Clinical trials: Phase 1, open to volunteers
Neuroendocrine Tumors, Progression
Everolimus 5 mg vs 10 mg/Daily for Patients With Neuroendocrine Tumors
Everolimus is approved in many countries to treat patients with advanced/metastatic well-differentiated neuroendocrine tumors (NET), providing median progression-free survival times of approximately 12 months across different…
- Conditions
- Neuroendocrine Tumors, Progression, Neuroendocrine Tumor Grade 1, Neuroendocrine Tumor Grade 2
- Trial name
- EVENET (NCT06472388)
- Testing
- Everolimus 5 MG
- Where
- 2 sites in Brazil
- Volunteers
- 100 people
- Sponsor
- AC Camargo Cancer Center
SMARD1, CMT2S
Gene Therapy for IGHMBP2-Related Diseases
Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.
- Trial name
- NCT05152823
- Testing
- Gene Therapy
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Megan Waldrop
Liver Cancer (Fibrolamellar Hepatocellular Cancer (FLC))
A DNAJB1-PRKACA Fusion Kinase Peptide Vaccine Combined With Glutamine Antagonist DRP-104, Nivolumab, and Ipilimumab in Patients With Advanced Stage Fibrolamellar Carcinoma (FLC)
The purpose of this study is to determine whether the combination of a neoantigen vaccine targeting the DNAJB1-PRKACA fusion kinase in combination with DRP-104, Nivolumab and Ipilimumab is safe and yields a clinically compelling…
- Trial name
- NCT07430202
- Testing
- DNAJB1-PRKACA Peptide Vaccine, DRP-104, Nivolumab
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Leukaemia (Acute Lymphoblastic), Leukaemia (Acute Myeloid)
Phase I Clinical Trial of ThINKK Adoptive Immunotherapy After Allogeneic Hematopoietic Transplantation in Children With Leukemia or Neuroblastoma
A first-in-class adoptive immunotherapy we called ThINKK, for Therapeutic Inducers of Natural Killer (NK) cell Killing, have been designed for use after hematopoietic stem cell transplantation (HSCT), where the proper stimulation…
- Conditions
- Leukaemia (Acute Lymphoblastic), Leukaemia (Acute Myeloid), Neuroblastoma, Neuroblastoma, Metastatic
- Trial name
- ThINKK-01 (NCT07518654)
- Testing
- Therapeutic Inducers of Natural Killer Killing (ThINKK)
- Where
- 2 sites in Canada
- Volunteers
- 12 people
- Sponsor
- Michel Duval
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
Brain Tumor, Pediatric, Medulloblastoma, Childhood
GD2-CAR T Cells for Pediatric Brain Tumours
The purpose of this study is to test the safety and efficacy of iC9-GD2-CAR T-cells, a third generation (4.1BB-CD28) CAR T cell treatment targeting GD2 in paediatric or young adult patients affected by relapsed/refractory…
- Conditions
- Brain Tumor, Pediatric, Medulloblastoma, Childhood, Embryonal Tumor, High Grade Glioma
- Trial name
- NCT05298995
- Testing
- GD2-CART01 (iC9-GD2-CAR T-cells)
- Where
- Italy
- Volunteers
- 54 people
- Sponsor
- Bambino Gesù Hospital and Research Institute
Hematologic Malignancy, AML
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Tagraxofusp is a protein-drug conjugate consisting of a diphtheria toxin redirected to target CD123 has been approved for treatment in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN).
- Conditions
- Hematologic Malignancy, AML, ALL, BPDCN
- Trial name
- NCT05476770
- Testing
- Tagraxofusp, Fludarabine, Cytarabine
- Where
- 31 sites in United States, Australia
- Volunteers
- 54 people
- Sponsor
- Therapeutic Advances in Childhood Leukemia Consortium
Rhabdomyosarcoma, Pediatric Cancer
Effectiveness of Pucotenlimab Combined With Standard Chemotherapy Regimen
This study comprises both Phase I and Phase II research. This phase focuses on safety, tolerability, and pharmacokinetics using a "3+3" dose escalation design with three dose groups: 1 mg/kg, 3 mg/kg, and 6 mg/kg.
- Trial name
- NCT06456892
- Testing
- Pucotenlimab
- Where
- China
- Volunteers
- 82 people
- Sponsor
- Sun Yat-sen University
Respiratory Syncytial Virus Infections
A Study of BLB-201 RSV Vaccine in Infants and Children
This Phase 1/2a trial is a randomized, placebo-controlled trial to evaluate the safety, tolerability and immunogenicity of two ascending doses (10^6 PFU and 10^7 PFU) of intranasal BLB-201 (a recombinant parainfluenza virus type…
- Trial name
- NCT05655182
- Testing
- PIV5-vectored RSV Vaccine (BLB-201) Low Dose, PIV5-vectored RSV Vaccine (BLB-201) High Dose
- Where
- 18 sites in United States
- Volunteers
- 137 people, healthy volunteers welcome
- Sponsor
- Blue Lake Biotechnology Inc.
