Clinical trials: Genetic Mutation
Pro-opiomelanocortin (POMC) Deficiency Obesity
Setmelanotide for the Treatment of Early-Onset Pro-Opiomelanocortin (POMC) Deficiency Obesity
To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with pro-opiomelanocortin (POMC) deficiency or proprotein convertase subtilisin/kexin type…
- Trial name
- NCT02896192
- Testing
- Setmelanotide
- Where
- 7 sites in United States, Belgium and 5 more
- Volunteers
- 15 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Carcinoma, Non-Small-Cell Lung With EGFR Mutation Positive
Phase I, Study in Chinese NSCLC Patients
A Phase I, Open-Label, Two Parts Study to Assess the Safety, Tolerability,Pharmacokinetics and Preliminary Anti-tumour Activity of AZD9291 in Chinese Patients with Advanced Non-Small Cell Lung Cancer who have Progressed Following…
- Trial name
- AURAChinaPK (NCT02529995)
- Testing
- AZD9291 40 mg, AZD9291 80 mg
- Where
- 2 sites in China
- Volunteers
- 31 people
- Sponsor
- AstraZeneca
Solid Tumor, Breast Tumor
Niraparib in the Treatment of Patients With Advanced PALB2 Mutated Tumors
The purpose of this study is to further evaluate the efficacy and safety of niraparib in patients with locally advanced or metastatic solid tumors and a pathogenic or likely pathogenic tumor PALB2 (tPALB2) mutation.
- Conditions
- Solid Tumor, Breast Tumor, Colon Tumor, Malignant, Lung Tumor
- Trial name
- PAVO (NCT05169437)
- Testing
- Niraparib
- Where
- 80 sites in United States
- Volunteers
- 22 people
- Sponsor
- Tempus AI
Newly Diagnosed, Advanced Ovarian Cancer
Olaparib Maintenance Monotherapy in Patients With BRCA Mutated Ovarian Cancer Following First Line Platinum Based Chemotherapy.
Olaparib Monotherapy in Patients with BRCA Mutated Ovarian Cancer following First Line Platinum Based Chemotherapy.
- Conditions
- Newly Diagnosed, Advanced Ovarian Cancer, FIGO Stage III-IV, BRCA Mutation
- Trial name
- SOLO-1 (NCT01844986)
- Testing
- Olaparib 300mg tablets
- Where
- 177 sites in United States, Australia and 13 more
- Volunteers
- 450 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR Mutation
The study will evaluate the impact of smart adherence technology for monitoring on lumacaftor/ivacaftor (LUM/IVA) adherence rates among subjects 16 years of age and older with Cystic Fibrosis (CF) who are homozygous for the…
- Trial name
- NCT02823470
- Testing
- LUM/IVA, activated smart device, de-activated smart device
- Where
- 11 sites in United States, Canada
- Volunteers
- 24 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non Small Cell Lung Cancer (Stage III)
A Global Study to Assess the Effects of Osimertinib Following Chemoradiation in Patients With Stage III Unresectable Non-small Cell Lung Cancer (LAURA)
A global study to assess the efficacy and safety of osimertinib following chemoradiation in patients with stage III unresectable Epidermal Growth Factor Receptor Mutation Positive non-small cell lung cancer
- Trial name
- LAURA (NCT03521154)
- Testing
- Osimertinib 80mg/40mg
- Where
- 118 sites in United States, Argentina and 15 more
- Volunteers
- 216 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)…
- Trial name
- STRIVE (NCT00909532)
- Testing
- Ivacaftor
- Where
- 65 sites in United States, Australia and 6 more
- Volunteers
- 167 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Lung Cancer
T790M Plasma Testing Methodology Comparison and Clinical Validation
The aim of this study is to evaluate concordance of T790M mutation plasma testing between the Cobas test and each of other platforms: Super-ARMS, digital PCR or NGS.
- Trial name
- ADELOS (NCT02997501)
- Testing
- T790M+ Testing, Baseline Visit Blood & Urine Testing, Baseline ECG
- Where
- 7 sites in China
- Volunteers
- 256 people
- Sponsor
- AstraZeneca
Cholangiocarcinoma Non-resectable, Cholangiocarcinoma Metastatic
A Phase 2 Study of Ivosidenib in Previously Treated Japanese Subjects With Nonresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation
This study will enroll participants with nonresectable or metastatic cholangiocarcinoma with an Isocitrate dehydrogenase protein, 1 (IDH1) mutation, who have previously received at least 1, but no more than 2, prior regimens for…
- Trial name
- NCT06081829
- Testing
- Ivosidenib
- Where
- 7 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Servier
Carcinoma, Non-Small-Cell Lung
A Study of Dabrafenib in Combination With Trametinib in Chinese Patients With BRAF V600E Mutant Metastatic NSCLC
This was a single-arm, open label, multicenter phase II, study of dabrafenib in combination with trametinib in Chinese participants with BRAF V600E mutation positive, stage IV NSCLC (American joint committee on cancer staging 8th…
- Trial name
- NCT04452877
- Testing
- Dabrafenib, Trametinib
- Where
- 8 sites in China
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Solid Tumor, Lymphoma
Precision Medicine for Patients With Identified Actionable Mutations
The goal of the current pragmatic trial is to evaluate the impact of a simple method of selecting a treatment approach for identified mutations on participants' progression free survival (PFS).
