Clinical trials: Disorder, Pediatric
Pompe Disease (Late-onset)
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric…
- Trial name
- NCT03911505
- Testing
- Cipaglucosidase Alfa, Miglustat
- Where
- 17 sites in United States, Australia and 4 more
- Volunteers
- 21 people
- Sponsor
- Amicus Therapeutics
Atopic Dermatitis, Pediatric ALL
TRacking Atopic Dermatitis Infants and Their Microbiome (TRAM)
TRacking Atopic dermatitis Infants and their Microbiome: A study to primarily assess the user experience of parents of infants with early onset eczema and parents of healthy infants on OneBiome real-world evidence research…
- Trial name
- TRAM (NCT06578351)
- Where
- Singapore
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Danone Asia Pacific Holdings Pte, Ltd.
Central Nervous System Tumors
Study of BLZ-100 in Pediatric Subjects With CNS Tumors
Many types of cancer are primarily treated with surgery and patient survival is directly related to the extent to which the tumor is able to be removed.
- Trial name
- NCT02462629
- Testing
- BLZ-100
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Blaze Bioscience Inc.
Asthma
A Study to Investigate How Budesonide and Formoterol Move Through the Body (Pharmacokinetics) When Delivered With Different Devices in Participants Aged 4 to Less Than 12 Years Old With Asthma
The purpose of this study is to assess the pharmacokinetics (PK) and safety of symbicort aerosphere and symbicort pressurized metered dose inhaler (pMDI) in participants with asthma aged 4 to less than 12 years.
- Trial name
- COMPAIR (NCT07433569)
- Testing
- Budesonide/formoterol fumarate Aerosphere, Budesonide/formoterol fumarate pMDI
- Where
- 6 sites in United States
- Volunteers
- 16 people
- Sponsor
- AstraZeneca
Infection, Human Immunodeficiency Virus I
48-Week Study Of GW433908 And Ritonavir Or GW433908 Alone, Twice Daily In Pediatric Patients With HIV Infection
This is a 48-week study to collect information on the safety and activity of an investigational medicine in patients, ages 2 to 18 years old, with HIV infection .
- Trial name
- NCT00089583
- Testing
- LEXIVA (GW433908), Ritonavir
- Where
- 39 sites in United States, Belgium and 5 more
- Volunteers
- 110 people
- Sponsor
- ViiV Healthcare
Lennox Gastaut Syndrome
Carisbamate in Adult & Pediatric Subjects With Lennox-Gastaut Syndrome
This is an open-label, multi-center study of carisbamate in adult and pediatric subjects with LGS, with single- and multiple-dose PK assessments from Days 1 through 73.
- Trial name
- NCT03731715
- Testing
- Carisbamate
- Where
- 7 sites in United States
- Volunteers
- 18 people
- Sponsor
- SK Life Science, Inc.
Congenital Myasthenic Syndrome, CMS
A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)
The purpose of this study is to assess efficacy and safety of adimanebart in participants at least 12 years of age with DOK7-, MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS).
- Trial name
- CoMetS (NCT07746089)
- Testing
- Adimanebart IV
- Volunteers
- 105 people
- Sponsor
- argenx
End Stage Renal Disease
Hemoglobin Maintenance in Pediatric ESRD (End-stage Renal Disease) Patients by Ferric Pyrophosphate Citrate (FPC)
The main purpose is to determine the safety of Triferic iron administered via dialysate and intravenously in pediatric patients with chronic kidney disease on chronic hemodialysis (CKD-5HD).
- Trial name
- NCT04239391
- Testing
- Triferic
- Where
- 9 sites in United States, Puerto Rico
- Volunteers
- 150 people
- Sponsor
- Rockwell Medical Technologies, Inc.
Pediatric ALL
Investigation of Remimazolam in Children Undergoing Sedation for Medical Procedures
To assess the efficacy of intravenous remimazolam in inducing and maintaining suitable sedation levels for paediatric patients undergoing diagnostic and/or therapeutic procedures
- Trial name
- NCT04851717
- Testing
- Remimazolam
- Where
- 8 sites in United States, Denmark
- Volunteers
- 100 people
- Sponsor
- Acacia Pharma Ltd
Acute Graft Versus Host Disease
Study of Pharmacokinetics, Activity and Safety of Ruxolitinib in Pediatric Patients With Grade II-IV Acute Graft vs. Host Disease
The study was an open-label, single-arm, Phase I/II multi-center study to investigate the PK, activity and safety of ruxolitinib added to the patient's immunosuppressive regimen in infants, children, and adolescents ages ≥28 days…
- Trial name
- NCT03491215
- Testing
- Ruxolitinib
- Where
- 19 sites in Belgium, Canada and 6 more
- Volunteers
- 45 people
- Sponsor
- Novartis Pharmaceuticals
Psoriasis, Atopic Dermatitis
Study to Evaluate Effect of Pandel Cream 0.1% on HPA Axis in Pediatric and Adult Population
The purpose of this study is to determine the effect of Pandel® (hydrocortisone probutate cream) Cream 0.1% on the Hypothalamic Pituitary Adrenal (HPA) axis in pediatric and adult subjects with either psoriasis or atopic…
- Trial name
- NCT01137032
- Testing
- Pandel Cream 0.1%
- Where
- 3 sites in United States
- Volunteers
- 19 people
- Sponsor
- Fougera Pharmaceuticals Inc.
