Clinical trials: Mutations
X-linked Hypophosphatemia
Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH
This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab…
- Trial name
- NCT03920072
- Testing
- Burosumab
- Where
- 10 sites in France, Ireland and 2 more
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Advanced/Metastatic Solid Tumors With KRAS p.G12C Mutation
AMG 510 Ethnic Sensitivity Study (CodeBreaK 105).
To evaluate safety, tolerability, PK, and preliminary efficacy of AMG 510 PO QD in subjects of Chinese descent with KRAS p.G12C-mutant advanced/metastatic solid tumors.
- Trial name
- NCT04380753
- Testing
- AMG 510
- Where
- 4 sites in Hong Kong, Taiwan
- Volunteers
- 12 people
- Sponsor
- Amgen
Neoplasms, Advanced Solid Tumors
First-in-Human Study of JNJ-74699157 in Participants With Tumors Harboring the KRAS G12C Mutation
The purpose of this study is to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of JNJ-74699157 in participants with advanced solid tumors harboring a kirsten rat sarcoma virus homolog (KRAS)…
- Conditions
- Neoplasms, Advanced Solid Tumors, Non-small Cell Lung Cancer, Colorectal Cancer
- Trial name
- NCT04006301
- Testing
- JNJ-74699157
- Where
- 7 sites in United States, France
- Volunteers
- 10 people
- Sponsor
- Janssen Research & Development, LLC
Cystic Fibrosis
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
The purpose of this study was to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in participants with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an…
- Trial name
- NCT02725567
- Testing
- IVA
- Where
- 23 sites in United States, Australia and 3 more
- Volunteers
- 57 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cancer
Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations
This was a 4-part (Part A, Part B, Part C and Part D), Phase I/IIa, multi-center, open label, study in pediatric subjects with refractory or recurrent tumors.
- Trial name
- NCT02124772
- Testing
- Trametinib, Dabrafenib
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 139 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis
The goal of this clinical trial is to learn if drug SPL84 is safe for adult patients with cystic fibrosis (CF). It will also learn if the drug works to treat works to treat CF with a specific mutation (3849 +10kb C-->T).
- Trial name
- NCT06429176
- Testing
- SPL84
- Where
- 3 sites in United States
- Volunteers
- 64 people
- Sponsor
- SpliSense Ltd.
Muscular Dystrophy, Duchenne, Muscular Dystrophies
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT02090959
- Testing
- Ataluren
- Where
- 58 sites in United States, Australia and 17 more
- Volunteers
- 219 people
- Sponsor
- PTC Therapeutics
C-Met Exon 14 Mutation
Assessment of Anti-tumor and Safety in Glumetinib in Patients With c-MET-positive Non-Small Cell Lung Cancer
Indication:Patients with Advanced c-MET-positive Non-Small Cell Lung Cancer Phase Ib (China only): Approximately 90 patients Phase Ⅱ (globally): Approximately 78 evaluable patients; addition of at least 6 patients in Safety…
- Trial name
- NCT04270591
- Testing
- Glumetinib
- Where
- 44 sites in United States, China and 1 more
- Volunteers
- 183 people
- Sponsor
- Haihe Biopharma Co., Ltd.
Advanced or Metastatic NSCLC Harboring Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertion (ex20ins) Mutations
A Study of Zipalertinib in Patients With Advanced Non-Small Cell Lung Cancer With Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertions or Other Uncommon Mutation.
The purpose of this study is to evaluate the safety, efficacy and pharmacokinetics (PK) of zipalertinib in participants with locally advanced or metastatic Non-Small Cell Lung Cancer (NSCLC) harboring EGFR ex20ins mutations and…
- Trial name
- REZILIENT2 (NCT05967689)
- Testing
- TAS6417, CYP Cocktail, Transporter Cocktail
- Where
- 80 sites in United States, Australia and 10 more
- Volunteers
- 220 people
- Sponsor
- Taiho Oncology, Inc.
Ovarian Cancer
FLABRA: Frontline Approach for BRCA Testing in OC Treatment naïve Population. A LATIN AMERICA Epidemiologic Study
This study aims to answer very important questions regarding the prevalence of somatic and germline mutations (sBRCAm and gBRCAm) in a population of newly diagnosed ovarian cancer patients from Latin America.
- Trial name
- FLABRA (NCT02984423)
- Where
- 27 sites in Argentina, Brazil and 4 more
- Volunteers
- 471 people
- Sponsor
- AstraZeneca
Cancer, Breast Cancer
A Study to Learn About Study Medicine ALTA2618 in Adults With AKT1 E17K-Mutant Solid Tumors
The purpose of this study is to characterize the safety and tolerability of ALTA2618 in adults with AKT1 E17K-mutant advanced solid tumors.
