Clinical trials: Gene Mutations
Metastatic Colorectal Cancer
Study of WNT974 in Combination With LGX818 and Cetuximab in Patients With BRAF-mutant Metastatic Colorectal Cancer (mCRC) and Wnt Pathway Mutations
The purpose of this study is to assess the safety and anti-tumor activity of the triple combination of WNT974, LGX818 and cetuximab in BRAFV600-mutant mCRC with RNF43 mutations or RSPO fusions.
- Trial name
- NCT02278133
- Testing
- WNT974, LGX818, Cetuximab
- Where
- 19 sites in United States, Australia and 8 more
- Volunteers
- 20 people
- Sponsor
- Array BioPharma
Cystic Fibrosis
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D).
- Trial name
- VOCAL (NCT02445053)
- Testing
- ivacaftor
- Where
- 15 sites in Italy, Netherlands and 1 more
- Volunteers
- 75 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Metastatic Non-Small Cell Lung Cancer
Pre-Approval Access With Amivantamab (JNJ-61186372) in Participants With Metastatic Non-Small Cell Lung Cancer
The purpose of this expanded access program (EAP) is to provide amivantamab for the treatment of participants with metastatic non-small cell lung cancer who have epidermal growth factor receptor exon 20 insertion mutations, and…
- Trial name
- NCT04599712
- Testing
- Amivantamab
- Sponsor
- Janssen Research & Development, LLC
Carcinoma, Non-Small-Cell Lung
An Expanded Access Protocol for Mobocertinib in Refractory Non-small Cell Lung Cancer (NSCLC) Participants With Epidermal Growth Factor Receptor (EGFR) Exon20 Insertion Mutations
The purpose of this study is to provide expanded compassionate use access to mobocertinib, in participants with locally advanced or metastatic NSCLC harboring EGFR in-frame exon 20 insertion mutations and who have received at…
- Trial name
- NCT04535557
- Testing
- Mobocertinib 160 mg
- Sponsor
- Millennium Pharmaceuticals, Inc.
GBA1 Parkinson Disease
A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease
The goal of this clinical trial is to learn about the safety of CAP-003 gene therapy in adults with GBA1 associated Parkinson's Disease.
- Trial name
- NCT07011771
- Testing
- gene therapy
- Where
- 5 sites in United States
- Sponsor
- Capsida Biotherapeutics, Inc.
X-linked Hypophosphatemia
A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
A first-in-human study of KK8123 in adults with X-linked hypophosphatemia.
- Trial name
- NCT06525636
- Testing
- KK8123
- Where
- 10 sites in United States, Canada and 3 more
- Volunteers
- 39 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Advanced Solid Tumor, Ovarian Cancer
A Phase 1 Clinical Study of NXP800 in Subjects With Advanced Cancers and Expansion in Subjects With Ovarian Cancer
The purpose of the dose escalation phase is to evaluate the safety profile of escalating doses and dose schedules of NXP800. In the expansion phase the preliminary efficacy in subjects with ARID1a mutated ovarian clear cell and…
- Conditions
- Advanced Solid Tumor, Ovarian Cancer, Ovarian Clear Cell Carcinoma, Ovarian Clear Cell Tumor
- Trial name
- NCT05226507
- Testing
- NXP800
- Where
- 22 sites in United States, United Kingdom
- Volunteers
- 45 people
- Sponsor
- Nuvectis Pharma, Inc.
Carcinoma, Non-Small-Cell Lung
Afatinib in NSCLC With HER2 Mutation
to investigate effectiveness and safety of afatinib in the advanced NSCLC patients with HER2 mutations, previously treated with 1 or 2 chemotherapy regimens
- Trial name
- NCT02597946
- Testing
- afatinib, paclitaxel
- Where
- 9 sites in China, Malaysia
- Volunteers
- 18 people
- Sponsor
- Boehringer Ingelheim
Mutations in Exons 18 to 21
Pathomolecular Analysis of Rare EGFR Mutations in Advanced NSCLC
While current mutational analyses comprise exons 19 and 21 in which the majority of EGFR-mutations occur, this study aims at investigating the relevance of exon 18 and 20 mutations.
- Trial name
- NCT01215474
- Where
- Germany
- Volunteers
- 500 people
- Sponsor
- Provitro GmbH
Non Small Cell Lung Cancer, EGFR Exon 20 Mutation
A Phase 1/2 Trial of CLN-081 in Patients With Non-Small Cell Lung Cancer
CLN-081-001 is a Phase 1/2, open label, multi-center study of CLN-081 in patients with non-small cell lung cancer (NSCLC) harboring EGFR (epidermal growth factor receptor) exon 20 insertion mutations, to characterize the safety…
- Trial name
- REZILIENT1 (NCT04036682)
- Testing
- CLN-081
- Where
- 64 sites in United States, Hong Kong and 7 more
- Volunteers
- 284 people
- Sponsor
- Cullinan Therapeutics Inc.
