Clinical trials: Offspring, Adult
Dravet Syndrome
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
This (DEEp SEA Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS.
- Trial name
- NCT06660394
- Testing
- LP352
- Where
- 105 sites in United States, Australia and 16 more
- Volunteers
- 108 people
- Sponsor
- Longboard Pharmaceuticals
Leukemia, Myeloid, Acute
A Study of Safety, Efficacy and Pharmacodynamics of Azacitidine in Children and Young Adults With Acute Myeloid Leukemia.
This study is a randomized, multicenter, open-label, Phase 2 study that will be run in 2 parts: a safety run-in part to determine the dose of azacitidine and then a second part to determine the efficacy of that dose in children…
- Trial name
- NCT02450877
- Testing
- Azacitidine, Control Arm
- Where
- 8 sites in Denmark, Germany and 1 more
- Volunteers
- 7 people
- Sponsor
- Celgene
Respiratory Tract Infections
A Multifaceted Intervention to Improve Prescribing for Acute Respiratory Infection for Adults and Children in Emergency Department and Urgent Care Settings
Inappropriate antibiotic use is a major public health concern. Excessive exposure to antibiotics results in emergence and spread of drug-resistant bacteria, potentially avoidable adverse drug reactions, and increased healthcare…
- Trial name
- MITIGATE (NCT03022929)
- Testing
- Enhanced Antimicrobial Stewardship Commitment and Feedback
- Where
- 3 sites in United States
- Volunteers
- 257 people
- Sponsor
- University of California, Davis
Autism Spectrum Disorder
A Study to Evaluate Scales for Repetitive and Restricted Behaviors in Children, Adolescents, and Adults With Autism Spectrum Disorder (ASD)
This is a non-drug study seeking to characterize different scales to measure repetitive and restrictive behaviors in different ASD sub-populations over time. This study will also explore the use of digital biomarkers.
- Trial name
- NCT03611075
- Testing
- No Intervention
- Where
- 18 sites in United States, Canada and 1 more
- Volunteers
- 144 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Pediatric Solid Tumor, Neuroblastoma
B7-H3.CD28Z.CART in Solid Tumors
The goal of this research study is to test if a new cell therapy (B7-H3.CD28Z.CART / B7-H3 CAR T cells) is safe and effective in treating children and young adults with solid cancers whose tumors have returned or stopped…
- Trial name
- NCT07358260
- Testing
- B7-H3.CD28Z.CART, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Robbie Majzner
Central Nervous System Malignancies, Ependymoma
Phase I Study of 5-Fluorouracil in Children and Young Adults With Recurrent Ependymoma
This is a phase I study to investigate the safety and pharmacokinetics of weekly 5-fluorouracil (5-FU) administered as a bolus dose in children and young adults with recurrent or refractory ependymoma.
- Trial name
- SJREFU (NCT01498783)
- Testing
- 5-fluorouracil
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- St. Jude Children's Research Hospital
Lymphoblastic Leukemia, Lymphoblastic Leukemia, Acute, Childhood
Dual Anti-CD22/CD19 Chimeric Antigen Receptor-directed T Cells (CART2219.1) for Relapsed Refractory B-Lineage Leukaemia
The purpose of this study is to deliver dual-targeting CAR-T cell therapy (CART 2219.1) as a salvage treatment to patients with relapsed/refractory B-lineage leukaemia in place of stem cell transplant or irradiation.
- Conditions
- Lymphoblastic Leukemia, Lymphoblastic Leukemia, Acute, Childhood, Lymphoblastic Leukemia in Children, Lymphoblastic Leukemia, Acute Adult
- Trial name
- NCT05429905
- Testing
- Phase I, Phase II
- Where
- Singapore
- Volunteers
- 48 people
- Sponsor
- KK Women's and Children's Hospital
Atopic Dermatitis
Tapinarof for the Treatment of Atopic Dermatitis in Children and Adults (DMVT-505-3102)
This is a double-blind, randomized, vehicle controlled Phase 3 study to evaluate the efficacy and safety of topical tapinarof cream, 1% compared to vehicle control cream in pediatric and adult subjects with atopic dermatitis.
- Trial name
- NCT05032859
- Testing
- tapinarof cream, 1%, vehicle cream
- Where
- 62 sites in United States, Canada
- Volunteers
- 406 people
- Sponsor
- Organon and Co
Leber Congenital Amaurosis
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
- Trial name
- OPTIRPE65 (NCT02781480)
- Testing
- AAV RPE65
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 15 people
- Sponsor
- MeiraGTx UK II Ltd
High Risk Neuroblastoma, Recurrent Neuroblastoma
Testing the Combination of Two Immunotherapy Drugs (Magrolimab and Dinutuximab) in Patients With Relapsed or Refractory Neuroblastoma or Relapsed Osteosarcoma
This phase I trial is to find out the best dose, possible benefits and/or side effects of magrolimab in combination with dinutuximab in treating patients with neuroblastoma that has come back (relapsed) or does not respond to…
- Conditions
- High Risk Neuroblastoma, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Refractory Neuroblastoma
- Trial name
- NCT04751383
- Testing
- Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy
- Where
- 11 sites in United States, Canada
- Volunteers
- 12 people
- Sponsor
- National Cancer Institute (NCI)
Ewing Sarcoma, Osteosarcoma
Osteosarcoma and Ewing Sarcoma Treatment Response Assessment With Functional MRI Imaging in Children and Young Adults
The purpose of the study was to investigate whether functional MRI imaging (diffusion weighted imaging) is useful for monitoring the therapeutic response of bone sarcomas in children and young adults.
