Clinical trials: Gene Mutations
Familial Erythromelalgia
A Phase 1b Study of BHV-7000 in Participants With Inherited Erythromelalgia
The purpose of this study is to test the potential benefits of BHV-7000 in reducing chronic pain in participants with IEM with a previously demonstrated gain of function mutation in the SCN9A gene.
- Trial name
- NCT07262268
- Testing
- BHV-7000
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Biohaven Therapeutics Ltd.
Endometrial Cancer
Sapanisertib and Serabelisib (PIKTOR) With Paclitaxel and a Substudy With an Insulin-Suppressing Diet in Patients With Advanced/Recurrent Endometrial Cancer
This is a Phase 2, multicenter, open-label, single-arm study to evaluate the efficacy and safety of sapanisertib and serabelisib (PIKTOR) with paclitaxel in participants with advanced or recurrent endometrial cancer.
- Trial name
- NCT06463028
- Testing
- Sapanisertib, Serabelisib, Paclitaxel
- Where
- 18 sites in United States
- Volunteers
- 40 people
- Sponsor
- Faeth Therapeutics
Solid Tumors, EGFR Exon 20 Insertion Mutations
ORIC-114 in Combination With Subcutaneous Amivantamab in Patients With EGFR Exon20 Insertion Mutant NSCLC
The purpose of this study is to establish the recommended phase 2 dose (RP2D), safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antitumor activity of ORIC-114 in combination with subcutaneous (SC) amivantamab…
- Conditions
- Solid Tumors, EGFR Exon 20 Insertion Mutations, NSCLC, EGFR-mutated NSCLC
- Trial name
- NCT06816992
- Testing
- ORIC-114 Dose 1 + amivantamab, ORIC-114 Dose 2 + amivantamab, ORIC-114 Dose 3 + amivantamab
- Where
- 4 sites in United States, Australia and 1 more
- Volunteers
- 76 people
- Sponsor
- ORIC Pharmaceuticals
Newly Diagnosed AML With FLT3 Activating Mutations
A Safety and Tolerability Study of Crenolanib in Combination With Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia Patients With FLT3 Mutations
This pilot study is designed to evaluate the safety and tolerability of oral crenolanib besylate given sequentially during standard induction and consolidation chemotherapy in patients with newly diagnosed AML with FLT3…
- Trial name
- NCT02283177
- Testing
- crenolanib, cytarabine, daunorubicin
- Where
- 9 sites in United States
- Volunteers
- 44 people
- Sponsor
- Arog Pharmaceuticals, Inc.
Non-Small Cell Lung Cancer, Advanced Non-Small Cell Squamous Lung Cancer
Phase 1/2 Study of Silevertinib (BDTX-1535) in Patients With Glioblastoma or Non-Small Cell Lung Cancer With EGFR Mutations
BDTX-1535-101 is an open-label, Phase 1 dose escalation and Phase 2 multiple cohort study designed to evaluate the safety, pharmacokinetics (PK), optimal dosage, central nervous system (CNS) activity, and antitumor activity of…
- Conditions
- Non-Small Cell Lung Cancer, Advanced Non-Small Cell Squamous Lung Cancer, Metastatic Lung Non-Small Cell Carcinoma, Metastatic Lung Cancer
- Trial name
- NCT05256290
- Testing
- silevertinib (BDTX-1535) monotherapy
- Where
- 25 sites in United States
- Volunteers
- 200 people
- Sponsor
- Black Diamond Therapeutics, Inc.
Solid Tumors, Triple-Negative Breast Cancer
Study of the Glutaminase Inhibitor CB-839 in Solid Tumors
Many tumor cells, in contrast to normal cells, have been shown to require the amino acid glutamine to produce energy for growth and survival.
