Clinical trials: Phase 1
Colorectal Carcinoma, Endometrial Carcinoma
Tegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors
This phase I/II trial evaluates the highest safe dose, side effects, and possible benefits of tegavivint in treating patients with solid tumors that has come back (recurrent) or does not respond to treatment (refractory).
- Conditions
- Colorectal Carcinoma, Endometrial Carcinoma, Melanoma, Neuroblastoma
- Trial name
- NCT04851119
- Testing
- Biospecimen Collection, Dual X-ray Absorptiometry, Tegavivint
- Where
- 21 sites in United States
- Volunteers
- 147 people
- Sponsor
- Children's Oncology Group
Ischemic Stroke
Safety, Dose-Finding, and Efficacy of Transcranial Pulse Stimulation for Post-Stroke Neurorehabilitation
This study aims to evaluate the safety, tolerability, dose-response, and preliminary efficacy of transcranial pulse stimulation (TPS) for neurorehabilitation in patients with ischemic stroke.
- Trial name
- TPS-STROKE (NCT07784751)
- Testing
- Required Field
- Where
- Taiwan
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Taipei Hospital, Ministry of Health and Welfare
ALK Positive, ALK Positive Non-small Cell Lung Cancer
Study of NXP900 With Lorlatinib in ALK+ Lung Cancer
This is a multi-center, open label, Phase 1b study of NXP900 in combination with lorlatinib in subjects with advanced, ALK-positive non-small cell lung cancer (NSCLC)
- Conditions
- ALK Positive, ALK Positive Non-small Cell Lung Cancer, NSCLC, ALK+ Non-Small-Cell Lung Carcinoma
- Trial name
- NCT07784959
- Testing
- NXP900, Lorlatinib
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- Nuvectis Pharma, Inc.
Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Flotetuzumab for the Treatment of Pediatric Recurrent or Refractory Acute Myeloid Leukemia
This phase I trial studies the side effects, best dose of flotetuzumab and how well it works in treating patients with acute myeloid leukemia (AML) that has come back (recurrent) or has not responded to treatment (refractory).
- Trial name
- NCT04158739
- Testing
- Cytarabine, Flotetuzumab
- Where
- 19 sites in United States
- Volunteers
- 16 people
- Sponsor
- Children's Oncology Group
Childhood Fibrolamellar Carcinoma, Recurrent Childhood Fibrolamellar Carcinoma
Testing the Addition of an Anti-cancer Drug, DT2216, to the Usual Chemotherapy Treatment for Relapsed or Refractory Solid Tumors and Fibrolamellar Carcinoma
This phase I/II trial tests the safety, side effects and best dose of DT2216 in combination with irinotecan and how well it works in treating children, adolescents and young adults with solid tumors and fibrolamellar cancer that…
- Conditions
- Childhood Fibrolamellar Carcinoma, Recurrent Childhood Fibrolamellar Carcinoma, Recurrent Childhood Malignant Solid Neoplasm, Recurrent Fibrolamellar Carcinoma
- Trial name
- NCT06620302
- Testing
- Bcl-XL Proteolysis Targeting Chimera DT2216, Biospecimen Collection, Irinotecan
- Where
- 21 sites in United States
- Volunteers
- 81 people
- Sponsor
- Children's Oncology Group
Duchenne Muscular Dystrophy (DMD)
Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy
This is a pharmacokinetic study (PK Study) to better understand empagliflozin dosing in pediatric Duchenne muscular dystrophy patients.
- Trial name
- CHERISH (NCT07172971)
- Testing
- SGLT-2 inhibitor, SGLT2 inhibitor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Vanderbilt University Medical Center
Acute Lymphoblastic Leukemia ALL
Ribociclib in Combination With Everolimus and Dexamethasone in Relapsed ALL
This research study is evaluating a drug called ribociclib (LEE011) given in combination with everolimus and other standard of care chemotherapy drugs as a possible treatment for relapsed or refractory ALL.
- Trial name
- NCT03740334
- Testing
- Ribociclib, Dexamethasone, Everolimus
- Where
- 12 sites in United States
- Volunteers
- 45 people
- Sponsor
- Dana-Farber Cancer Institute
Carcinoma, Non-Small-Cell Lung, Thyroid Neoplasms
A Study of LOXO-260 in Cancer Patients With a Change in a Particular Gene (RET) That Has Not Responded to Treatment
The main purpose of this study is to learn more about the safety, side effects, and effectiveness of LOXO-260. LOXO-260 may be used to treat cancer that has a change in a particular gene (known as the RET gene).
- Trial name
- NCT05241834
- Testing
- LOXO-260
- Where
- 16 sites in United States, France
- Volunteers
- 70 people
- Sponsor
- Eli Lilly and Company
Propionic Acidemia
AAVrh10-PCCA Gene Therapy for Propionic Acidemia
Propionic acidemia is a genetic metabolic disorder characterized by metabolic acidosis, ketosis, vomiting, lethargy, cognitive impairment, and risk of death.
