Clinical trials: Pediatric Disease
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Insomnia
Dose-finding Study Assessing the Efficacy, Safety, and Pharmacokinetics of Daridorexant in Subjects Aged 10 to < 18 Years With Insomnia Disorder
This study assesses the efficacy, safety, and pharmacokinetics of multiple-dose oral administration of daridorexant in pediatric subjects aged 10 to < 18 years with insomnia disorder.
- Trial name
- NCT05423717
- Testing
- Daridorexant 10 mg, Daridorexant 25 mg, Daridorexant 50 mg
- Where
- 35 sites in United States, Bulgaria and 4 more
- Volunteers
- 166 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Von Willebrand Disease, Blood Coagulation Disorders
Study of Safety and Efficacy of Antihemophilic Factor/Von Willebrand Factor Complex in Surgical Subjects With Von Willebrand Disease (vWD)
The purpose of this study is to test the safety and effectiveness of Humate-P® to prevent bleeding in patients with von Willebrand Disease who are undergoing surgery.
- Conditions
- Von Willebrand Disease, Blood Coagulation Disorders, Blood Platelet Disorders, Hematologic Disease
- Trial name
- NCT00168090
- Testing
- Blood coagulation Factor VIII and vWF, human
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- CSL Behring
Asthma
Montelukast Post-marketing Comparative Study With Theophyline Added to Inhaled Corticosteroid (0476-396)
A clinical study explores the safety and efficacy of montelukast and to compare the safety and efficacy of montelukast with those of theophylline in pediatric bronchial asthma patients when they are used in conjunction with…
- Trial name
- NCT00756418
- Testing
- montelukast, Theophylline
- Volunteers
- 84 people
- Sponsor
- Organon and Co
Atopic Dermatitis
Tapinarof for the Treatment of Atopic Dermatitis in Children and Adults
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
- NCT05014568
- Testing
- tapinarof cream, 1%, Vehicle cream
- Where
- 67 sites in United States, Canada
- Volunteers
- 407 people
- Sponsor
- Organon and Co
Cystic Fibrosis
A Study in Adult and Pediatric Patients With Cystic Fibrosis
The purpose of this ascending dose group study is to assess the safety, tolerability, and effects of multiple dose levels of INS37217 and placebo followed by five days twice daily treatment with maximum tolerated dose…
- Trial name
- NCT00034515
- Testing
- denufosol tetrasodium (INS37217) Inhalation Solution
- Volunteers
- 63 people
- Sponsor
- Merck Sharp & Dohme LLC
Pediatric Glaucoma, Elevated IOP in Pediatric Patients
Study of Travoprost Ophthalmic Solution, 0.004% Compared to Timolol (0.5% or 0.25%) in Pediatric Glaucoma Patients
The purpose of this study is to evaluate the intraocular pressure (IOP)-lowering efficacy of Travoprost 0.004% POLYQUAD (PQ) ophthalmic solution compared to Timolol ophthalmic solution (0.5% or 0.25%) in pediatric glaucoma…
- Conditions
- Pediatric Glaucoma, Elevated IOP in Pediatric Patients, Ocular Hypertension in Pediatric Patients
- Trial name
- NCT01652664
- Testing
- Travoprost 0.004% PQ ophthalmic solution, Timolol, 0.5% or 0.25% ophthalmic solution, Travoprost Vehicle
- Volunteers
- 184 people
- Sponsor
- Alcon Research
Kawasaki Disease
Study of Efficacy and Safety of Canakinumab in Pediatric Patients With Kawasaki Disease
The purpose of this trial is to evaluate whether, in Intravenous Immunoglobulin-naïve or refractory patients diagnosed with active Kawasaki disease, administration of canakinumab controls fever and acute phase reactants.
- Trial name
- NCT02980263
- Testing
- Canakinumab
- Sponsor
- Novartis Pharmaceuticals
Non-hodgkin Lymphoma
Study of the Adverse Events and Change in Disease State of Pediatric Participants (and Young Adults Between the Ages of 18-25) With Relapsed/Refractory Aggressive Mature B-cell Neoplasms Receiving Subcutaneous (SC) Injections of Epcoritamab
The most common types of mature B-cell lymphomas (MBLs) in children are Burkitt lymphoma (BL) and diffuse large B-cell lymphoma (DLBCL).
- Trial name
- NCT05206357
- Testing
- Epcoritamab
- Where
- 41 sites in United States, Australia and 12 more
- Volunteers
- 17 people
- Sponsor
- Genmab
Respiratory Syncytial Virus (RSV)
Study to Learn About a Respiratory Syncytial Virus Vaccine in Children at High Risk of RSV Disease
The purpose of the study is to evaluate the safety, tolerability, and immunogenicity of RSVpreF in children 5 to <18 years of age at high risk for RSV disease, including those who are immunocompromised.
- Trial name
- NCT07839871
- Testing
- RSVpreF
- Where
- 2 sites in United States, Japan
- Volunteers
- 920 people, healthy volunteers welcome
- Sponsor
- Pfizer
Asthma
Efficacy and Safety Comparison of Albuterol Spiromax® and ProAir® Hydrofluoroalkane (HFA) in Pediatric Patients
This is a multicenter, randomized, double-blind, double-dummy, placebo-controlled, single-dose, 5-treatment, 5-period, 5-way crossover study in pediatric patients with persistent asthma.
