Clinical trials: Phase 1
Jansen's Metaphyseal Chondrodysplasia
Safety and Efficacy of PTH-IA
Jansen s Metaphyseal Chondrodysplasia (JMC) is a very rare disorder with only approximately 30 people known to have the disease worldwide.
- Trial name
- NCT07541209
- Testing
- PTH-IA
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Primary Hyperoxaluria Type 1 (PH1)
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
The goal of the redePHine study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ABO-101 in participants with primary hyperoxaluria type 1 (PH1). The trial will consist of 2 Study Periods.
- Trial name
- NCT06839235
- Testing
- ABO-101
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 23 people
- Sponsor
- Arbor Biotechnologies
Suicide
Army-SPRING Army-Suicide Prevention Research
Every unit in the Connecticut Army National Guard has a Suicide Intervention Officer to provide suicide intervention education awareness for their unit, monitor for soldiers in crisis and connect soldiers with helping resources…
- Trial name
- NCT06547008
- Testing
- Army-SPRING, Risk Reduction Group (Control)
- Where
- 3 sites in United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Duchenne Muscular Dystrophy
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT04906460
- Testing
- WVE-N531
- Where
- 6 sites in United States, Jordan and 1 more
- Volunteers
- 25 people
- Sponsor
- Wave Life Sciences USA, Inc.
Medulloblastoma, Childhood, Medulloblastoma Recurrent
Testing the Safety and Tolerability of CX-4945 in Patients With Recurrent Medulloblastoma Who May or May Not Have Surgery
This is a multi center, Phase I, Phase II and surgical study of the CX-4945 drug (silmitasertib sodium) for patients with recurrent SHH (Sonic Hedgehog) medulloblastoma
- Conditions
- Medulloblastoma, Childhood, Medulloblastoma Recurrent, Medulloblastoma
- Trial name
- NCT03904862
- Testing
- CX 4945
- Where
- 12 sites in United States
- Volunteers
- 21 people
- Sponsor
- Pediatric Brain Tumor Consortium
Acute Leukemia, Acute Leukemia of Ambiguous Lineage
Revumenib for the Treatment of Acute Leukemia in Patients Post-Allogeneic Stem Cell Transplant
This phase I trial tests the safety, side effects, best dose and effectiveness of revumenib in treating patients with acute leukemia after allogeneic stem cell transplant.
- Conditions
- Acute Leukemia, Acute Leukemia of Ambiguous Lineage, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT06575296
- Testing
- Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- City of Hope Medical Center
Healthy Controls
Intranasal Insulin and Olanzapine Study in Healthy Volunteers
Antipsychotic (AP) medications are considered to be the gold standard treatment for psychotic disorders including schizophrenia.
- Trial name
- INI/OLA (NCT03741478)
- Testing
- OLANZapine 2.5 MG, Insulin Lispro 100 UNT/ML, Saline
- Where
- 2 sites in Canada
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- Centre for Addiction and Mental Health
Osteosarcoma Recurrent, Osteosarcoma in Children
Tegavivint With Gemcitabine in Patients With Relapsed or Refractory Osteosarcoma
The goal of this clinical trial is to define the maximum tolerated dose (MTD) and/or Recommended phase 2 dose (RP2D) of Tegavivint in combination with Gemcitabine in patients with relapsed or refractory osteosarcoma (OS).
- Conditions
- Osteosarcoma Recurrent, Osteosarcoma in Children, Relapsed Osteosarcoma, Refractory Osteosarcoma
- Trial name
- NCT07144254
- Testing
- Tegavivint, Gemcitabine
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Emory University
Leukemia, Myelodysplastic Syndromes
Clofarabine and High-Dose Melphalan Followed by Donor Stem Cell Transplant in Patients With Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, or Myelodysplastic Syndromes
RATIONALE: Giving chemotherapy, such as clofarabine and melphalan, before a donor stem cell transplant helps stop the growth of cancer or abnormal cells.
- Trial name
- NCT00641030
- Testing
- clofarabine, melphalan, gene expression analysis
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- City of Hope Medical Center
Vanishing White Matter Disease
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants.
- Trial name
- NCT05757141
- Testing
- Fosigotifator
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Calico Life Sciences LLC
Rhabdomyosarcoma
FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma
Background: Rhabdomyosarcoma (RMS) is a cancer of soft tissues. It is the most common soft tissue sarcoma seen in children. RMS cancer cells have a protein called FGFR4 on their surface.
