Clinical trials: Phase 1
Sickle Cell Disease
Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function
Background: Sickle cell disease (SCD) is a genetic disorder where red blood cells, that carry oxygen, are stiff and become stuck in small blood vessels.
- Trial name
- NCT06145282
- Testing
- Briquilimab, Filgrastim, Abatacept
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Neuroblastoma
A Clinical Study Evaluating the Safety of Peptide Receptor Radionuclide Therapy (PRRT) With 177Lu-DOTA0-Tyr3-Octreotate in Children With Refractory or Recurrent Neuroblastoma Expressing Somatostatin Receptors.
This study is a multicenter, open label phase I dose escalation trial designed to define the Maximum Tolerated Dose (MTD) of 177Lu-DOTATATE in children with refractory or recurrent neuroblastoma.
- Trial name
- NEUROBLU 02 (NCT03966651)
- Testing
- PRRT with 177Lu-DOTATATE
- Where
- 14 sites in France
- Volunteers
- 1 people
- Sponsor
- Institut Claudius Regaud
Dental Pulp Exposure
Comparative Evaluation of Clinical and Radiographic Outcomes of Mustard Gel (Brassica Nigra) and Mineral Trioxide Aggregate (MTA) as Pulpotomy Agents in Deciduous Molars- A Randomized Controlled Trial
The aim of this clinical trial is to compare two materials to treat baby molars with deep cavities. When the cavity is very deep and reaches the nerve of a baby tooth, dentist perform a treatment called "Pulpotomy".
- Trial name
- MG-MTA-RCT2026 (NCT07557290)
- Testing
- Intervention 1: Mustard Gel, Intervention 2 : Mineral Trioxide Aggregate (MTA)
- Volunteers
- 72 people
- Sponsor
- Dr Nida Farooq
Rhabdomyosarcoma, Non-Rhabdo. Soft Tissue Sarcoma
Molecular-Functional Imaging of Hypoxia in Childhood Sarcomas
This study will test the feasibility of using novel/existing imaging technologies focused on hypoxia measurements to determine "response to therapy" in pediatric soft tissue sarcomas as a pilot study.
- Trial name
- NCT03054792
- Testing
- Blood Oxygen Level Dependent [BOLD] MRI, Diffusion-Weighted [DW] MRI, Magnetic Resonance Spectroscopy [MRS]
- Where
- Canada
- Volunteers
- 18 people
- Sponsor
- The Hospital for Sick Children
Pregnant Women, Pregnant Women and Their Offspring
Effectiveness of MMS Compared With IFS Among Pregnant Women in Bangladesh: an Experimental Study
Background Maternal undernutrition and micronutrient deficiencies remain highly prevalent in Bangladesh and contribute to maternal anaemia, low birth weight, preterm birth, and adverse neonatal outcomes.
- Trial name
- NCT07540130
- Testing
- Multiple micronutrient supplementation
- Where
- Bangladesh
- Volunteers
- 667 people
- Sponsor
- National Institute of Neurosciences and Hospital, Dhaka
Pediatric Solid Tumor, Germ Cell Tumor
B7H3 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults
This is a phase I, open-label, non-randomized study that will enroll pediatric and young adult research participants with relapsed or refractory non-CNS solid tumors to evaluate the safety, feasibility, and efficacy of…
- Conditions
- Pediatric Solid Tumor, Germ Cell Tumor, Retinoblastoma, Hepatoblastoma
- Trial name
- NCT04483778
- Testing
- second generation 4-1BBζ B7H3-EGFRt-DHFR, second generation 4-1BBζ B7H3-EGFRt-DHFR(selected) and a second generation 4-1BBζ CD19-Her2tG, Pembrolizumab
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Seattle Children's Hospital
Schizophrenia
Study to Assess the Safety, Tolerability and Pharmacokinetics of Oral Brexpiprazole (OPC- 34712) in Adolescents With Schizophrenia
The purpose of this study is to assess the safety, tolerability and pharmacokinetics (PK) of oral brexpipirazole in adolescent subjects with schizophrenia or Other Related Psychiatric Disorders.
- Trial name
- NCT02411695
- Testing
- Brexpiprazole (OPC-34712)
- Where
- 10 sites in United States
- Volunteers
- 43 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Neuroblastoma
Phase I Study of 131-I mIBG Followed by Nivolumab & Dinutuximab Beta Antibodies in Children With Relapsed/Refractory Neuroblastoma
Neuroblastoma, the most common extra-cranial solid tumour in children, remains one of the major challenges in paediatric oncology.
- Trial name
- MiniVan (NCT02914405)
- Testing
- Nivolumab, Ch14.18/CHO
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- University Hospital Southampton NHS Foundation Trust
NHL, Large Cell Lymphoma
Anti-CD22 Chimeric Receptor T Cells in Pediatric and Young Adults With Recurrent or Refractory CD22-expressing B Cell Malignancies
Background: One type of cancer therapy takes blood cells from a person, changes them in a lab, then gives the cells back to the person.
- Conditions
- NHL, Large Cell Lymphoma, Follicular Lymphoma, ALL
- Trial name
- NCT02315612
- Testing
- CD22-CAR
- Where
- United States
- Volunteers
- 134 people
- Sponsor
- National Cancer Institute (NCI)
Type1diabetes
Mesenchymal Stromal Cells to Treat Type 1 Diabetes in Children and Adolescents
This is a combined phase 1 and 2 study in 66 subjects, male or female, between 7-21 years of age that have recently (< 6 months) been diagnosed with type 1 diabetes.
