Clinical trials: Genetic Change
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Anatomic Stage IV Breast Cancer AJCC v8, Metastatic HER2-Negative Breast Carcinoma
Testing the Use of Fulvestrant and Binimetinib Targeted Treatment for NF1 Mutation in Hormone Receptor-Positive Metastatic Breast Cancer (A ComboMATCH Treatment Trial)
This phase II ComboMATCH treatment trial compares the usual treatment alone (fulvestrant) to using binimetinib plus the usual treatment in patients with hormone receptor positive breast cancer that has spread from where it first…
- Conditions
- Anatomic Stage IV Breast Cancer AJCC v8, Metastatic HER2-Negative Breast Carcinoma, Metastatic Hormone Receptor-Positive Breast Carcinoma
- Trial name
- NCT05554354
- Testing
- Binimetinib, Biopsy Procedure, Biospecimen Collection
- Where
- 196 sites in United States, Puerto Rico
- Volunteers
- 1 people
- Sponsor
- National Cancer Institute (NCI)
Cancer, Cancer Metastatic
A Basket Trial of an ERK1/2 Inhibitor (LY3214996) in Combination With Abemaciclib.
The purpose of CTO-IUSCC-0730 study is to assess the clinical efficacy of LY3214996 in combination with abemaciclib at the recommended phase 2 dose of LY3214996 200 mg orally daily and abemaciclib 150 mg orally twice daily.
- Conditions
- Cancer, Cancer Metastatic, BRAF V600E, MEK1 Gene Mutation
- Trial name
- NCT04534283
- Testing
- Abemaciclib, LY3214996
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Anita Turk
Clinical Stage IV Cutaneous Melanoma AJCC v8, Metastatic Melanoma
Testing the Addition of the Anti-cancer Drug, Tazemetostat, to the Usual Treatment (Dabrafenib and Trametinib) for Metastatic Melanoma That Has Progressed on the Usual Treatment
This phase I/II trial investigates the best dose, possible benefits and/or side effects of tazemetostat in combination with dabrafenib and trametinib in treating patients with melanoma that has a specific mutation in the BRAF…
- Trial name
- NCT04557956
- Testing
- Biopsy Procedure, Computed Tomography, Dabrafenib Mesylate
- Where
- 17 sites in United States
- Volunteers
- 16 people
- Sponsor
- National Cancer Institute (NCI)
Diffuse Large B Cell Lymphoma
Dose Escalation Study in Patients With Relapsed or Refractory DLBCL and MyD88 L265P Mutation
Recent reports have identified a specific oncogenic mutation L265P of the MYD88 gene in approximately 30% of the patients with the activated B-cell (ABC) type of Diffuse Large B Cell Lymphoma (DLBCL).
- Trial name
- NCT02252146
- Testing
- IMO-8400
- Where
- 12 sites in United States
- Volunteers
- 6 people
- Sponsor
- Idera Pharmaceuticals, Inc.
Locally Advanced Bladder Urothelial Carcinoma, Metastatic Transitional Cell Carcinoma
Sapanisertib in Treating Patients With Locally Advanced or Metastatic Bladder Cancer With TSC1 and/or TSC2 Mutations
This pilot phase II trial studies how well sapanisertib works in treating patients with bladder cancer that has spread from where it started to nearby tissue or lymph nodes (locally advanced) or other places in the body…
- Conditions
- Locally Advanced Bladder Urothelial Carcinoma, Metastatic Transitional Cell Carcinoma, Metastatic Urothelial Carcinoma, Recurrent Bladder Carcinoma
- Trial name
- NCT03047213
- Testing
- Sapanisertib
- Where
- 20 sites in United States
- Volunteers
- 17 people
- Sponsor
- National Cancer Institute (NCI)
Carcinoma, Non-Small-Cell Lung
A Study of Combination Amivantamab and Carboplatin-Pemetrexed Therapy, Compared With Carboplatin-Pemetrexed, in Participants With Advanced or Metastatic Non-Small Cell Lung Cancer Characterized by Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertions
The purpose of this study is to compare the efficacy, as demonstrated by progression-free survival (PFS), in participants treated with amivantamab in combination with chemotherapy, versus chemotherapy alone in participants with…
- Trial name
- PAPILLON (NCT04538664)
- Testing
- Amivantamab, Pemetrexed, Carboplatin
- Where
- 232 sites in United States, Australia and 24 more
- Volunteers
- 308 people
- Sponsor
- Janssen Research & Development, LLC
Colorectal Cancer
Panitumumab and Irinotecan as Third-Line Therapy in Treating Patients With Metastatic Colorectal Cancer
RATIONALE: Monoclonal antibodies, such as panitumumab, can block tumor growth in different ways. Some block the ability of tumor cells to grow and spread.
- Trial name
- PIMABI (NCT00655499)
- Testing
- Panitumumab, Irinotecan hydrochloride, Chromogenic in situ hybridization
- Where
- 9 sites in France
- Volunteers
- 65 people
- Sponsor
- GERCOR - Multidisciplinary Oncology Cooperative Group
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
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
Carcinoma, Non-Small-Cell Lung, ErbB Receptors
Afatinib Treatment for Patients With EGFR Mutation Positive NSCLC Who Are Age 70 or Older
Continuous treatment until progression or occurence of intolerable Adverse Event (AE) or end of trial. The end of trial is one year after the last patient has entered the study.