- Trial name
- NCT07643844
- Testing
- AAVrh10-PCCA low dose, AAVrh10-PCCA middle dose, AAVrh10-PCCA high dose
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Mayo Clinic
Neuroblastoma, Solid Tumor
APG-115 Alone or in Combination With APG-2575 in Recurrent or Refractory Neuroblastoma or Solid Tumors
An open, non-randomized Phase Ib-II trial of dose-escalation and cohorts expansion to evaluate the safety, pharmacokinetic profile and initial efficacy of APG-115 alone or in combination with APG-2575 in the treatment of…
- Trial name
- NCT05701306
- Testing
- APG-115, APG-2575
- Where
- 3 sites in China
- Volunteers
- 100 people
- Sponsor
- Ascentage Pharma Group Inc.
X-Linked Chronic Granulomatous Disease
Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease
The goal of this clinical trial is to evaluate the safety and potential efficacy of the EN-374 treatment regimen and identify a dose level for further evaluation in participants with x-linked chronic granulomatous disease.
- Trial name
- NCT06876363
- Testing
- EN-374
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 15 people
- Sponsor
- Ensoma
Neuroblastoma
A Study of LY3295668 Erbumine in Participants With Relapsed/Refractory Neuroblastoma
The reason for this study is to see if the study drug LY3295668 erbumine is safe in participants with relapsed/refractory neuroblastoma.
- Trial name
- NCT04106219
- Testing
- LY3295668 Erbumine, Topotecan, Cyclophosphamide
- Where
- 17 sites in United States, Australia and 7 more
- Volunteers
- 71 people
- Sponsor
- Eli Lilly and Company
Acute Leukemia, Myelodysplastic Syndrome
Study of QUAIL-100 in Pediatric and Young Adult Participants With High-Risk Hematologic Malignancies Who Have Received a Hematopoietic Stem Cell Transplantation
Study of QUAIL-100 in Patients With High Risk Acute Leukemia and Myelodysplastic Syndrome Who Have Received Hematopoietic Stem Cell Transplant
- Trial name
- NCT07573111
- Testing
- QUAIL-100
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Laguna Biotherapeutics, Inc.
Adenosine Deaminase Severe Combined Immune Deficiency
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human…
- Trial name
- NCT05432310
- Testing
- A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, Los Angeles
Neurofibromatosis Type 1
Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
This study in adolescent participants with NF1 who have inoperable PN is designed to evaluate the effect of a low fat meal on steady state selumetinib exposure; to assess the effect on GI tolerability when selumetinib is dosed…
- Trial name
- NCT05101148
- Testing
- Selumetinib
- Where
- 9 sites in United States, Poland and 2 more
- Volunteers
- 24 people
- Sponsor
- AstraZeneca
Recurrent B Acute Lymphoblastic Leukemia, Recurrent Mixed Phenotype Acute Leukemia
Blinatumomab and Nivolumab With or Without Ipilimumab in Treating Patients With Poor-Risk Relapsed or Refractory CD19+ Precursor B-Lymphoblastic Leukemia
This phase I trial studies the side effects and best dose of blinatumomab when given with nivolumab alone or nivolumab and ipilimumab in treating patients with poor-risk CD19+ precursor B-lymphoblastic leukemia that has come back…
- Conditions
- Recurrent B Acute Lymphoblastic Leukemia, Recurrent Mixed Phenotype Acute Leukemia, Refractory B Acute Lymphoblastic Leukemia, Refractory Mixed Phenotype Acute Leukemia
- Trial name
- NCT02879695
- Testing
- Biospecimen Collection, Blinatumomab, Bone Marrow Aspiration and Biopsy
- Where
- 7 sites in United States
- Volunteers
- 28 people
- Sponsor
- National Cancer Institute (NCI)
Primary Hyperoxaluria Type 1, PH1
Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)
This is a single-arm, open-label, single-dose dose-escalation study. It aims to evaluate the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to preliminarily assess the effect…
- Trial name
- NCT07776626
- Testing
- YOLT-203
- Where
- China
- Volunteers
- 9 people
- Sponsor
- YolTech Therapeutics Co., Ltd
Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma
Rituximab + High-Dose Methylprednisolone Debulking Prior to Venetoclax for CLL & SLL Patients
The purpose of the study is to investigate whether the combination of rituximab and high dose methylprednisolone can be given together, can reduce the amount of cancer cells that are present prior to starting venetoclax, and…
- Trial name
- NCT04981912
- Testing
- HDMP + rituximab as a means of debulking prior to initiating venetoclax.
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of California, San Diego
Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IIIA Breast Cancer AJCC v8
Personalized Neoantigen Peptide-Based Vaccine in Combination With Pembrolizumab for Treatment of Advanced Solid Tumors
This phase I/II trial tests the safety and tolerability of an experimental personalized vaccine when given by itself and with pembrolizumab in treating patients with solid tumor cancers that have spread to other places in the…
- Conditions
- Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IIIA Breast Cancer AJCC v8, Anatomic Stage IIIB Breast Cancer AJCC v8, Anatomic Stage IIIC Breast Cancer AJCC v8
- Trial name
- PNeoVCA (NCT05269381)
- Testing
- Cyclophosphamide, Neoantigen Peptide Vaccine, Pembrolizumab
- Where
- United States
- Volunteers
- 138 people
- Sponsor
- Mayo Clinic
Angelman Syndrome
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
- Trial name
- NCT05127226
- Testing
- ION582
- Where
- 11 sites in United States, Australia and 4 more
- Volunteers
- 71 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
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