Clinical trials: Genetic Mutation
Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)
The purpose of this study is to learn about the natural progression of DCM (dilated cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is weakened and the heart becomes enlarged.
- Trial name
- NCT05981092
- Where
- 15 sites in United States, Netherlands and 3 more
- Volunteers
- 35 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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
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.
Cystic Fibrosis
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03525444
- Testing
- VX-445/TEZ/IVA, IVA
- Where
- 115 sites in United States, Australia and 11 more
- Volunteers
- 405 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non-small Cell Lung Cancer, Brain Metastases
High Dose Icotinib With Sequential SRS For NSCLC Patients Harboring EGFR Mutation With Brain Metastases
This trail is designed to assess the efficacy and safety of high dose Iconitib combined with SRS for NSCLC patients harboring EGFR mutation with brain metastases.
- Trial name
- NCT02726568
- Testing
- Icotinib, SRS
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Advanced Cancer, Metastatic Cancer
A Study of ART0380 for the Treatment of Advanced or Metastatic Solid Tumors
This clinical trial is evaluating a drug called ART0380 in participants with advanced or metastatic solid tumors. The main goals of this study are to: Find the recommended dose of ART0380 that can be given safely to participants…
- Conditions
- Advanced Cancer, Metastatic Cancer, Ovarian Cancer, Primary Peritoneal Cancer
- Trial name
- NCT04657068
- Testing
- ART0380, Gemcitabine, Irinotecan
- Where
- 88 sites in United States, France and 2 more
- Volunteers
- 542 people
- Sponsor
- Artios Pharma Ltd
Carcinoma, Non-Small-Cell Lung
Study Evaluating Efficacy and Safety of Capmatinib in Combination With Osimertinib in Adult Subjects With Non-small Cell Lung Cancers as Second Line Therapy
This study aimed to evaluate the anticancer activity of capmatinib in combination with osimertinib compared to platinum-pemetrexed based doublet chemotherapy as second line treatment in patients with advanced or metastatic…
- Trial name
- GEOMETRY-E (NCT04816214)
- Testing
- Capmatinib, Osimertinib, Pemetrexed
- Where
- 4 sites in Japan, Singapore and 1 more
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals
Advanced NRAS-Mutant Melanoma, Advanced NRAS-Mutant Solid Tumors
A First-in-human Study of RLY-8161 in Advanced NRAS-Mutant Solid Tumors
This is a Phase 1/2 first-in-human, open-label multicenter study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary antitumor activity of RLY-8161, an NRAS-selective inhibitor…
- Conditions
- Advanced NRAS-Mutant Melanoma, Advanced NRAS-Mutant Solid Tumors, NRAS Mutation, NRAS G12D
- Trial name
- NCT07584226
- Testing
- RLY-8161
- Where
- 9 sites in United States
- Volunteers
- 180 people
- Sponsor
- Relay Therapeutics, Inc.
Cystic Fibrosis
ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation
The purpose of this program is to provide elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) to CF patients in critical need who are 6 to 11 years of age with at least one F508del mutation in response to unsolicited physician…
- Trial name
- NCT04702360
- Testing
- ELX/TEZ/IVA, IVA
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non Small Cell Lung Cancer
Epidemiologic Study of KRAS Mutation in Brazilian Patients With Advanced or Metastatic Non Small Cell Lung Cancer
The purpose of this study is to characterize the demographic and clinical aspects, and describe the frequency and type of KRAS mutation in a Brazilian population sample with advanced non-small cell lung cancer (NSCLC).
- Trial name
- NCT00956280
- Where
- 5 sites in Brazil
- Volunteers
- 100 people
- Sponsor
- AstraZeneca
AML, AML With Mutated NPM1
Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed/Refractory Acute Myeloid Leukemia
The safety, tolerability, and antileukemic response of ziftomenib in combination with standard of care treatments for patients with relapsed/refractory acute myeloid leukemia will be examined with the following agents: FLAG-IDA…
- Conditions
- AML, AML With Mutated NPM1, Hematologic Malignancy, KMT2Ar
- Trial name
- NCT06001788
- Testing
- Ziftomenib, Fludarabine, Idarubicin
- Where
- 45 sites in United States, Italy and 1 more
- Volunteers
- 171 people
- Sponsor
- Kura Oncology, Inc.
