Clinical trials: Phase 1
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
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)
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
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
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
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
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)
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
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
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
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)
Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia
The goal of this clinical trial is to learn if sonrotoclax (BGB-11417) is safe and may help treat children and adolescents with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) that has come back after treatment…
- Conditions
- Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, B-cell Acute Lymphoblastic Leukemia, Pediatric Cancer
- Trial name
- NCT07770698
- Testing
- sonrotoclax, Azacitidine, Inotuzumab ozogamicin
- Volunteers
- 30 people
- Sponsor
- BeOne Medicines
Myeloid Chimerism
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some…
- Trial name
- NCT04008368
- Testing
- CliniMACS CD34 Reagent
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
B-NHL, B-Non Hodgkin Lymphoma
CD19/CD22 Bicistronic Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory B Cell Malignancies
Background: Acute lymphoblastic leukemia (ALL) is the most common cancer in children. About 90% of children and young adults who are treated for ALL can now be cured.
- Conditions
- B-NHL, B-Non Hodgkin Lymphoma, Acute Lymphocytic Leukemia, Acute Lymphoblastic Leukemia
- Trial name
- NCT05442515
- Testing
- CD19/CD22-CAR-transduced T cells, cyclophosphamide, fludarabine
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Cancer Institute (NCI)
Catecholaminergic Polymorphic Ventricular Tachycardia
A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
This is a Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in participants with catecholaminergic polymorphic ventricular tachycardia (CPVT).
- Trial name
- ARTEMIS (NCT07148089)
- Testing
- SGT-501
- Where
- 5 sites in United States, Canada
- Volunteers
- 18 people
- Sponsor
- Solid Biosciences Inc.
Sickle Cell Disease
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell…
- Trial name
- NCT03279094
- Testing
- Hematopoietic stem cell transplantation
- Sponsor
- City of Hope Medical Center
Nicotine Vaping, Nicotine Use Disorder
Testing the Feasibility and Acceptability of Social Media and Digital Therapeutics to Decrease Vaping Behaviors
Use of vaping products (e.g., electronic nicotine delivery systems, e-cigarettes) has been increasing rapidly, particularly among teens and young adults.
- Trial name
- NCT05994209
- Testing
- quitSTART Mobile Application, Chatbot Feature in quitSTART Mobile Application
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Washington University School of Medicine