Clinical trials: Gene Mutations
AMN, AMN Gene Mutation
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy…
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- PROPEL (NCT05394064)
- Testing
- SBT101 Treatment
- Where
- 2 sites in United States, Netherlands
- Volunteers
- 9 people
- Sponsor
- SwanBio Therapeutics, Inc.
Extrahepatic Bile Duct Cancer, Gallbladder Cancer
Anonymous Testing of Pathology Specimens for BRCA Mutations in Ashkenazi Jewish Individuals Who Have Cancer
The intent of the proposed study is to describe the prevalence of the most common recurring mutations in BRCA1 and BRCA2, blmAsh , and the A636P MSH2 mutation among Ashkenazi Jewish individuals with a variety of cancer diagnoses.
- Conditions
- Extrahepatic Bile Duct Cancer, Gallbladder Cancer, Gastric Cancer, Lung Cancer
- Trial name
- NCT00588263
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Advanced Solid Tumors
MEK162 and RAF265 in Adult Patients With Advanced Solid Tumors Harboring RAS or BRAFV600E Mutations
This is a multi- center, open-label, dose finding, Phase Ib study to be conducted in two stages: a dose escalation part to determine the maximum tolerated dose (MTD) safety and tolerability of concurrent administration of MEK162…
- Trial name
- NCT01352273
- Testing
- MEK162 + RAF265
- Where
- 8 sites in United States, Canada and 3 more
- Volunteers
- 69 people
- Sponsor
- Array Biopharma, now a wholly owned subsidiary of Pfizer
Non Small Cell Lung Cancer
Trametinib Plus Anlotinib in Non-G12C KRAS-Mutant NSCLC Patients
This is a Phase I exploratory study. The study is divided into two parts (A/B).In part A, the primary endpoint is the determination of the recommended phase 2 dose (RP2D).
- Trial name
- NCT04967079
- Testing
- Trametinib, Anlotinib
- Where
- China
- Volunteers
- 33 people
- Sponsor
- Shanghai Chest Hospital
Clonal Cytopenia of Undetermined Significance
Ivosidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH1
This is an open-label, multicenter study exploring the efficacy of ivosidenib in patients with clonal cytopenia of undetermined significance (CCUS) with mutations in IDH1.
- Trial name
- NCT05030441
- Testing
- Ivosidenib
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Washington University School of Medicine
KRAS Gene Mutation, Recurrent Lung Non-Small Cell Carcinoma
Trametinib and Docetaxel in Treating Patients With Recurrent or Stage IV KRAS Mutation Positive Non-small Cell Lung Cancer
This phase II trial studies how well trametinib and docetaxel work in treating patients with stage IV KRAS mutation positive non-small cell lung cancer or cancer that has come back.
- Conditions
- KRAS Gene Mutation, Recurrent Lung Non-Small Cell Carcinoma, Stage IV Lung Non-Small Cell Cancer AJCC v7
- Trial name
- NCT02642042
- Testing
- Docetaxel, Laboratory Biomarker Analysis, Trametinib
- Where
- 596 sites in United States
- Volunteers
- 60 people
- Sponsor
- National Cancer Institute (NCI)
Retinitis Pigmentosa
A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
The purpose of this first-in-human study is to explore the maximum tolerated dose (MTD) of CPK850 as determined by the single ascending dose ranging portion of the study.
- Trial name
- NCT03374657
- Testing
- CPK850
- Where
- Sweden
- Volunteers
- 12 people
- Sponsor
- Novartis Pharmaceuticals
Newly Diagnosed AML With FLT3 Activating Mutations
A Safety and Tolerability Study of Crenolanib in Combination With Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia Patients With FLT3 Mutations
This pilot study is designed to evaluate the safety and tolerability of oral crenolanib besylate given sequentially during standard induction and consolidation chemotherapy in patients with newly diagnosed AML with FLT3…
- Trial name
- NCT02283177
- Testing
- crenolanib, cytarabine, daunorubicin
- Where
- 9 sites in United States
- Volunteers
- 44 people
- Sponsor
- Arog Pharmaceuticals, Inc.
Locally Advanced or Metastatic Non-Small Cell Lung Cancer, EGFR Exon20 Insertion Mutations
Efficacy and Safety of JMT101 Combined With Osimertinib in Patients With Non-Small Cell Lung Cancer
This study is a phase II, open label, multi-center study to evaluate the efficacy and safety of JMT101 combined with Osimertinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring EGFR…
- Trial name
- NCT05132777
- Testing
- JMT101, Osimertinib Mesylate Tablets
- Volunteers
- 155 people
- Sponsor
- Shanghai JMT-Bio Inc.
Locally Advanced, Metastatic, or Recurrent Non-small Cell Lung Cancer (NSCLC) Harboring Epidermal Growth Factor Receptor (EGFR) Mutations
JS212 Plus JS111 in EGFR-Mutant Advanced NSCLC: A Phase II Study
This is a Phase II study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of JS212 in combination with JS111 in patients with advanced or metastatic non-small cell lung cancer (NSCLC) harboring…
- Trial name
- NCT07518160
- Testing
- JS212 for Injection＋JS111 capsules (AP-L1898)
- Where
- 2 sites in China
- Volunteers
- 35 people
- Sponsor
- Shanghai Junshi Bioscience Co., Ltd.
