Clinical trials: Gene Mutation
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
SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant)
An Open-Label Study of Diazoxide Choline in Patients With Genetic Obesities
This is a phase 2 open-label, one-arm, clinical study to evaluate the efficacy and safety of DCCR (diazoxide choline) extended-release tablets over 1 year of treatment, in patients with genetic obesities.
- Conditions
- SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant), SIM1 Deficiency Obesity
- Trial name
- NCT05532020
- Testing
- DCCR (diazoxide choline) extended-release tablets
- Sponsor
- Soleno Therapeutics, Inc.
Non-small-cell Lung Cancer
ARQ 197 Plus Erlotinib in Patient With Locally Advanced or Metastatic EGFR Mutation-positive Non-small-cell Lung Cancer
The primary objective of this study is to examine if the combination regimen of ARQ 197 with erlotinib is active in subjects with locally advanced or metastatic NSCLC with activating mutation EGFR who progressed on EGFR-TKI…
- Trial name
- NCT01580735
- Testing
- ARQ 197
- Where
- Japan
- Volunteers
- 40 people
- Sponsor
- Kyowa Kirin Co., Ltd.
NF type1 With Inoperable Plexiform Neurofibromas
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1
This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable…
- Trial name
- NCT03259633
- Testing
- Selumetinib
- Where
- United States
- Sponsor
- AstraZeneca
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
FGFR2 Amplification, FGFR2 Gene Mutation
REFOCUS: A First-in-Human Study of Highly Selective FGFR2 Inhibitor, RLY-4008, in Patients With ICC and Other Advanced Solid Tumors
This is a Phase 1/2, open-label, FIH study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDy), and antineoplastic activity of RLY-4008, a potent and highly selective FGFR2 inhibitor, in…
- Conditions
- FGFR2 Amplification, FGFR2 Gene Mutation, FGFR2 Gene Fusion/Rearrangement, FGFR2 Gene Translocation
- Trial name
- NCT04526106
- Testing
- RLY-4008
- Where
- 48 sites in United States, Australia and 11 more
- Volunteers
- 490 people
- Sponsor
- Elevar Therapeutics
Breast Cancer
Study of Safety and Efficacy of Dapagliflozin + Metformin XR Versus Metformin XR in Participants With HR+, HER2-, Advanced Breast Cancer While on Treatment With Alpelisib and Fulvestrant
This study was designed to assess the safety and efficacy of the combination of dapagliflozin plus metformin extended release (XR) compared with metformin XR during treatment with alpelisib plus fulvestrant in participants with…
- Trial name
- EPIK-B4 (NCT04899349)
- Testing
- Alpelisib, Fulvestrant, Metformin XR
- Where
- 2 sites in United States, Malaysia
- Volunteers
- 2 people
- Sponsor
- Novartis Pharmaceuticals
Metastatic Colorectal Cancer
Sym004 in Metastatic Colorectal Cancer and ECD-EGFR Patients
This is a single arm, phase 2, open-label, multicenter trial in patients with metastatic colorectal cancer (mCRC) and acquired resistance to anti-epidermal growth factor receptor (EGFR) monoclonal antibodies (mAbs) and documented…
- Trial name
- NCT02965417
- Testing
- Sym004
- Sponsor
- Symphogen A/S
CLL / SLL, CLL (Chronic Lymphocytic Leukemia)
ROCKET-CLL Global Phase 3 Study: Rocbrutinib vs Pirtobrutinib in cBTKi-Pretreated R/R CLL/SLL
This is a Phase 3, randomized, open-label, multicenter study comparing rocbrutinib (LP-168) versus pirtobrutinib in adult participants with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma…
- Conditions
- CLL / SLL, CLL (Chronic Lymphocytic Leukemia), CLL, Refractory, CLL, Relapsed
- Trial name
- NCT07342478
- Testing
- Rocbrutinib, Pirtobrutinib
- Where
- 6 sites in United States
- Volunteers
- 306 people
- Sponsor
- Newave Pharmaceutical Inc
X-Linked Retinitis Pigmentosa (XLRP)
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene…
- Trial name
- NCT06492850
- Testing
- FT-002
- Where
- China
- Volunteers
- 32 people
- Sponsor
- Frontera Therapeutics
Acute Myeloid Leukemia, Acute Myelogenous Leukemia
Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation
This Phase 1/2 study will evaluate the safety, efficacy, PK, and PD of FT-2102 (olutasidenib) as a single agent or in combination with azacitidine or cytarabine.
