Clinical trials: Phase 1
Bronchopulmonary Dysplasia
Inhaled Ciclesonide Study in Preterm Infants
Our overall objective is to conduct a safety study with inhaled ciclesonide to evaluate known glucocorticoids (sGC)-related acute and intermediate toxic effects while measuring for the first time in neonates its systemic…
- Trial name
- NCT06589245
- Testing
- Alvesco Inhalant Product
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Venkatesh Sampath
HIV Infection
Very Early Intensive Treatment of Infants Living With HIV to Achieve HIV Remission
The study will explore the effects of early intensive antiretroviral therapy (ART) with or without a broadly neutralizing antibody (bNAb) on achieving HIV remission (HIV RNA below the limit of detection of the assay) among…
- Trial name
- NCT02140255
- Testing
- Nucleoside Reverse Transcriptase Inhibitors (NRTIs), Nevirapine (NVP), Lopinavir/Ritonavir (LPV/r)
- Where
- 47 sites in United States, Argentina and 11 more
- Volunteers
- 1,120 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Congenital Hemolytic Anemia, Sickle Cell Disease
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT).
- Trial name
- NCT00061568
- Testing
- Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis
- Where
- United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Diffuse Midline Glioma or Diffuse Intrinsic Pontine Glioma, High Grade Gliomas
NgFUS NIVO: NeuroNavigation-Guided Focused Ultrasound With Nivolumab in Relapsed and Progressive DMG and Other High Grade Brain Tumors
This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for progressive and relapsed diffuse midline glioma (DMG) and other progressive and relapsed high-grade brain tumors.
- Conditions
- Diffuse Midline Glioma or Diffuse Intrinsic Pontine Glioma, High Grade Gliomas, Medulloblastoma Recurrent, Ependymoma Recurrent
- Trial name
- NCT07664176
- Testing
- NeuroNavigation-Guided Focused Ultrasound (NgFUS), Lumason, Nivolumab
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Children's National Research Institute
Acute Leukemia, Myelodysplastic Syndromes
Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled.
- Conditions
- Acute Leukemia, Myelodysplastic Syndromes, Severe Aplastic Anemia (SAA)
- Trial name
- NCT07796620
- Testing
- Sirolimus, Ruxolitinib, MMF
- Volunteers
- 40 people
- Sponsor
- Peking University People's Hospital
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented…
- Trial name
- NCT05683860
- Testing
- WVE-004 10 mg Q12W
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Wave Life Sciences USA, Inc.
PANS Pediatric Acute-Onset Neuropsychiatric Syndrome
Investigating The Effects Of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) On Paediatric Acute-onset Neuropsychiatric Syndrome (PANS)
This study investigates the effectiveness of a medicinal cannabis extract (NTI164) with 0.08% THC in treating children with pediatric acute-onset neuropsychiatric syndrome (PANS) over a period of 18 to 54 weeks.
- Trial name
- CannaPANS (NCT06621888)
- Testing
- NTI164
- Where
- 2 sites in Australia
- Volunteers
- 18 people
- Sponsor
- Fenix Innovation Group
Sickle Cell Disease, Executive Dysfunction
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork…
- Conditions
- Sickle Cell Disease, Executive Dysfunction, Cognitive Impairment, Attention Deficit/Hyperactivity Disorder (ADHD)
- Trial name
- NCT07226219
- Testing
- Extended-Release Methylphenidate
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- St. Jude Children's Research Hospital
JAK1 GOF, STAT1 GOF
Upadacitinib in Treatment of JAK/STAT Pathway Disorders With Activating Mutations
This study focuses on a genetic condition that affects the Janus Kinase-Signal Transducer and Activator of Transcription (JAK-STAT) immune signaling pathway.
- Conditions
- JAK1 GOF, STAT1 GOF, STAT3 GOF, STAT5B GOF
- Trial name
- SAFE-JAKi (NCT07670156)
- Testing
- Upadacitinib
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Lisa Satter
Systemic Sclerosis (SSc)
Clinical Study of CD19 CAR-T in the Treatment of Refractory Systemic Sclerosis
This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of childhood-onset systemic sclerosis
- Trial name
- NCT06792344
- Testing
- anti-CD19 CAR-T cells
- Where
- 2 sites in China, Christmas Island
- Volunteers
- 12 people
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine
Autism Spectrum Disorder
Safety and Efficacy of Oral Full-Spectrum Medicinal Cannabis Plant Extract in Children With Autism Spectrum Disorder.
This is a 20-week open-label study to evaluate the safety and efficacy of full-spectrum medicinal cannabis plant extract < 0.08% THC (FEN164) in children with Autism Spectrum Disorder.
