Clinical trials: Gene
Low-grade NMIBC, FGFR Gene Amplification
Efficacy and Safety of TYRA-300 in Participants With FGFR3 Altered Low Grade, Intermediate Risk Non-Muscle Invasive Bladder Cancer
Phase 2 Study of TYRA-300 in FGFR3 Altered Low Grade, Intermediate Risk NMIBC
- Conditions
- Low-grade NMIBC, FGFR Gene Amplification, FGFR Gene Alterations, FGFR3 Gene Alteration
- Trial name
- SURF302 (NCT06995677)
- Testing
- TYRA-300 60mg, TYRA-300 50mg, TYRA-300 Dose 70 mg
- Where
- 48 sites in United States, Australia and 2 more
- Volunteers
- 90 people
- Sponsor
- Tyra Biosciences, Inc
Locally Advanced or Metastatic Solid Tumor Harboring an NTRK Gene Fusion
Study to Learn More About the Safety and Effectiveness of the Drug VITRAKVI During Routine Use in Patients With TRK Fusion Cancer Which is Locally Advanced or Spread From the Place Where it Started to Other Places in the Body
In this observational study researcher want to learn more about the effectiveness of drug VITRAKVI (generic name: larotrectinib) and how well the drug is tolerated during routine use in patients with TRK fusion cancer which is…
- Trial name
- ON-TRK (NCT04142437)
- Testing
- larotrectinib(Vitrakvi, BAY2757556)
- Where
- 47 sites in United States, Argentina and 13 more
- Volunteers
- 54 people
- Sponsor
- Bayer
Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
This is a phase 2, multicenter, randomized, placebo-controlled, double-blind, parallel-group, proof-of-concept study to assess vamifeport in adult participants with homeostatic iron regulator gene-related hereditary…
- Trial name
- NCT07332091
- Testing
- Vamifeport
- Where
- 100 sites in United States, Australia and 16 more
- Volunteers
- 84 people
- Sponsor
- CSL Behring
Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation
Study to Learn If Tofersen (BIIB067) Can Delay ALS Symptoms and About Its Safety in Adults With a SOD1 Gene Mutation Who Have Not Yet Developed ALS Symptoms
In this study, researchers will learn more about the effects of a drug called tofersen, also known as BIIB067 and QALSODY®, in adults with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene…
- Trial name
- ATLAS (NCT04856982)
- Testing
- Tofersen
- Where
- 32 sites in United States, Australia and 12 more
- Volunteers
- 158 people
- Sponsor
- Biogen
Gene Mutations, Pseudoxanthoma Elasticum
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to…
- Conditions
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
- Trial name
- NCT06462547
- Testing
- INZ-701
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 22 people
- Sponsor
- Inozyme Pharma
Metastatic Hepatocellular Carcinoma, Solid Tumors
Safety and Preliminary Anti-Tumor Activity of TYRA-430 in Advanced Hepatocellular Carcinoma and Other Solid Tumors With Activating FGF/FGFR Pathway Aberrations
A Phase 1 study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamic (PD), and preliminary antitumor activity of TYRA-430 in cancers with FGF/FGFR pathway aberrations, including locally advanced/metastatic…
- Conditions
- Metastatic Hepatocellular Carcinoma, Solid Tumors, Solid Tumor, Adult, FGFR Gene Amplification
- Trial name
- SURF431 (NCT06915753)
- Testing
- TYRA-430
- Where
- 16 sites in United States, Canada and 2 more
- Volunteers
- 125 people
- Sponsor
- Tyra Biosciences, Inc
Epidermal Growth Factor, Epidermal Growth Factor Receptor
A Phase 1 Study of EPI-326 in EGFR-mutant NSCLC and HNSCC
A phase 1 study to determine the safety, tolerability, PK, PD, and preliminary anti-tumor activity of ascending doses of EPI-326 administered to patients with locally advanced or metastatic HNSCC and to patients with any…
- Conditions
- Epidermal Growth Factor, Epidermal Growth Factor Receptor, Epidermal Growth Factor Receptor Gene Mutation, Non Small Cell
- Trial name
- NCT07462377
- Testing
- EPI-326
- Where
- 8 sites in United States, South Korea and 1 more
- Volunteers
- 110 people
- Sponsor
- EpiBiologics
Patients With Locally Advanced or Metastatic Non-Squamous NSCLC Requiring First-Line Treatment and no Driver Gene Alterations Confirmed by Histology or Cytology
Phase II Study to Evaluate Telisotuzumab Adizutecan in Combination With RC148 as First-Line Treatment for Patients With Non-Squamous Non-Small-Cell Lung Cancer
To Evaluate the Safety, Tolerability, and Preliminary Antitumor Efficacy of RC148 in Combination with Telisotuzumab adizutecan as First-line Treatment for Locally Advanced or Metastatic Non-Squamous Non-Small-Cell Lung Cancer
- Trial name
- NCT07874282
- Testing
- Telisotuzumab adizutecan plus RC148
- Where
- China
- Volunteers
- 200 people
- Sponsor
- RemeGen Co., Ltd.
Breast Neoplasms, Solid Tumors
A Phase 1/2a Study of K-1728 in Advanced Solid Tumors
This first-in-human, open-label, multicenter study evaluates K-1728 administered as monotherapy and in combination in participants with advanced or metastatic solid tumors.
- Conditions
- Breast Neoplasms, Solid Tumors, PIK3CA Gene Mutation
- Trial name
- PIKYMA-01 (NCT07861932)
- Testing
- K-1728, Fulvestrant, Palbociclib
- Volunteers
- 110 people
- Sponsor
- KymaThera, Inc.
