Clinical trials: Genetic Mutation
Common Variable Immunodeficiency (CVID), APDS / PASLI
Study of Efficacy of CDZ173 in Patients With APDS/PASLI
This study was designed to explore CDZ173, a selective PI3Kδ inhibitor, in patients with genetically activated PI3Kδ, i.e., patients with Activated phosphoinositide 3-kinase delta syndrome/ p110δ-activating mutation causing…
- Trial name
- NCT02435173
- Testing
- CDZ173
- Where
- 10 sites in United States, Belarus and 7 more
- Volunteers
- 37 people
- Sponsor
- Novartis Pharmaceuticals
Genetic Disease, Nonsense Mutation
Phase 1 Study of ELX-02 in Healthy Adults
Phase 1 Single Ascending Dose Study in Normal Healthy Volunteers
- Trial name
- NCT03292302
- Testing
- ELX-02
- Where
- Belgium
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Cystic Fibrosis
A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation
To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation
- Trial name
- NCT02514473
- Testing
- VX-809, VX-770
- Where
- 53 sites in United States, Australia and 7 more
- Volunteers
- 206 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Glioblastoma
CM93 Treatment in Subjects With Epidermal Growth Factor Receptor (EGFR)-Modified Recurrent Glioblastoma (rGBM)
This is a first-in-human study of CM93, an oral investigational drug, in adults with Epidermal Growth Factor Receptor-modified glioblastoma.
- Trial name
- NCT04933422
- Testing
- CM93
- Volunteers
- 72 people
- Sponsor
- Crimson Biopharm Inc.
Ovarian Cancer, TNBC, SCLC, Other Solid Tumours
Safety, Tolerance, PK, and Anti-tumour Activity of AZD1775 Monotherapy in Patients With Advanced Solid Tumours
This is an open-label, multi-centre, Phase Ib study of AZD1775 designed to assess the safety, tolerability, pharmacokinetics, and anti-tumour activity of AZD1775 monotherapy in patients with advanced solid tumours.
- Trial name
- NCT02482311
- Testing
- AZD 1775
- Where
- 16 sites in United States, Canada
- Volunteers
- 92 people
- Sponsor
- AstraZeneca
BRCA1 Mutation, BRCA2 Mutation
Pre-Salpingo-Oophorectomy Pilot Study of MAKO 7 Device Performance
This study evaluates the ability of the MAKO 7 device to collect various cells
- Conditions
- BRCA1 Mutation, BRCA2 Mutation, Atypia Suspicious for Malignancy
- Trial name
- NCT02974842
- Testing
- MAKO 7
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- nVision Medical
Stargardt Disease, Stargardt Macular Degeneration
A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)
This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1).
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
- Trial name
- ASTRA (NCT06942572)
- Testing
- SB-007
- Where
- 5 sites in United States
- Volunteers
- 57 people
- Sponsor
- Splice Bio
Congenital Myasthenic Syndrome
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7…
- Trial name
- NCT06078553
- Where
- 33 sites in United States, Austria and 10 more
- Volunteers
- 100 people
- Sponsor
- argenx
Advanced Solid Tumors With an Alteration of the PIK3CA Gene, Estrogen Receptor Positive Breast Cancer
A Study of BYL719 in Adult Patients With Advanced Solid Malignancies, Whose Tumors Have an Alteration of the PIK3CA Gene
This is a first-in-man trial, in which BYL719 will be administered to adult patients with advanced solid tumors, whose tumors have an alteration of the PIK3CA gene and whose disease has progressed despite standard therapy or for…
- Trial name
- NCT01219699
- Testing
- BYL719, Fulvestrant
- Where
- 11 sites in United States, Germany and 3 more
- Volunteers
- 221 people
- Sponsor
- Novartis Pharmaceuticals
T790M Positive NSCLC Patients
Effect of Osimertinib in Ethnic Chinese With EGFR and T790M Mutated NSCLC
To assess the effectiveness and safety of osimertinib treatment in a real world setting.
- Trial name
- NCT03133234
- Testing
- osimertinib
- Where
- China
- Volunteers
- 47 people
- Sponsor
- AstraZeneca
Cancer, Colorectal Cancer
FOG-001 in Locally Advanced or Metastatic Solid Tumors
The goal of this clinical trial is to determine if FOG-001 is safe and effective in participants with locally advanced or metastatic solid tumors or in participants with familial adenomatous polyposis (FAP).
- Conditions
- Cancer, Colorectal Cancer, Solid Tumor, Locally Advanced Solid Tumor
- Trial name
- NCT05919264
- Testing
- FOG-001, mFOLFOX-6, Nivolumab
- Where
- 33 sites in United States, Australia
- Volunteers
- 619 people
- Sponsor
- Parabilis Medicines, Inc.
