Clinical trials: Phase 1b
Duchenne Muscular Dystrophy (DMD)
A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
The primary purpose of this study is to evaluate the safety and tolerability of ASP0367. This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367.
- Trial name
- NCT04184882
- Testing
- Bocidelpar
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Astellas Pharma Inc
Diffuse Large B-Cell Lymphoma, Mediastinal B-cell Lymphoma
Idelalisib With Rituximab, Ifosfamide, Carboplatin, Etoposide (RICE) in Children and Adolescents
The primary objectives of this study are to evaluate safety, tolerability, pharmacokinetics (PK) and preliminary efficacy of idelalisib; and to establish recommended phase 2 doses (RP2D) of idelalisib in combination with…
- Trial name
- NCT03349346
- Testing
- Idelalisib, Rituximab, Ifosfamide
- Where
- 7 sites in France, Italy and 2 more
- Sponsor
- Gilead Sciences
Pre-Symptomatic Type 1 Diabetes
A Study to Evaluate SIMPONI (Golimumab) Therapy in Children, Adolescents and Young Adults With Pre-Symptomatic Type 1 Diabetes
The purpose of this study is to determine the safety and tolerability of golimumab in children, adolescents, and young adults with pre-symptomatic stage 2 type 1 diabetes mellitus (T1D).
- Trial name
- NCT03298542
- Testing
- Golimumab
- Where
- 8 sites in United States, Finland and 1 more
- Volunteers
- 8 people
- Sponsor
- Janssen Research & Development, LLC
Pediatric Growth Hormone Deficiency
A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)
This is a two-part clinical study for children with growth hormone deficiency. In the first part, participants will be randomly assigned to receive different doses of an oral treatment (GS3-007a dry suspension) or a placebo for…
- Trial name
- NCT07264595
- Testing
- GS3-007a dry suspension, rhGH injection
- Where
- China
- Volunteers
- 88 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Advanced Solid Tumor, CNS Tumor
Study With Infigratinib in Subjects With Advanced Solid and CNS Tumors or Recurrent or Progressive Low-Grade Glioma With Selected FGFR1-3 Alterations
The phase 1b study is aimed at determining the pediatric recommended phase 2 dose (RP2D) of Infigratinib. The phase 2 study will evaluate efficacy and safety of infigratinib.
- Conditions
- Advanced Solid Tumor, CNS Tumor, Recurrent WHO Grade II Glioma
- Trial name
- NEWEL (NCT05222165)
- Testing
- Infigratinib
- Where
- 10 sites in United States, Canada and 1 more
- Sponsor
- Helsinn Healthcare SA
FSHD, FSHD1
Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- FSHD, FSHD1, FSHD2, FMD
- Trial name
- FORTITUDE-OLE (NCT06547216)
- Testing
- AOC 1020
- Where
- 17 sites in United States, Canada and 1 more
- Volunteers
- 84 people
- Sponsor
- Avidity Biosciences, Inc.
Pneumococcal Infectious Disease
A Phase 1b Clinical Trial of 13-valent Pneumococcal Polysaccharide Conjugate Vaccine
A phase 1b clinical trial of 13-valent Pneumococcal Polysaccharide Conjugate Vaccine (PCV13) developed by Sinovac Life Science Co., Ltd will be conducted in children aged 2 months (42-89 days) and 2 to 5 years.
- Trial name
- NCT06183216
- Testing
- Sinovac PCV13, PREVNAR 13
- Where
- China
- Volunteers
- 140 people, healthy volunteers welcome
- Sponsor
- Sinovac Research and Development Co., Ltd.
Metastatic Solid Tumors
Study to Investigate the Safety and Activity of Aldoxorubicin Plus Gemcitabine in Subjects With Metastatic Solid Tumors
This is a Phase 1 study to investigate the safety and activity of aldoxorubicin plus gemcitabine in Subjects with solid tumors.
- Trial name
- NCT02235688
- Testing
- aldoxorubicin, gemcitabine
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- ImmunityBio, Inc.
Relapsed Non Hodgkin Lymphoma, Relapsed Adult ALL
Anti-CD19, Dual Co-stimulatory (4-1BB, CD3ζ) Chimeric Antigen Receptor T-cells in Patients With Relapsed/Refractory Aggressive Lymphoma or Acute Lymphoblastic Leukemia (ALL)
Autologous, unselected CD3+ lymphocytes collected from apheresis, transfected with a lentiviral vector containing a 2nd generation chimeric antigen receptor (CAR) consisting of a scFv recognizing CD19 and dual co-stimulatory…
- Conditions
- Relapsed Non Hodgkin Lymphoma, Relapsed Adult ALL, Relapsed Pediatric ALL
- Trial name
- ACIT001/EXC002 (NCT03938987)
- Testing
- autologous CD19-directed chimeric antigen receptor (CAR) T-cells
- Where
- 6 sites in Canada
- Volunteers
- 63 people
- Sponsor
- University of Alberta
Malaria
L9LS MAb in Malian Infants
The purpose of this study is to assess the safety, tolerability, and pharmacokinetics of L9LS in infants in Mali and to evaluate the impact of L9LS on subsequent R21/Matrix-MTM vaccine immunogenicity.