Activated PI3Kdelta Syndrome (APDS); PASLI Disease
Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI
This study is designed to provide long-term CDZ173 treatment, a selective PI3Kδ inhibitor, to the patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI who participated in the CCDZ173X2201 study or who were…
- Trial name
- NCT02859727
- Testing
- CDZ173
- Where
- 8 sites in United States, Belarus and 5 more
- Volunteers
- 37 people
- Sponsor
- Pharming Technologies B.V.
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy
The purpose of this study is to provide safety and efficacy data for voretigene neparvovec, administered as subretinal injection, in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy.
- Trial name
- NCT04516369
- Testing
- voretigene neparvovec
- Where
- Japan
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
IDH1-mutant Cholangiocarcinoma
Ivosidenib, Nivolumab, and Ipilimumab Combination in Previously Treated Subjects With Nonresectable or Metastatic IDH1 Mutant Cholangiocarcinoma
This is a Phase 1/2 study evaluating the safety, tolerability, and activity of ivosidenib in combination with immunotherapy in participants with nonresectable or metastatic cholangiocarcinoma.
- Trial name
- NCT05921760
- Testing
- Ivosidenib, Nivolumab, Ipilimumab
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Servier Bio-Innovation LLC
Cystic Fibrosis
A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein.
- Trial name
- NCT00458341
- Testing
- Ataluren
- Where
- 3 sites in Belgium, France
- Volunteers
- 30 people
- Sponsor
- PTC Therapeutics
HRAS Gene Mutation, HNSCC
Safety and Efficacy of Tipifarnib in Head and Neck Cancer With HRAS Mutations and Impact of HRAS on Response to Therapy
An international, multicenter, open-label, 2 cohort, non-comparative, pivotal study evaluating the efficacy of tipifarnib in HRAS mutant HNSCC (AIM-HN).
- Trial name
- AIM-HN/SEQ-HN (NCT03719690)
- Testing
- Tipifarnib, HRAS Detection Assay
- Where
- 102 sites in United States, Australia and 14 more
- Volunteers
- 296 people
- Sponsor
- Kura Oncology, Inc.
Melanoma
A Study Comparing 3 Study Medicines (Encorafenib, Binimetinib, Pembrolizumab) to 2 Study Medicines (Ipilimumab and Nivolumab) in Patients With Advanced Melanoma
The purpose of this study is to learn about the effects of 3 study medicines (encorafenib, binimetinib, pembrolizumab) compared to 2 study medicines (ipilimumab and nivolumab) given for the treatment of melanoma.
- Trial name
- PORTSIDE (NCT05926960)
- Testing
- encorafenib, binimetinib, pembrolizumab
- Where
- 24 sites in Germany, Italy and 3 more
- Volunteers
- 38 people
- Sponsor
- Pfizer
Advanced Solid Tumor, BRAF Gene Mutation
Study of Ulixertinib for Patients With Advanced Malignancies Harboring MEK or Atypical BRAF Alterations
This BVD-523-ABC study builds on the safety and clinical activity experience of previous studies that have evaluated ulixertinib as a novel targeted cancer treatment in cohorts of patients with specific genetic alterations and…
- Conditions
- Advanced Solid Tumor, BRAF Gene Mutation, BRAF Gene Alteration, MEK Mutation
- Trial name
- NCT04488003
- Testing
- Ulixertinib, Physician's Choice
- Where
- 22 sites in United States
- Volunteers
- 104 people
- Sponsor
- BioMed Valley Discoveries, Inc
Pulmonary Cancer
First-line Treatment for Adenocarcinoma Patients With Epidermal Growth Factor Receptor (EGFR) Mutation
The purpose of this study is to evaluate the overall objective tumor response rate (ORR) of Gefitinib.
- Trial name
- NCT00344773
- Testing
- Gefitinib
- Where
- 2 sites in South Korea
- Volunteers
- 46 people
- Sponsor
- AstraZeneca
NSCLC
Phase I Study of HSK40118 in NSCLC Patients With EGFR Mutation
This is a phase I, open-label, dose-escalation and expansion study to evaluate the safety, tolerability, PK and PD of HSK40118 when given orally in patients with active EGFR mutation locally advanced or metastatic non-small cell…
- Trial name
- NCT06050980
- Testing
- HSK40118
- Where
- 12 sites in China
- Volunteers
- 220 people
- Sponsor
- Haisco Pharmaceutical Group Co., Ltd.
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