Clinical trials: Gene Mutations
Advanced Solid Malignancies, Breast Cancer - ER+, HER2 -
Phase I, Dose Study to Look at the Safety and Pharmacokinetics of AZD8835 in Patients With Advanced Solid Tumours
First time in patients study of AZD8835. The study has four parts. Part A AZD8835 is administered as a single agent in a multiple ascending dose escalation phase to investigate dose level for monotherapy.
- Conditions
- Advanced Solid Malignancies, Breast Cancer - ER+, HER2 -, Breast Cancer - ER+, HER2-, PIK3CA Gene Mutation
- Trial name
- NCT02260661
- Testing
- AZD8835, AZD8835 in combination with fulvestrant
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 20 people
- Sponsor
- AstraZeneca
Non-small Cell Lung Cancer (NSCLC)
A Study of ASP8273 vs. Erlotinib or Gefitinib in First-line Treatment of Patients With Stage IIIB/IV Non-small Cell Lung Cancer Tumors With EGFR Activating Mutations
The purpose of the study was to evaluate the progression free survival (PFS), based on independent radiologic review (IRR), of ASP8273 compared to erlotinib or gefitinib in patients with locally advanced, metastatic or…
- Trial name
- SOLAR (NCT02588261)
- Testing
- naquotinib mesilate, Erlotinib, Gefitinib
- Where
- 167 sites in United States, Australia and 21 more
- Volunteers
- 530 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
- Trial name
- NCT05858983
- Testing
- FT-001 Low Dose, FT-001 Mid Dose, FT-001 High Dose
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Frontera Therapeutics
AML, MDS
A Study of E6201 for the Treatment of Advanced Hematologic Malignancies With FLT3 and/or Ras Mutations
This is a Phase 1/2a dose-escalation study of E6201, a dual mitogen-activated protein kinase/extracellular-signal regulated kinase 1 (MEK1) and FMS-like tyrosine kinase 3 (FLT3) inhibitor, in subjects with advanced hematologic…
- Conditions
- AML, MDS, CMML
- Trial name
- NCT02418000
- Testing
- E6201
- Where
- 4 sites in United States
- Volunteers
- 27 people
- Sponsor
- Spirita Oncology, LLC
Non Small Cell Lung Cancer
Prospective Cohort of Locally Advanced and Metastatic Non-Small Cell Lung Cancer Patients With Activating EGFR Mutations
This is an observational multicenter study. A cross-sectional descriptive study of patients with locally advanced or metastatic Non Small Cell Lung Cancer with activating EGFR mutation treated by any first line therapy will be…
- Trial name
- POSITHES (NCT05103605)
- Testing
- Osimertinib
- Where
- 46 sites in France
- Volunteers
- 274 people
- Sponsor
- AstraZeneca
LMNA-Related Dilated Cardiomyopathy
A Rollover Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy
This is a rollover study designed to investigate the safety and effectiveness of investigational study drug ARRY-371797 in patients who previously received ARRY-371797 in a study for patients with LMNA-related dilated…
- Trial name
- NCT02351856
- Testing
- ARRY-371797, p38 inhibitor, oral
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Pfizer
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
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
Breast Neoplasms, BRCA 1 Gene Mutation
A Phase 2, 2-Stage, 2-Cohort Study of Talazoparib (BMN 673), in Locally Advanced and/or Metastatic Breast Cancer Patients With BRCA Mutation (ABRAZO Study)
The purpose of this 2-stage, 2-cohort Phase 2 trial is to evaluate the safety and efficacy of talazoparib (also known as BMN 673) in subjects with locally advanced or metastatic breast cancer with a deleterious germline BRCA 1 or…
- Conditions
- Breast Neoplasms, BRCA 1 Gene Mutation, BRCA 2 Gene Mutation
- Trial name
- ABRAZO (NCT02034916)
- Testing
- talazoparib
- Where
- 71 sites in United States, France and 3 more
- Volunteers
- 84 people
- Sponsor
- Pfizer
NF type1 With Inoperable Plexiform Neurofibromas
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1
This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable…
- Trial name
- NCT03259633
- Testing
- Selumetinib
- Where
- United States
- Sponsor
- AstraZeneca
Non-Squamous Non-Small Cell Lung Cancer
A Study of Tarceva (Erlotinib) in Patients With Locally Advanced or Metastatic Non-Small Cell Lung Cancer (TRIGGER)
This single-arm, open-label study evaluated the efficacy and safety of Tarceva (erlotinib) in participants with locally advanced or metastatic non-small cell lung cancer. Participants received daily oral doses of 150 mg Tarceva.
