Clinical trials: Pediatric Disease
Fabry Disease
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to < 12 years of age with Fabry disease and with amenable GLA variants.
- Trial name
- NCT06904261
- Testing
- Migalastat HCl 20 mg
- Where
- 12 sites in United States, Belgium and 4 more
- Volunteers
- 8 people
- Sponsor
- Amicus Therapeutics
Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)
Intermediate-size Expanded Access Program (EAP), Mesenchymal Stromal Cells (MSC) for Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)
The objectives of this intermediate-size expanded access protocol are to assess the safety and efficacy of remestemcel-L in participants with MIS-C associated with COVID-19.
- Trial name
- NCT04456439
- Testing
- Remestemcel-L, Hydrocortisone, Diphenhydramine
- Sponsor
- Mesoblast International Sàrl
Pediatric Malignant Solid Tumor, Febrile Neutropenia (FN)
Phase II Study of QLC2519 in Pediatric Solid Tumor Participants
QLC2519 (Mai Li Sheng®) is a new protein drug created by fusing the N-terminal of highly active modified G-CSF with the C-terminal of HSA.
- Conditions
- Pediatric Malignant Solid Tumor, Febrile Neutropenia (FN), Neutropenia
- Trial name
- NCT07724756
- Testing
- Albipagrastim alf
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Attention Deficit Hyperactivity Disorder
Safety and Efficacy of Switching From a Stimulant Medication to Atomoxetine in Children and Adolescents With ADHD
The purpose of this study is to assess changes in ADHD symptoms and tolerability of medication in children and adolescents switching from a stimulant to atomoxetine.
- Trial name
- NCT00485875
- Testing
- Atomoxetine Hydrochloride
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Eli Lilly and Company
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
Gastroesophageal Reflux Disease
A Study to Evaluate the Effect of Lansoprazole on Infants With Gastroesophageal Reflux Disease.
The purpose of this study is to understand how quickly lansoprazole, once daily (QD), improves feeding in premature babies or babies less than 28 days of age.
- Trial name
- NCT00220818
- Testing
- Lansoprazole
- Volunteers
- 24 people
- Sponsor
- Takeda
Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia, Relapsed or Refractory B Cell Non-Hodgkin Lymphoma
A Study of CD19 Targeted CAR T Cell Therapy in Pediatric Patients With Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL)
This is a Phase 1b/2 study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting cluster of differentiation (CD)19 in pediatric patients with relapsed or refractory…
- Trial name
- NCT06173518
- Testing
- AUTO1
- Where
- 14 sites in United States, Spain and 1 more
- Volunteers
- 30 people
- Sponsor
- Autolus Limited
Urea Cycle Disorder
UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder
UNLOCKED: A Phase 2 Trial to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
- Trial name
- UNLOCKED (NCT03933410)
- Testing
- KB195
- Where
- 19 sites in United States, Belgium and 5 more
- Volunteers
- 13 people
- Sponsor
- Kaleido Biosciences
Primary Immune Deficiency Disorder
Study of Immune Globulin Intravenous (Human) GC5101F in Subjects With Primary Humoral Immunodeficiency
To assess the safety, efficacy, and pharmacokinetics of IGIV-SN in pediatric subjects with primary immunodeficiency humoral diseases (PHID)
- Trial name
- NCT03492710
- Testing
- Immunoglobulin
- Sponsor
- Green Cross Corporation
Healthy
Normative Oculomotor and Vestibular Data in Pediatric Population
The purpose of this study is to use a device called I-Portal® Portable Assessment System (I-PAS) to record eye movements in response to a number of visual and auditory stimuli designed to evaluate neural brain pathways related to…
- Trial name
- NCT04107610
- Testing
- I-PAS
- Where
- 2 sites in United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Neurolign
Dermatitis Atopic
Observational Evaluation of Atopic Dermatitis in Pediatric Patients
Primary Objectives: To describe the characteristics of pediatric patients with moderate to severe atopic dermatitis (AD) whose disease is not adequately controlled with topical therapies or when those therapies are not medically…
- Trial name
- PEDISTAD (NCT03687359)
- Testing
- Standard of care
- Where
- 182 sites in United States, Argentina and 18 more
- Volunteers
- 1,860 people
- Sponsor
- Sanofi
Chronic Kidney Disease Stage 3 and 4
Safety and Efficacy of Paricalcitol Capsules in Decreasing Serum Parathyroid Hormone Levels in Children Aged 10-16 With Chronic Kidney Disease (CKD)
Part 1: To determine the safety, tolerability, and pharmacokinetics of a single dose of 3 μg paricalcitol capsules in children ages 10 to 16 years with moderate to severe chronic kidney disease (CKD Stages 3 and 4).
