Clinical trials: Gene Mutation
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00847379
- Testing
- Ataluren
- Where
- 37 sites in United States, Australia and 9 more
- Volunteers
- 173 people
- Sponsor
- PTC Therapeutics
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
Melanoma, Solid Tumor
Tovorafenib (DAY101) or in Combination With Pimasertib for Participants With Melanoma and Other Solid Tumors
This is a subprotocol of Master Protocol DAY101-102 and is a Phase 1b/2, multi-center, open label subprotocol of participants ≥12 years of age, with recurrent or progressive solid tumors with alterations in the key proteins of…
- Conditions
- Melanoma, Solid Tumor, Pilocytic Astrocytoma, Non Small Cell Lung Cancer
- Trial name
- NCT07121829
- Testing
- Tovorafenib, Tovorafenib Drug: Pimasertib
- Where
- 10 sites in United States, Canada
- Volunteers
- 44 people
- Sponsor
- Day One Biopharmaceuticals, Inc.
Cystic Fibrosis
Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR Mutation
This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter, crossover study that will evaluate the efficacy of LUM/IVA in subjects with CF 12 years of age and older who have at least one A455E mutation.
- Trial name
- NCT03061331
- Testing
- LUM/IVA
- Where
- 2 sites in Netherlands
- Volunteers
- 20 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Gaucher Disease, Type 2
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2).
- Trial name
- NCT04411654
- Testing
- LY3884961, Methylprednisolone, Sirolimus
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Prevail Therapeutics
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
ESR1 Gene Mutation, Advanced Breast Cancer
Current Status of Treatment for Chinese Patients With ESR1-mutated HR+/HER2-advanced Breast Cancer
This is a prospective, non-interventional real-world study to observe the efficacy and safety of different treatment regimens in patients with ESR1-mutated HR+/HER2-advanced breast cancer after failure of endocrine therapy.
- Trial name
- NCT06548919
- Testing
- Endocrine therapy, Chemotherapy Prednisone
- Where
- China
- Volunteers
- 450 people
- Sponsor
- SciClone Pharmaceuticals
NSCLC
Phase Ib Study of FURMONERTINIB in Patients with NSCLC Having Exon 20 Insertion Mutation
This is a phase 1 multi-center clinical study. To explore the efficacy and safety of Furmonertinib Mesilate at different doses in locally advanced or metastatic NSCLC patients with EGFR exon 20 insertion mutation.
- Trial name
- NCT04958967
- Testing
- Furmonertinib 240mg, Furmonertinib 160mg
- Where
- China
- Sponsor
- Allist Pharmaceuticals, Inc.
Non-small Cell Lung Cancer
A Study of JAB-21822 in Advanced or Metastatic NSCLC With KRAS p.G12C and STK11 Co-mutation and Wild-type KEAP1
Evaluate the safety and tolerability, drug levels, and clinical activity of JAB-21822 in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) whose tumors with KRAS p.G12C mutation and a…
- Trial name
- NCT05276726
- Testing
- JAB 21822
- Where
- 21 sites in China
- Volunteers
- 104 people
- Sponsor
- Allist Pharmaceuticals, Inc.
Cystic Fibrosis
PTC124 for the Treatment of Cystic Fibrosis
In some patients 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
- NCT00351078
- Testing
- PTC124
- Where
- Israel
- Volunteers
- 19 people
- Sponsor
- PTC Therapeutics
Heart Failure
Vivio At-Home Study
The purpose of the study is to examine the Vivio System's ability to track heart failure status in subjects, and compare the Vivio System's measurements to CardioMEMS measurements.
- Trial name
- NCT07730268
- Testing
- Vivio measurement
- Where
- 4 sites in United States
- Volunteers
- 500 people
- Sponsor
- Ventric Health, LLC
Advanced Solid Tumor, NSCLC
JAB-21822 Activity in Adult Patients With Advanced Solid Tumors Harboring KRAS G12C Mutation
This study is to evaluate the safety and tolerability of JAB-21822 monotherapy and combination therapy in adult participants with advanced solid tumors harboring KRAS G12C mutation.