- Conditions
- Solid Tumor, Lymphoma, Multiple Myeloma, Malignant Neoplasm
- Trial name
- NCT04111107
- Testing
- Investigational Agent, Supportive Care Regimens, Next Gen Sequencing Report
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Wake Forest University Health Sciences
Cystic Fibrosis
Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation in the cystic fibrosis transmembrane conductance…
- Trial name
- NCT00457821
- Testing
- Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg, Ivacaftor 150 mg or 250 mg
- Where
- 15 sites in United States, Canada and 1 more
- Volunteers
- 39 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
RET-altered Non Small Cell Lung Cancer, Medullary Thyroid Cancer
Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors
This is a Phase 1/2, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of pralsetinib (BLU-667)…
- Conditions
- RET-altered Non Small Cell Lung Cancer, Medullary Thyroid Cancer, RET-altered Papillary Thyroid Cancer, RET-altered Colon Cancer
- Trial name
- ARROW (NCT03037385)
- Testing
- pralsetinib (BLU-667)
- Where
- 71 sites in United States, Belgium and 11 more
- Volunteers
- 590 people
- Sponsor
- Hoffmann-La Roche
Carcinoma, Non-Small-Cell Lung
A Study of Combination Amivantamab and Carboplatin-Pemetrexed Therapy, Compared With Carboplatin-Pemetrexed, in Participants With Advanced or Metastatic Non-Small Cell Lung Cancer Characterized by Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertions
The purpose of this study is to compare the efficacy, as demonstrated by progression-free survival (PFS), in participants treated with amivantamab in combination with chemotherapy, versus chemotherapy alone in participants with…
- Trial name
- PAPILLON (NCT04538664)
- Testing
- Amivantamab, Pemetrexed, Carboplatin
- Where
- 232 sites in United States, Australia and 24 more
- Volunteers
- 308 people
- Sponsor
- Janssen Research & Development, LLC
Chronic Myelogenous Leukemia (CML), Metastatic Gastrointestinal Stromal Tumors (GIST)
Study to Allow Access to Nilotinib for Patients Who Are on Nilotinib Treatment in a Novartis-sponsored Study
The purpose of this study was to allow continued use of nilotinib in patients who were on nilotinib treatment in a Novartis-sponsored, Oncology Clinical Development \& Medical Affairs (CD\&MA) study and were benefiting from the…
- Conditions
- Chronic Myelogenous Leukemia (CML), Metastatic Gastrointestinal Stromal Tumors (GIST), Acute Lymphoblastic Leukemia (ALL), Receptor Tyrosine Kinase (KIT) Mutated Melanoma
- Trial name
- NCT01735955
- Testing
- Nilotinib
- Where
- 33 sites in United States, Austria and 14 more
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Leukemia, Leukemia, Myeloid
Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation
This is a phase 1b/2, open-label, single arm study to evaluate if enasidenib is safe and effective in improving anemia and decreasing transfusion needs in subjects diagnosed with lower risk myelodysplastic syndrome (MDS) or…
- Conditions
- Leukemia, Leukemia, Myeloid, Monocytic Leukemia
- Trial name
- NCT05282459
- Testing
- Enasidenib mesylat dose escalation
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Tian Yi Zhang
Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Neutrophilic Leukemia
Ruxolitinib Phosphate in Treating Patients With Chronic Neutrophilic Leukemia or Atypical Chronic Myeloid Leukemia
This phase II trial studies how well ruxolitinib phosphate works in treating patients with chronic neutrophilic leukemia (CNL) or atypical chronic myeloid leukemia (aCML).
- Trial name
- NCT02092324
- Testing
- Laboratory Biomarker Analysis, Quality-of-Life Assessment, Questionnaire Administration
- Where
- 7 sites in United States
- Volunteers
- 51 people
- Sponsor
- OHSU Knight Cancer Institute
Small-cell Lung Cancer
Berzosertib + Topotecan in Relapsed Platinum-Resistant Small-Cell Lung Cancer (DDRiver SCLC 250)
The main purpose of this study is to assess efficacy, safety, tolerability and pharmacokinetics (PK) of Berzosertib in combination with Topotecan in participants with relapsed, platinum-resistant small-cell lung cancer (SCLC).
- Trial name
- NCT04768296
- Testing
- Berzosertib, Topotecan
- Where
- 51 sites in United States, Belgium and 5 more
- Volunteers
- 76 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Cystic Fibrosis
A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).
- Trial name
- NCT05033080
- Testing
- VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA
- Where
- 139 sites in United States, Australia and 10 more
- Volunteers
- 435 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Melanoma
GSK1120212 vs Chemotherapy in Advanced or Metastatic BRAF V600E/K Mutation-positive Melanoma
This is a two-arm, open-label, randomized Phase III study comparing single agent GSK1120212 to chemotherapy (either dacarbazine or paclitaxel) in subjects with Stage IIIc or Stage IV malignant cutaneous melanoma.
- Trial name
- NCT01245062
- Testing
- GSK1120212, Chemotherapy
- Where
- 112 sites in United States, Argentina and 17 more
- Volunteers
- 322 people
- Sponsor
- GlaxoSmithKline