Clinical trials: Pediatric Safety and Effectiveness
Biliary Atresia
Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OCA) Compared to Placebo in Pediatric Participants With Biliary Atresia, Post-hepatoportoenterostomy
This study will evaluate the efficacy, safety and tolerability, as well as PK/PD of OCA in eligible pediatric participants with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterostomy).
- Trial name
- NCT06121375
- Testing
- OCA
- Where
- 24 sites in Australia, Canada and 8 more
- Volunteers
- 28 people
- Sponsor
- Intercept Pharmaceuticals
Poor Graft Function, Hematopoietic Stem Cell Transplantation Complications
Expanded Donor Stem Cells for Poor Graft Function After Stem Cell Transplantation in Children
Poor graft function (PGF) is a serious complication after allogeneic hematopoietic stem cell transplantation (HSCT) and may cause persistent low blood cell counts despite donor-cell engraftment.
- Trial name
- NCT07907172
- Testing
- Ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cells
- Volunteers
- 3 people
- Sponsor
- wang xiaodong
Asthma
Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Glycopyrronium (Bromide) in Children (6 to Less Than 12 Years) With Asthma
The purpose of this study is to characterize the bronchodilator effect, systemic exposure and safety/tolerability of two different doses of inhaled glycopyrronium, when compared to placebo.
- Trial name
- NCT05222529
- Testing
- Glycopyrronium bromide 25ug, Glycopyrronium bromide 12.5ug
- Where
- 23 sites in Bulgaria, Colombia and 6 more
- Volunteers
- 42 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Non CNS Tumors
Study of Talimogene Laherparepvec In Children With Advanced Non CNS Tumors
This is a phase 1 study to evaluate the safety of intralesional talimogene laherparepvec administration in pediatric subjects with advanced non-CNS tumors that are amenable to direct injection
- Trial name
- NCT02756845
- Testing
- Talimogene Laherparepvec
- Where
- 22 sites in United States, Belgium and 5 more
- Volunteers
- 15 people
- Sponsor
- Amgen
Down Syndrome, Cognitive Dysfunction
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17
The purpose of this study is to determine the efficacy and safety of donepezil hydrochloride (Aricept) in the treatment of the cognitive dysfunction shown by children with Down syndrome, aged 11 to 17.
- Trial name
- NCT00754052
- Testing
- Aricept (donepezil hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Eisai Inc.
Psoriasis
A Study of Subcutaneous Risankizumab Injection for Pediatric Participants With Moderate to Severe Plaque Psoriasis to Assess Change in Disease Symptoms
Psoriasis is a chronic, systemic, inflammatory disease in which skin cells build up and develop thick, red and white scaly patches on the skin.
- Trial name
- OptIMMize-1 (NCT04435600)
- Testing
- Risankizumab, Ustekinumab
- Where
- 53 sites in United States, Canada and 5 more
- Volunteers
- 139 people
- Sponsor
- AbbVie
Familial Hypercholesterolemia - Homozygous
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein…
- Trial name
- ORION-19 (NCT06597006)
- Testing
- Inclisiran
- Where
- 19 sites in United States, Austria and 9 more
- Volunteers
- 9 people
- Sponsor
- Novartis Pharmaceuticals
Bipolar Disorder
Safety and Efficacy Study of Lithium for the Treatment of Pediatric Mania.
Study Design This is the second study of a multiphase, multicenter trial that will comprehensively examine lithium in the treatment of pediatric participants with bipolar I disorder.
- Trial name
- Lithium2 (NCT01166425)
- Testing
- Lithium Carbonate
- Where
- 11 sites in United States
- Volunteers
- 81 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Sickle Cell Disease, Hydroxyurea Failure
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human…
- Conditions
- Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies
- Trial name
- NCT05329649
- Testing
- CTX001
- Where
- 7 sites in United States, Germany and 2 more
- Volunteers
- 13 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Type 1 Diabetes Mellitus
Study of Secukinumab in Patients With Newly-diagnosed Type 1 Diabetes Mellitus
This study will assess the safety and efficacy of secukinumab on the preservation of pancreatic beta cells in patients with newly-diagnosed type 1 diabetes mellitus.
