Clinical trials: Mutation Abnormality
Inherited Retinal Dystrophy Associated With RPE65 Mutations
Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.
- Trial name
- NCT06196827
- Testing
- LX101
- Where
- 2 sites in China
- Volunteers
- 9 people
- Sponsor
- Innostellar Biotherapeutics Co.,Ltd
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.
Melanoma
MEK Inhibitor FCN-159 To Treat Advanced Melanoma With NRAS-aberrant (Ia) and NRAS-mutant (Ib)or NF1-mutant(1b)
Melanoma is one of the most common cutaneous cancers worldwide. Activating mutations in RAS oncogenes are found in a third of all human cancers and NRAS mutations are found in 15%-20% of melanomas.
- Trial name
- NCT03932253
- Testing
- FCN-159
- Where
- China
- Volunteers
- 79 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Farber Disease, Farber's Disease
Farber Disease Natural History Study
The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber…
- Conditions
- Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency
- Trial name
- NCT03233841
- Where
- 15 sites in United States, Argentina and 7 more
- Volunteers
- 45 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Melanoma
Ph I Ipilimumab Vemurafenib Combo in Patients With v-Raf Murine Sarcoma Viral Oncogene Homolog B1 (BRAF)
Treatment of subjects who have metastatic melanoma that expresses an activated mutant form of the BRAF oncogene (V600E) with a combination of the specific BRAF inhibitor, Vemurafenib, and the Cytotoxic T Lymphocyte Antigen 4…
- Trial name
- NCT01400451
- Testing
- Ipilimumab (BMS-734016), Vemurafenib
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Bristol-Myers Squibb
Non-small Cell Lung Cancer
Phase II of Neoadjuvant and Adjuvant Capmatinib in NSCLC
This study was planned to determine if neoadjuvant capmatinib could improve the major pathological response (MPR) in patients with Stage IB-IIIA, N2 and selected IIIB (T3N2 or T4N2) lung cancers with Mesenchymal Epithelial…
- Trial name
- Geometry-N (NCT04926831)
- Testing
- capmatinib
- Where
- 6 sites in United States
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Non Small Cell Lung Cancer
XZP-5809-TT1 Tablets in Patients With T790M Mutation-positive Locally Advanced or Metastatic Non-small Cell Lung Cancer
The trial is divided into two parts, one is dose escalation phase, the second one is dose expansion phase. For dose escalation phase, the main purpose is to evaluate safety and tolerability of XZP-5809-TT1 tablets after treatment…
- Trial name
- NCT04622072
- Testing
- XZP-5809-TT1 Tablet
- Where
- 5 sites in China
- Volunteers
- 21 people
- Sponsor
- Sihuan Pharmaceutical Holdings Group Ltd.
Myeloproliferative Neoplasms
A Study to Evaluate INCA033989 Administered as a Monotherapy or in Combination With Ruxolitinib in Participants With Myeloproliferative Neoplasms
This study is being conducted to evaluate the safety, tolerability, dose-limiting toxicity (DLT) and determine the maximum tolerated dose (MTD) and/or recommended dose(s) for expansion (RDE) of INCA033989 administered as a…
- Trial name
- NCT06034002
- Testing
- INCA033989, Ruxolitinib
- Where
- 13 sites in United States
- Volunteers
- 290 people
- Sponsor
- Incyte Corporation
Cystic Fibrosis
Study of GLPG1837 in Subjects With Cystic Fibrosis (G551D Mutation)
32 cystic fibrosis patients with the G551D mutation will be treated for 4 weeks, consisting of three consecutive treatment periods: two 1-week periods followed by one 2-week period, evaluating one dose of GLPG1837 each.
- Trial name
- SAPHIRA1 (NCT02707562)
- Testing
- GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3
- Where
- 16 sites in Australia, Czechia and 3 more
- Volunteers
- 26 people
- Sponsor
- Lakefront Biotherapeutics NV
WHIM Syndrome
A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome
This natural history study is a prospective and retrospective, observational study of WHIM patients. WHIM syndrome is a rare, genetic, primary immunodeficiency disorder (a disorder in which the body's immune system does not…
- Trial name
- NCT03087370
- Testing
- No intervention
- Sponsor
- X4 Pharmaceuticals
Colorectal Neoplasms, Advanced Metastatic Colorectal Cancer
Biomarker Analysis for Patients With Metastatic Colorectal Cancer (MCC)
Patients are being asked to participate in this study who have colorectal cancer that has come back after initial treatment. The investigators want to improve treatment in patients with this disease.