- Trial name
- NCT01020487
- Testing
- Paricalcitol
- Volunteers
- 47 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease.
- Trial name
- HOPE Kids 2 (NCT04218084)
- Testing
- Voxelotor
- Where
- 35 sites in United States, Egypt and 7 more
- Volunteers
- 236 people
- Sponsor
- Pfizer
Systemic Lupus Erythematosus
Study of Subcutaneous (SC) Belimumab in Pediatric Participants With Systemic Lupus Erythematosus (SLE)
The purpose of this study is to evaluate the pharmacokinetics (PK), safety, and pharmacodynamics (PD) of repeat doses of 200 milligrams per milliliter (mg/mL) belimumab administered via SC injection in pediatric participants 5 to…
- Trial name
- NCT04179032
- Testing
- Belimumab
- Where
- 11 sites in United States, Argentina and 5 more
- Volunteers
- 25 people
- Sponsor
- GlaxoSmithKline
Bipolar Disorder, Pediatric
Efficacy and Safety of Asenapine Treatment for Pediatric Bipolar Disorder (P06107 Has an Extension [P05898; NCT01349907])(P06107)
Efficacy and safety of asenapine for the treatment of bipolar I disorder (manic or mixed episodes) will be evaluated in participants between 10 and 17 years old, who are either hospitalized or non-hospitalized.
- Trial name
- NCT01244815
- Testing
- asenapine, Rescue medication
- Volunteers
- 404 people
- Sponsor
- Organon and Co
Hereditary Angioedema (HAE)
Donidalorsen Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).
- Trial name
- NCT07298447
- Testing
- Donidalorsen
- Where
- 18 sites in United States, Australia and 3 more
- Volunteers
- 20 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
PFIC1, PFIC2
This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with progressive familial intrahepatic cholestasis (PFIC)…
- Trial name
- PEDFIC 1 (NCT03566238)
- Testing
- A4250 (odevixibat)
- Where
- 45 sites in United States, Australia and 13 more
- Volunteers
- 62 people
- Sponsor
- Albireo
Focal Segmental Glomerulosclerosis
Post Approval Study for Treatment of Drug-resistant Adult and Pediatric Primary FSGS Using the LIPOSORBER® LA-15 System
This multicenter, prospective, single-arm clinical study will evaluate the probable benefit and safety of the LIPOSORBER® LA-15 System for the treatment of adult patients with nephrotic syndrome associated with primary focal…
- Trial name
- FSGSALLAGE (NCT04065438)
- Testing
- LIPOSORBER® LA-15
- Where
- 10 sites in United States
- Volunteers
- 35 people
- Sponsor
- Kaneka Medical America LLC
Secondary Hyperparathyroidism, End-Stage Renal Disease
Study to Evaluate Safety of Vitamin D Receptor Activators in Patients Ages 0 to 16 With Chronic Kidney Disease Stage 5 Receiving Peritoneal Dialysis Within Current Clinical Practice
The objective of this study was to observe the safety of paricalcitol utilization in pediatric participants (ages 0 to 16 years old) being treated for secondary hyperparathyroidism (SHPT).
- Trial name
- NCT01134315
- Testing
- Paricalcitol, Calcitriol
- Where
- 18 sites in United States
- Volunteers
- 61 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Epilepsy
Efficacy and Tolerability of Levetiracetam Add-On Treatment in Refractory Pediatric Patients With Partial Onset Seizures
Double-blind, randomized, placebo-controlled, multi-center clinical trial conducted to evaluate levetiracetam as adjunctive therapy in children (4-16 years) with refractory partial onset seizures.
- Trial name
- NCT00615615
- Testing
- Levetiracetam
- Volunteers
- 216 people
- Sponsor
- UCB Pharma
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