- Trial name
- NCT06865664
- Testing
- fludarabine, cyclophosphamide, cetuximab
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Cancer Institute (NCI)
Parkinson Disease
Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cell Exosomes for Parkinson's Disease
This trial will study the safety and efficacy of intranasal instillation of cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cell exosomes for the treatment of Parkinson's Disease
- Trial name
- NCT05152394
- Testing
- cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cell exosomes
- Where
- 2 sites in United States, Greece
- Volunteers
- 20 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
DMG, DIPG
Blood-Brain Barrier Disruption Using Exablate Magnetic Resonance-Guided Low Intensity Focused Ultrasound With Microbubbles in Combination With T-Cell Infusion in Patients With Newly Diagnosed Diffuse Midline Glioma (LIFT)
This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for Diffuse Midline Glioma (DMG), an aggressive brain tumor with a very poor prognosis - an average one-year overall survival.
- Trial name
- LIFT (NCT07796061)
- Testing
- TAA-T Cell, BBB Disruption
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Children's National Research Institute
Long COVID, Post COVID-19 Condition
Taurine Supplementation in Adolescents With Post-COVID Condition
The COVID-19 pandemic has swept across the globe, affecting millions of individuals with varying degrees of severity. While many individuals recover from the acute phase of the infection, a significant proportion continue to…
- Conditions
- Long COVID, Post COVID-19 Condition, PASC Post Acute Sequelae of COVID 19
- Trial name
- TaurineLCPeds (NCT07682402)
- Testing
- Taurine
- Volunteers
- 30 people
- Sponsor
- University of Alberta
Soft Tissue Sarcoma, Primary Central Nervous System Carcinoma
Study of Autologous Tumor-Infiltrating Lymphocytes in Pediatric, Adolescent, and Young Adult Participants
This study is planned to test the safety and tolerability of the TIL regimen. The study will also test how well TIL fights cancer.
- Conditions
- Soft Tissue Sarcoma, Primary Central Nervous System Carcinoma, Melanoma, Rhabdomyosarcoma
- Trial name
- NCT06566092
- Testing
- LN-145/LN-144
- Where
- 5 sites in United States
- Volunteers
- 5 people
- Sponsor
- Iovance Biotherapeutics, Inc.
Neurofibromatosis 1
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
Background: NF1 is a genetic disease that causes tumors called atypical neurofibromas. These tumors, which arise from nerves, can cause serious medical problems. The only treatment is surgery.
- Trial name
- NCT04750928
- Testing
- Abemaciclib
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Amyotrophic Lateral Sclerosis
Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cell Intrathecal Injection for ALS
This trial will study the safety and efficacy of intrathecal injection of cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cells for the treatment of amyotrophic lateral sclerosis
- Trial name
- NCT05003921
- Testing
- cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cells
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Recurrent B Acute Lymphoblastic Leukemia, Refractory B Acute Lymphoblastic Leukemia
Recombinant Erwinia Asparaginase and Venetoclax in Combination With Blinatumomab for the Treatment of Relapsed or Refractory CD19 Positive B-cell Acute Lymphoblastic Leukemia
This phase I/Ib trial tests the safety and side effects of asparaginase Erwinia chrysanthemi-recombinant-rywn (recombinant Erwinia asparaginase) and venetoclax in combination with blinatumomab and how well the combination works…
- Trial name
- NCT07133997
- Testing
- Asparaginase Erwinia chrysanthemi, Biospecimen Collection, Blinatumomab
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- City of Hope Medical Center
Peripheral Blood Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation
The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
Background: Blood cancers (such as leukemias or lymphomas) often do not respond to standard treatments. A transplant of blood stem cells from a healthy donor can help people with these cancers.
- Trial name
- NCT05436418
- Testing
- Melphalan, Sirolimus, Total Body Irradiation (TBI)
- Where
- 2 sites in United States
- Volunteers
- 260 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Hematologic Neoplasms
Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies
Background: People with blood cancers often receive blood or bone marrow transplants. But even with these treatments, the risk of relapse is high.
- Trial name
- NCT05327023
- Testing
- donor lymphocyte infusion, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 430 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
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