AML With Gene Mutations
Dose Escalation and Expansion Study to Evaluate the Safety, PK, PD and Efficacy of ZE46-0134 in Adults With FLT3 Mutated or Spliceosome Mutated Relapsed or Refractory Acute Myeloid Leukemia
This is a clinical study aiming to assess pharmacokinetics, pharmacodynamics and preliminary efficacy of ZE46-0134 in patients with FLT3 and spliceosome mutated Relapsed or Refractory Acute Myeloid Leukemia
- Trial name
- NCT06366789
- Testing
- ZE46-0134
- Where
- 23 sites in United States, Australia and 2 more
- Volunteers
- 150 people
- Sponsor
- Lomond Therapeutics Holdings, Inc.
AMN, AMN Gene Mutation
Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments.
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- CYGNET (NCT05008874)
- Testing
- Natural History Observation
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 65 people
- Sponsor
- SwanBio Therapeutics, Inc.
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT04927247
- Testing
- Inclacumab
- Where
- 59 sites in United States, Brazil and 12 more
- Volunteers
- 72 people
- Sponsor
- Pfizer
Lung Neoplasm Malignant
A Clinical Study of MK-1084 With Other Treatments for Non-small Cell Lung Cancer (MK-3475-01F)
Researchers want to learn if MK-1084, the study medicine, can treat advanced or metastatic non-squamous NSCLC. MK-1084 is a targeted therapy, which is a treatment that works to control how specific types of cancer cells grow and…
- Trial name
- NCT07286149
- Testing
- MK-1084, Patritumab deruxtecan, Sacituzumab tirumotecan
- Where
- 29 sites in United States, Brazil and 9 more
- Volunteers
- 140 people
- Sponsor
- Merck Sharp & Dohme LLC
NSCLC (Non-small Cell Lung Cancer)
Real World Study of Icotinib in Adjuvant Post Surgical Treatment for Stage IA2 High Risk to IB EGFR Mutant Non Small Cell Lung Cancer
This is a multicenter, retrospective, observational real-world study designed based on real-world data. Eligible medical institutions that meet standardized data quality requirements and have an adequate sample size will be…
- Trial name
- NCT07806604
- Testing
- Icotinib, Routine Observational
- Where
- 2 sites in China
- Volunteers
- 2,572 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Advanced Solid Tumor
A Phase I/IIa Study of JAB-23E73 in Patients With Advanced Solid Tumors Harboring KRAS Gene Alteration
This is a multicenter, open-label, phase I/IIa to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary antitumor activity of pan-KRAS inhibitor JAB-23E73 in patients with advanced solid…
- Trial name
- NCT06959615
- Testing
- JAB-23E73
- Where
- 32 sites in China
- Volunteers
- 334 people
- Sponsor
- Jacobio Pharmaceuticals Co., Ltd.
Metastatic Breast Cancer, Breast Neoplasms
Treatment Patterns and Clinical Outcomes Among Talazoparib-Treated Adults With HER2-Negative mBC With gBRCA1/2m
This non-interventional retrospective study will describe real-world treatment patterns and clinical outcomes among adults with HER2-negative metastatic breast cancer with germline BRCA1/2 mutations who initiated talazoparib as a…
- Trial name
- NCT05141708
- Testing
- Talazoparib
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Pfizer
Carcinoma, Non-small Cell Lung
Phase III Study of Nazartinib (EGF816) Versus Erlotinib/Gefitinib in First-line Locally Advanced / Metastatic NSCLC With EGFR Activating Mutations
This is a phase III, open label, randomized controlled multi-center global study designed to evaluate the safety and efficacy of single agent nazartinib (EGF816) compared with investigator's choice (erlotinib or gefitinib) in…
- Trial name
- NCT03529084
- Testing
- EFG816, erlotinib or gefitinib
- Sponsor
- Novartis Pharmaceuticals
Non-small Cell Lung Cancer (NSCLC), Colorectal Cancer (CRC)
Study of BPI-572270 in Patients With Advanced Solid Tumors Harboring Specific Mutations in RAS
Evaluate the safety and tolerability of BPI-572270 in adult patients with specific RAS mutant advanced solid tumors.
- Conditions
- Non-small Cell Lung Cancer (NSCLC), Colorectal Cancer (CRC), Pancreatic Ductal Adenocarcinoma (PDAC), Advanced Solid Tumors
- Trial name
- NCT07435038
- Testing
- BPI-572270
- Where
- 7 sites in Australia, China
- Volunteers
- 120 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Urothelial Carcinoma
A Study of Disitamab Vedotin Alone or With Pembrolizumab in Urothelial Cancer That Expresses HER2
This study is being done to see if a drug called disitamab vedotin, alone or with pembrolizumab, works to treat HER2 expressing urothelial cancer. It will also test how safe the drug is for participants.
- Trial name
- NCT04879329
- Testing
- disitamab vedotin, pembrolizumab
- Where
- 221 sites in United States, Argentina and 10 more
- Volunteers
- 372 people
- Sponsor
- Seagen, a wholly owned subsidiary of Pfizer