Clinical trials: Gene Mutation
Cystic Fibrosis
GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis
This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d.
- Trial name
- PELICAN (NCT03474042)
- Testing
- GLPG2737
- Where
- 9 sites in Germany
- Volunteers
- 22 people
- Sponsor
- Lakefront Biotherapeutics NV
Advanced Non-squamous Non-small-cell Lung Cancer, Metastatic Non-squamous Non Small Cell Lung Cancer
JAB-21822 Combined With JAB-3312 Compared SOC in the First Line for Treatment of Advanced Non-small Cell Lung Cancer With KRAS p.G12C Mutation
This Phase 3 study will evaluate the efficacy of JAB-21822+JAB-3312 versus tislelizumab (PD-1 Ab) combined with pemetrexed+carboplatin as the first line treatment in subjects with KRAS G12C mutated locally advanced or metastatic…
- Trial name
- NCT06416410
- Testing
- JAB-21822, Tislelizumab, JAB-3312
- Where
- 37 sites in China
- Volunteers
- 392 people
- Sponsor
- Allist Pharmaceuticals, Inc.
Carcinoma, Non-Small-Cell Lung
Survey on the Treatment Reality of Patients With Epidermal Growth Factor Receptor (EGFR) Mutation-positive Non-small Cell Lung Cancer(NSCLC)
According to the recent report using EGFR tyrosine kinase inhibitors (EGFR-TKI), there is all over survival of the EGFR gene mutation-positive NSCLC in a tendency to extension.
- Trial name
- NCT02475720
- Where
- Japan
- Volunteers
- 2,000 people
- Sponsor
- AstraZeneca
Non-small Cell Lung Cancer
Icotinib as Adjuvant Therapy Compared With Standard Chemotherapy in Stage II-IIIA NSCLC With EGFR-mutation
Adjuvant therapy has been proved effective in treating earlier stage or less advanced non-small-cell lung cancer. This study is designed to evaluate the efficacy of icotinib as adjuvant therapy in treating stage II-IIIA non-small…
- Trial name
- EVIDENCE (NCT02448797)
- Testing
- Icotinib, Chemotherapy
- Where
- 8 sites in China
- Volunteers
- 320 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Low-grade Glioma
Expanded Access Program (EAP) for Tovorafenib (DAY101) in RAF-Altered, Relapsed or Refractory Low-Grade Glioma
The DAY101-EAP is a multicenter, open-label, expanded access treatment protocol designed to provide access to tovorafenib (DAY101) for eligible patients.
- Trial name
- NCT05760586
- Testing
- Tovorafenib
- Sponsor
- Day One Biopharmaceuticals, Inc.
Clonal Mast Cell Disease, KIT D816V Mutation
Screening Study for KIT D816V Mutated Mast Cell Disease in Select Populations
This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.
- Conditions
- Clonal Mast Cell Disease, KIT D816V Mutation, Suspected KITD816V Mutated Clonal Mast Cell Disease
- Trial name
- POLARIS (NCT07143669)
- Testing
- Screening
- Where
- 26 sites in United States, Belgium
- Volunteers
- 750 people
- Sponsor
- Blueprint Medicines Corporation
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
Acalabrutinib in Patients With Relapsed/Refractory and Treatment naïve Deletion 17p CLL/SLL
This study is to determine the response to acalabrutinib in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
- Trial name
- NCT02337829
- Testing
- Acalabrutinib (Arm A), Acalabrutinib (Arm B)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Acerta Pharma BV
Advanced or Metastatic Solid Tumors, Breast Cancer
A Study of PARG Inhibitor IDE161 in Participants With Advanced Solid Tumors
The purpose of this study is to characterize the safety, tolerability, and efficacy of IDE161 as a single agent and in combination with pembrolizumab.
- Conditions
- Advanced or Metastatic Solid Tumors, Breast Cancer, Ovarian Cancer, Prostate Cancer
- Trial name
- NCT05787587
- Testing
- IDE-161, Pembrolizumab
- Where
- 27 sites in United States
- Volunteers
- 216 people
- Sponsor
- IDEAYA Biosciences
Schizophrenia
A Placebo-Controlled Study of Physiologic Effects of L-methylfolate in Schizophrenia Patients
This study is a 12-week, double-blind, placebo-controlled trial of L-methylfolate 15 mg/d supplementation in schizophrenia patients with mild or greater negative symptoms.
- Trial name
- NCT01091506
- Testing
- L-methylfolate
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- Pamlab, Inc.
Non-Small Cell Lung Cancer, Brain Metastases
Icotinib Combined With Radiation Therapy For NSCLC Patients With Brain Metastases and EGFR Mutation
The purpose of this study is to evaluate the efficacy of icotinib in combination with radiotherapy for NSCLC patients with brain metastases. The primary endpoint is PFS of intracranial lesions
- Trial name
- NCT03754530
- Testing
- Icotinib, WBRT or SRS
- Volunteers
- 162 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Advanced Cancer, Metastatic Cancer
Adagrasib in Combination With TNO155 in Patients With Cancer (KRYSTAL 2)
This study will evaluate the safety, tolerability, drug levels, molecular effects, and clinical activity of MRTX849 in combination with TNO155 in patients with advanced solid tumors that have a KRAS G12C mutation.
- Conditions
- Advanced Cancer, Metastatic Cancer, Malignant Neoplastic Disease
- Trial name
- NCT04330664
- Testing
- MRTX849, TNO155
- Where
- 17 sites in United States
- Volunteers
- 86 people
- Sponsor
- Mirati Therapeutics Inc.