HER2 Mutation-Related Tumors, HER2
A Phase 1/1b Study of IAM1363 in HER2 Cancers
This is a Phase 1/1b open-label, multi-center dose escalation and dose optimization study designed to evaluate the safety and preliminary efficacy of IAM1363 in participants with advanced cancers that harbor HER2 alterations.
- Conditions
- HER2 Mutation-Related Tumors, HER2, HER2-positive Breast Cancer, HER2 + Breast Cancer
- Trial name
- NCT06253871
- Testing
- IAM1363
- Where
- 53 sites in United States, France and 6 more
- Volunteers
- 383 people
- Sponsor
- Iambic Therapeutics, Inc
Urothelial Carcinoma, Bladder Cancer
Diagnostic Evaluation of Urine DNA Methylation/Somatic Mutation Profiling for the Detection of Urothelial Carcinoma
Clinical trial to evaluate the performance characteristics(sensitivity and specificity) of AnchorDx's urine DNA methylation/somatic mutation profiling assay for detecting urothelial carcinoma compared to pathology in patients.
- Trial name
- NCT04314245
- Testing
- Active Comparator: Pathology, Active Comparator: Clinical Diagnosis, Genetic test: AnchorDx's urine DNA methylation/somatic mutation profiling assay
- Where
- China
- Volunteers
- 1,170 people, healthy volunteers welcome
- Sponsor
- AnchorDx Medical Co., Ltd.
Small Cell Lung Carcinoma
Managed Access Program Cohort Treatment Plan CTMT212X2002I to Provide Access to Trametinib and Dabrafenib Combination Therapy for Patients With BRAF V600 Mutation-positive Advanced Non-Small Cell Lung Cancer
The purpose of this Managed Access Program (MAP) Cohort Treatment Plan is to provide guidance to the Physician for the treatment and monitoring of eligible patients diagnosed with BRAF V600E/K activating mutation-positive…
- Trial name
- NCT04507919
- Testing
- Dabrafenib, Trametininb
- Sponsor
- Novartis Pharmaceuticals
Non-small-cell Lung Cancer
Efficacy and Safety of HL-085 Combined With Vemurafenib in BRAF V600E Patients With Non-small Cell Lung Cancer: a Phase II Clinical Study
This is a single-arm, open, multicenter phase II clinical study to evaluate the efficacy and safety of HL-085 capsules combined with Vemurafenib in the treatment of BRAF V600E mutated patients with unresectable locally advanced…
- Trial name
- NCT05900219
- Testing
- HL-085+Vemurafenib
- Volunteers
- 75 people
- Sponsor
- Shanghai Kechow Pharma, Inc.
Metastatic Non-small Cell Lung Cancer, BRAF V600 Mutation
A Study in Patients With BRAF V600E-mutant Metastatic Non-small Cell Lung Cancer (OCTOPUS)
This study aims to describe the treatment patterns in clinical practice in adult patients with mNSCLC with a BRAF V600E mutation.
- Trial name
- NCT05546905
- Where
- 39 sites in France, Germany and 3 more
- Volunteers
- 206 people
- Sponsor
- Pierre Fabre Medicament
Leukemia, Acute Myeloid (AML)
A Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase (FLT3) Mutation
The purpose of this study was to determine the clinical benefit of ASP2215 therapy in participants with FMS-like tyrosine kinase (FLT3) mutated acute myeloid leukemia (AML) who were refractory to or had relapsed after first-line…
- Trial name
- NCT02421939
- Testing
- gilteritinib, LoDAC (Low Dose Cytarabine), Azacitidine
- Where
- 126 sites in United States, Belgium and 12 more
- Volunteers
- 371 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Advanced Cholangiocarcinoma, FGFR2 Gene Mutation
Phase 3 Study of BGJ398 (Oral Infigratinib) in First Line Cholangiocarcinoma With FGFR2 Gene Fusions/Translocations
Infigratinib is an oral drug which selectively binds to fibroblast growth factor receptor (FGFR) 2 and is being developed to treat participants with FGFR2 mutated cholangiocarcinoma.
- Trial name
- NCT03773302
- Testing
- BGJ398, Gemcitabine, Cisplatin
- Where
- 116 sites in United States, Australia and 13 more
- Volunteers
- 48 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Advanced Solid Tumors Cancer, PDAC
A Study of GFH375 Combined With Cetuximab or Chemotherapy in Participants With Solid Tumors Harboring KRAS G12D Mutation
This is a Phase Ib/II clinical study aimed at exploring the safety and efficacy of Regimen A (GFH375 in combination with Cetuximab) and Regimen B (GFH375 in combination with AG) in participants with solid tumors.Phase Ib: To…
- Conditions
- Advanced Solid Tumors Cancer, PDAC, CRC (Colorectal Cancer)
- Trial name
- NCT07259590
- Testing
- GFH375
- Where
- 4 sites in China
- Volunteers
- 126 people
- Sponsor
- Genfleet Therapeutics (Shanghai) Inc.
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.
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