- Trial name
- NCT05061030
- Testing
- the ATMP Protrans
- Where
- Sweden
- Volunteers
- 66 people
- Sponsor
- Uppsala University Hospital
Sickle Cells Disease
RH Genotype Matched RBC Transfusions
To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.
- Trial name
- RBC (NCT04156893)
- Testing
- Red cell units that are genotype matched at the RHD and RHCE loci
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Children's Hospital of Philadelphia
Autism Spectrum Disorder, Autism
Autism Spectrum Disorder (ASD) Neurodevelopmental Disorder With Issues Social Behavior, Communication Issues, GI Dysfunction. Study is Multimodal Interventions Targeting These Pathways With cSVF, Stored MSCs, FMT and Diet Modification. Role of Autoimmunity, Gut-brain Issues, & Issues Examined.
Autism spectrum disorder (ASD) is a neurodevelopmental condition with core deficits in social communication and behavior, often accompanied by gastrointestinal (GI) and metabolic dysfunction.
- Conditions
- Autism Spectrum Disorder, Autism, Autism Spectral Disorder
- Trial name
- GARM-Autism (NCT07546942)
- Testing
- Autologous MSC isolation, concentration, and cryopreservation of adult mesenchymal stem cells, Fecal Biomicrome Transplantation (FMT), Ketogenic RESET Diet Protocol
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- Black Tie Medical, Inc.
Tetralogy of Fallot, Pulmonary Insufficiency
Contrast Enhanced 3D Echocardiographic Quantification of Right Ventricular Volumes in Repaired CHD
We propose the novel integration of two echocardiographic technologies - three-dimensional echocardiography using semi-automated right ventricular analysis coupled with the administration of ultrasound enhancing agents - to…
- Trial name
- NCT05186415
- Testing
- Sulfur Hexafluoride Lipid Type A Microspheres 25 MG Injection Powder for Suspension [LUMASON]
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Nemours Children's Clinic
Neuroblastoma
Multi-modular Chimeric Antigen Receptor Targeting GD2 in Neuroblastoma
MAGNETO is a single-centre, non-randomised, open label Phase I clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children and teenagers aged 1-16 years with relapsed or refractory neuroblastoma.
- Trial name
- MAGNETO (NCT05990751)
- Testing
- GD2 CAR T cells
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- University College, London
Malnutrition, Severe Acute, Malnutrition, Child
Slow Introduction of Nutrition for Ill Malnourished Children
The goal of this clinical trial is to learn if giving lower calorie feeds during the first stage of treatment helps improve survival in severely malnourished children who are ill.
- Conditions
- Malnutrition, Severe Acute, Malnutrition, Child, Malnutrition, Infant
- Trial name
- F50 (NCT06846749)
- Testing
- F75 milk feeds, F50 milk feeds, F35 milk feeds
- Where
- 2 sites in Bangladesh, Malawi
- Volunteers
- 135 people
- Sponsor
- The Hospital for Sick Children
Malignant Epithelial Neoplasms
Personalized TCR-T: Study of Adoptively Transferred T-cell Receptor Gene-engineered T Cells (TCR-T)
This is a phase I/Ib study of adoptively transferred T-cell receptor gene-engineered T cells (TCR-T) targeting tumor-specific antigens, with in vivo CD40 activation and PD-1 blockade, for patients with incurable cancers.
- Trial name
- NCT05349890
- Testing
- TCR-transduced T cells, CDX-1140, Pembrolizumab
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Providence Health & Services
Peanut Allergy
Study of ENP-501 in Peanut-Allergic and Non-Allergic Participants
This is a Phase 1/2 study to evaluate the safety and clinical activity of ENP-501 in non-peanut allergic (NPA) healthy participants and participants with an established peanut allergy (PA)
- Trial name
- NCT07117669
- Testing
- ENP-501
- Where
- United States
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- N-Fold, LLC
Hemophagocytic Lymphohistiocytosis
Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis
This study is a multi-site Phase Ib/II, 2-arm non-randomized clinical trial to determine the efficacy and tolerability of a response-adapted regimen combining ruxolitinib, dexamethasone, and etoposide as Frontline therapy for…
- Trial name
- NCT04551131
- Testing
- Ruxolitinib, Dexamethasone, Etoposide
- Where
- 13 sites in United States
- Volunteers
- 10 people
- Sponsor
- St. Jude Children's Research Hospital
Pediatric Brain Tumor, Recurrent Pediatric Brain Tumor
Intra-Tumoral Injections of Natural Killer Cells for Recurrent Malignant Pediatric Brain Tumors
This phase I trial tests the safety, side effects, and best dose of ex vivo expanded natural killer cells in treating patients with cancerous (malignant) tumors affecting the upper part of the brain (supratentorial) that have…
- Conditions
- Pediatric Brain Tumor, Recurrent Pediatric Brain Tumor, Pediatric Neoplasm
- Trial name
- PNOC028 (NCT05887882)
- Testing
- Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, Implantation, Magnetic Resonance Imaging (MRI)
- Where
- 10 sites in United States
- Volunteers
- 24 people
- Sponsor
- Sabine Mueller, MD, PhD
CAR T Cell Therapy, CD33 Positive Acute Myelogenous Leukemia
Functionally Optimized CD33 CAR-T Cell Therapy Targeting Recurrent/Refractory Acute Myeloid Leukemia
Relapsed/refractory acute myeloid leukemia (R/R AML) currently lacks effective CAR-T therapeutic agents due to the absence of tumor-specific target antigens.
- Trial name
- NCT07538713
- Testing
- Functionally optimized CD33 CAR-T
- Volunteers
- 18 people
- Sponsor
- Qi deng
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