- Trial name
- NCT01899144
- Testing
- Albuterol Spiromax, ProAir HFA
- Where
- 22 sites in United States
- Volunteers
- 61 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Congenital Hearing Loss Secondary to Biallelic Mutations in the Otoferlin Gene (OTOF), Biallelic Mutations in the Gap Junction Beta 2 (GJB2) Gene
A Natural History Study in Pediatric Participants With Hearing Loss Due to OTOF, GJB2, or GJB2/GJB6 Mutations
This is an observational study to examine the characteristics of gene-related hearing loss in pediatric participants with biallelic otoferlin (OTOF) Mutations, Gap Junction Beta 2 (GJB2) Mutations, or Digenic GJB2/Gap Junction…
- Conditions
- Congenital Hearing Loss Secondary to Biallelic Mutations in the Otoferlin Gene (OTOF), Biallelic Mutations in the Gap Junction Beta 2 (GJB2) Gene, Digenic Mutations in GJB2/Gap Junction Beta 6 (GJB6) Genes
- Trial name
- ENSEMBLE (NCT06019481)
- Where
- 2 sites in United States
- Sponsor
- Regeneron Pharmaceuticals
Rhinitis
Safety Study of Levocetirizine Oral Solution for Japanese Pediatrics
To evaluate the safety of treatment with levocetirizine oral solution in pediatric patients aged form 6 months to 2 years old with allergic rhinitis or pruritus associated with the skin diseases.
- Trial name
- NCT01563081
- Testing
- Levocetirizine
- Where
- 7 sites in Japan
- Volunteers
- 60 people
- Sponsor
- GlaxoSmithKline
Growth Disorders
To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in Mexico
This is a Mexican, Multicentre, Observational Study to evaluate Adherence and Long Term Outcomes of Therapy in paediatric subjects using easypod™ electromechanical device for growth hormone treatment to assess the level of…
- Trial name
- ECOS MEX (NCT01555528)
- Testing
- easypod™
- Where
- Mexico
- Volunteers
- 193 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Acute Otitis Media
Efficacy and Safety of DF289 Plus DF277 Otic Solution in the Treatment of Middle Ear Infections in Pediatric Patients
The purpose of this study is to determine if a combination of an antibiotic plus a corticosteroid is safe and effective in treating middle ear infections in children with ear tubes.
- Trial name
- NCT01404611
- Testing
- DF289, DF277, DF289 plus DF277
- Where
- Spain
- Volunteers
- 331 people
- Sponsor
- Salvat
Gastroenteritis
Gastroenteritis in Pediatric Population of Qatar
Comparison of clinical and laboratory diagnosis for cause of Gastroenteritis(GE) depending on the clinical manifestation.Identifying the sources of viral, bacterial and parasitic GE in Pediatric population of Qatar
- Trial name
- GE (NCT03046342)
- Testing
- Molecular method (PCR)
- Where
- Qatar
- Volunteers
- 3,000 people
- Sponsor
- Hamad Medical Corporation
Pediatrics, Muscle Spasticity
BOTOX® Treatment in Pediatric Upper Limb Spasticity
This study will evaluate the safety and efficacy of BOTOX® (botulinum toxin Type A) in pediatric patients with upper limb spasticity.
- Conditions
- Pediatrics, Muscle Spasticity, Cerebral Palsy, Stroke
- Trial name
- NCT01603602
- Testing
- botulinum toxin Type A
- Where
- 45 sites in United States, Canada and 7 more
- Volunteers
- 235 people
- Sponsor
- Allergan
Neutropenia, Fever
Use of NGS Cell-free Pathogen Test for Identification of Low Risk Fever & Neutropenia in Pediatric Patients
Febrile neutropenia is a common complication in pediatric oncology patients. Standard of care requires admission of all patients for intravenous antibiotics until cultures are negative, patients are afebrile and there are signs…
- Trial name
- NCT03530072
- Testing
- Karius Test
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Karius, Inc.
Psoriasis
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneously Administered Guselkumab for the Treatment of Chronic Plaque Psoriasis in Pediatric Participants
The purpose of this study is to evaluate the efficacy and safety of guselkumab in pediatric participants aged greater than or equal to 6 through less than 18 years with chronic plaque psoriasis.
- Trial name
- PROTOSTAR (NCT03451851)
- Testing
- Guselkumab, Etanercept
- Where
- 39 sites in United States, Australia and 7 more
- Volunteers
- 120 people
- Sponsor
- Janssen Research & Development, LLC
Tinea Capitis
A Study Of Diflucan In Children With Ringworm Of The Scalp
The purpose of this study is to evaluate the safety and efficacy of fluconazole for the treatment of tinea capitis in pediatric patients aged 3 to 12 years.
- Trial name
- NCT00645242
- Testing
- fluconazole
- Where
- 31 sites in United States, Puerto Rico
- Volunteers
- 185 people
- Sponsor
- Pfizer
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