Clinical trials: Genetic Mutation
Leber Congenital Amaurosis 10, Blindness
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- ILLUMINATE (NCT03913143)
- Testing
- sepofarsen
- Where
- 14 sites in United States, Belgium and 7 more
- Volunteers
- 36 people
- Sponsor
- ProQR Therapeutics
Non-small Cell Lung Cancer
Ningetinib (CT053PTSA) Plus Gefitinib in Stage IIIB or IV NSCLC Patients With EGFR Mutation and T790M Negative
This is a phase Ib, multi-center, open label study evaluating the safety and efficacy of CT053PTSA in combination with gefitinib in patients with EGFR mutation, T790M negative NSCLC who have progressed after EGFR TKI treatment.
- Trial name
- NCT03758287
- Testing
- CT053PTSA, Gefitinib
- Where
- China
- Volunteers
- 108 people
- Sponsor
- Sunshine Lake Pharma Co., Ltd.
Lung Cancer
Osimertinib Induction and Maintenance for Chemo-ineligible Stage III Unresectable EGFR+ NSCLC: Single-arm Study
The purpose of this study is to measure efficacy and safety of osimertinib as induction therapy prior to curative intent RT and maintenance osimertinib in chemotherapy ineligible or refusal adult patients with Stage III…
- Trial name
- NSCLC (NCT07295821)
- Testing
- Osimertinib
- Where
- 20 sites in China
- Volunteers
- 60 people
- Sponsor
- AstraZeneca
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003
This is a Phase 1, First-in-Human study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and immunogenicity of PYC-003 in healthy adult participants and adult participants with confirmed PKD1…
- Trial name
- NCT06714006
- Testing
- PYC-003
- Where
- 12 sites in Australia, Hong Kong and 1 more
- Volunteers
- 166 people, healthy volunteers welcome
- Sponsor
- PYC Therapeutics
Stargardt, Stargardt's Disease
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
This is an Observational Study to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene This is a multicenter study which will enroll approximately 75…
- Conditions
- Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
- Trial name
- POLARIS (NCT06435000)
- Where
- 20 sites in United States, Germany and 1 more
- Volunteers
- 75 people
- Sponsor
- Splice Bio
Solid Tumors, KRAS Mutation
A Study to Test Different Doses of BI 3011441 in Japanese People With Different Types of Advanced Cancer (NRAS/KRAS Mutation Positive)
This study is open to Japanese adults with different types of advanced cancer that are positive for NRAS/KRAS mutations. This is a study in people for whom previous treatment was not successful or no standard treatment exists.
- Trial name
- NCT04742556
- Testing
- BI 3011441
- Where
- 3 sites in Japan
- Volunteers
- 15 people
- Sponsor
- Boehringer Ingelheim
KRAS G12D-Mutant Advanced or Metastatic Pancreatic Cancer in the First-line Setting
HRS 4642 Injection Combined With AG Versus Placebo Combined With AG Therapy in First-Line Advanced or Metastatic Pancreatic Cancer
This study investigates the first-line treatment of advanced pancreatic cancer with KRAS G12D mutation. Patients were randomized 1:1 to receive either the experimental regimen HRS-4642 plus AG or the control regimen AG alone.
- Trial name
- NCT07232875
- Testing
- HRS-4642 + AG
- Where
- China
- Volunteers
- 588 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Melanoma
Ph I Ipilimumab Vemurafenib Combo in Patients With v-Raf Murine Sarcoma Viral Oncogene Homolog B1 (BRAF)
Treatment of subjects who have metastatic melanoma that expresses an activated mutant form of the BRAF oncogene (V600E) with a combination of the specific BRAF inhibitor, Vemurafenib, and the Cytotoxic T Lymphocyte Antigen 4…
- Trial name
- NCT01400451
- Testing
- Ipilimumab (BMS-734016), Vemurafenib
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Bristol-Myers Squibb
Bladder Cancer, Urothelial Carcinoma
Biomarker Research Study for Patients With FGFR-Mutant Bladder Cancer Receiving Erdafitinib
Bladder cancers are associated with genetic mutations that are present in the patient's bladder or urothelium, the lining of the lower urinary tract.
- Conditions
- Bladder Cancer, Urothelial Carcinoma, Transitional Cell Carcinoma, Platinum-Resistant Urothelial Carcinoma
- Trial name
- NCT05052372
- Testing
- Balversa
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- xCures
Non-small Cell Lung Cancer
Icotinib With Concurrent Radiotherapy Versus Chemotherapy With Concurrent Radiotherapy in Non-small Cell Lung Cancer
This randomised, multi-center, controlled trial is designed to assess the efficacy and safety of icotinib with concurrent radiotherapy versus chemotherapy with concurrent radiotherapy in non-small cell lung cancer
- Trial name
- NCT02215356
- Testing
- Icotinib with concurrent radiotherapy, Chemoradiotherapy
- Where
- 26 sites in China
- Volunteers
- 120 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Leber Hereditary Optic Neuropathy
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.
