Clinical trials: Phase 1
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma
Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...
Background: Some people with cancer have solid tumors. Others have refractory leukemia. This may not go away after treatment. Researchers want to see if a drug called TURALIO(R) can shrink tumors or stop them from growing.
- Conditions
- Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Promyelocytic, Acute, Sarcoma
- Trial name
- NCT02390752
- Testing
- TURALIO(R)
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- National Cancer Institute (NCI)
Graft Versus Host Disease (cGvHD)
Ex Vivo-Expanded Regulatory T Cells Plus Low-Dose Interleukin-2 for Steroid-Refractory Chronic GVHD
Hematopoietic Stem Cell Transplant (HCT) has been used to treat children and adults who have leukemia, lymphoma and other cancers of blood and immune cells since the 1970s. For many of these forms of cancer, HCT works well.
- Trial name
- EVENT (NCT06991361)
- Testing
- EVE-Treg
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Sarah Nikiforow, MD
High-risk GD2 Positive Ewing Sarkoma
International Phase I Trial of Dinutuximab Beta With VDC/IE in GD2-Positive Ewing Sarkoma
The goal of the study is to investigate the feasibility, toxicity, and biological activity of the treatment with dinutuximab beta in combination with standard chemotherapy in EWS to give high-risk patients with a GD2 positiv…
- Trial name
- NCT06839703
- Testing
- Dinutuximab beta
- Where
- 5 sites in Germany
- Volunteers
- 18 people
- Sponsor
- Prof. Dr. Dr. Birgit Burkhardt
Stargardt Disease, Stargardt Macular Dystrophy
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye.
- Conditions
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- Trial name
- DEVOTE (NCT07734064)
- Testing
- ASP2020
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Seasonal Influenza
A Study to Evaluate the Safety, Reactogenicity, and Immunogenicity of mRNA-1010 in Healthy Children
The purpose of this study is to evaluate safety, reactogenicity, and immunogenicity of mRNA-1010 in healthy children (9 to <18 years of age).
- Trial name
- NCT07870330
- Testing
- mRNA-1010, Fluarix Trivalent
- Where
- 6 sites in United States
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- ModernaTX, Inc.
Malignant Glioma
A Study of the Drug Selinexor With Radiation Therapy in Patients With Newly-Diagnosed Diffuse Intrinsic Pontine (DIPG) Glioma and High-Grade Glioma (HGG)
This phase I/II trial tests the safety, side effects, and best dose of selinexor given in combination with standard radiation therapy in treating children and young adults with newly diagnosed diffuse intrinsic pontine glioma…
- Trial name
- NCT05099003
- Testing
- Biopsy Procedure, Magnetic Resonance Imaging, Radiation Therapy
- Where
- 129 sites in United States, Australia and 2 more
- Volunteers
- 132 people
- Sponsor
- National Cancer Institute (NCI)
Advanced Solid Tumor
Study of Lunresertib Alone or in Combination With RP-3500 or Zedoresertib (Debio 0123) in Patients With Advanced Solid Tumors
The primary purpose of this study is to assess the safety and tolerability of lunresertib alone and in combination with RP-3500 or in combination with zedoresertib in patients with eligible advanced solid tumors, determine the…
- Trial name
- MYTHIC (NCT04855656)
- Testing
- Lunresertib, RP-3500, Zedoresertib
- Where
- 26 sites in United States, Canada and 3 more
- Volunteers
- 464 people
- Sponsor
- Debiopharm International SA
Duchenne Muscular Dystrophy
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
- Trial name
- ASCEND (NCT06817382)
- Testing
- INS1201
- Where
- 10 sites in United States
- Volunteers
- 12 people
- Sponsor
- Insmed Gene Therapy LLC
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
Alpha/Beta CD19+ Depleted Haploidentical Transplantation + Zometa for Pediatric Hematologic Malignancies and Solid Tumors
This phase I trial studies the safety of transplantation with a haploidentical donor peripheral blood stem cell graft depleted of TCRαβ+ cells and CD19+ cells in conjunction with the immunomodulating drug, Zoledronate, given in…
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Hodgkin Lymphoma, Non-Hodgkin Lymphoma
- Trial name
- NCT02508038
- Testing
- TCRαβ+/CD19+ depleted Haploidentical HSCT, Zoledronate
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of Wisconsin, Madison
WHIM, Warts
Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM
Background: Warts, hypogammaglobulinemia, infections and myelokathexis syndrome (WHIMs) is a rare disorder that affects the immune system. People with WHIMs can have severe infections all over their body.
- Conditions
- WHIM, Warts, Hypogammaglobulinemia, Immunodeficiency
- Trial name
- NCT07775313
- Testing
- Busulfan, Palifermin, Plerixafor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Neuroblastoma (NBL), Ewing Sarcoma
GD2IL18CART Combined With Bevacizumab for Relapsed and Refractory GD2-positive Solid Tumors
This study will test a CAR T-cell treatment combined with bevacizumab in children and young adults with relapsed or refractory neuroblastoma, Ewing sarcoma, or osteosarcoma.
