Clinical trials: Gene Mutations
Completely Resected NSCLC With Common EGFR Mutations
A Phase III, Randomized, Multi-center Study to Determine the Efficacy of the Intercalating Combination Treatment of Chemotherapy and Gefitinib or Chemotherapy as Adjuvant Treatment in NSCLC With Common EGFR Mutations.
This study aims to compare the efficacy of intercalating chemotherapy (gefitinib and pemetrexed/cisplatin) and chemotherapy (navelbine/cisplatin) in completely resected NSCLC with common EGFR mutations.
- Trial name
- NCT03381066
- Testing
- gefitinib, pemetrexed,cisplatin, Vinorelbine, cisplatin
- Where
- South Korea
- Volunteers
- 225 people
- Sponsor
- Yonsei University
Breast Cancer
Real-World Outcomes of US Talazoparib-Treated Patients With Locally Advanced or Metastatic Breast Cancer
This study is a retrospective, multi-site, patient-level medical chart review of US adult patients with locally advanced or metastatic breast cancer (ABC) who initiated talazoparib on or after October 16, 2018 and were managed by…
- Trial name
- NCT04987931
- Testing
- Talazoparib
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- Pfizer
Non-Small-Cell Lung Cancer
Vebreltinib Plus Furmonertinib in Patients With EGFR-mutated Advanced Non-small Cell Lung Cancer and High PD-L1 Expression
This is a single-arm, exploratory phase Ib/II study with a seamless design to evaluate the safety and efficacy of Vebreltinib combined with furmonertinib as first-line treatment in patients with locally advanced or metastatic…
- Trial name
- DUAL-THRUST (NCT07655622)
- Testing
- Vebreltinib, Furmonertinib 80mg
- Where
- China
- Volunteers
- 45 people
- Sponsor
- Second Affiliated Hospital of Nanchang University
Acute Myeloid Leukemia, AML, Adult
Revumenib in Combination With 7+3 + Midostaurin in AML
This research is being conducted to determine a safe and effective dose of revumenib that can be given in combination with standard induction (initial therapy to induce a remission) + FLT3 targeted therapy (midostaurin) and a…
- Conditions
- Acute Myeloid Leukemia, AML, Adult, AML With Gene Mutations, AML
- Trial name
- NCT06313437
- Testing
- Revumenib, Midostaurin, Cytarabine
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Richard Stone, MD
Non-small Cell Lung Cancer, Effects of Chemotherapy
Vinorelbine-ifosfamide Versus Gefitinib for EGFR Gene Mutation Negative Non-small Cell Lung Cancer Patients
In the National Comprehensive Cancer Network (NCCN) guideline for NSCLC, epidermal growth factor receptor-tyrosine kinase inhibitor (EGFR-TKI) is recommended as the third-line treatment for EGFR gene mutation negative NSCLC…
- Trial name
- NCT01749072
- Testing
- Gefitinib group, Vinorelbine, Ifosfamide, Mesna
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Peking Union Medical College Hospital
Charcot Marie Tooth Disease, DIFGAN
Genotype/Phenotype Correlation of MORC2 Mutations
The Microrchidia CW-type zinc finger 2 (MORC2) gene encodes a protein expressed in all tissues and enriched in the brain. It is involved in Charcot-Marie-Tooth disease, with mire than 30 families presenting MORC2 mutations.
- Conditions
- Charcot Marie Tooth Disease, DIFGAN, Developmental Delay (Disorder), Impaired Growth
- Trial name
- PhenoMORC2 (NCT07038239)
- Testing
- Skin biopsy, Blood sample
- Where
- 12 sites in France
- Volunteers
- 45 people
- Sponsor
- Hospices Civils de Lyon
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
IDH2-Post-Allo-Trial for Patients with IDH2-mut Myeloid Neoplasms After Allo-SCT
This is a prospective, open label, single arm, multi-centre phase II trial aiming to evaluate the safety and efficacy of Enasidenib (investigational product) as prophylactic consolidation in patients with IDH2-mutated MDS, CMML…
- Conditions
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, IDH2 Gene Mutation
- Trial name
- NCT04522895
- Testing
- Enasidenib
- Where
- 11 sites in Germany
- Volunteers
- 50 people
- Sponsor
- Heinrich-Heine University, Duesseldorf
Malignant Melanoma Stage IV, Malignant Melanoma Stage III
Detection of Plasmatic Cell-free BRAF and NRAS Mutations : a New Tool for Monitoring Patients With Metastatic Malignant Melanoma Treated With Targeted Therapies or Immunotherapy ( MALT )
The main objective of this project is to perform a longitudinal monitoring of BRAF and NRAS cell-free DNA in a large cohort of metastatic melanomas patients before treatment and during the follow-up.
- Trial name
- MALT (NCT03493230)
- Testing
- quantification of BRAF and NRAS mutation
- Where
- France
- Volunteers
- 35 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
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.
Limb-girdle Muscular Dystrophy
MRI and Muscle Involvement in Patients With Mutations in GMPPB
Limb girdle muscular dystrophies (LGMD) are a very heterogeneous group of muscle disorders characterized by muscle weakness and atrophy of the proximal muscles of the shoulder and pelvic girdles.
- Trial name
- NCT02635321
- Where
- Denmark
- Volunteers
- 4 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Duchenne Muscular Dystrophy, Exon 44
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants…
- Trial name
- EXPLORE44 (NCT05670730)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Avidity Biosciences, Inc.
