Clinical trials: Disorder, Pediatric
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Hemophilia A
Russian Kogenate Pediatric Study
A prospective study to evaluate the effect of rFVIII-FS in different prophylactic regimens on bleeding events frequency and development of arthropathy in Previously Treated and Minimally treated Hemophilia A pediatric population.
- Trial name
- NCT00632814
- Testing
- rFVIII-FS (Kogenate FS, BAY14-2222) 70 IU/kg, dosing once per week, rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg twice per week (30 IU/kg + 40 IU/kg), rFVIII-FS (Kogenate FS, BAY14-2222) 75 IU/kg, dosing three times per week (3 x 25 IU/kg)
- Where
- 4 sites in Russia
- Volunteers
- 32 people
- Sponsor
- Bayer
Vernal Keratoconjunctivitis
Double-Masked Trial of NOVA22007 (1mg/mL Ciclosporin/Cyclosporine) Versus Vehicle in Pediatric Patients With Active Severe Vernal Keratoconjunctivitis
The objective of this study is to compare the efficacy of two different dosing regimen of NOVA22007 (1mg/ml ciclosporin/cyclosporine) eye drops, emulsion versus placebo (vehicle of the formulation) administered four times a day…
- Trial name
- NCT01751126
- Testing
- NOVA22007 ''Ciclosporin''
- Where
- 46 sites in United States, Croatia and 9 more
- Volunteers
- 169 people
- Sponsor
- Santen SAS
Mycobacterium Avium-intracellulare Infection, HIV Infections
The Safety and Effectiveness of Clarithromycin Plus Zidovudine or Dideoxyinosine in the Treatment of Mycobacterium Avium Complex (MAC) Infections in Children With AIDS
To evaluate three doses of clarithromycin in children with AIDS and Mycobacterium avium complex (MAC) infection who are receiving concurrent antiretroviral therapy.
- Trial name
- NCT00000971
- Testing
- Clarithromycin, Zidovudine, Didanosine
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Abbott
Attention Deficit Hyperactivity Disorder
Open-label, Single-dose Trial to Assess the Pharmacokinetics of Centanafadine Extended-release Capsules in Pediatric Participants With Attention-deficit Hyperactivity Disorder (ADHD)
With the pharmacokinetics (PK) of centanafadine currently being evaluated in adults. The PK of extended-release centanafadine may differ in children compared to adults due to physiological differences in the gastrointestinal…
- Trial name
- NCT04081363
- Testing
- Centanafadine
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Autism Spectrum Disorder
Study of Cariprazine Oral Capsules or Solution to Assess Adverse Events and Change in Irritability Due to Autism Spectrum Disorder (ASD) in Participants Aged 5-17 Years With ASD
Autism spectrum disorder (ASD) consists of deficits in social, communication, and cognitive development, repetitive and stereotypic behaviors.
- Trial name
- NCT05439616
- Testing
- Cariprazine
- Where
- 36 sites in United States, Puerto Rico
- Volunteers
- 161 people
- Sponsor
- AbbVie
Cradle Cap, Infantile Seborrheic Dermatitis
Promiseb Topical Cream for Cradle Cap
The purpose of this study is to evaluate the efficacy and safety of Promiseb Topical Cream in cradle cap.
- Trial name
- NCT01214434
- Testing
- Promiseb Topical Cream, Bland emollient
- Where
- 4 sites in United States
- Volunteers
- 42 people
- Sponsor
- Promius Pharma, LLC
Fragile x Syndrome
Ganaxolone Treatment in Children With Fragile X Syndrome
This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, crossover study to investigate ganaxolone treatment in children with fragile x syndrome (FXS).
- Trial name
- NCT01725152
- Testing
- Ganaxolone
- Where
- 2 sites in United States, Belgium
- Volunteers
- 59 people
- Sponsor
- Marinus Pharmaceuticals
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
HIV Infections
Pediatric Expanded Access Program-Oral Solution (0831-908)
Monitor the safety and tolerability of efavirenz oral solution in combination with Antiretroviral Therapy for the treatment of patients age 3 to 16 years who have failed therapy or who are intolerant to their current therapy.