- Trial name
- NCT02044848
- Testing
- Secukinumab
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Novartis Pharmaceuticals
Gram-negative Bacterial Infections
Study of 2 Medicines (Aztreonam and Avibactam) Compared to Best Available Therapy for Serious Gram-negative Infections
The purpose of this study is to evaluate how Aztreonam (ATM) and Avibactam (AVI) are processed in pediatric participants. This study also aims to understand participant safety and effects in pediatric participants.
- Trial name
- NCT05639647
- Testing
- ATM-AVI, BAT
- Where
- 22 sites in United States, China and 6 more
- Volunteers
- 48 people
- Sponsor
- Pfizer
Lymphatic Malformations
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.
- Trial name
- EPIK-L1 (NCT05948943)
- Testing
- Alpelisib
- Where
- 61 sites in United States, Argentina and 10 more
- Volunteers
- 232 people
- Sponsor
- Novartis Pharmaceuticals
Pediatric Cancer, Solid Tumor Pediatric
Durvalumab and Tremelimumab for Pediatric Malignancies
The purpose of the study is to determine the recommended dose of durvalumab and tremelimumab (immunotherapy drugs) in pediatric patients with advanced solid and hematological cancers and expand in a second phase to test the…
- Conditions
- Pediatric Cancer, Solid Tumor Pediatric, Hematological Malignancies
- Trial name
- NCT03837899
- Testing
- Durvalumab / Tremelimumab Combination Therapy
- Where
- 19 sites in United States, France and 5 more
- Volunteers
- 50 people
- Sponsor
- AstraZeneca
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD
- Trial name
- FORZETTO (NCT07608432)
- Testing
- Zeleciment Rostudirsen (DYNE-251)
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Dyne Therapeutics
Generalized Myasthenia Gravis, gMG
Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis
The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
- Trial name
- NCT05644561
- Testing
- Ravulizumab
- Where
- 11 sites in United States, Italy and 3 more
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Tourette Syndrome
Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a…
- Trial name
- NCT03325010
- Testing
- Valbenazine
- Where
- 35 sites in United States, Puerto Rico
- Volunteers
- 127 people
- Sponsor
- Neurocrine Biosciences
Malaria, Vivax
A Pharmacokinetics, Safety and Efficacy Study of Tafenoquine (TQ) in Pediatric Subjects With Plasmodium Vivax (P. Vivax) Malaria
This is a prospective, open-label, multicenter, non-comparative, single arm study of pediatric subjects with Plasmodium vivax (P. vivax) malaria, aged 6 months to <16 years of age. A total of 60 subjects will be enrolled.
- Trial name
- NCT02563496
- Testing
- Tafenoquine, Chloroquine
- Where
- 4 sites in Colombia, Vietnam
- Volunteers
- 60 people
- Sponsor
- GlaxoSmithKline
Hemophilia A
Efficacy and Safety Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients in Canada With Hemophilia A - A Continuation of Baxter Study 060101
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of children with hemophilia A. The study is open to pediatric patients in Canada who completed Baxter Study 060101.
- Trial name
- NCT00189982
- Testing
- Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- Baxalta now part of Shire
Respiratory Viral Infection
Phase III Clinical Study Evaluating the Safety, Pharmacokinetics, and Efficacy of JKN2301 Dry Suspension in Pediatric Patients Aged 2 to Under 12 Years With Influenza
The goal of this phase III study is to learn if JKN2301 Dry Suspension works to treat uncomplicated influenza in Pediatric Participants aged 2 to 11 years.
- Trial name
- NCT07357051
- Testing
- JKN2301 Dry Suspension, Oseltamivir
- Where
- China
- Volunteers
- 177 people
- Sponsor
- Joincare Pharmaceutical Group Industry Co., Ltd
Duchenne Muscular Dystrophy (DMD)
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
This Phase 2 clinical trial will evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of tegacorat oral solution in male participants 4 to less than 7 years of age with Duchenne muscular dystrophy…
- Trial name
- NCT07911995
- Testing
- Tegacorat
- Volunteers
- 36 people
- Sponsor
- Grünenthal GmbH
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