Clinical trials: Genetic Mutation
Cystic Fibrosis
Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation
This Phase 1b study in F508del-CFTR homozygous CF patients is being conducted to assess the safety of N91115 as the sole cystic fibrosis transmembrane conductance regulator (CFTR) modulator at doses near the expected therapeutic…
- Trial name
- SNO4 (NCT02275936)
- Testing
- N91115
- Where
- 19 sites in United States
- Volunteers
- 51 people
- Sponsor
- Nivalis Therapeutics, Inc.
Advanced Solid Tumors
A Rollover Study Evaluating Sotorasib With or Without Panitumumab in Participants With KRAS p.G12C Mutation
The primary objective of the study is to evaluate the safety and tolerability of the continuation of sotorasib with or without panitumumab and/or other anti-cancer therapies in participants continuing to derive benefit in…
- Trial name
- NCT07172919
- Testing
- Sotorasib, Panitumumab
- Where
- 11 sites in United States, Australia and 7 more
- Volunteers
- 14 people
- Sponsor
- Amgen
Intrahepatic Cholangiocarcinoma, Combined Hepatocellular and Cholangiocarcinoma
Derazantinib in Subjects With FGFR2 Gene Fusion-, Mutation- or Amplification- Positive Inoperable or Advanced Intrahepatic Cholangiocarcinoma
This Phase II, open-label, single-arm study evaluated the anti-cancer activity of derazantinib in subjects with inoperable or advanced intrahepatic cholangiocarcinoma (iCCA) who received at least one prior regimen of systemic…
- Trial name
- FIDES-01 (NCT03230318)
- Testing
- derazantinib
- Where
- 41 sites in United States, Belgium and 9 more
- Volunteers
- 148 people
- Sponsor
- Basilea Pharmaceutica
Advanced Solid Tumors With MAPK Pathway Mutations
A Phase 1/2 Study of D3S-002 as Monotherapy or Combination Therapy in Adult Subjects With Advanced Solid Tumors With MAPK Pathway Mutations
This first-in-human (FIH) study aims to assess the safety, tolerability, pharmacokinetics, and recommended phase 2 dose (RP2D) of D3S-002 given orally daily for 21-day cycles in adult subjects with advanced solid tumors with…
- Trial name
- NCT05886920
- Testing
- D3S-002, D3S-001
- Where
- 18 sites in United States, Australia and 2 more
- Volunteers
- 67 people
- Sponsor
- D3 Bio (Wuxi) Co., Ltd
Solid Tumors
A Study to Evaluate the Safety and Effectiveness of Different Doses of RO7836748 Alone or in Combination With Other Cancer Therapies in People With Solid Tumors With a Rat Sarcoma Viral Oncogene Homolog Glycine 12 Valine (RAS G12V) Mutation
The study will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of RO7836748 as monotherapy and in combination with other anti-cancer therapies, including…
- Trial name
- NCT07905924
- Testing
- RO7836748, Pembrolizumab, mFOLFIRINOX
- Volunteers
- 540 people
- Sponsor
- Hoffmann-La Roche
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
Metastatic Non Small Cell Lung Cancer, Advanced Non Small Cell Lung Cancer
Phase 3 Study of MRTX849 (Adagrasib) vs Docetaxel in Patients With Advanced Non-Small Cell Lung Cancer With KRAS G12C Mutation
This Phase 3 study will evaluate the efficacy of the investigational agent MRTX849 (adagrasib) versus docetaxel in patients who have been previously treated for metastatic NSCLC with a KRAS G12C mutation.
- Trial name
- KRYSTAL-12 (NCT04685135)
- Testing
- MRTX849, Docetaxel
- Where
- 380 sites in United States, Australia and 22 more
- Volunteers
- 453 people
- Sponsor
- Mirati Therapeutics Inc.