Asthma
Efficacy and Safety of Fluticasone Propionate(FP)/ Salmeterol Xinafoate (SLM) Hydro Fluoro Alkane (HFA) Metered Dose Inhaler (MDI) in Pediatric Patients With Bronchial Asthma
This study is a multicenter, stratified, randomized, active control, double-blinded, parallel-group comparative study with an open-label extension period.
- Trial name
- NCT02113436
- Testing
- FP/ SLM HFA MDI 50/25 mcg, FP HFA MDI 50 mcg
- Where
- 69 sites in Japan
- Volunteers
- 300 people
- Sponsor
- GlaxoSmithKline
Asthma
Pediatric Study to Evaluate Treatment of Persistent Moderate or Severe Asthma With the Association Fluticasone 250 mcg/Dose and Salmeterol 50 mcg/Dose - ADERE PROJECT
Prospective, parallel-group, randomized study to evaluate the compliance to the treatment of children aged between 6 and 14 years old with persistent moderate or severe asthma, receiving the association Fluticasone 250 mcg/dose…
- Trial name
- ADERE (Ped) (NCT01476059)
- Testing
- Telephone follow-up, No telephone follow-up
- Volunteers
- 472 people
- Sponsor
- GlaxoSmithKline
Myeloid Leukemia, Philadelphia Positive
Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
The aim of this study is to support development of asciminib in the pediatric population (1 to <18 years) previously treated with one or more TKIs.
- Trial name
- NCT04925479
- Testing
- Asciminib Pediatric formulation group, Asciminib Adult formulation group
- Where
- 39 sites in United States, China and 12 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
HIV Infections
The Safety and Effectiveness of Two Forms of Saquinavir Combined With Other Anti-HIV Drugs in HIV-Infected Infants and Children
To determine the pharmacokinetic properties, tolerance and safety of 2 formulations of saquinavir, given in combination with 2 nucleoside antiretroviral drugs (part 1) or in combination with nelfinavir and nucleoside…
- Trial name
- NCT00002380
- Testing
- Nelfinavir mesylate, Saquinavir
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study.
- Trial name
- ENVISION (NCT05881408)
- Testing
- delandistrogene moxeparvovec
- Where
- 46 sites in United States, Australia and 12 more
- Volunteers
- 148 people
- Sponsor
- Sarepta Therapeutics, Inc.
Otitis Media, Infections, Respiratory Tract
Study to Evaluate the Safety and Clinical Efficacy of Augmentin® Extra Strength-600 in Children With Acute Otitis Media in India
Augmentin (ES)-600 is a high-dose amoxicillin/clavulanic acid 14:1 formulation that allows administration at 90/6.4 milligrams (mg)/kilograms (kg)/day in two divided doses.
- Conditions
- Otitis Media, Infections, Respiratory Tract, Respiratory Tract Infections
- Trial name
- NCT04600752
- Testing
- Augmentin (ES)-600
- Where
- 13 sites in India
- Volunteers
- 310 people
- Sponsor
- GlaxoSmithKline
Dravet Syndrome
An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome
Stoke Therapeutics is evaluating the long-term safety \& tolerability of repeated doses of zorevunersen (STK-001) in patients with Dravet syndrome who previously participated in studies of zorevunersen.
- Trial name
- NCT04740476
- Testing
- zorevunersen (STK-001)
- Where
- 17 sites in United States
- Volunteers
- 60 people
- Sponsor
- Stoke Therapeutics, Inc
Growth Disorders
A Canadian, Multi-centre, Observational Registry to Study Adherence and Long Term Outcomes of Therapy in Pediatric Subjects Using SAIZEN® Via Easypod™ Auto-injector for Growth Hormone Treatment
This is a Canadian, Multicentre, Observational Registry to study Adherence and Long Term Outcomes of Therapy in paediatric subjects using Easypod™ electromechanical device for growth hormone treatment from hospital and to assess…
- Trial name
- ECOS CAN (NCT01267526)
- Testing
- Easypod™
- Where
- 9 sites in Canada
- Volunteers
- 205 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Crohn's Disease, Ulcerative Colitis
Expanded Access to Vedolizumab for Children and Teenagers With Ulcerative Colitis or Crohn's Disease in the USA
The expanded access program (EAP) allows people to gain access to an unlicensed treatment on compassionate grounds. This EAP provides children and teenagers with Ulcerative Colitis (UC) or Crohn's Disease (CD) who completed the…
- Trial name
- NCT06856135
- Testing
- Vedolizumab IV
- Where
- 3 sites in United States
- Sponsor
- Takeda
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