- Trial name
- NCT06461026
- Testing
- L9LS, R21/Matrix-MTM
- Where
- 3 sites in Mali
- Volunteers
- 327 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Advanced Soft Tissue Sarcoma
A Phase 1B Dose-escalation and Phase 2a Study of Carotuximab (TRC105) in Combination With Pazopanib in Patients With Advanced Soft Tissue Sarcoma
The purpose of the phase 1b portion is to evaluate safety and tolerability and determine a recommended phase 2 dose for TRC105 when added to standard dose pazopanib in patients with advanced soft tissue sarcoma.
- Trial name
- NCT01975519
- Testing
- TRC105 and Pazopanib
- Where
- 9 sites in United States
- Volunteers
- 111 people
- Sponsor
- Tracon Pharmaceuticals Inc.
Netherton Syndrome
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
This is a first-in-human, Phase 1/1b, 4-part study that includes the evaluation of safety, tolerability, pharmacokinetics (PK), and immunogenicity of BCX17725 when administered via single and multiple doses in healthy adult…
- Trial name
- NCT06539507
- Testing
- BCX17725
- Where
- 12 sites in United States, Australia and 3 more
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Diabetes type1
A Study to Assess the Safety and Tolerability of Different Doses of AG019 Administered Alone or in Combination With Teplizumab in Participants With Recent-onset Diagnosed Type 1 Diabetes (T1D)
The purpose of this study is to assess the safety and tolerability of different doses of AG019 administered alone or in combination with teplizumab in participants with recent-onset type 1 diabetes (T1D).
- Trial name
- NCT03751007
- Testing
- AG019 - Low Dose, Teplizumab, AG019 - High Dose
- Where
- 18 sites in United States, Belgium
- Volunteers
- 45 people
- Sponsor
- Precigen Actobio T1D, LLC
High-Grade Glioma
Novel Targeted Radiotherapy in Pediatric Patients With Inoperable Relapsed or Refractory HGG
The purpose of this dose finding study is to evaluate the safety and efficacy of 2 different dose levels of CLR 131 in children, adolescents and young adults with relapsed or refractory high-grade glioma (HGG).
- Trial name
- NCT05610891
- Testing
- CLR 131
- Where
- 8 sites in United States, Canada
- Volunteers
- 50 people
- Sponsor
- Cellectar Biosciences, Inc.
Respiratory Syncytial Virus Infections
A Study to Learn About the Amount of the Study Medicine (Sisunatovir) in Blood and Its Safety in Infants and Children With Pneumonia Caused by RSV
The purpose of the study is to learn about the safety and amount of sisunatovir in the blood of infants and children up to age 60 months.
- Trial name
- NCT06102174
- Testing
- Active
- Where
- 9 sites in United States, Japan and 1 more
- Volunteers
- 10 people
- Sponsor
- Pfizer
Focal Seizures
A Study to Evaluate the Efficacy and Safety of Cannabidiol Oral Solution (CBD-OS [GWP42003-P, JZP926]) for the Treatment of Focal-Onset Seizures
Cannabidiol oral solution (CBD-OS) is approved in the US for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or Tuberous sclerosis complex (TSC) in patients 1 year of age and older.
- Trial name
- NCT07233239
- Testing
- CBD-OS
- Where
- 24 sites in United States
- Volunteers
- 100 people
- Sponsor
- Jazz Pharmaceuticals
Leukemia, Acute Myeloid Leukemia
A Phase 1 Dose Escalation and Expanded Cohort Study of EPZ-5676 in the Treatment of Pediatric Patients With Relapsed/Refractory Leukemias Bearing a Rearrangement of the MLL Gene
A subset of patients with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) harbor rearrangements of the MLL gene, which are detected either by cytogenetic or fluorescent in situ hybridization evaluation at the…
- Conditions
- Leukemia, Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Acute Leukemias
- Trial name
- NCT02141828
- Testing
- EPZ-5676
- Where
- 9 sites in United States, Canada
- Volunteers
- 18 people
- Sponsor
- Epizyme, Inc.
COVID-19
COVID-19 Study of Pharmacokinetics, Safety, Tolerability, and Efficacy of Intravenous Anti-Spike(s) SARS-CoV-2 Monoclonal Antibodies (Casirivimab+Imdevimab) for the Treatment of Pediatric Patients Hospitalized Due to COVID-19
The primary objectives of the study are: To characterize the concentrations of casirivimab+imdevimab in serum over time To evaluate the safety and tolerability of casirivimab+imdevimab The secondary objective of the study is: To…
- Trial name
- NCT05092581
- Testing
- casirivimab+imdevimab
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Regeneron Pharmaceuticals
Classical Hodgkin Lymphoma
CD30 CAR T-cells Post AutoHSCT for Poor-risk Hodgkin Lymphoma
Patients with poor risk classical Hodgkin Lymphoma (cHL) will undergo myeloablative chemotherapy (MAC) with autologous stem cell transplantation (AutoHSCT) and subsequently receive autologous CD30+ CAR T-cells.
- Trial name
- NCT06617286
- Testing
- CD30 CAR T-cell
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- New York Medical College
Cystic Fibrosis
"Safety, Tolerability and Pharmacokinetics of MP-376 Administered for 14 Days to Stable Pediatric (CF) Patients"
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- NCT00840333
- Testing
- MP-376 (Levofloxacin solution for Inhalation)
- Where
- 6 sites in United States
- Volunteers
- 27 people
- Sponsor
- Amgen