Non-small Cell Lung Cancer, EGFR Gene Mutation
First Line Treatment in EGFR Mutation Positive Advanced NSCLC Patients With Central Nervous System (CNS) Metastases
The first-line treatment with single agent AZD3759 results in superior Progression Free Survival (PFS) compared to Standard of Care (SoC) Epidermal Growth Factor Receptor Tyrosine Kinase Inhibitors (EGFR-TKI), in patients with…
- Conditions
- Non-small Cell Lung Cancer, EGFR Gene Mutation, Brain Metastases
- Trial name
- BM (NCT03653546)
- Testing
- AZD3759, Erlotinib, Gefitinib
- Where
- 58 sites in China, Singapore and 2 more
- Volunteers
- 492 people
- Sponsor
- Alpha Biopharma (Jiangsu) Co., Ltd.
Gene Mutation-Related Cancer, Traditional Chinese Medicine
Study on the Correlation Between Gene Mutation and TCM Syndrome Types in Metastatic Colorectal Cancer
To explore the correlation between gene mutations of metastatic colorectal cancer and TCM syndrome types based on Second-generation sequencing technology.
- Trial name
- NCT04661046
- Testing
- Traditional Chinese Medicine
- Volunteers
- 100 people
- Sponsor
- Shanghai University of Traditional Chinese Medicine
DFNB9, Cochlear Implantation
Safety, Tolerability, Efficacy of EA0010 in OTOF Patients With CI
This study will evaluate the safety, tolerability, and efficacy of EA0010 injection in patients with OTOF-related hearing loss who have already undergone cochlear implantation.
- Trial name
- NCT07752888
- Testing
- EA0010 Injection
- Where
- China
- Volunteers
- 1 people
- Sponsor
- Shanghai EmayGene Technology Co., Ltd
EGFR-TKI-naïve Patients With NSCLC Harboring EGFR Activating Mutations
A Study of ASP8273 in Epidermal Growth Factor Receptor (EGFR) Tyrosine Kinase Inhibitor-Naïve Patients With Non-Small Cell Lung Cancer Harboring EGFR Mutations
The purpose of this study is to determine the safety, the antitumor activity and the pharmacokinetics of ASP8273 in EGFR tyrosine kinase inhibitor (EGFR-TKI)-naïve patients with non-small cell lung cancer (NSCLC) harboring EGFR…
- Trial name
- NCT02500927
- Testing
- ASP8273 Capsules, ASP8273 Capsules A
- Where
- 11 sites in Japan
- Volunteers
- 31 people
- Sponsor
- Astellas Pharma Inc
HIV Infections
Development of Resistance Mutations in Pregnant HIV-positive (+) Women Following Perinatal Antiretroviral Therapy in Israel
It has been shown that treatment of HIV-positive women with antiretroviral medication during pregnancy reduces the risk of HIV transmission to the child.
- Trial name
- NCT00197366
- Where
- 5 sites in Israel
- Volunteers
- 50 people
- Sponsor
- Hadassah Medical Organization
Epidermolysis Bullosa
Topical Gentamicin Nonsense Suppression Therapy of EB
The overall purpose of this study is to address whether topical gentamicin therapy is an effective and feasible treatment. Specifically, we will investigate the effect of non-intensive treatment (once daily or every other day) on…
- Trial name
- GENTELBULL (NCT04644627)
- Testing
- Gentamicin Sulfate
- Where
- Norway
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Oslo University Hospital
Breast Cancer, Hereditary Breast and Ovarian Cancer
Triple Negative Breast Cancer and Germline Hereditary Breast and Ovarian Cancer Mutation Carrier Registry
PROGECT is a registry for patients with Triple Negative breast cancer (TNBC) or patients who have an identified germline mutations (such as a mutation on the BRCA1 or BRCA2 genes).
- Trial name
- PROGECT (NCT02302742)
- Where
- 11 sites in United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
HIV, HIV Antiretroviral Therapy (ART) Adherence
Selection of Resistant Mutations to Dolutegravir in PLWH Treated in Mozambique
The HIV infection not fully controlled, despite being under antiretroviral treatment, could make virus resistance against the antiretroviral treatments, making hardest the well control of HIV infection.
- Trial name
- REDOCH (NCT06607588)
- Testing
- Not applicable- observational study
- Where
- Mozambique
- Volunteers
- 200 people
- Sponsor
- Fundación FLS de Lucha Contra el Sida, las Enfermedades Infecciosas y la Promoción de la Salud y la Ciencia
Neuroaxonal Dystrophy, Atypical
Phenotypic Description of Patients With Atypical Clinical Forms of PLA2G6 Mutations
Mutations in the PLA2G6 gene are well known in the classical phenotype called infantile neuro-axonal dystrophy (INAD), a severe neurodegenerative disease starting in infancy with homogeneous clinical, radiological…
- Trial name
- ATYPICPLA2G6 (NCT05440994)
- Where
- 7 sites in France
- Volunteers
- 20 people
- Sponsor
- University Hospital, Clermont-Ferrand
Patients With Parkinson's Disease (PD) Caused by the p.Gly2019Ser (G2019S) Pathogenic Mutation of the Leucine-Rich Repeat Kinase 2 (LRRK2) Gene
A Natural History Study of Patients With G2019S LRRK2 Parkinson's Disease
To characterize using a participant centered decentralized (at home) study featuring wearable technology and telemedicine to study disease change over time in patients with PD caused by the G2019S mutation in the LRRK2 gene and…
- Trial name
- NCT05349019
- Testing
- Observational
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Escape Bio, Inc.
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