Clinical trials: Pediatric Disease
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Asthma
FLOVENT Hydrofluoroalkane (HFA) In 6-12 Month Old Subjects
The purpose of this study is to address an Food Drug and Administration (FDA) request to provide safety data in pre-asthmatic patients, ages 6 months to <12 months, following administration of fluticasone propionate HFA.
- Trial name
- NCT00370097
- Testing
- FLOVENT (fluticasone propionate) HFA
- Where
- 7 sites in United States
- Volunteers
- 16 people
- Sponsor
- GlaxoSmithKline
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
Growth Hormone Deficiency (GHD)
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
The OraGrowtH Phase 3 Trial is a multi-national trial. The goals of the trial are to study LUM-201 as a treatment for Pediatric Growth Hormone Deficiency (PGHD) in naive to treatment children and validate the LUM-201 predictive…
- Trial name
- NCT06948214
- Testing
- LUM-201
- Where
- 55 sites in United States, Argentina and 6 more
- Volunteers
- 150 people
- Sponsor
- Lumos Pharma
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
Asthma
An Open-label Study to Evaluate Correct Use and Ease of Use of the ELLIPTA DPI in Pediatric Subjects With Asthma
Asthma is a chronic inflammatory disease of airways and lungs that results in hyper-reactivity and clinically relevant episodes of wheezing.
- Trial name
- NCT03478657
- Testing
- ELLIPTA DPI, Ease of use questionnaires
- Where
- 19 sites in United States, Canada
- Volunteers
- 222 people
- Sponsor
- GlaxoSmithKline
Still´s Disease
A Phase II Trial to Evaluate the Clinical Efficacy, Safety and Tolerability of MAS825 in Pediatric and Adult Participants With Still's Disease
The study is a phase II trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 (arumakimig) in pediatric and adult participants with Still's disease
- Trial name
- NCT07203001
- Testing
- MAS825
- Where
- 23 sites in United States, Canada and 6 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Type 2 Diabetes Mellitus
A Study to Evaluate Pharmacokinetics of LY3209590 in Pediatric Participants With Type 2 Diabetes Mellitus
The main purpose of this study is to evaluate how much of LY3209590 gets into the blood stream after a single dose and how long it takes the body to remove it in pediatric participants with Type 2 Diabetes Mellitus (T2DM).
- Trial name
- NCT06362265
- Testing
- LY3209590
- Where
- 10 sites in United States
- Volunteers
- 15 people
- Sponsor
- Eli Lilly and Company
Solid Tumor, Lymphoma
A Study to Evaluate Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Tripegfilgrastim in Pediatric Solid Tumor/Lymphoma Patients
This is an open-label, single ascending dose study to evaluate safety, tolerability, and pharmacokinetics/pharmacodynamics of Tripegfilgrastim in pediatric solid tumor/lymphoma patients
- Trial name
- NCT02963389
- Testing
- Tripegfilgrastim
- Where
- South Korea
- Volunteers
- 27 people
- Sponsor
- Dong-A ST Co., Ltd.
Heart Decompensation
Levosimendan Compassionate Use in Pediatric Patients With Advanced Decompensated Heart Failure
Open label study of levosimendan; planned comparison for effectiveness and safety to historic matched case controls from participating sites.
- Trial name
- NCT02973620
- Testing
- Levosimendan
- Sponsor
- Tenax Therapeutics, Inc.
Community-acquired Pneumonia (CAP), Hospital-acquired Pneumonia (HAP)
Ceftobiprole in the Treatment of Pediatric Patients With Pneumonia
This was a study of the safety and efficacy of ceftobiprole medocaril compared with intravenous (IV) standard-of-care cephalosporin treatment with or without vancomycin in pediatric patients with either hospital-acquired…
- Trial name
- NCT03439124
- Testing
- ceftobiprole medocaril, IV standard-of-care cephalosporin
- Where
- 19 sites in Bulgaria, Georgia and 2 more
- Volunteers
- 138 people
- Sponsor
- Basilea Pharmaceutica
Mucopolysaccharidosis II
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant…
- Trial name
- COMPASS (NCT05371613)
- Testing
- tividenofusp alfa, idursulfase
- Where
- 30 sites in United States, Argentina and 12 more
- Volunteers
- 65 people
- Sponsor
- Denali Therapeutics Inc.
Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome
Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)
- Conditions
- Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease
- Trial name
- NCT01302860
- Testing
- ACZ885
- Where
- 12 sites in Belgium, Canada and 5 more
- Volunteers
- 17 people
- Sponsor
- Novartis Pharmaceuticals
Acne Vulgaris
A Study of Acne Treatment in Children Ages 9 to 11
A study to determine if using Retin-A Micro 0.04% as facial acne treatment in patients ages 9 to 11 is safe and efficacious.
- Trial name
- NCT00907335
- Testing
- Retin-A Micro 0.04% facial acne treatment, Vehicle control
- Where
- 12 sites in United States
- Volunteers
- 110 people
- Sponsor
- Bausch Health Americas, Inc.
Asthma
A Study In Pediatric Subjects With Asthma Using ADVAIR HFA, ADVAIR HFA With Spacer, And ADVAIR DISKUS
Collect PK/PD data for this age group to compare ADVAIR DISKUS with ADVAIR HFA
- Trial name
- NCT00400608
- Testing
- ADVAIR HFA, ADVAIR DISKUS
- Where
- 3 sites in United States
- Volunteers
- 28 people
- Sponsor
- GlaxoSmithKline
Sickle Cell Anemia
Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia
The study assesses the efficacy, safety and tolerability of ACZ885 (canakinumab) in pediatric and young adult patients with sickle cell anemia (SCA).
- Trial name
- NCT02961218
- Testing
- ACZ885
- Where
- 15 sites in United States, Canada and 5 more
- Volunteers
- 49 people
- Sponsor
- Novartis Pharmaceuticals
Multiple Sclerosis
Efficacy, Safety and Pharmacokinetics of Teriflunomide in Pediatric Patients With Relapsing Forms of Multiple Sclerosis
Primary Objective: To assess the effect of teriflunomide in comparison to placebo on disease activity measured by time to first clinical relapse after randomization in children and adolescents 10 to 17 years of age with relapsing…
- Trial name
- TERIKIDS (NCT02201108)
- Testing
- Teriflunomide
- Where
- 57 sites in United States, Belgium and 20 more
- Volunteers
- 166 people
- Sponsor
- Genzyme, a Sanofi Company
Immune Thrombocytopenia (ITP), ITP - Immune Thrombocytopenia
A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)
The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP).
- Conditions
- Immune Thrombocytopenia (ITP), ITP - Immune Thrombocytopenia, ITP, Immune Thrombocytopenic Purpura
- Trial name
- Advance Jr (NCT07194850)
- Testing
- Efgartigimod IV
- Where
- 26 sites in France, Germany and 8 more
- Volunteers
- 24 people
- Sponsor
- argenx
Amblyopia, Strabismus
Amblyopia and Strabismus Detection Using a Pediatric Vision Scanner
The purpose of this project is to compare the performance of the Pediatric Vision Scanner (PVS) with the Gold Standard Examination to determine the effectiveness of the PVS at detecting amblyopia and strabismus.
- Trial name
- NCT01706991
- Testing
- Pediatric Vision Scanner eye scan
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Rebiscan, Inc.
Infection, Human Immunodeficiency Virus, HIV Infections
Study Of An Investigational Regimen Including FDA Approved HIV Drugs In HIV-Infected Pediatric Subjects
This is a 48-week study to evaluate the safety, tolerability, pharmacokinetics, and antiviral activity of an investigational regimen including FDA approved HIV drugs in HIV-infected pediatric subjects, ages 4 weeks to < 2 years…
- Trial name
- NCT00071760
- Testing
- GW433908, ritonavir
- Where
- 15 sites in United States, Argentina and 5 more
- Volunteers
- 59 people
- Sponsor
- ViiV Healthcare
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