- Conditions
- Solid Tumors, Triple-Negative Breast Cancer, Non Small Cell Lung Cancer, Renal Cell Carcinoma
- Trial name
- NCT02071862
- Testing
- CB-839, Pac-CB, CBE
- Where
- 13 sites in United States
- Volunteers
- 210 people
- Sponsor
- Calithera Biosciences, Inc
Advanced Solid Tumor, Non Small Cell Lung Cancer
Phase 1b/2 Study to Evaluate ABT-101 in Solid Tumor and NSCLC Patients
A Phase 1b/2, open-label, multicenter study to determine the recommended phase 2 (RP2D) of ABT-101in solid tumor and to explore antitumor activities of ABT-101 in patients with HER 2 mutated non-small cell lung cancer (NSCLC)
- Conditions
- Advanced Solid Tumor, Non Small Cell Lung Cancer, HER2 Mutations
- Trial name
- NCT05532696
- Testing
- ABT-101
- Where
- 5 sites in Taiwan
- Volunteers
- 61 people
- Sponsor
- Anbogen Therapeutics, Inc.
Tumor, Tumor, Solid
Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)
A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes
- Conditions
- Tumor, Tumor, Solid, Metastasis, Metastatic Cancer
- Trial name
- NCT05103358
- Testing
- nab-sirolimus
- Where
- 166 sites in United States, Puerto Rico and 1 more
- Volunteers
- 128 people
- Sponsor
- Aadi Bioscience, Inc.
Advanced Solid Tumor
Study of RP-3500, Camonsertib, in Advanced Solid Tumors
The primary purpose of this study is to define the maximum tolerated dose (MTD) and determine a recommended Phase 2 dose (RP2D) and schedule of orally-administered RP-3500 (camonsertib) alone or in combination with talazoparib, a…
- Trial name
- TRESR (NCT04497116)
- Testing
- RP-3500 (camonsertib), Talazoparib, Gemcitabine Injection
- Where
- 13 sites in United States, Canada and 2 more
- Volunteers
- 276 people
- Sponsor
- Repare Therapeutics
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
Neoplasms, Breast
Efficacy and Safety Comparison of Niraparib to Placebo in Participants With Human Epidermal Growth Factor 2 Negative (HER2-) Breast Cancer Susceptibility Gene Mutation (BRCAmut) or Triple-Negative Breast Cancer (TNBC) With Molecular Disease
This study will assess the efficacy and safety of Niraparib in participants with either tumor mutation in the BRCA gene (tBRCAmut) HER2- breast cancer (Independent of hormone receptor [HR] status, including HR positive [+] and…
- Trial name
- ZEST (NCT04915755)
- Testing
- Niraparib
- Where
- 198 sites in United States, Argentina and 25 more
- Volunteers
- 40 people
- Sponsor
- GlaxoSmithKline
Carcinoma, Non-Small Cell Lung
Quaratusugene Ozeplasmid (Reqorsa) and Osimertinib in Patients With Advanced Lung Cancer Who Progressed on Osimertinib
The purpose of this randomized study is to determine the safety and efficacy of quaratusugene ozeplasmid (Reqorsa) added to osimertinib in NSCLC patients with activating EGFR mutations who have progressed while on treatment with…
- Trial name
- Acclaim-1 (NCT04486833)
- Testing
- quaratusugene ozeplasmid, osimertinib, Platinum-Based Chemotherapy
- Where
- 10 sites in United States
- Volunteers
- 158 people
- Sponsor
- Genprex, Inc.
Advanced Solid Tumor, BRAF V600 Mutation
Safety and Tolerability of ABM-1310 in Patients With Advanced Solid Tumors
This is a Phase I, First-In-Human, open label, dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-cancer activity of ABM-1310 in adult patients with locally…
- Trial name
- NCT04190628
- Testing
- ABM-1310, Cobimetinib
- Where
- 8 sites in United States
- Volunteers
- 53 people
- Sponsor
- ABM Therapeutics Corporation
Carcinoma, Non-Small-Cell Lung
Detection of Either the EML4-ALK Gene Rearrangements or the T790M EGFR Mutation in the Plasma of Advanced NSCLC Patients
Demonstrate feasibility of detection of EML4-ALK fusion transcripts and T790M EGFR mutation from exosomes in the circulation of Non-Small Cell Lung Cancer (NSCLS) patients.