- Conditions
- Neuroblastoma (NBL), Ewing Sarcoma, Osteosarcoma
- Trial name
- GD2CART-BEV (NCT07897968)
- Testing
- Chimeric Antigen Receptor (CAR) Therapy Infusion, Bevacizumab (Avastin)
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Universität Münster
Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency
Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)
The goal of this clinical trial is to learn if the treatment is a safe, tolerable, and efficacious treatment for adults and children with Aspartylglucosaminuria (AGU).
- Trial name
- NCT07530796
- Testing
- scAAV9/AGA
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Rare Trait Hope
X-linked Severe Combined Immunodeficiency (XSCID)
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
This Phase I/II clinical trial will study the safety and potential benefits of gene therapy for up to 35 people aged 2 to 50 with X-linked severe combined immunodeficiency (XSCID), a rare genetic condition that makes it difficult…
- Trial name
- NCT01306019
- Testing
- Campath, Palifermin, Busulfan
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Advanced Solid Tumors, Ewing Sarcoma
A Phase 1/1B Study of ST-01156, a Small Molecule RBM39 Degrader, in Patients With Advanced Solid Malignancies
A Phase 1/1B Study of ST-01156 in Patients with Advanced Solid Malignancies
- Conditions
- Advanced Solid Tumors, Ewing Sarcoma, Hepatocellular Carcinoma (HCC), Biliary Tract Cancer (BTC)
- Trial name
- NCT07197554
- Testing
- ST-01156
- Where
- 6 sites in United States
- Volunteers
- 171 people
- Sponsor
- SEED Therapeutics, Inc.
Depression - Major Depressive Disorder, Generalized Anxiety Disorder (GAD)
Microbiota Modulation for Psychiatry Patients
The investigators aim to confirm pilot study findings (REB 119638) on the effects of delayed release apple cider vinegar (drACV).
- Conditions
- Depression - Major Depressive Disorder, Generalized Anxiety Disorder (GAD), Anxiety
- Trial name
- ACV-MTPIES-002 (NCT07899424)
- Testing
- Dietary Supplement: Acetate (Apple Cider Vinegar)
- Volunteers
- 62 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Somatostatin Receptor Positive, NETs
Phase I Trial to Determine the Dose and Evaluate the PK and Safety of Lutetium Lu 177 Edotreotide Therapy in Pediatric Participants With SSTR-positive Tumors
The purpose of the study is to determine the appropriate pediatric dosage and evaluate the pharmacokinetics (PK) and safety of Lutetium Lu 177 Edotreotide Targeted Radiopharmaceutical Therapy (RPT) as a monotherapy or following…
- Conditions
- Somatostatin Receptor Positive, NETs, Lymphoma, Solid Tumor
- Trial name
- KinLET (NCT06441331)
- Testing
- Lutetium Lu 177-Edotreotide, Amino Acid Solution
- Where
- 9 sites in United States, France and 1 more
- Volunteers
- 20 people
- Sponsor
- ITM Solucin GmbH
Sickle Cell Disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512.
- Trial name
- NCT06546670
- Testing
- ITU512
- Where
- 14 sites in United States, Brazil and 1 more
- Volunteers
- 161 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Healthy
A Study to Evaluate Safety, Tolerability, and Immunogenicity of V350A and V350B in Healthy Participants (V350-001).
Epstein Barr virus (EBV) is a virus which can cause infectious mononucleosis and is associated with certain kinds of cancer and multiple sclerosis.
- Trial name
- NCT06655324
- Testing
- V350A, V350B
- Where
- 9 sites in United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Neurofibromatosis 1, Neurofibromatosis Type 1
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Background: Plexiform neurofibromas are tumors that grow in and around nerves. The only way to treat them is with surgery. Some of these tumors cannot be completely removed.
- Conditions
- Neurofibromatosis 1, Neurofibromatosis Type 1, NF1, Neurofibroma, Plexiform
- Trial name
- NCT01362803
- Testing
- AZD6244
- Where
- 4 sites in United States
- Volunteers
- 99 people
- Sponsor
- National Cancer Institute (NCI)
Glioblastoma (GBM), Diffuse Midline Glioma (DMG)
Intrathecal Azacitidine and Nivolumab in Patients With Recurrent High-grade Glioma
This is a single-arm open-label phase 1 dose escalation/expansion trial assessing the safety and efficacy of concurrent intrathecal azacitidine and intrathecal nivolumab in recurrent high-grade glioma.
- Conditions
- Glioblastoma (GBM), Diffuse Midline Glioma (DMG), Astrocytoma, IDH-Mutant, Grade 4, Diffuse Hemispheric Glioma, H3 G34-Mutant
- Trial name
- NCT06896110
- Testing
- Nivolumab, Azacitidine (AZA), lumbar puncture
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Andrew P. Groves