Desmoid Tumor, NOTCH Gene Mutation Positive Tumors
Individual Patient Compassionate Use of Nirogacestat
This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee, and applicable competent authority approval.
- Trial name
- NCT05041036
- Testing
- Nirogacestat (Gamma Secretase Inhibitor)
- Where
- 2 sites in United States, Germany
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
glp1 Agonist, Gene Expression
Ellacor® Micro-Coring Technology® in Fitzpatrick Skin Types I-VI, Including Patients Receiving Glucagon-Like Peptide-1 Receptor Agonists
The main purpose of the study is to collect information about how the device functions in a variety of treatment areas and skin types and to find out what changes, if any, occur in the skin treated with the study device compared…
- Conditions
- glp1 Agonist, Gene Expression, Skin Laxity
- Trial name
- NCT07224880
- Testing
- ellacor
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Cytrellis Biosystems, Inc.
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 51 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
Advanced or Metastatic Solid Tumors With NTRK, ROS1 or ALK Gene Fusion
An Open Label, Multi-center, Phase I Clinical Study to Evaluate the Safety, Effectiveness and Pharmacokinetic Characteristics of SIM1803-1A in Patients With Locally Advanced/Metastatic Solid Tumors With NTRK, ROS1 or ALK Gene Fusion Mutations.
This research study is done to test the safety, effectiveness and pharmacokinetic characteristics of SIM1803-1A in patients with locally advanced/metastatic solid tumors with NTRK, ROS1 or ALK gene fusion mutations.
- Trial name
- NCT04671849
- Testing
- SIM1803-1A
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Jiangsu Simcere Pharmaceutical Co., Ltd.
HER2 Gene Mutation, Advanced Solid Tumors
HEAT Trial (HER2 Antibody Therapy With Lutetium-177)
This is a first-in-human, Phase 0/1, open-label study of177Lu-RAD202 to determine the recommended dose(s) for future exploration of 177Lu-RAD202 in participants with HER2 expressing advanced solid tumours.
- Trial name
- RAD202 (NCT06824155)
- Testing
- 177Lu-RAD202
- Where
- 5 sites in Australia
- Volunteers
- 30 people
- Sponsor
- Radiopharm Theranostics, Ltd
Breast Cancer Stage III, HER2-positive Metastatic Breast Cancer
Evaluate BL-M17D1 in Patients w/HER2-Expressing/Mutant Advanced or Metastatic Solid Tumors
The objective of this study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of BL-M17D1 in patients with HER2-Expressing or HER2-Mutant Advanced or Metastatic Solid Tumors.
- Conditions
- Breast Cancer Stage III, HER2-positive Metastatic Breast Cancer, Unresectable Breast Carcinoma, Her2-Positive
- Trial name
- NCT06714617
- Testing
- BL-M17D1
- Where
- 9 sites in United States
- Volunteers
- 3 people
- Sponsor
- SystImmune Inc.
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
Amyotrophic Lateral Sclerosis (ALS), Mutation in the Superoxide Dismutase-1 (SOD1) Gene
First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1)…
- Trial name
- GOALS (NCT06351592)
- Testing
- ALN-SOD, Diluent
- Where
- 22 sites in Australia, Belgium and 7 more
- Volunteers
- 54 people
- Sponsor
- Regeneron Pharmaceuticals
Advanced Solid Tumor, Adult, ERBB2 Altered Breast Cancer
A Study of a Selective ERBB2 Inhibitor (CGT4255), in Patients With Advanced Solid Tumors
This is an open-label, phase 1/1b study evaluating the safety, tolerability, pharmacokinetic (what the body does to the drug), pharmacodynamic (what the drug does to the body), and antitumor activity of CGT4255 in adult…
- Conditions
- Advanced Solid Tumor, Adult, ERBB2 Altered Breast Cancer, ERBB2 Gene Amplification, HER2 Overexpression
- Trial name
- NCT07361562
- Testing
- CGT4255
- Where
- 15 sites in United States
- Volunteers
- 100 people
- Sponsor
- Cogent Biosciences, Inc.
FGFR2 Gene Fusion/Rearrangement, Other Solid Tumors, Adult
A Study of Lirafugratinib in Non-CCA Solid Tumors With FGFR2 Fusion or Rearrangement
The goal of this clinical trial is to evaluate if lirafugratinib is efficacious and safe to treat adult patients with previously treated, unresectable, locally advanced or metastatic solid tumors (excluding cholangiocarcinoma)…
- Trial name
- ReFocus202 (NCT07359820)
- Testing
- Lirafugratinib
- Where
- 19 sites in United States, France and 3 more
- Volunteers
- 30 people
- Sponsor
- Elevar Therapeutics
Advanced Cholangiocarcinoma, FGFR2 Fusions
Study of Futibatinib in Patients With Advanced Cholangiocarcinoma With FGFR2 Fusion or Rearrangement
This is an open-label, multinational, randomized Phase 2 study confirming the clinical benefit of 20 mg futibatinib and evaluating the safety and efficacy of 16 mg futibatinib in previously treated CCA harboring FGFR2 gene…
- Conditions
- Advanced Cholangiocarcinoma, FGFR2 Fusions, Gene Rearrangement
- Trial name
- FOENIX-CCA4 (NCT05727176)
- Testing
- TAS-120
- Where
- 65 sites in United States, Argentina and 11 more
- Volunteers
- 120 people
- Sponsor
- Taiho Oncology, Inc.