Vascular Ehlers-Danlos Syndrome
Investigate Efficacy, Safety, and Pharmacokinetics of Enzastaurin for the Prevention of Arterial Events in Patients With Vascular Ehlers-Danlos Syndrome.
The purpose of this study is to investigate the efficacy of enzastaurin compared to placebo in preventing arterial events (rupture, dissection, pseudoaneurysm, carotid-cavernous sinus fistula, or aneurysm, fatal or not) leading…
- Trial name
- PREVEnt (NCT05463679)
- Testing
- Enzastaurin
- Where
- United States
- Volunteers
- 260 people
- Sponsor
- Aytu BioPharma, Inc.
Cancer
A Study Comparing GSK2118436 to Dacarbazine (DTIC) in Previously Untreated Subjects With BRAF Mutation Positive Advanced (Stage III) or Metastatic (Stage IV) Melanoma
BRF113683 is a Phase III, randomized, open-label study comparing the efficacy, safety, and tolerability of GSK2118436 to dacarbazine (DTIC), in subjects with BRAF mutant advanced (Stage III) or metastatic (Stage IV) melanoma.
- Trial name
- NCT01227889
- Testing
- GSK2118436, Dacarbazine (DTIC)
- Where
- 94 sites in United States, Australia and 10 more
- Volunteers
- 251 people
- Sponsor
- GlaxoSmithKline
PEComa, Malignant, TSC1
Expanded Access for ABI-009 in Patients With Advanced PEComa and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation
Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients with a…
- Conditions
- PEComa, Malignant, TSC1, TSC2, mTOR Pathway Abberation
- Trial name
- NCT03817515
- Testing
- ABI-009
- Sponsor
- Aadi Bioscience, Inc.
Melanoma, Stage II, HER2-positive
A Study of RC48-ADC in Advanced Melanoma Subjects With HER2 Variant (Mutation, Amplification, Overexpression)
This is a Phase IIa, single-arm, multicentre, open-label clinical trial aims to evaluate the effectiveness and safety of RC48-ADC in the treatment of HER2 Variant (Mutation, Amplification, Overexpression) advanced melanoma.
- Conditions
- Melanoma, Stage II, HER2-positive, Advanced Melanoma
- Trial name
- NCT05135715
- Testing
- RC48-ADC
- Where
- 4 sites in China
- Volunteers
- 50 people
- Sponsor
- RemeGen Co., Ltd.
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
This is a Study to Investigate the Effects and Safety of Codabakalner for the Treatment of Fragile X Syndrome in Male Participants and Adult Participants with Autism Spectrum Disorders
- Trial name
- NCT07439510
- Testing
- Codabakalner
- Volunteers
- 120 people
- Sponsor
- Spinogenix
Locally Advanced or Metastatic KRAS G12C-mutated NSCLC With a PD-L1 Expression <1% or a PD-L1 Expression ≥ 1% and an STK11 Co-mutation
Study of Efficacy and Safety of JDQ443 Single-agent as First-line Treatment for Patients With Locally Advanced or Metastatic KRAS G12C- Mutated Non-small Cell Lung Cancer With a PD-L1 Expression < 1% or a PD-L1 Expression ≥ 1% and an STK11 Co-mutation.
This study aims to evaluate the antitumor activity and safety of JDQ443 single-agent as first-line treatment for participants with locally advanced or metastatic non-small cell lung cancer (NSCLC) whose tumors harbor a KRAS G12C…
- Trial name
- KontRASt-06 (NCT05445843)
- Testing
- JDQ443
- Where
- 45 sites in United States, Argentina and 17 more
- Volunteers
- 95 people
- Sponsor
- Novartis Pharmaceuticals
Breast Cancer Female
Pembrolizumab And Tamoxifen Among Women With Advanced Hormone Receptor Positive Breast Cancer And Esr1 Mutation
Pembrolizumab is a potent and highly selective humanized monoclonal antibody (mAb) designed to directly block the interaction between PD-1 and its ligands and enable the T cell to remain active and co-ordinate an attack on tumor…
- Trial name
- Pembro (NCT03879174)
- Testing
- Pembrolizumab + Tamoxifen
- Where
- United Arab Emirates
- Volunteers
- 25 people
- Sponsor
- Mediclinic Middle East
Myeloproliferative Neoplasms
A Study to Evaluate INCA035784 in Participants With Myeloproliferative Neoplasms
This study is being conducted to evaluate the safety and tolerability of INCA035784 in participants with myeloproliferative neoplasms.
