Clinical trials: Gene Mutations
Solid Tumors, Ovarian
First-in-Human, Multicenter, Phase I Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Preliminary Antitumor Activity of YL-18319 Monotherapy in Patients With Locally Advanced/Metastatic Solid Tumors
First-in-Human, multicenter, phase I study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of YL-18319 monotherapy treatment in patients with advanced solid tumors who…
- Trial name
- NCT07903207
- Testing
- YL-18319
- Volunteers
- 45 people
- Sponsor
- Shanghai YingLi Pharmaceutical Co. Ltd.
Sleep Wake Disorders, Sleep Disorders, Circadian Rhythm
Evaluating the Effects of Tasimelteon Vs. Placebo in Delayed Sleep-Wake Phase Disorder (DSWPD) and the CRY1Δ11 Variant
This is a double-blind, randomized, two-period crossover study to evaluate the efficacy and safety of a single oral dose of tasimelteon and matching placebo in male and female subjects with DSWPD and the CRY1Δ11 variant.
- Conditions
- Sleep Wake Disorders, Sleep Disorders, Circadian Rhythm, Chronobiology Disorders, Gene Mutations and Other Alterations Nec
- Trial name
- NCT06701396
- Testing
- Tasimelteon
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Vanda Pharmaceuticals
Breast Cancer
Study of PIK3CA Mutations and Effectiveness and Tolerability Outcomes of Alpelisib in Real-world
SPEAR is a non-interventional / observational, prospective, multicenter study planned to be conducted across ~ 30 sites in India, among HR-positive and HER2-negative ABC/MBC patients.
- Trial name
- SPEAR (NCT05022342)
- Testing
- alpelisib plus fulvestrant
- Where
- 28 sites in India
- Volunteers
- 595 people
- Sponsor
- Novartis Pharmaceuticals
Non-small Cell Lung Cancer
Study to Assess the Efficacy and Safety of Adjuvant Osimertinib in NSCLC With Uncommon EGFRm
This is an open-label, multi-centre, single-arm study assessing the efficacy and safety of osimertinib as adjuvant treatment in stage IB-IIIB (8th AJCC) NSCLC with uncommon EGFRm after receiving complete surgical resection with…
- Trial name
- NCT05546866
- Testing
- Osimertinib
- Where
- 10 sites in China
- Volunteers
- 51 people
- Sponsor
- AstraZeneca
Amyotrophic Lateral Sclerosis
FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)
The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).
- Trial name
- NCT04768972
- Testing
- ION363
- Where
- 25 sites in United States, Belgium and 14 more
- Volunteers
- 89 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Centronuclear Myopathy
Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies
There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety and tolerability as well as pharmacokinetics (PK), pharmacodynamics (PD) and…
- Trial name
- DyNaMic (NCT04743557)
- Testing
- DYN101
- Where
- 2 sites in France, Germany
- Sponsor
- Dynacure
NSCLC
JS111 in Patients With Advanced NSCLC Harboring EGFR Mutations
To evaluate the safety, tolerability, and preliminary efficacy of JS111 capsules in patients with locally advanced, metastatic, or recurrent NSCLC harboring EGFR mutations;to determine the recommended Phase II dose (RP2D).
- Trial name
- NCT06940401
- Testing
- JS111 capsules (AP-L1898)
- Where
- China
- Volunteers
- 44 people
- Sponsor
- Suzhou Junjing BioSciences Co., Ltd.
Frontotemporal Dementia
A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)
A Phase 2 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.
- Trial name
- NCT03987295
- Testing
- AL001
- Where
- 12 sites in United States, Canada and 4 more
- Volunteers
- 33 people
- Sponsor
- Alector Inc.
Solid Tumors, ER+ Breast Cancer
JAB-2485 Activity in Adult Patients With Advanced Solid Tumors
This study is to evaluate the safety and tolerability of JAB-2485 monotherapy in adult participants with advanced solid tumors.
- Conditions
- Solid Tumors, ER+ Breast Cancer, Triple Negative Breast Cancer, TNBC, ARID1A Gene Mutation
- Trial name
- NCT05490472
- Testing
- JAB-2485 (Aurora A inhibitor)
- Where
- 8 sites in United States, China
- Volunteers
- 102 people
- Sponsor
- Jacobio Pharmaceuticals Co., Ltd.
Solid Tumor (Phase I), RAS Mutation
A Study of AN4035 in Advanced Cancers With RAS Mutations and High CEACAM5 Expression
The goal of this clinical trial is to determine whether AN4035 is safe and tolerable in people with advanced or metastatic solid tumors that have Rat Sarcoma oncogene (RAS) mutated solid tumors and high levels of the CEACAM5…
- Conditions
- Solid Tumor (Phase I), RAS Mutation, CEACAM5
- Trial name
- NCT07686445
- Testing
- Intravenous AN4035, Intravenous AN4035 followed by intravenous EGFR Inhibitor.
- Where
- 2 sites in Australia
- Volunteers
- 228 people
- Sponsor
- Adlai Nortye Biopharma Co., Ltd.
Venous Malformation, Lymphatic Malformation
First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations
VT30-101 is a 2-part first-in-human trial of topically administered VT30 to subjects with cutaneous venous malformations, lymphatic malformations, or mixed venolymphatic malformations associated with PIK3CA or TEK mutations.