- Trial name
- NCT01909362
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- US Oncology Research
Metastatic Melanoma
HD IL-2 + Vemurafenib in Patients With BRAF Mutation Positive Metastatic Melanoma
This is a research study to evaluate treatment of metastatic melanoma patients with a combination of drugs. The combination being studied is vemurafenib (also known as Zelboraf®) and High Dose Interleukin-2 (abbreviated as HD…
- Trial name
- PROCLIVITY01 (NCT01683188)
- Testing
- vemurafenib + HD IL-2
- Where
- 21 sites in United States
- Volunteers
- 53 people
- Sponsor
- Clinigen, Inc.
FLT3-ITD Mutation, FLT3/TKD Mutation
Individual Patient Compassionate Use of Crenolanib
Compassionate use of crenolanib for patients with serious life-threatening illness that have exhausted all available therapies used to treat the disease, with no other viable therapy options, who is not eligible for clinical…
- Conditions
- FLT3-ITD Mutation, FLT3/TKD Mutation, PDGFR-Alpha D842V, PDGFRA Gene Amplification
- Trial name
- NCT03620318
- Testing
- Crenolanib besylate
- Where
- Italy
- Sponsor
- Arog Pharmaceuticals, Inc.
Cystic Fibrosis
GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis
This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d.
- Trial name
- PELICAN (NCT03474042)
- Testing
- GLPG2737
- Where
- 9 sites in Germany
- Volunteers
- 22 people
- Sponsor
- Lakefront Biotherapeutics NV
Advanced Non-squamous Non-small-cell Lung Cancer, Metastatic Non-squamous Non Small Cell Lung Cancer
JAB-21822 Combined With JAB-3312 Compared SOC in the First Line for Treatment of Advanced Non-small Cell Lung Cancer With KRAS p.G12C Mutation
This Phase 3 study will evaluate the efficacy of JAB-21822+JAB-3312 versus tislelizumab (PD-1 Ab) combined with pemetrexed+carboplatin as the first line treatment in subjects with KRAS G12C mutated locally advanced or metastatic…
- Trial name
- NCT06416410
- Testing
- JAB-21822, Tislelizumab, JAB-3312
- Where
- 37 sites in China
- Volunteers
- 392 people
- Sponsor
- Allist Pharmaceuticals, Inc.
Carcinoma, Non-Small-Cell Lung
Survey on the Treatment Reality of Patients With Epidermal Growth Factor Receptor (EGFR) Mutation-positive Non-small Cell Lung Cancer(NSCLC)
According to the recent report using EGFR tyrosine kinase inhibitors (EGFR-TKI), there is all over survival of the EGFR gene mutation-positive NSCLC in a tendency to extension.
- Trial name
- NCT02475720
- Where
- Japan
- Volunteers
- 2,000 people
- Sponsor
- AstraZeneca
Non-small Cell Lung Cancer
Icotinib as Adjuvant Therapy Compared With Standard Chemotherapy in Stage II-IIIA NSCLC With EGFR-mutation
Adjuvant therapy has been proved effective in treating earlier stage or less advanced non-small-cell lung cancer. This study is designed to evaluate the efficacy of icotinib as adjuvant therapy in treating stage II-IIIA non-small…
- Trial name
- EVIDENCE (NCT02448797)
- Testing
- Icotinib, Chemotherapy
- Where
- 8 sites in China
- Volunteers
- 320 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Low-grade Glioma
Expanded Access Program (EAP) for Tovorafenib (DAY101) in RAF-Altered, Relapsed or Refractory Low-Grade Glioma
The DAY101-EAP is a multicenter, open-label, expanded access treatment protocol designed to provide access to tovorafenib (DAY101) for eligible patients.
- Trial name
- NCT05760586
- Testing
- Tovorafenib
- Sponsor
- Day One Biopharmaceuticals, Inc.
Clonal Mast Cell Disease, KIT D816V Mutation
Screening Study for KIT D816V Mutated Mast Cell Disease in Select Populations
This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.
- Conditions
- Clonal Mast Cell Disease, KIT D816V Mutation, Suspected KITD816V Mutated Clonal Mast Cell Disease
- Trial name
- POLARIS (NCT07143669)
- Testing
- Screening
- Where
- 26 sites in United States, Belgium
- Volunteers
- 750 people
- Sponsor
- Blueprint Medicines Corporation
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
Acalabrutinib in Patients With Relapsed/Refractory and Treatment naïve Deletion 17p CLL/SLL
This study is to determine the response to acalabrutinib in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
- Trial name
- NCT02337829
- Testing
- Acalabrutinib (Arm A), Acalabrutinib (Arm B)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Acerta Pharma BV