A Clinical Study of Intismeran Autogene (V940) Treatment and Pembrolizumab in People With Bladder Cancer (V940-005/INTerpath-005)
NCT06305767
Recruiting
Researchers are looking for new ways to treat people with high-risk muscle-invasive urothelial carcinoma (MIUC). Urothelial carcinoma is a type of bladder cancer that begins in cells that line the inside of the bladder and other parts of the urinary tract, such as part of the kidneys, ureters, and urethra. People with MIUC usually have chemotherapy before surgery, then surgery to remove the cancer. Chemotherapy is a type of medicine to destroy cancer cells or stop them from growing. After surger... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
07/16/2025
Locations: UCLA Hematology/Oncology - Westwood (Building 200 Suite 140)-Department of Urology/Institute of Uro ( Site 0104), Los Angeles, California +73 locations
Conditions: Bladder Cancer
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EPIC-Peds: A Study to Learn About the Study Medicine Called PF-07321332 (Nirmatrelvir)/Ritonavir in Patients Under 18 Years of Age With COVID-19 That Are Not Hospitalized But Are at Risk for Severe Disease
NCT05261139
Recruiting
The purpose of this clinical trial is to learn about the safety, pharmacokinetics (pharmacokinetics helps us understand how the drug is changed and eliminated from your body after you take it), and efficacy (how well a study treatment works in the study) of the study medicine (called nirmatrelvir/ritonavir) for potential treatment of coronavirus disease 2019 (COVID-19). The study medicine will be given to patients under 18 years of age with COVID-19 that are not hospitalized but are at risk for... Read More
Gender:
ALL
Ages:
Between 0 years and 17 years
Trial Updated:
07/16/2025
Locations: Phoenix Children's Hospital, Phoenix, Arizona +72 locations
Conditions: COVID-19
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Mitochondrial Donation: An 18 Month Outcome Study.
NCT04113447
Recruiting
The Investigator proposes to record the fetal and postnatal development of children conceived using Mitochondrial Donation (MD) and to perform expert assessment of development at 18 months (corrected for gestational age) using the internationally validated Bayley-III developmental assessment tool.
Gender:
FEMALE
Ages:
All
Trial Updated:
07/16/2025
Locations: The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle Upon Tyne, Tyne & Wear
Conditions: Mitochondrial Diseases
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Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
NCT04833894
Recruiting
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. Trial details include: * The maximum trial duration for each individual participant will be approximately 28 weeks * The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)
Gender:
ALL
Ages:
Between 2 years and 18 years
Trial Updated:
07/16/2025
Locations: Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital, Chicago, Illinois +23 locations
Conditions: Generalized Myasthenia Gravis
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A Comparison of THD® Anopress With High Resolution Anorectal Manometry
NCT07083908
Recruiting
Currently, high resolution anorectal manometry (HDARM) is used as gold standard to measure anal sphincter pressures in women who have sustained a third or fourth degree perineal tears (obstetric anal sphincter injuries). This test is performed to help advise patients on future pregnancies or deliveries. However, HDRM catheters are very expensive and they also need to be performed in a dedicated procedure room. THD® Anopress in comparison is a lightweight, portable device which has a comparativel... Read More
Gender:
FEMALE
Ages:
18 years and above
Trial Updated:
07/16/2025
Locations: Kings College Hospital, London, Not set
Conditions: Obstetric Anal Sphincter Injury
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A Study for GSK3862995B in Healthy Participants and Participants With Chronic Obstructive Pulmonary Disease
NCT06154837
Recruiting
The primary objective of the study is to investigate the safety and tolerability of ascending doses of GSK3862995B following single dose in healthy participants and repeat doses in participants with Chronic obstructive pulmonary disease (COPD).
Gender:
ALL
Ages:
Between 18 years and 75 years
Trial Updated:
07/16/2025
Locations: GSK Investigational Site, Yuma, Arizona +31 locations
Conditions: Pulmonary Disease, Chronic Obstructive
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A Phase 3 Study to Evaluate Petosemtamab Plus Pembrolizumab vs Pembrolizumab in First-line Treatment of Head and Neck Cancer (LiGeR - HN1)
NCT06525220
Recruiting
This is a Phase 3 randomized, open-label study to evaluate the efficacy and safety of petosemtamab plus pembrolizumab vs pembrolizumab in first-line treatment of recurrent or metastatic PD-L1+ head and neck squamous cell carcinoma.
Gender:
ALL
Ages:
18 years and above
Trial Updated:
07/16/2025
Locations: Site 36, La Jolla, California +143 locations
Conditions: Head and Neck Squamous Cell Carcinoma
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Efficacy and Safety of Depemokimab Compared With Mepolizumab in Adults With Relapsing or Refractory Eosinophilic Granulomatosis With Polyangiitis (EGPA)
NCT05263934
Recruiting
This study aims to investigate the efficacy and safety of depemokimab compared with mepolizumab in adults with relapsing or refractory EGPA receiving SoC therapy.
Gender:
ALL
Ages:
18 years and above
Trial Updated:
07/16/2025
Locations: GSK Investigational Site, Denver, Colorado +80 locations
Conditions: Eosinophilic Granulomatosis With Polyangiitis
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A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
NCT06283212
Recruiting
EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to \< 48 months. The study follows and open-label, dose-escalation design.
Gender:
ALL
Ages:
Between 6 months and 47 months
Trial Updated:
07/16/2025
Locations: Queen Elizabeth Hospital, Glasgow, Not set +2 locations
Conditions: Dravet Syndrome
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A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
NCT07082543
Recruiting
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis
Gender:
ALL
Ages:
4 years and above
Trial Updated:
07/16/2025
Locations: Children's Hospital and Research Center at Oakland, Oakland, California +30 locations
Conditions: Gangliosidoses, GM2, Gangliosidosis, GM1
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A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
NCT07082725
Recruiting
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease
Gender:
ALL
Ages:
4 years and above
Trial Updated:
07/16/2025
Locations: Children's Hospital and Research Center at Oakland, Oakland, California +26 locations
Conditions: Niemann-Pick Type C Disease
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Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
NCT06553768
Recruiting
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric and adult participants who have cholestatic liver disease with pruritus that has been refractory to other therapies, and who have no other treatment options.
Gender:
ALL
Ages:
6 months and above
Trial Updated:
07/16/2025
Locations: Children's Hospital Los Angeles (CHLA), Los Angeles, California +34 locations
Conditions: Cholestatic Liver Disease (except ALGS, PFIC, PBC and PSC)
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