- Trial name
- NCT05516407
- Testing
- FEN164
- Where
- Australia
- Volunteers
- 18 people
- Sponsor
- Fenix Innovation Group
Chronic Granulomatous Disease (CGD)
Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
Background: X-Linked Chronic Granulomatous Disease (X-CGD) is caused by a gene mutation that makes the immune system to not work properly.
- Trial name
- NCT07113743
- Testing
- Busulfan, Tocilizumab, Eltrombopag
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Sanfilippo Syndrome Type A
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device…
- Trial name
- NCT06567769
- Testing
- GC1130A
- Where
- 4 sites in United States, South Korea
- Volunteers
- 9 people
- Sponsor
- GC Biopharma Corp
Acute Skin Graft Versus Host Disease (GVHD)
Topical Ruxolitinib in Acute Skin GVHD (Graft Versus Host Disease)
The purpose of this study is to see if ruxolitinib, a Janus Kinase (JAK) 1/2 inhibitor, is safe for people who have acute skin graft versus host disease (GVHD) following allogenic hematopoietic stem cell transplantation (HSCT).
- Trial name
- NCT07801248
- Testing
- Ruxolitinib 1.5% cream
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Ewing Sarcoma, Colorectal Adenocarcinoma
Phase 1 Study of INBRX-109 in Subjects With Locally Advanced or Metastatic Solid Tumors Including Sarcomas
This is a first-in-human, open-label, non-randomized, three-part phase 1 trial of INBRX-109, which is a recombinant humanized tetravalent antibody targeting the human death receptor 5 (DR5).
- Trial name
- NCT03715933
- Testing
- INBRX-109, Irinotecan, Temozolomide
- Where
- 37 sites in United States, France and 4 more
- Volunteers
- 411 people
- Sponsor
- Inhibrx Biosciences, Inc
Neoplasms, Brain, Glioblastoma Multiforme
HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors
This study is a clinical trial to determine the safety of inoculating G207 (an experimental virus therapy) into a recurrent or refractory cerebellar brain tumor.
- Conditions
- Neoplasms, Brain, Glioblastoma Multiforme, Glioblastoma of Cerebellum, Neoplasms
- Trial name
- NCT03911388
- Testing
- G207
- Where
- 3 sites in United States
- Volunteers
- 24 people
- Sponsor
- M.D. Anderson Cancer Center
Recurrent Gliosarcoma, Recurrent Supratentorial Glioblastoma
Phase I CB-NK-TGF-ßR2-/NR3C1- in rGBM
This phase I trial is to find out the best dose, possible benefits and/or side effects of engineered natural killer (NK) cells containing deleted TGF-betaR2 and NR3C1 (cord blood [CB]-NK-TGF-betaR2-/NR3C1-) in treating patients…
- Conditions
- Recurrent Gliosarcoma, Recurrent Supratentorial Glioblastoma, Supratentorial Gliosarcoma
- Trial name
- NCT04991870
- Testing
- Cord Blood-derived Expanded Allogeneic Natural Killer Cells, Resection
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- M.D. Anderson Cancer Center
Allogeneic Stem Cell Transplant, Viral Infection
Donor-Derived Viral Specific T-cells (VSTs)
In this research study, the investigators want to learn more about the use of donor-derived viral specific T-cells (VSTs) to treat viral infections that occur after allogeneic stem cell transplant.
- Conditions
- Allogeneic Stem Cell Transplant, Viral Infection, Viral Reactivation
- Trial name
- VSTs (NCT02048332)
- Testing
- Viral specific VST Infusion
- Where
- 3 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Hematopoietic and Lymphoid Cell Neoplasm, Immunocompromised
Donor T Cell Therapy in Treating Immunocompromised Patients With Adenovirus-Related Disease
This phase I trial studies the side effects of allogeneic adenovirus-specific cytotoxic T lymphocytes (donor T cell therapy) and to see how well they work in treating patients with a weakened immune system (immunocompromised) and…
- Trial name
- NCT03425526
- Testing
- Allogeneic Adenovirus-specific Cytotoxic T Lymphocytes
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- M.D. Anderson Cancer Center
Bowel Preparation
Study Assessing CLENPIQ as Bowel Preparation for Pediatric Colonoscopy
Bowel preparation for pediatric colonoscopy.
- Trial name
- PICCOLO (NCT04113382)
- Testing
- CLENPIQ, MIRALAX, SENNA
- Where
- 4 sites in United States
- Volunteers
- 39 people
- Sponsor
- Ferring Pharmaceuticals
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.