- Trial name
- NCT01378962
- Testing
- erlotinib
- Where
- 10 sites in Italy
- Volunteers
- 50 people
- Sponsor
- Hoffmann-La Roche
BRCA Mutations
The Use of Digital Genetic Counselor (DGC) for BRCA1/2 Mutation Carrier Testing
The DGC provides an end-to-end solution to the Oncogenetics screening process from participant registration to receipt of the genetic test results and their interpretation.
- Trial name
- NCT04038905
- Volunteers
- 300 people
- Sponsor
- Igentify Ltd
Treatment in HER2-negative Metastatic Breast Cancer Patients With Germline BRCA Mutation
A Study of Fluzoparib±Apatinib Versus Chemotherapy Treatment of Physician's Choice in HER2-negative Metastatic Breast Cancer Patients With Germline BRCA Mutation
This is a multicenter, randomized, open-label, 3-arm Phase 3 study to evaluate the efficacy and safety of Fluzoparib alone or with Apatinib versus Physicians Choice Chemotherapy, as treatment, in patients with a Germline BRCA…
- Trial name
- NCT04296370
- Testing
- Fluzoparib; Apatinib, Fluzoparib, Physician's choice chemotherapy
- Where
- China
- Volunteers
- 474 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Ovarian Cancer, Breast Cancer
Roll Over StudY for Patients Who Have Completed a Previous Oncology Study With Olaparib
The rationale of the ROSY-O study is to continue to provide study treatment for patients who have participated in a parent study with olaparib and who are continuing to derive clinical benefit from treatment at the end of such…
- Trial name
- ROSY-O (NCT04421963)
- Testing
- Olaparib
- Where
- 107 sites in United States, Belgium and 21 more
- Volunteers
- 185 people
- Sponsor
- AstraZeneca
OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)
A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.
- Conditions
- OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
- Trial name
- NCT06461286
- Testing
- PYC-001
- Where
- 2 sites in Australia
- Volunteers
- 18 people
- Sponsor
- PYC Therapeutics
Non Small Cell Lung Cancer, Non-Small Cell Lung Cancer
A Study of TPX-0131, a Novel Oral ALK Tyrosine Kinase Inhibitor, in Patients With ALK+ Advanced or Metastatic NSCLC
A phase 1, first-in-human, open-label study to evaluate the safety, tolerability, PK, and efficacy of the novel ALK inhibitor TPX-0131 in pretreated subjects with ALK+ advanced or metastatic non-small cell lung cancer (NSCLC).
- Conditions
- Non Small Cell Lung Cancer, Non-Small Cell Lung Cancer, NSCLC, Advanced Solid Tumor
- Trial name
- NCT04849273
- Testing
- TPX-0131
- Where
- 15 sites in United States, Australia and 1 more
- Volunteers
- 11 people
- Sponsor
- Turning Point Therapeutics, Inc.
GIST
A Study of Ripretinib vs Sunitinib in Patients With Advanced GIST With Specific KIT Exon Mutations Who Were Previously Treated With Imatinib
This is a Phase 3, 2-arm, randomized, open-label, global, multicenter study comparing the efficacy of ripretinib to sunitinib in participants with GIST who progressed on first-line treatment with imatinib, harbor co-occurring KIT…
- Trial name
- INSIGHT (NCT05734105)
- Testing
- Ripretinib, Sunitinib
- Where
- 70 sites in United States, Australia and 13 more
- Volunteers
- 54 people
- Sponsor
- Deciphera Pharmaceuticals, LLC
Acute Myeloid Leukemia
Efficacy Study for AC220 to Treat Acute Myeloid Leukemia (AML)
AC220 will be administered as a once daily oral solution given continuously as 28-day treatment cycles, without any rest periods, until disease progression, relapse, intolerance to the drug, or elective allogeneic hematopoietic…
- Trial name
- ACE (NCT00989261)
- Testing
- Compound AC220
- Where
- 85 sites in United States, Canada and 7 more
- Volunteers
- 333 people
- Sponsor
- Daiichi Sankyo
Advanced Solid Tumors Harboring RAS Mutations or Amplifications
A Phase I Study of HRS-7172 in Participants With Advanced Solid Tumors Harboring RAS Mutations or Amplifications
This is a phase I study of HRS-7172 to evaluate safety, tolerability and pharmacokinetics in participants with advanced solid tumors harboring RAS mutations or amplifications.
- Trial name
- NCT07142980
- Testing
- HRS-7172 Tablets
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Severe Familial Hypercholesterolemia
Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders
A study to assess the long term safety and tolerability of evolocumab (AMG 145) in adolescents and adults with severe familial hypercholesterolemia.
- Trial name
- TAUSSIG (NCT01624142)
- Testing
- Evolocumab
- Where
- 44 sites in United States, Australia and 16 more
- Volunteers
- 300 people
- Sponsor
- Amgen