- Trial name
- NCT07008118
- Testing
- INCA035784
- Where
- 26 sites in United States, Australia and 3 more
- Volunteers
- 120 people
- Sponsor
- Incyte Corporation
ALK-activated Tumors
Phase I Study of LDK378 in Pediatric, Malignancies With a Genetic Alteration in Anaplastic Lymphoma Kinase (ALK)
The purpose of this study was to estimate the maximum tolerated dose and/or recommended dose for expansion of LDK378 as a single agent, assess safety, tolerability and anti-tumor activity and characterize single and multiple-dose…
- Trial name
- NCT01742286
- Testing
- Ceritinib
- Where
- 23 sites in United States, Australia and 8 more
- Volunteers
- 83 people
- Sponsor
- Novartis Pharmaceuticals
HER2 Mutation-Related Tumors, HER2 Amplified Solid Tumors
ARX788 in Selected HER2-mutated or HER2-amplified/Overexpressed Solid Tumors (ACE-Pan Tumor-02)
A Global Phase 2 Study to Evaluate the Efficacy and Safety of ARX788 for Selected HER2-mutated or HER2-amplified/overexpressed Solid Tumors (ACE-Pan tumor-02)
- Trial name
- NCT05041972
- Testing
- ARX788
- Where
- 2 sites in United States
- Sponsor
- Ambrx, Inc.
Cancer, Solid Tumor Cancer
Clinical Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of MPD-1 in Patients With Advanced Solid Tumor
A Phase I, Open-label, Single-center, Dose-escalation and Dose-finding Clinical trial to evaluate the safety, tolerability and pharmacokinetics of MPD-1 in patients with advanced solid tumor
- Conditions
- Cancer, Solid Tumor Cancer, Biomarkers
- Trial name
- NCT06944457
- Testing
- MPD-1
- Where
- South Korea
- Volunteers
- 24 people
- Sponsor
- Pharosgen Co.,Ltd
Advanced or Metastatic Breast Cancer, ER+ve Advanced or Metastatic Breast Cancer
Investigating Safety, Tolerability and Efficacy of AZD5363 When Combined With Paclitaxel in Breast Cancer Patients
The purpose of this study is to investigate the safety and efficacy of different doses and schedules of AZD5363, when in combination with paclitaxel, in treatment of patients with advanced or metastatic breast cancer.
- Trial name
- BEECH (NCT01625286)
- Testing
- AZD5363 when combined with weekly paclitaxel., AZD5363when combined with weekly paclitaxel.
- Where
- 41 sites in Bulgaria, Canada and 9 more
- Volunteers
- 148 people
- Sponsor
- AstraZeneca
Relapsed Solid Tumors, Refractory Solid Tumors
A Study of KB-0742 in Participants With Relapsed or Refractory Solid Tumors Including Platinum Resistant High Grade Serous Ovarian Cancer (HGSOC)
Part 1: Dose Escalation. The primary objective of Part 1 of this study is to evaluate the safety and tolerability of KB-0742 in participants with relapsed or refractory (R/R) solid tumors or non-Hodgkin lymphoma (NHL).
- Conditions
- Relapsed Solid Tumors, Refractory Solid Tumors, Non-Hodgkin Lymphoma, HGSOC
- Trial name
- NCT04718675
- Testing
- KB-0742
- Where
- 24 sites in United States, Spain and 1 more
- Volunteers
- 135 people
- Sponsor
- Kronos Bio
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