- Conditions
- Advanced Solid Tumor, NSCLC, CRC
- Trial name
- NCT05002270
- Testing
- JAB-21822 (KRAS G12C inhibitor), Cetuximab (EGFR inhibitor)
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Jacobio Pharmaceuticals Co., Ltd.
Cancer
A Rollover Study to Provide Continued Treatment With GSK2118436 to Subjects With BRAF Mutation-Positive Tumors
This rollover study is designed to provide continued access to GSK2118436 for eligible subjects with BRAF mutation-positive tumors who have previously participated in a GlaxoSmithKline (GSK)-sponsored GSK2118436 study (parent…
- Trial name
- NCT01231594
- Testing
- GSK2118436, GSK1120212, Other approved anti-cancer agent
- Where
- 44 sites in United States, Australia and 7 more
- Volunteers
- 214 people
- Sponsor
- Novartis Pharmaceuticals
Ovarian Cancer
A Study of Adavosertib (MK-1775) in Combination With Paclitaxel and Carboplatin Versus Paclitaxel and Carboplatin Alone for Participants With Platinum-Sensitive Ovarian Tumors With the P53 Gene Mutation (MK-1775-004)
This is a study of the safety and efficacy of adavosertib in combination with paclitaxel plus carboplatin in the treatment of ovarian, fallopian tube, and primary peritoneal tumors with the P53 mutation.
- Trial name
- NCT01357161
- Testing
- adavosertib, paclitaxel, carboplatin
- Volunteers
- 136 people
- Sponsor
- Merck Sharp & Dohme LLC
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
- Trial name
- NCT05858983
- Testing
- FT-001 Low Dose, FT-001 Mid Dose, FT-001 High Dose
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Frontera Therapeutics
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.
Locally Advanced or Metastatic EGFR(+) NSCLC Patients
ARTEMIS DIANE T790M (An Amino Acid Substitution at Position 790 in EGFR, From a Threonine (T) to a Methionine (M)) Mutation at Hospital Laboratories in Comparison With Central Laboratory
The study primary objective is to assess the concordance of T790M resistance mutation testing from hospital-based laboratories with T790M resistance mutation testing from a central laboratory.
- Trial name
- NCT02991274
- Testing
- genomic testing of T790M mutation
- Where
- 31 sites in China
- Volunteers
- 897 people
- Sponsor
- AstraZeneca
Vaping, Nicotine Absorption
Study to Investigate the Nicotine Pharmacokinetic Profiles and Pharmacodynamic Effects of P4M3 Variants
This is a single-center, open-label, concentration-ranging study to evaluate the nicotine PK profile and PD effects in healthy adult experienced users of closed tank/cartridge e-cigarettes using four different variants of P4M3…
- Trial name
- NCT03379740
- Testing
- E-cigarette, P4M3-1.7%, P4M3-1.7%LA
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Philip Morris Products S.A.
Advanced NSCLC Patients With T790M
Efficacy and Safety of Alflutinib in Locally Advanced or Metastatic Non-Small Cell Lung Cancer Patients With T790M
This study is conducted to evaluate the efficacy and safety of Alflutinib in locally advanced or metastatic non-small cell lung cancer patients harbouring T790M mutation
- Trial name
- NCT03452592
- Testing
- Alflutinib
- Where
- China
- Volunteers
- 220 people
- Sponsor
- Allist Pharmaceuticals, Inc.
Solid Tumors
Alternative Dosing for CRLX101(NLG207) Alone, With Avastin and With mFOLFOX6 in Advanced Solid Tumors
The purpose of this study is to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of weekly dosing of CRLX101 (both as monotherapy; (Schedule 1) and in combination with bevacizumab every 2 weeks…
- Trial name
- NCT02648711
- Testing
- CRLX101, Bevacizumab, mFOLFOX6
- Where
- 3 sites in United States
- Volunteers
- 41 people
- Sponsor
- NewLink Genetics Corporation
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