- Conditions
- Cancer, Breast Cancer, Endometrial Cancer, Metastatic Cancer
- Trial name
- NCT06533059
- Testing
- ALTA2618
- Where
- 31 sites in United States, Australia and 6 more
- Volunteers
- 110 people
- Sponsor
- Alterome Therapeutics, Inc.
Advanced or Metastatic Solid Tumors, FGFR Mutations
Efficacy and Safety of Pemigatinib in Participants With Solid Tumors With FGFR Mutations or Translocations (FIGHT-208)
The purpose of this study is to evaluate the efficacy and safety of pemigatinib in participants with previously treated locally advanced/metastatic or surgically unresectable solid tumors harboring activating FGFR mutations or…
- Conditions
- Advanced or Metastatic Solid Tumors, FGFR Mutations, FGFR Translocations
- Trial name
- NCT04003623
- Testing
- Pemigatinib
- Where
- 11 sites in United States
- Volunteers
- 1 people
- Sponsor
- Incyte Corporation
X-Linked Hypophosphataemia
A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH
This study aims to provide Health-related Quality of Life (HRQoL) data from children and adolescents with growing skeletons in the United Kingdom (UK), including those treated with burosumab or alternative XLH treatment, as part…
- Trial name
- NCT04819490
- Where
- 6 sites in United Kingdom
- Volunteers
- 32 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Ovarian Cancer, Breast Cancer
Roll Over StudY for Patients Who Have Completed a Previous Oncology Study With Olaparib
The rationale of the ROSY-O study is to continue to provide study treatment for patients who have participated in a parent study with olaparib and who are continuing to derive clinical benefit from treatment at the end of such…
- Trial name
- ROSY-O (NCT04421963)
- Testing
- Olaparib
- Where
- 107 sites in United States, Belgium and 21 more
- Volunteers
- 185 people
- Sponsor
- AstraZeneca
PIK3CA-related Overgrowth Spectrum (PROS)
Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)
This Phase II, multicenter, open-label study, preceded by a retrospective non-interventional period, is assessing the long-term safety and efficacy of alpelisib in pediatric and adult participants with PIK3CA-related overgrowth…
- Trial name
- EPIK-P3 (NCT04980833)
- Testing
- Alpelisib
- Where
- 6 sites in United States, France and 2 more
- Volunteers
- 41 people
- Sponsor
- Novartis Pharmaceuticals
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
- Trial name
- NCT06333249
- Testing
- rAAV2tYF-GRK1-RPGR
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- Beacon Therapeutics
Metastatic Colorectal Cancer
Study of Sotorasib, Panitumumab and FOLFIRI Versus FOLFIRI With or Without Bevacizumab-awwb in Treatment-naïve Participants With Metastatic Colorectal Cancer With KRAS p.G12C Mutation
The aim of this study is to compare progression free survival (PFS) in treatment-naïve participants with KRAS p.G12C mutated metastatic colorectal cancer (mCRC) receiving sotorasib, panitumumab and FOLFIRI vs FOLFIRI with or…
- Trial name
- CodeBreaK 301 (NCT06252649)
- Testing
- FOLFIRI Regimen, Sotorasib, Panitumumab
- Where
- 291 sites in United States, Argentina and 33 more
- Volunteers
- 450 people
- Sponsor
- Amgen
NSCLC
A Study of BPI-7711 Capsule in Non-small Cell Lung Cancer Patients
A randomized, double-blind, positive controlled phase III study to evaluate the efficacy and safety of BPI-7711 capsule in locally advanced or recurrent/metastatic treatment-naïve non-small cell lung cancer patients with EGFR…
- Trial name
- NCT03866499
- Testing
- BPI-7711, Gefitinib
- Where
- 3 sites in China
- Volunteers
- 369 people
- Sponsor
- Beta Pharma Shanghai
Advanced or Metastatic NSCLC
Phase 1/2 Study of HS-10375 in Patients with Advanced or Metastatic Non-Small-Cell Lung Cancer(NSCLC)
HS-10375 is an oral, highly selective, small molecular inhibitor of EGFR C797S. This study will evaluate the safety, tolerability, pharmacokinetics and clinical activity of HS-10375 in Chinese advanced or metastatic NSCLC.
- Trial name
- NCT05435248
- Testing
- HS-10375
- Where
- China
- Volunteers
- 354 people
- Sponsor
- Jiangsu Hansoh Pharmaceutical Co., Ltd.
Non Small Cell Lung Cancer
Phase II Trial of HM61713 for the Treatment of ≥2nd Line T790M Mutation Positive Adenocarcinoma of the Lung
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of HM61713 in patients with T790M-positive non-small cell lung cancer (NSCLC) after treatment with an epidermal growth factor receptor-tyrosine…
- Trial name
- NCT02485652
- Testing
- HM61713
- Where
- 72 sites in United States, Australia and 8 more
- Volunteers
- 162 people
- Sponsor
- Hanmi Pharmaceutical Company Limited