Chronic Myeloid Leukemia
Clinical Outcomes of 3L+ Therapies Among Patients With Chronic Myeloid Leukemia and Those With T315I Mutation
The study was a retrospective, non-interventional patient chart review and used a panel of oncologists/hematologists from the US to collect real-world clinical outcomes of patients with CML-CP in 3L+ and those with the T315I…
- Trial name
- NCT05611216
- Where
- United States
- Volunteers
- 164 people
- Sponsor
- Novartis Pharmaceuticals
PIK3CA Mutation, HER2- Negative Breast Cancer
Phase 3 Study of RLY-2608 + Fulvestrant vs Capivasertib + Fulvestrant as Treatment for Locally Advanced or Metastatic PIK3CA-mutant HR+/HER2- Breast Cancer
This is a global, multicenter, open-label, randomized Phase 3 study comparing the efficacy and safety of RLY-2608 (zovegalisib) + fulvestrant to capivasertib + fulvestrant for the treatment of patients with HR+/HER2- ABC with…
- Conditions
- PIK3CA Mutation, HER2- Negative Breast Cancer, Hormone Receptor Positive Tumor, Breast Cancer
- Trial name
- ReDiscover-2 (NCT06982521)
- Testing
- Zovegalisib, Capivasertib, Fulvestrant
- Where
- 243 sites in United States, Argentina and 25 more
- Volunteers
- 540 people
- Sponsor
- Relay Therapeutics, Inc.
Advanced or Metastatic Solid Tumors
A Study to Assess Naporafenib (ERAS-254) Administered With Trametinib in Patients With RAS Q61X Mutations
To evaluate the efficacy of naporafenib administered with trametinib in patients with rat sarcoma viral oncogene (RAS) Q61X solid tumors To evaluate the safety and tolerability of naporafenib administered with trametinib in…
- Trial name
- SEACRAFT-1 (NCT05907304)
- Testing
- Naporafenib, Trametinib
- Where
- 29 sites in United States, Australia and 3 more
- Volunteers
- 86 people
- Sponsor
- Erasca, Inc.
Solid Tumor
Dose-Esc/Exp RMC4630 & Cobi in Relapsed/Refractory Solid Tumors & RMC4630& Osi in EGFR+ Locally Adv/Meta NSCLC
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles of RMC-4630 and cobimetinib in adult participants with relapsed/refractory solid tumors with specific…
- Trial name
- NCT03989115
- Testing
- RMC-4630, Cobimetinib, Drug: Osimertinib
- Where
- 24 sites in United States, South Korea
- Volunteers
- 113 people
- Sponsor
- Revolution Medicines, Inc.
Advanced Solid Tumor, Unresectable Solid Tumor
A Beta-only IL-2 ImmunoTherapY Study
This is a Phase 1/2, multi-center, open-label, dose-escalation and expansion study to evaluate safety and tolerability, PK, pharmacodynamic, and early signal of anti-tumor activity of MDNA11 alone or in combination with a…
- Conditions
- Advanced Solid Tumor, Unresectable Solid Tumor, Clear Cell Renal Cell Carcinoma, Triple Negative Breast Cancer
- Trial name
- ABILITY-1 (NCT05086692)
- Testing
- MDNA11, Pembrolizumab (KEYTRUDA®)
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 160 people
- Sponsor
- Medicenna Therapeutics, Inc.
Solid Tumor, Adult, NSCLC
Adagrasib in Combination With Nab-Sirolimus in Patients With Advanced Solid Tumors and Non-Small Cell Lung Cancer With a KRAS G12C Mutation (KRYSTAL -19)
This study will evaluate the safety, MTD and/or RP2D, PK, and clinical activity of the combination of adagrasib with nab-sirolimus in patients with advanced solid tumors/NSCLC with a KRAS G12C mutation.
- Conditions
- Solid Tumor, Adult, NSCLC, Advanced Cancer, Metastatic Cancer
- Trial name
- NCT05840510
- Testing
- Adagrasib, nab-Sirolimus
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Mirati Therapeutics Inc.
NSCLC, EGFR Activating Mutation
TY-9591 in the Patients With EGFR Mutations in Advanced NSCLC With Brain Metastases
This study is to evaluate the efficacy and safety of TY-9591 in first-line treatment of patients with EGFR-sensitive mutation-positive non-small cell lung cancer with brain metastases compared to Osimertinib.
- Conditions
- NSCLC, EGFR Activating Mutation, Brain Metastases
- Trial name
- NCT05948813
- Testing
- TY-9591, Osimertinib
- Where
- China
- Volunteers
- 420 people
- Sponsor
- TYK Medicines, Inc
Non-Small Cell Lung Cancer, Medullary Thyroid Cancer
A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001)
This is an open-label, first-in-human study designed to evaluate the safety, tolerability, pharmacokinetics (PK) and preliminary anti-tumor activity of selpercatinib (also known as LOXO-292) administered orally to participants…
- Conditions
- Non-Small Cell Lung Cancer, Medullary Thyroid Cancer, Colon Cancer, Any Solid Tumor
- Trial name
- LIBRETTO-001 (NCT03157128)
- Testing
- LOXO-292
- Where
- 85 sites in United States, Australia and 14 more
- Volunteers
- 857 people
- Sponsor
- Eli Lilly and Company
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.