Non-Small Cell Lung Cancer, Brain Metastases
Comparator-Controlled Study for EGFR(+) Patients With Multiple BMs From NSCLC (BROKE) (EGFR-epidermal Growth Factor Receptor;BM-brain Metastases)
1. Compare the effect and safety of gefitinib alone with gefitinib plus concomitant WBRT(whole-brain radiotherapy ) in treatment of NSCLC patients harboring an EGFR mutation with multiple BM. 2.
- Conditions
- Non-Small Cell Lung Cancer, Brain Metastases, EGFR Gene Mutation
- Trial name
- EGFR (NCT02338011)
- Testing
- Gefitinib, WBRT
- Where
- China
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- Shanghai Chest Hospital
Cohort 1a and 1b: Glioma (Advanced Gliomas and Glioblastoma Multiforme), Cohort 2a and 2b: Hepatobiliary Tumors (Hepatocellular Carcinoma, Bile Duct Carcinoma, Intrahepatic Cholangiocarcinoma, Other Hepatobiliary Carcinomas)
A Study of FT-2102 in Patients With Advanced Solid Tumors and Gliomas With an IDH1 Mutation
This Phase 1/2 study will evaluate the safety, efficacy, PK, and PD of FT-2102 as a single agent and in combination with other anti-cancer drugs in patients with advanced solid tumors and gliomas.
- Conditions
- Cohort 1a and 1b: Glioma (Advanced Gliomas and Glioblastoma Multiforme), Cohort 2a and 2b: Hepatobiliary Tumors (Hepatocellular Carcinoma, Bile Duct Carcinoma, Intrahepatic Cholangiocarcinoma, Other Hepatobiliary Carcinomas), Cohort 3a and 3b: Chondrosarcoma, Cohort 4a and 4b: Intrahepatic Cholangiocarcinoma
- Trial name
- NCT03684811
- Testing
- FT-2102, Azacitidine, Nivolumab
- Where
- 26 sites in United States, Australia and 4 more
- Volunteers
- 93 people
- Sponsor
- Forma Therapeutics, Inc.
X-Linked Retinitis Pigmentosa
Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
The purpose of this study is to evaluate subjects with X-linked retinitis pigmentosa caused by RPGR-ORF15 mutations in a clinical setting to fully characterize their condition, measure testing variability, and estimate rates of…
- Trial name
- NCT03314207
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- Beacon Therapeutics
Prostate Cancer, Prostate Carcinoma
The Study of Olaparib in Newly Diagnosed mCRPC Patients With HRR Gene Mutation
This is a multi-center, single-arm, prospective study to assess the efficacy and safety of Olaparib in men with newly diagnosed metastatic castration-resistant prostate cancer (mCRPC) who carried homologous recombination repair…
- Conditions
- Prostate Cancer, Prostate Carcinoma, Metastatic Castration-resistant Prostate Cancer
- Trial name
- PROspect (NCT05262608)
- Testing
- Olaparib
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Stargardt Disease
A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene.
- Trial name
- CELESTE (NCT07161544)
- Testing
- AAVB-039
- Where
- 11 sites in United States, Norway and 1 more
- Volunteers
- 75 people
- Sponsor
- AAVantgarde Bio Srl
Acute Myeloid Leukemia, IDH1 Gene Mutation
a New Treatment of Newly Diagnosed IDH1 Mutation Acute Myeloid Leukemia
This is a single arm, open-label, multicenter clinical trial to evaluate the efficacy and safety of ivosidenib+venetoclax+ azacitidine in adult Chinese subjects with newly diagnosed IDH1m AML.A total of approximately 42 China…
- Conditions
- Acute Myeloid Leukemia, IDH1 Gene Mutation, Ivosidenib
- Trial name
- NCT07007949
- Testing
- Ivosidenib combined with venetoclax and azacitidine
- Where
- China
- Volunteers
- 42 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Advanced Non-small Cell Lung Cancer
A Phase I/II, Multicenter, Open-label Study of EGFRmut-TKI EGF816, Administered Orally in Adult Patients With EGFRmut Solid Malignancies
This is a Phase I/II, multi-center, open-label study, composed with a Phase I part (dose-escalation phase) followed by a Phase II part (expansion phase).
- Trial name
- NCT02108964
- Testing
- EGF816
- Where
- 14 sites in United States, Canada and 7 more
- Volunteers
- 225 people
- Sponsor
- Novartis Pharmaceuticals
Lysosomal Acid Lipase Deficiency
Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)
Familial Hypercholesterolemia (FH) is a monogenic autosomal dominant disease also known as Autosomal Dominant Hypercholesterolemia - ADH) that leads to dramatically increased levels of Low Density Lipoprotein (LDL) and total…
- Trial name
- NCT03984149
- Testing
- Observational study
- Where
- 4 sites in Italy
- Volunteers
- 1,000 people
- Sponsor
- Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)
HIV-1-infection
Dynamics of Drug Resistance-associated Mutations in HIV-1 DNA Reverse Transcriptase Clearance During Effective Antiretroviral Therapy
In view of the prolongation of patients living with HIV's life expectancy, the question of optimization of ART, which is still a life-long treatment, becomes central.
- Trial name
- MUTARESERVOIR (NCT04448158)
- Testing
- Genotypic Resistance Test
- Where
- France
- Volunteers
- 79 people
- Sponsor
- Association de Recherche en Virologie et Dermatologie
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