- Trial name
- NCT00543530
- Testing
- MK0831
- Sponsor
- Merck Sharp & Dohme LLC
HIV Infections
Pharmacokinetics and Safety Study of Tipranavir in Combination With Low Dose Ritonavir in Human Immunodeficiency Virus (HIV)-Infected Children
The primary objective of this study is to assess the safety and tolerability of tipranavir (TPV) oral formulation and soft gelatin capsules together with low-dose ritonavir in HIV-infected children and adolescents, to provide…
- Trial name
- NCT00076999
- Testing
- TPV oral solution, RTV oral solution
- Where
- 30 sites in United States, Argentina and 8 more
- Volunteers
- 115 people
- Sponsor
- Boehringer Ingelheim
Asthma
Efficacy of Ciclesonide Inhaled Once Daily Versus Fluticasone Propionate Inhaled Twice Daily in Children With Asthma (4 to 15 y) (BY9010/M1-205)
The aim of the study is to compare the efficacy of ciclesonide versus fluticasone propionate on lung function, symptoms, and use of rescue medication in children with persistent asthma.
- Trial name
- NCT00163410
- Testing
- Ciclesonide
- Where
- 16 sites in India
- Volunteers
- 500 people
- Sponsor
- AstraZeneca
Pupillary Dilation Effect During Cycloplegic Refraction
Comparing Efficacy and Safety of CTO0303 in Pediatric Subjects
This is a prospective randomized study compared with active control arm.
- Trial name
- NCT06609525
- Testing
- CTO0303-A, CTO0303-B, CTO0303-C
- Where
- South Korea
- Volunteers
- 156 people, healthy volunteers welcome
- Sponsor
- Taejoon Pharmaceutical Co., Ltd.
Crohn Disease
A Pharmacokinetic Study of Ustekinumab in Pediatric Subjects With Moderately to Severely Active Crohn's Disease
The purpose of this study is to evaluate the pharmacokinetics (PK) of ustekinumab in subjects from 2 through less than (<) 18 years old in the USA, or 6 through less than (<) 18 years old in other countries and determine if it is…
- Trial name
- STELARA (NCT02968108)
- Testing
- Ustekinumab
- Where
- 26 sites in United States, Belgium and 4 more
- Volunteers
- 45 people
- Sponsor
- Janssen Research & Development, LLC
Growth Disorders
To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in Greece
This is a National, Multicentre, Observational Registry to study adherence and long term outcomes of therapy in paediatric subjects using Easypod™ electromechanical device for growth hormone treatment from hospitals in Greece and…
- Trial name
- ECOS GRE (NCT01363674)
- Testing
- Easypod™
- Where
- Greece
- Volunteers
- 180 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Febrile Neutropenia, Neutropenia
Prevention of Febrile Neutropenia by Synbiotics in Pediatric Cancer Patients
Febrile neutropenia (FN) is a major life-threatening treatment complication in cancer patients undergoing intensive chemotherapy. Endogenous flora is considered to be one of the main sources of infections during neutropenia.
- Conditions
- Febrile Neutropenia, Neutropenia, Infection in an Immunocompromised Host, Cancer
- Trial name
- FENSY (NCT02544685)
- Testing
- Probio-Fix Inum, Beneo Synergy 1
- Where
- Slovakia
- Volunteers
- 120 people
- Sponsor
- S&D Pharma SK s.r.o.
Heart Failure, Left Ventricular Systolic Dysfunction
Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036)
This study aims to compare the efficacy of vericiguat versus placebo on change in n-terminal pro-brain natriuretic peptide (NTproBNP) from baseline to Week 16 of the Base Period.
- Trial name
- NCT05714085
- Testing
- Vericiguat tablet, Vericiguat suspension
- Where
- 111 sites in United States, Belgium and 28 more
- Volunteers
- 342 people
- Sponsor
- Merck Sharp & Dohme LLC
Healthy Volunteers (Meningococcal Infection)
Immunogenicity and Safety of a Quadrivalent Meningococcal Conjugate Vaccine When Administered Concomitantly With Routine Pediatric Vaccines in Healthy Infants and Toddlers in the US
The purpose of this study was to compare the immunogenicity and describe the safety of MenACYW conjugate vaccine and MENVEO® when both are administered concomitantly with routine pediatric vaccines to healthy infants and toddlers…
- Trial name
- NCT03537508
- Testing
- MenACYW conjugate vaccine, MenACYW-135 conjugate vaccine, DTaP-IPV//Hib vaccine
- Where
- 70 sites in United States, Puerto Rico
- Volunteers
- 2,627 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Healthy Subjects, Purpura, Thrombocytopenic, Idiopathic
Eltrombopag Taste Testing in Healthy Adult Volunteers
Based on a need for a pediatric formulation of eltrombopag for the treatment of pediatric thrombocytopenia, this study will help determine the best tasting pediatric formulation of eltrombopag.
- Trial name
- NCT00487968
- Testing
- eltrombopag
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
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
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