- Trial name
- FUBEO (NCT01780779)
- Testing
- MRI
- Where
- Belgium
- Volunteers
- 25 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Achromatopsia
Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia…
- Trial name
- NCT03278873
- Testing
- Prior exposure to AAV-CNGA3 or AAV-CNGB3
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 34 people
- Sponsor
- MeiraGTx UK II Ltd
COVID-19, SARS-CoV-2
Clinical and Immunological Characterisation of COVID-19 in Children, Adolescents and Adults
The investigators aim to characterise Coronavirus Disease 2019 (COVID-19) in every age group. On the one hand, emphasis is put on the initial presentation, clinical course, outcome and the therapy used.
- Trial name
- NCT04341168
- Testing
- this study is non- interventional
- Where
- Germany
- Volunteers
- 160 people
- Sponsor
- University Hospital of Cologne
Nephrolithiasis, Ureterolithiases
Validation of Low Dose CT for Diagnosis of Urolithiasis
The investigators have developed an extremely low dose renal computed tomography (CT) protocol that on preliminary testing has an effective dose in the range of a single view abdominal radiograph.
- Trial name
- NCT03656913
- Testing
- Extremely low dose CT
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Respiratory Distress Syndrome, Adult
Understanding the Role of Genes and Biomarkers in the Inflammation and Blood Clotting Process in Children With Acute Lung Injury
Acute lung injury (ALI)/Acute Respiratory Distress Syndrome (ARDS) is a severe lung condition that causes respiratory failure. This study will examine if differences in genes and biomarkers involved in the inflammation and blood…
- Trial name
- PALI (NCT00605527)
- Where
- 5 sites in United States
- Volunteers
- 396 people
- Sponsor
- University of California, San Francisco
Viral Infection, Primary Immune Deficiency Disorder
Treatment of Refractory BK Infections With Related Donor BK Specific Cytotoxic T-cells (CTLs)
BK cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be safe and effective in decreasing specific viral load in children, adolescents and young adults (CAYA) with refractory BK…
- Trial name
- NCT04197596
- Testing
- BK CTL
- Where
- 7 sites in United States
- Volunteers
- 40 people
- Sponsor
- New York Medical College
Sepsis
Adaptive Platform Trial for Personnalisation of Sepsis Treatment in Children and Adults: a Multi-national, Treatable Traits-guided, Adaptive, Exploratory, Bayesian Basket Trial
PALETTE is a perpetual adaptive platform to efficiently study sepsis interventions within 'treatable traits' in all-ages patients enabling prompt evaluation of pandemic treatments.
- Trial name
- PALETTE (NCT06381661)
- Testing
- Tocilizumab, Baricitinib, Anakinra
- Where
- 19 sites in France
- Volunteers
- 2,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cystic Fibrosis, Pancreatic Insufficiency
Evaluation of the Safety and Efficacy of Pancrecarb® MS-16 in Cystic Fibrosis
The primary objective of this study is to determine if PANCRECARB® MS-16 (pancrelipase) is safe and effective in reducing steatorrhea (as measured by 72-hour stool fat determinations) in children and adults with cystic fibrosis…
- Trial name
- NCT00432861
- Testing
- PANCRECARB® (pancrelipase)
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Digestive Care, Inc.
Refractory Acute Lymphoblastic Leukemia, Relapsed Acute Lymphoblastic Leukemia
Trial Treating Relapsed Acute Lymphoblastic Leukemia With Venetoclax and Navitoclax
This is a phase I/II clinical trial evaluating the activity of combination chemotherapy with venetoclax and navitoclax in children with relapsed or refractory acute lymphoblastic leukemia or lymphoma (rALL) and assessing the…
- Trial name
- NCT05192889
- Testing
- Venetoclax, Navitoclax, Dexamethasone
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- St. Jude Children's Research Hospital
Epilepsy, Lennox-Gastaut Syndrome
A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
To evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo, in participants with Lennox-Gastaut Syndrome (LGS).
- Trial name
- GWPCARE4 (NCT02224690)
- Testing
- GWP42003-P 20 mg/kg/day Dose
- Where
- 24 sites in United States, Netherlands and 1 more
- Volunteers
- 171 people
- Sponsor
- Jazz Pharmaceuticals