- Trial name
- NCT02064569
- Testing
- GS010
- Where
- France
- Volunteers
- 19 people
- Sponsor
- GenSight Biologics
Melanoma and Brain Metastases
A Study of GSK2118436 in BRAF Mutant Metastatic Melanoma to the Brain
This study is designed to assess the efficacy, pharmacokinetics, safety, and tolerability of an oral, twice daily dose of 150 mg GSK2118436 administered to subjects with BRAF V600E or V600K mutation-positive metastatic melanoma…
- Trial name
- Break MB (NCT01266967)
- Testing
- GSK2118436
- Where
- 24 sites in United States, Australia and 4 more
- Volunteers
- 172 people
- Sponsor
- GlaxoSmithKline
Non-small Cell Lung Cancer
To Evaluate the Efficacy of QLH11811 in Advanced NSCLC Patients With EGFR Mutation
This is a phase 1, open-label, dose escalation and cohort expansion study and conducted in China and the United States to investigate the safety, tolerability and preliminary efficacy of QLH11811 in advanced or metastatic NSCLC…
- Trial name
- NCT05555212
- Testing
- QLH11811
- Where
- China
- Volunteers
- 72 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Advanced Cancer, Breast Cancer
First-in-Human Study of OKI-219 in Advanced Solid Tumors and Advanced Breast Cancer
OKI-219-101 is a Phase 1a/1b, open-label, multicenter, dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of OKI-219 as monotherapy and in combination…
- Conditions
- Advanced Cancer, Breast Cancer, Advanced Solid Tumors, PI3K Gene Mutation
- Trial name
- PIKture-01 (NCT06239467)
- Testing
- OKI-219, Fulvestrant, Trastuzumab
- Where
- 33 sites in United States, Belgium and 4 more
- Volunteers
- 200 people
- Sponsor
- OnKure, Inc.
Retinitis Pigmentosa 11, Retinal Degeneration
A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001
This is a Phase 1/2 repeat-dose, open-label, two-arm, parallel group safety and efficacy study of two doses of VP-001 (30 μg and 75 μg) in participants with confirmed PRPF31 mutation-associated retinal dystrophy, including…
- Conditions
- Retinitis Pigmentosa 11, Retinal Degeneration, Retinal Disease, Eye Diseases Hereditary
- Trial name
- DINGO (NCT06852963)
- Testing
- VP-001
- Where
- 6 sites in United States
- Volunteers
- 17 people
- Sponsor
- PYC Therapeutics
HIV Infections
Study of Once Daily Elvucitabine Versus Lamivudine in Participants With a Documented M184V Mutation
Human immunodeficiency virus (HIV)-1 infected participants receiving long-term therapy with lamivudine or emtricitabine (nucleoside reverse transcriptase inhibitors [NRTIs]) are at risk for the development of a mutation at…
- Trial name
- Resistance (NCT00312039)
- Testing
- Elvucitabine, Lamivudine
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Non-small Cell Lung Cancer
H002 in Patients With EGFR Mutation Locally Advanced or Metastatic NSCLC
This is a phase I/IIa, open-label, dose-escalation and expansion study to evaluate the safety, tolerability, PK and preliminary anti-tumor activity of H002 when given orally in patients with EGFR mutation-positive locally…
- Trial name
- NCT05552781
- Testing
- H002 capsule
- Where
- 8 sites in China
- Volunteers
- 76 people
- Sponsor
- RedCloud Bio
Non-Small Cell Lung Cancer
A Study of Atezolizumab as Neoadjuvant and Adjuvant Therapy in Resectable Non-Small Cell Lung Cancer (NSCLC) - Lung Cancer Mutation Consortium (LCMC3)
This study was designed to evaluate the safety and efficacy of neoadjuvant and adjuvant atezolizumab in participants with resectable Non-Small Cell Lung Cancer (NSCLC).
- Trial name
- NCT02927301
- Testing
- Atezolizumab (MPDL3280A), an engineered anti-PD-L1 antibody
- Where
- 19 sites in United States
- Volunteers
- 181 people
- Sponsor
- Genentech, Inc.
Metastatic Breast Cancer
Minimally Interventional Study on Prevalence of Emerging ESR1 Mutations in Liquid Biopsy in Three Cohorts of Patients With Breast Cancer in Comparison With Patient's Baseline ESR1 Mutation Status as Defined by Tissue Profiling.
Minimally interventional study on prevalence of emerging ESR1 mutations in liquid biopsy in three cohorts of patients with breast cancer (with and without prior therapies in metastatic setting, and during first-line aromatase…
- Trial name
- Pangeia-2 (NCT06417801)
- Where
- 6 sites in Brazil
- Volunteers
- 170 people
- Sponsor
- AstraZeneca
Breast Neoplasms, Neoplasm Metastasis
A Study of Tersolisib (LY4064809/STX-478) With Other Anti-Cancer Treatments in Participants With Advanced Breast Cancer With a Genetic Change (PIK3CA)
The purpose of the study is to assess the efficacy and safety of the addition of Tersolisib (LY4064809/STX-478) to other anti-cancer drugs as first treatment for advanced hormone receptor-positive (HR+)/human epidermal growth…
- Trial name
- NCT07174336
- Testing
- LY4064809, Ribociclib, Palbociclib
- Where
- 364 sites in United States, Argentina and 17 more
- Volunteers
- 800 people
- Sponsor
- Eli Lilly and Company
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