Clinical trials: Genetic Mutation
Cystic Fibrosis
Study of GLPG1837 in Subjects With Cystic Fibrosis (S1251N Mutation)
At least 6 cystic fibrosis patients with the S1251N mutation will be treated for 4 weeks, consisting of two consecutive treatment periods of two weeks evaluating one dose of GLPG1837 each.
- Trial name
- SAPHIRA2 (NCT02690519)
- Testing
- GLPG1837 dose 1, GLPG1837 dose 2
- Where
- 5 sites in Belgium, Netherlands
- Volunteers
- 7 people
- Sponsor
- Lakefront Biotherapeutics NV
Chronic Myelogenous Leukemia - Chronic Phase
Asciminib in Monotherapy for Chronic Myeloid Leukemia in Chronic Phase (CML-CP) With and Without T315I Mutation
This study was a multicenter Phase IIIb open-label, three-cohort study of asciminib in patients with CML-CP without T315I mutation who have had at least 2 prior TKIs and CML-CP harboring the T315I mutation with at least 1 prior…
- Trial name
- AIM4CML (NCT04666259)
- Testing
- ABL001
- Where
- 26 sites in United States
- Volunteers
- 56 people
- Sponsor
- Novartis Pharmaceuticals
Breast Cancer
Phase I Study of the HS-10352 in Patients With Advanced Breast Cancer
HS-10352 is a highly potent and selective small molecule inhibitor of phosphoinositide 3-kinase (p110α). In preclinical studies, it demonstrated strong activity against PI3K p110α in vitro and in vivo, and inhibited tumor cell…
- Trial name
- NCT04631835
- Testing
- HS-10352
- Where
- 3 sites in China
- Volunteers
- 54 people
- Sponsor
- Jiangsu Hansoh Pharmaceutical Co., Ltd.
Metastatic Colorectal Cancer
Sym004 in Metastatic Colorectal Cancer and ECD-EGFR Patients
This is a single arm, phase 2, open-label, multicenter trial in patients with metastatic colorectal cancer (mCRC) and acquired resistance to anti-epidermal growth factor receptor (EGFR) monoclonal antibodies (mAbs) and documented…
- Trial name
- NCT02965417
- Testing
- Sym004
- Sponsor
- Symphogen A/S
NUT Midline Carcinoma, Triple Negative Breast Cancer
A Dose-Finding Study of Birabresib (MK-8628), a Small Molecule Inhibitor of the Bromodomain and Extra-Terminal (BET) Proteins, in Adults With Selected Advanced Solid Tumors (MK-8628-003)
Open-label, phase I, non-randomized, multicentric study of single-agent birabresib (MK-8628) (formerly known as OTX015) administered according to two distinct regimens to participants with selected advanced tumors.
- Conditions
- NUT Midline Carcinoma, Triple Negative Breast Cancer, Non-small Cell Lung Cancer With Rearranged ALK Gene/Fusion Protein or KRAS Mutation, Castrate-resistant Prostate Cancer
- Trial name
- NCT02259114
- Testing
- Birabresib
- Volunteers
- 47 people
- Sponsor
- Oncoethix GmbH, a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
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.
Advanced Non-small Cell Lung Cancer, EGFR Mutation
A Study to Learn About How Food and Esomeprazole Affect the Level of BAY2927088 in the Blood When Taken Together in Healthy Participants
Researchers are looking for a better way to treat people who have advanced non-small cell lung cancer (NSCLC) with specific genetic changes called epidermal growth factor receptor (EGFR) and human epidermal growth factor receptor…
- Conditions
- Advanced Non-small Cell Lung Cancer, EGFR Mutation, HER2 Mutation, Healthy Volunteers
- Trial name
- NCT06378658
- Testing
- BAY2927088, Esomeprazole, Food
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Bayer
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENEREXT Study
This is an open-label, multi-centre study in subjects with a genetically confirmed mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation who completed study KH176-202.
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS)
- Trial name
- NCT04604548
- Testing
- Oral administration of 100 mg KH176 twice daily
- Where
- 4 sites in Denmark, Germany and 2 more
- Volunteers
- 11 people
- Sponsor
- Khondrion BV
Cerebrovascular Accident, Stroke, Acute
Fabry and Cardiomyopathy (FaCard)
Primary objective and endpoint is the analysis of the long-term course of lyso-Gb3 and its clinical correlation to the progression of the cardiomyopathy in N215S-Fabry patients.
- Conditions
- Cerebrovascular Accident, Stroke, Acute, Cerebral Stroke
- Trial name
- FaCard (NCT01429597)
- Where
- Germany
- Sponsor
- CENTOGENE GmbH Rostock
KRAS G12C Mutation Advanced Solid Tumor
Phase 1 Study of HS-10541 as Monotherapy or in Combination With Other Anti-cancer Therapies in Patients With KRAS G12C Mutation Advanced Solid Tumors.
This is a multicenter, open-label phase I clinical trial to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of HS-10541 as monotherapy or in combination with other anti-cancer therapies in…
- Trial name
- NCT07615413
- Testing
- HS-10541
- Volunteers
- 636 people
- Sponsor
- Jiangsu Hansoh Pharmaceutical Co., Ltd.
Sensorineural Hearing Loss, Bilateral, AUNB1
Natural History in Children up to 16 Years With Mild to Profound Hearing Loss Due to Mutations in GJB2 / OTOF Genes
The purpose of this study is to follow the natural history of non-syndromic hearing loss caused by mutations in two genes (GJB2 or OTOF) in children up to 16 years of age.
