more
- Volunteers
- 223 people
- Sponsor
- Hoffmann-La Roche
Advanced Solid Tumors Cancer, PDAC
A Study of GFH375 Combined With Cetuximab or Chemotherapy in Participants With Solid Tumors Harboring KRAS G12D Mutation
This is a Phase Ib/II clinical study aimed at exploring the safety and efficacy of Regimen A (GFH375 in combination with Cetuximab) and Regimen B (GFH375 in combination with AG) in participants with solid tumors.Phase Ib: To…
- Conditions
- Advanced Solid Tumors Cancer, PDAC, CRC (Colorectal Cancer)
- Trial name
- NCT07259590
- Testing
- GFH375
- Where
- 4 sites in China
- Volunteers
- 126 people
- Sponsor
- Genfleet Therapeutics (Shanghai) Inc.
Gastrointestinal Neoplasms
Individual Patient Expanded Access for Velzatinib (GSK6042981) in Patients With Gastrointestinal Stromal Tumours With KIT Mutations
Compassionate Use, Individual Patient access to velzatinib (GSK6042981) for eligible patients with Gastrointestinal Stromal Tumours (GIST) who have KIT mutations and at least three prior lines of therapy
- Trial name
- NCT07825701
- Testing
- Velzatinib (GSK6042981)
- Sponsor
- GlaxoSmithKline
Stargardt Disease Type 1 (STGD1)
An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease
Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence.
- Trial name
- START (NCT07063251)
- Testing
- HG005
- Where
- China
- Volunteers
- 6 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Fabry Disease
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
- Trial name
- NCT00925301
- Testing
- migalastat hydrochloride
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 67 people
- Sponsor
- Amicus Therapeutics
Advanced Solid Tumors
MEK162 and RAF265 in Adult Patients With Advanced Solid Tumors Harboring RAS or BRAFV600E Mutations
This is a multi- center, open-label, dose finding, Phase Ib study to be conducted in two stages: a dose escalation part to determine the maximum tolerated dose (MTD) safety and tolerability of concurrent administration of MEK162…
- Trial name
- NCT01352273
- Testing
- MEK162 + RAF265
- Where
- 8 sites in United States, Canada and 3 more
- Volunteers
- 69 people
- Sponsor
- Array Biopharma, now a wholly owned subsidiary of Pfizer
Non-Small Cell Lung Cancer, Advanced Solid Tumours
Phase I Dose-Escalation Study of AZD4785 in Patients With Advanced Solid Tumours
A Phase I, open-label, multicentre, dose-escalation study to investigate the safety, pharmacokinetics and maximum tolerated dose (MTD) of AZD4785 in patients with advanced solid tumours where KRAS may be an important driver of…
- Trial name
- NCT03101839
- Testing
- AZD4785
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 28 people
- Sponsor
- AstraZeneca
Epidermal Growth Factor, Epidermal Growth Factor Receptor
A Phase 1 Study of EPI-326 in EGFR-mutant NSCLC and HNSCC
A phase 1 study to determine the safety, tolerability, PK, PD, and preliminary anti-tumor activity of ascending doses of EPI-326 administered to patients with locally advanced or metastatic HNSCC and to patients with any…
- Conditions
- Epidermal Growth Factor, Epidermal Growth Factor Receptor, Epidermal Growth Factor Receptor Gene Mutation, Non Small Cell
- Trial name
- NCT07462377
- Testing
- EPI-326
- Where
- 8 sites in United States, South Korea and 1 more
- Volunteers
- 110 people
- Sponsor
- EpiBiologics
Low-grade NMIBC, FGFR Gene Amplification
Efficacy and Safety of TYRA-300 in Participants With FGFR3 Altered Low Grade, Intermediate Risk Non-Muscle Invasive Bladder Cancer
Phase 2 Study of TYRA-300 in FGFR3 Altered Low Grade, Intermediate Risk NMIBC
- Conditions
- Low-grade NMIBC, FGFR Gene Amplification, FGFR Gene Alterations, FGFR3 Gene Alteration
- Trial name
- SURF302 (NCT06995677)
- Testing
- TYRA-300 60mg, TYRA-300 50mg, TYRA-300 Dose 70 mg
- Where
- 48 sites in United States, Australia and 2 more
- Volunteers
- 90 people
- Sponsor
- Tyra Biosciences, Inc
Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
The objective of this early access program (EAP) is to provide access to tofersen to eligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene prior to an…
- Trial name
- NCT04972487
- Testing
- Tofersen
- Where
- 19 sites in United States
- Sponsor
- Biogen