- Trial name
- NCT03236675
- Where
- 2 sites in United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Exosome Diagnostics, Inc.
Non-Small Cell Lung Cancer
A Study of SKB264 in Combination With Osimertinib Versus Osimertinib in Patients With Epidermal Growth Factor Receptor (EGFR) Mutations, Locally Advanced or Metastatic Non-Squamous Non-Small Cell Lung Cancer
The aim of the study is to evaluate the efficacy and safety of SKB264 in combination with osimertinib as first-Line treatment for patients with epidermal growth factor receptor (EGFR) mutations, locally advanced or metastatic…
- Trial name
- NCT06670196
- Testing
- SKB264, Osimertinib
- Where
- China
- Volunteers
- 420 people
- Sponsor
- Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd.
Melanoma, Solid Tumor
Tovorafenib (DAY101) or in Combination With Pimasertib for Participants With Melanoma and Other Solid Tumors
This is a subprotocol of Master Protocol DAY101-102 and is a Phase 1b/2, multi-center, open label subprotocol of participants ≥12 years of age, with recurrent or progressive solid tumors with alterations in the key proteins of…
- Conditions
- Melanoma, Solid Tumor, Pilocytic Astrocytoma, Non Small Cell Lung Cancer
- Trial name
- NCT07121829
- Testing
- Tovorafenib, Tovorafenib Drug: Pimasertib
- Where
- 10 sites in United States, Canada
- Volunteers
- 44 people
- Sponsor
- Day One Biopharmaceuticals, Inc.
Carcinoma, Pancreatic Ductal, Colorectal Neoplasms
A Study to Learn About the Study Medicine PF-07934040 When Given Alone or With Other Anti-cancer Therapies in People With Advanced Solid Tumors That Have a Genetic Mutation.
The purpose of this study is to learn about the safety and effects of the study medicine alone or when given together with other anti-cancer therapies. This study also aims to find the best dose.
- Conditions
- Carcinoma, Pancreatic Ductal, Colorectal Neoplasms, Carcinoma, Non-Small-Cell Lung
- Trial name
- NCT06447662
- Testing
- PF-07934040, Gemcitabine, Nab-paclitaxel
- Where
- 27 sites in United States, Puerto Rico
- Volunteers
- 64 people
- Sponsor
- Pfizer
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation
Phase 1 dose escalation will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of revumenib in participants with acute leukemia.
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Mixed Lineage Acute Leukemia, Mixed Phenotype Acute Leukemia
- Trial name
- AUGMENT-101 (NCT04065399)
- Testing
- revumenib, cobicistat
- Where
- 57 sites in United States, Australia and 9 more
- Volunteers
- 447 people
- Sponsor
- Syndax Pharmaceuticals
Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
- Trial name
- NCT05906953
- Testing
- HG004
- Where
- 3 sites in United States, China
- Volunteers
- 20 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Solid Tumors With Documented BRCA, BARD, or PALB or Other Acceptable DNA Mutations or Anomalies That Are Scientifically Sound, Triple Negative Breast Cancer (TNBC)
Expanded Access to Veliparib
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to veliparib prior to approval by the local regulatory agency. Availability will depend on territory eligibility.
- Conditions
- Solid Tumors With Documented BRCA, BARD, or PALB or Other Acceptable DNA Mutations or Anomalies That Are Scientifically Sound, Triple Negative Breast Cancer (TNBC), High Grade Serous Ovarian Cancer, Patients Requiring Veliparib Suspension Formulation
- Trial name
- NCT03123211
- Testing
- Veliparib
- Sponsor
- AbbVie