- Conditions
- Sensorineural Hearing Loss, Bilateral, AUNB1, DFNB1A, Congenital Deafness
- Trial name
- Otoconex (NCT05402813)
- Testing
- Pure Tone Audiometry Assessment, Quality of Life Questionnaires
- Where
- France
- Volunteers
- 180 people
- Sponsor
- Sensorion
Non-Small Cell Lung Cancer
Combination Study of RMC-4630 and Sotorasib for NSCLC Subjects With KRASG12C Mutation After Failure of Prior Standard Therapies
The purpose of this study is to evaluate the antitumor effects of sotorasib and RMC-4630 in subjects with KRASG12C mutant NSCLC
- Trial name
- NCT05054725
- Testing
- RMC-4630, Sotorasib
- Where
- 60 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Revolution Medicines, Inc.
Locally Advanced or Metastatic Non Small Cell Lung Cancer Stage IIIb - IV
Assess Efficacy & Safety of Selumetinib in Combination With Docetaxel in Patients Receiving 2nd Line Treatment for v-Ki-ras2 Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS) Positive NSCLC
The purpose of this study is to assess the efficacy of selumetinib in combination with docetaxel (75mg/m2) vs placebo in combination with docetaxel (75mg/m2) in patients with locally advance or metastatic NSCLCs that harbor…
- Trial name
- SELECT-1 (NCT01933932)
- Testing
- Selumetinib, Docetaxel, Pegylated G-CSF
- Where
- 197 sites in United States, Argentina and 24 more
- Volunteers
- 510 people
- Sponsor
- AstraZeneca
Solid Tumor, Adult, BRCA1 Mutation
Study of ACE-86225106 to Treat Patients With Advanced Solid Tumors
The purpose of this study is to determine if the experimental treatment with poly-ADP ribose polymerase (PARP) inhibitor, ACE-86225106 is safe, tolerable and has anti-cancer activity in adult patients with advanced solid tumors.
- Conditions
- Solid Tumor, Adult, BRCA1 Mutation, BRCA2 Mutation, Ovarian Cancer
- Trial name
- NCT06380660
- Testing
- ACE-86225106 tablet
- Where
- 14 sites in China
- Volunteers
- 298 people
- Sponsor
- Acerand Therapeutics (Shanghai) Limited
Epidermolysis Bullosa Dystrophica, Recessive, Epidermolysis Bullosa Dystrophica, Dominant
A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene
A double-blind, randomized, intra-subject placebo-controlled, multicenter, multiple dose study, evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with confirmed DDEB or RDEB diagnosis…
- Trial name
- NCT03605069
- Testing
- QR-313
- Where
- 6 sites in United States, France and 1 more
- Volunteers
- 2 people
- Sponsor
- Phoenicis Therapeutics
Advanced or Metastatic Solid Tumor
The Clinical Trial to Evaluate the Pharmacokinetics and Safety of MRTX849 in Patients With Advanced Solid Tumors
This is a phase 1, open-label, single-arm study in Chinese patients with unresectable, locally advanced or metastatic solid tumor with KRAS G12C mutation, for which treatment with curative intent is not available.
- Trial name
- NCT05263986
- Testing
- MRTX849
- Where
- 8 sites in China
- Volunteers
- 22 people
- Sponsor
- Zai Lab (Shanghai) Co., Ltd.
Advanced Solid Tumors
A Study of Safety, Tolerability and Preliminary Efficacy of NTS071 in Subjects With Advanced Solid Tumors Harboring a TP53 Y220C Mutation
This study is to evaluate the safety, tolerability, PK, and preliminary efficacy of NTS071 in adults with TP53 Y220C-mutated solid tumors.
- Trial name
- NTS071 (NCT07060989)
- Testing
- NTS071
- Where
- 10 sites in United States, China
- Volunteers
- 220 people
- Sponsor
- Nutshell Therapeutics (Shanghai) Co., LTD.
HER2-positive Breast Cancer, HER2 Gene Mutation
Study of A166 in Patients With Relapsed/Refractory Cancers Expressing HER2 Antigen or Having Amplified HER2 Gene
Open-label, Phase I-II, first-in-human (FIH) study for A166 monotherapy in HER2-expressing or amplified patients who progressed on or did not respond to available standard therapies.
- Conditions
- HER2-positive Breast Cancer, HER2 Gene Mutation, HER-2 Gene Amplification, HER2 Positive Gastric Cancer
- Trial name
- NCT03602079
- Testing
- A166
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Klus Pharma Inc.
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies
Single Ascending Dose Study in Participants With LCA10
The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound…
- Conditions
- Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease
- Trial name
- NCT03872479
- Testing
- EDIT-101
- Where
- 5 sites in United States
- Volunteers
- 34 people
- Sponsor
- Editas Medicine, Inc.
Advanced or Metastatic Solid Tumors Irrespective of Gene Alterations, Advanced or Metastatic Solid Tumors With Germline PTEN Inactivating Mutations
TAS-117 in Patients With Advanced Solid Tumors Harboring Germline PTEN Mutations
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity of TAS-117 in patients with advanced or metastatic solid tumors (excluding primary brain tumors)…
- Trial name
- NCT04770246
- Testing
- TAS-117
- Where
- 8 sites in United States, Austria and 2 more
- Volunteers
- 17 people
- Sponsor
- Taiho Oncology, Inc.
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