Desmoplastic Small Round Cell Tumor, Osteosarcoma
Trastuzumab Deruxtecan (DS-8201a) for the Treatment of Recurrent or Refractory Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
This phase I/II trial studies the effects of trastuzumab deruxtecan (DS-8201a) in treating patients with osteosarcoma, Wilms tumor (WT) or desmoplastic small round cell tumor (DSRCT) that is newly diagnosed or has come back after…
- Conditions
- Desmoplastic Small Round Cell Tumor, Osteosarcoma, Recurrent Desmoplastic Small Round Cell Tumor, Recurrent Kidney Wilms Tumor
- Trial name
- NCT04616560
- Testing
- Biospecimen Collection, Computed Tomography, Echocardiography Test
- Where
- 32 sites in United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Type 1 Diabetes Mellitus, Stage 2 Type 1 Diabetes
Information, Follow-up and Early Diagnosis of Children at Risk for Type 1 Diabetes
The aim is to investigate whether screening for islet autoantibodies, regular follow-up, and increased knowledge in families about the progression of the disease in children at high genetic risk or with single or multiple islet…
- Conditions
- Type 1 Diabetes Mellitus, Stage 2 Type 1 Diabetes, Stage 1 Type 1 Diabetes, Stage 3 Type 1 Diabetes
- Trial name
- iT1D (NCT06676566)
- Where
- Sweden
- Volunteers
- 600 people
- Sponsor
- Lund University
Hypertrophic Obstructive Cardiomyopathy
Transapical Beating-heart Septal Myectomy in Patient With Hypertrophic Obstructive Cardiomyopathy
The primary purpose of this study is to evaluate the feasibility, the safety and the efficacy of the transapical beating-heart septal myectomy for the treatment of hypertrophic obstructive cardiomyopathy.
- Trial name
- NCT05332691
- Testing
- Transapical beating-heart septal myectomy
- Where
- China
- Volunteers
- 1,000 people
- Sponsor
- Xiang Wei
Bronchopulmonary Dysplasia (BPD)
Metformin for Premature Infants With Bronchopulmonary Dysplasia
The overall objective of this study is to investigate the role of metformin in decreasing lung injury and promoting lung growth in premature infants. There are two phases to this pilot study.
- Trial name
- NCT07120971
- Testing
- Metformin (open-label)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Medical College of Wisconsin
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)
Poliomyelitis
Study of Co-administered (Types 1 & 2) Novel Oral Poliomyelitis Vaccines Evaluation
The purpose of this study is to evaluate the safety and tolerability of co-administration of nOPV1 + nOPV2 in infants, relative to those receiving monovalent nOPV vaccines alone and whether two and/or three doses of…
- Trial name
- SCOPE (NCT06895486)
- Testing
- Novel Oral Polio Vaccine Type 1 (nOPV1), Novel Oral Polio Vaccine Type 2 (nOPV2)
- Where
- 3 sites in Panama
- Volunteers
- 675 people, healthy volunteers welcome
- Sponsor
- PATH
Brain Tumor, Cancer
000860: A Phase I/II Study of Zotiraciclib for Recurrent Malignant Gliomas With Isocitrate Dehydrogenase 1 or 2 (IDH1 or IDH2) Mutations
Protocol # 000860 Background: Diffuse gliomas are tumors that affect the brain and spinal cord. Gliomas that develop in people with certain gene mutations (IDH1 or IDH2) are especially aggressive. Better treatments are needed.
- Trial name
- NCT05588141
- Testing
- Zotiraciclib
- Where
- 2 sites in United States
- Volunteers
- 96 people
- Sponsor
- National Cancer Institute (NCI)
Recurrent Solid Tumor, Recurrent Ewing Sarcoma
Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma
The phase I portion of this study is designed for children or adolescents and young adults (AYA) with a diagnosis of a solid tumor that has recurred (come back after treatment) or is refractory (never completely went away).
- Conditions
- Recurrent Solid Tumor, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Malignant Germ Cell Tumor
- Trial name
- NCT04901702
- Testing
- Onivyde, Talazoparib, Temozolomide
- Where
- 10 sites in United States, Canada
- Volunteers
- 90 people
- Sponsor
- St. Jude Children's Research Hospital
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
Duchene Muscular Dystrophy
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin.
- Trial name
- NCT07856329
- Testing
- GNT0004 (1x10e13 vg/kg), GNT0004 (3x10e13 vg/kg)
- Where
- 14 sites in Belgium, France and 2 more
- Volunteers
- 77 people
- Sponsor
- Genethon
Type 1 Diabetes
A Phase I/II Trial to Preserve Residual Insulin Secretion in Children With Recent Onset Type 1 Diabetes by Giving Verapamil
The objective of this trial is first to evaluate safety and then the effect on preservation of residual beta cell function also clinical efficacy by treatment with Verapamil in children with recent onset Type 1 diabetes.
- Trial name
- VERADIAB-1 (NCT07199946)
- Testing
- Verapamil (Part A)
- Where
- 2 sites in Sweden
- Volunteers
- 36 people
- Sponsor
- Johnny Ludvigsson