- Conditions
- Acute Myeloid Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT02719574
- Testing
- FT-2102 (olutasidenib), Azacitidine, Cytarabine
- Where
- 60 sites in United States, Australia and 7 more
- Volunteers
- 336 people
- Sponsor
- Forma Therapeutics, Inc.
Cystic Fibrosis
A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation
The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual…
- Trial name
- NCT05076149
- Testing
- VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA
- Where
- 170 sites in United States, Australia and 18 more
- Volunteers
- 597 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cardiovascular Diseases, Arrhythmias, Cardiac
Intracardiac Echocardiography Catheter Supply-Chain Reliability and Clinical Access Study
The ICE-ACCESS study is a prospective observational study designed to evaluate how procurement reliability, distribution restrictions, supply-chain interruptions, and device availability affect access to intracardiac…
- Conditions
- Cardiovascular Diseases, Arrhythmias, Cardiac, Atrial Fibrillation
- Trial name
- ICE-ACCESS (NCT07788040)
- Testing
- Intracardiac Echocardiography Catheter
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Truway Health, Inc.
Multiple Myeloma
Study of Alternative and Approved Dosing Regimens of Belantamab Mafodotin, Bortezomib, and Dexamethasone (BVd) in Participants With Relapsed/Refractory Multiple Myeloma
The aim of this study is to assess safety, efficacy and pharmacokinetic (PK) parameters with alternative dosing schedules of belantamab mafodotin in combination with bortezomib and dexamethasone compared to the approved dosing…
- Trial name
- DREAMM-16 (NCT07614360)
- Testing
- Belantamab mafodotin, Dexamethasone, Bortezomib
- Volunteers
- 150 people
- Sponsor
- GlaxoSmithKline
Melanoma and Metastatic Colorectal Cancer
A Phase I Study of Oral LGX818 in Adult Patients With Advanced or Metastatic BRAF Mutant Melanoma
CLGX818X2101 is a first-time in-human, phase I study to establish the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of daily administered LGX818 (daily, twice daily and/or every-other-day), a RAF kinase…
- Trial name
- NCT01436656
- Testing
- LGX818
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 107 people
- Sponsor
- Pfizer
Severe Personality Disorder
The (Cost-)Effectiveness of Generalist Versus Specialist Treatment for Severe Personality Disorders'
The goal of this clinical trial is to investigate if generalist treatment (GIT-PD) is non-inferior in improving level op personality functioning compared to specialist treatment (MBT/ST) in patients with severe personality…
- Trial name
- P-DAET (NCT06789380)
- Testing
- Schema therapy, Mentalization-based treatment, Guideline-informed treatment for personality disorders
- Where
- 5 sites in Netherlands
- Volunteers
- 358 people
- Sponsor
- Arkin
Non-Small Cell Lung Cancer
Clinical Trial of YH25448(Lazertinib) as the First-line Treatment in Patients With EGFR Mutation Positive Locally Advanced or Metastatic NSCLC (LASER301)
This Phase III study will be conducted to evaluate the efficacy and safety of YH25448 as first-line treatment in locally advanced or metastatic Non-small Cell Lung Cancer (NSCLC) patients with EGFR mutations
- Trial name
- NCT04248829
- Testing
- Lazertinib 240 mg/160 mg, Gefitinib 250 mg
- Where
- 80 sites in Australia, Greece and 11 more
- Volunteers
- 393 people
- Sponsor
- Yuhan Corporation
Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
- Trial name
- NCT05906953
- Testing
- HG004
- Where
- 3 sites in United States, China
- Volunteers
- 20 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Amyotrophic Lateral Sclerosis
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for people with a certain type of amyotrophic lateral sclerosis, also known as…
- Trial name
- NCT07223723
- Testing
- Tofersen
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Biogen
Malignant Melanoma
A Study of Vemurafenib (RO5185426) in Comparison With Dacarbazine in Previously Untreated Patients With Metastatic Melanoma (BRIM 3)
This randomized, open-label study evaluated the efficacy, safety and tolerability of vemurafenib (RO5185426) as compared to dacarbazine in previously untreated patients with metastatic melanoma.
- Trial name
- NCT01006980
- Testing
- Vemurafenib, Dacarbazine
- Where
- 111 sites in United States, Australia and 10 more
- Volunteers
- 675 people
- Sponsor
- Hoffmann-La Roche
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