- Conditions
- Venous Malformation, Lymphatic Malformation, Venolymphatic Malformation
- Trial name
- NCT04409145
- Testing
- VT30
- Where
- 15 sites in United States
- Volunteers
- 15 people
- Sponsor
- Venthera, Inc., a BridgeBio company
Acute Myeloid Leukemia, Leukemia
A Phase I Study of AC220 in Patients With Relapsed/Refractory Acute Myeloid Leukemia Regardless of FLT3 Status
Patients received oral AC220 daily for 14 days to study the side effects, tolerability and best dose for treating relapsed or refractory acute myeloid leukemia, regardless of FLT3 status.
- Conditions
- Acute Myeloid Leukemia, Leukemia, Myelodysplastic Syndrome, AML
- Trial name
- NCT00462761
- Testing
- AC220
- Where
- 5 sites in United States, Georgia
- Volunteers
- 76 people
- Sponsor
- Daiichi Sankyo
Solid Tumor, Pancreatic Ductal Adenocarcinoma (PDAC)
Study of JYP0015 in Patients With Advanced Solid Tumors Harboring Specific Mutations in RAS
Evaluate the safety and antitumor activity of JYP0015 in adults with specific RAS mutant advanced solid tumors.
- Conditions
- Solid Tumor, Pancreatic Ductal Adenocarcinoma (PDAC), Non-small Cell Lung Cancer (NSCLC), Colorectal Cancer (CRC)
- Trial name
- STAR (NCT06895031)
- Testing
- JYP0015
- Where
- China
- Volunteers
- 210 people
- Sponsor
- Guangzhou JOYO Pharma Co., Ltd
Cancer, Solid Tumors
Vevorisertib (ARQ 751) as a Single Agent or in Combination With Other Anti-Cancer Agents, in Solid Tumors With PIK3CA / AKT / PTEN Mutations (MK-4440-001)
The primary objectives of this study are: Part 1 - Vevorisertib as single agent: To assess the safety and tolerability of vevorisertib in participants with advanced solid tumors with v-Akt murine thymoma viral oncogene homolog…
- Trial name
- NCT02761694
- Testing
- Vevorisertib, Fulvestrant, Paclitaxel
- Where
- 6 sites in United States
- Volunteers
- 78 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Metastatic Castration-resistant Prostate Cancer
Clinical Study to Assess the Efficacy and Safety of Olaparib in Chinese Patients With Metastatic Castration-Resistant Prostate Cancer Who Have Failed Prior Treatment With a New Hormonal Agent and Have BRCA1/2 Mutations
The purpose of this study is to evaluate the efficacy and safety of Olaparib compared with standard of care (Enzalutamide or Abiraterone Acetate) in Chinese men with metastatic castration-resistant prostate cancer who have failed…
- Trial name
- NCT05457257
- Testing
- olaparib, enzalutamide, abiraterone acetate
- Where
- 35 sites in China
- Volunteers
- 43 people
- Sponsor
- AstraZeneca
VHL Gene Mutation, VHL
MK-3795 (PT2385) for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma (MK-3795-003)
The primary objective of this study is to assess the overall response rate (ORR) of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC) tumors in VHL participants treated with MK-3795.
- Conditions
- VHL Gene Mutation, VHL, VHL Syndrome, VHL Gene Inactivation
- Trial name
- NCT03108066
- Testing
- MK-3795
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Malignant Melanoma
Study Of Zelboraf (Vemurafenib) in Patients With Locally-Advanced, Unresectable, Stage IIIc Or Metastatic Melanoma and Activating Exon 15 BRAF Mutations Other Than V600E
This is an open-label, multicenter, single-agent, phase II study of continuous oral Zelboraf (vemurafenib) in participants with locally-advanced, unresectable, stage IIIc or metastatic melanoma and activating exon 15 BRAF…
- Trial name
- NCT01586195
- Testing
- Vemurafenib
- Where
- 15 sites in United States
- Volunteers
- 31 people
- Sponsor
- Genentech, Inc.
Non-Small Cell Lung Cancer
A Phase II Trial of LM103 for Adjuvant Treatment in Patients With Non-small Cell Lung Cancer
After receiving neoadjuvant treatment with PD-1 antibody and undergoing radical resection, a total 36 to 45 NSCLC patients who met the inclusion criteria, will be randomly assigned in a 1:1:1 ratio to the experimental group 1…
- Trial name
- NCT07444437
- Testing
- LM103 TILs Injection, PD-1 / PD-L1 monoclonal antibody
- Where
- 5 sites in China
- Volunteers
- 45 people
- Sponsor
- Suzhou BlueHorse Therapeutics Co., Ltd.
Advanced Solid Tumor, Metastatic Solid Tumor
PRT3789 Monotherapy and in Combo w/Docetaxel in Participants w/Advanced or Metastatic Solid Tumors w/SMARCA4 Mutation
This is a Phase 1 dose-escalation study of PRT3789, a SMARCA2 degrader, in participants with advanced or metastatic solid tumors with loss of SMARCA4 due to truncating mutation and/or deletion.
- Conditions
- Advanced Solid Tumor, Metastatic Solid Tumor, Non-small Cell Lung Cancers, SMARCA4 Gene Mutation
- Trial name
- NCT05639751
- Testing
- PRT3789, Docetaxel
- Where
- 32 sites in United States, France and 3 more
- Volunteers
- 135 people
- Sponsor
- Prelude Therapeutics Incorporated
Cardiomyopathy
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality…
- Trial name
- MyCLIMB (NCT05112237)
- Where
- 29 sites in United States, Canada and 2 more
- Volunteers
- 200 people
- Sponsor
- Tenaya Therapeutics