- Trial name
- NCT02514174
- Testing
- Afatinib
- Where
- 10 sites in United States
- Volunteers
- 25 people
- Sponsor
- Boehringer Ingelheim
Carcinoma, Non-Small-Cell Lung
Evaluation of the Impact of Nurse-led Telephone on Treatment Compliance
The study will take place over a period of 27 months. The recruitment phase will last approximately 24 months and each patient will participate for approximately 3 months.
- Trial name
- NCT02483416
- Where
- 36 sites in France
- Volunteers
- 30 people
- Sponsor
- Boehringer Ingelheim
Hemochromatosis, Cerebrovascular Accident
Stroke With Transfusions Changing to Hydroxyurea
The purpose of this study is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iron overload in children with…
- Conditions
- Hemochromatosis, Cerebrovascular Accident, Anemia, Sickle Cell, Hematologic Diseases
- Trial name
- NCT00122980
- Testing
- Red Cell Transfusions, Iron Chelation, Hydroxyurea
- Where
- 28 sites in United States
- Volunteers
- 134 people
- Sponsor
- St. Jude Children's Research Hospital
Non-small Cell Lung Cancer
Study of Pemetrexed + Platinum Chemotherapy With or Without Pembrolizumab (MK-3475) in Adults With Tyrosine Kinase Inhibitor- (TKI)-Resistant Epidermal Growth Factor Receptor- (EGFR)-Mutated Metastatic Non-squamous Non-small Cell Lung Cancer (NSCLC) (MK-3475-789/KEYNOTE-789)
The purpose of this study is to evaluate the efficacy and safety of pemetrexed plus platinum chemotherapy (carboplatin or cisplatin) with or without pembrolizumab (MK-3475; KEYTRUDA®) in the treatment of adults with the following…
- Trial name
- NCT03515837
- Testing
- pembrolizumab, pemetrexed, carboplatin
- Where
- 158 sites in United States, Australia and 15 more
- Volunteers
- 492 people
- Sponsor
- Merck Sharp & Dohme LLC
Ewing Sarcoma, PEComa
Nivolumab (Opdivo®) Plus ABI-009 (Nab-rapamycin) for Advanced Sarcoma and Certain Cancers
This study investigates the safety/toxicity and potential anti-tumor activity of sequential administration of nivolumab and escalating doses of the mammalian target of rapamycin (mTOR) inhibitor nab-rapamycin (ABI-009) in…
- Conditions
- Ewing Sarcoma, PEComa, Epithelioid Sarcoma, Desmoid Tumor
- Trial name
- NCT03190174
- Testing
- Nab-Rapamycin, Nivolumab
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Sarcoma Oncology Research Center, LLC
Epilepsy
Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
This is a phase 2, crossover study of Ataluren for the treatment of nonsense mutation Dravet syndrome or cyclin-dependent kinase-like 5 (CDKL5) deficiency, resulting in drug-resistant epilepsy.
- Trial name
- NCT02758626
- Testing
- ataluren
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Duchenne Muscular Dystrophy
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) 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
- NCT00759876
- Testing
- Ataluren
- Where
- 3 sites in United States
- Volunteers
- 36 people
- Sponsor
- PTC Therapeutics
Atopic Dermatitis
A Study to Evaluate Adverse Events and Change in Disease Activity Comparing Oral Upadacitinib to Subcutaneous Dupilumab in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis
Atopic dermatitis (AD) is a skin condition that may cause a rash and itching due to inflammation of the skin. Therapies spread over the skin may not be enough to control the AD in trial participants who require systemic…
- Trial name
- Level Up (NCT05601882)
- Testing
- Dupilumab, Upadacitinib
- Where
- 257 sites in United States, Australia and 27 more
- Volunteers
- 920 people
- Sponsor
- AbbVie
Solid Tumours
Japan PhI/II of GSK2118436 and GSK1120212 Combination in Subjects With BRAF V600E/K Mutation Positive Advanced Solid Tumors (Phase I Part) or Cutaneous Melanoma (Phase II Part)
This is a Japanese Phase I/II, open-label, non-controlled study to evaluate the safety, tolerability, pharmacokinetic profile, and efficacy of the combination of GSK2118436 and GSK1120212 in subjects with BRAF V600E/K mutation…
- Trial name
- NCT01928940
- Testing
- dabrafenib, trametinib
- Where
- 2 sites in Japan
- Volunteers
- 12 people
- Sponsor
- GlaxoSmithKline
Chronic Myelogenous Leukemia - Chronic Phase
Asciminib in Monotherapy for Chronic Myeloid Leukemia in Chronic Phase (CML-CP) With and Without T315I Mutation
This study was a multicenter Phase IIIb open-label, three-cohort study of asciminib in patients with CML-CP without T315I mutation who have had at least 2 prior TKIs and CML-CP harboring the T315I mutation with at least 1 prior…
- Trial name
- AIM4CML (NCT04666259)
- Testing
- ABL001
- Where
- 26 sites in United States
- Volunteers
- 56 people
- Sponsor
- Novartis Pharmaceuticals