Clinical trials: Childhood
Idiopathic Thrombocytopenic Purpura
Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body.
The purpose of this study is to investigate the efficacy, safety and tolerability of eltrombopag in children with previously treated chronic immune thrombocytopenia who are between 1 and 17 years of age. This is a 2 part study.
- Trial name
- PETIT2 (NCT01520909)
- Testing
- Eltrombopag
- Where
- 51 sites in United States, Argentina and 11 more
- Volunteers
- 92 people
- Sponsor
- GlaxoSmithKline
Spinal Muscular Atrophy 1
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT02122952
- Testing
- AVXS-101
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novartis Gene Therapies
Heterozygous Familial Hypercholesterolemia
Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.
- Trial name
- HAUSER-RCT (NCT02392559)
- Testing
- Evolocumab
- Where
- 66 sites in United States, Australia and 24 more
- Volunteers
- 158 people
- Sponsor
- Amgen
Childhood Absence Epilepsy, Juvenile Absence Epilepsy
A Study to Test the Long-term Safety, Tolerability and Efficacy of Brivaracetam in Study Participants 2 to 26 Years of Age With Childhood Absence Epilepsy or Juvenile Absence Epilepsy
The purpose of the study is to investigate the long-term safety, tolerability and efficacy of brivaracetam in pediatric study participants with childhood absence epilepsy (CAE) or juvenile absence epilepsy (JAE).
- Trial name
- NCT05109234
- Testing
- Brivaracetam Film-coated tablet, Brivaracetam oral solution
- Where
- 24 sites in United States, Georgia and 4 more
- Volunteers
- 84 people
- Sponsor
- UCB Biopharma SRL
Attention Deficit/Hyperactivity Disorder
Efficacy and Safety Study of Methylphenidate Hydrochloride Extended Release in Adults With Childhood-onset Attention Deficit/Hyperactivity Disorder (ADHD)
This study will evaluate efficacy and safety of methylphenidate hydrochloride extended release compared to placebo in adult patients with childhood-onset attention deficit/hyperactivity disorder (ADHD).
- Trial name
- NCT01259492
- Testing
- Ritalin LA 20 mg, Ritalin LA 30 mg
- Where
- 68 sites in United States, Belgium and 7 more
- Volunteers
- 725 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy Type I
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic…
- Trial name
- NCT03837184
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 4 sites in Japan, South Korea and 1 more
- Volunteers
- 2 people
- Sponsor
- Novartis Gene Therapies
Childhood Idiopathic Nephrotic Syndrome
A Study to Evaluate the Efficacy and Safety of Obinutuzumab Versus MMF in Participants With Childhood Onset Idiopathic Nephrotic Syndrome
This open-label, randomized multicenter study is to assess the efficacy, safety, and pharmacokinetics (PK)/pharmacodynamics (PD) of obinutuzumab compared with mycophenolate mofetil (MMF) in children and young adults (aged >= 2-25…
- Trial name
- INShore (NCT05627557)
- Testing
- Obinutuzumab, MMF, Prednisone
- Where
- 35 sites in United States, Belgium and 7 more
- Volunteers
- 85 people
- Sponsor
- Hoffmann-La Roche
Varicella, Chickenpox Vaccines
Evaluate Vaccine Against Chickenpox and a Combined Vaccine Against 4 Viral Childhood Diseases: Measles, Mumps, Rubella and Chickenpox
An observer-blind study to evaluate GlaxoSmithKline Biologicals' live attenuated varicella vaccine and GlaxoSmithKline Biologicals' combined measles-mumps-rubella-varicella vaccine in the prevention of varicella disease in…
- Trial name
- NCT00226499
- Testing
- Priorix-tetra™, Priorix™, Varilrix™
- Where
- 104 sites in Czechia, Greece and 8 more
- Volunteers
- 5,803 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
Childhood-Onset Fluency Disorder (Stuttering)
Effects of Ecopipam or Placebo in Adults With Stuttering (Speak Freely)
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, Phase 2 exploratory study in adult subjects with childhood onset fluency disorder.
- Trial name
- NCT04492956
- Testing
- Ecopipam
- Where
- 9 sites in United States
- Volunteers
- 68 people
- Sponsor
- Emalex Biosciences Inc.
Childhood-Onset Fluency Disorder
A 10-week Efficacy Study of NOE-105 in Childhood Onset Fluency Disorder (Orpheus)
This study is designed to evaluate the effectiveness of NOE-105 on speech fluency without the known antipsychotic-induced side effects of commonly used treatments for childhood onset fluency disorder (COFD).
- Trial name
- NCT05583955
- Testing
- NOE-105
- Where
- 9 sites in United States, Australia
- Volunteers
- 75 people
- Sponsor
- Noema Pharma AG
B-cell Acute Lymphoblastic Leukemia
Study of Efficacy and Safety of CTL019 in Pediatric ALL Patients
This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of CTL019 in pediatric patients with r/r B-cell ALL.
- Trial name
- ELIANA (NCT02435849)
- Testing
- CTL019
- Where
- 23 sites in United States, Australia and 9 more
- Volunteers
- 80 people
- Sponsor
- Novartis Pharmaceuticals
Childhood Absence Epilepsy
Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence Seizures
The primary purpose of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in pediatric participants with treatment-resistant childhood absence seizures.
- Trial name
- NCT03355300
- Testing
- Cannabidiol Oral Solution
- Where
- 9 sites in United States
- Volunteers
- 11 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Childhood-onset Growth Hormone Deficiency, Pituitary Dwarfism
Cool.Click™ Adolescent Transition Study: Study of Saizen® in Subjects With Childhood-onset Growth Hormone Deficiency
The primary objective is to evaluate the efficacy and safety of two different dose regimens of r-hGH (Saizen®) in subjects with childhood-onset growth hormone deficiency (COGHD) during the transition phase from childhood to…
- Trial name
- NCT00109733
- Testing
- recombinant human growth hormone
- Where
- 9 sites in United States
- Volunteers
- 31 people
- Sponsor
- EMD Serono
Spinal Muscular Atrophy Type 3
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
- Trial name
- SMA3 (NCT03819660)
- Testing
- Amifampridine Phosphate 10 MG Oral Tablet
- Where
- Italy
- Volunteers
- 13 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Childhood Absence Epilepsy
Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence Seizures
The primary purpose of this study is to assess the efficacy of Cannabidiol Oral Solution in the treatment of pediatric participants with treatment-resistant childhood absence seizures.
- Trial name
- NCT03336242
- Testing
- Cannabidiol Oral Solution
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Meningococcal Disease
Persistence of Immunogenicity of MenACWY Conjugate Vaccine 5 Years After Childhood Vaccination, and Immune Response to a Booster Dose
In this extension study, V59P20E1, the sponsor was planning to assess 5-year antibody persistence in subjects who received one or two doses of MenACWY conjugate vaccine at 2 to 10 years of age, and to evaluate response to a…
- Trial name
- NCT01823536
- Testing
- Meningococcal (groups A, C, W, and Y) oligosaccharide diphtheria CRM-197 conjugate vaccine
- Where
- 16 sites in United States, Ukraine
- Volunteers
- 465 people, healthy volunteers welcome
- Sponsor
- Novartis Vaccines
B Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia
CD19 /22 CAR T Cells (AUTO3) for the Treatment of B Cell Acute Lymphoblastic Leukemia (ALL)
The purpose of this study is to test the safety and efficacy of AUTO3, a CAR T cell treatment targeting CD19 and CD22 in paediatric or young adult patients with relapsed or refractory B cell acute lymphoblastic leukaemia.
- Conditions
- B Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia, Refractory Childhood Acute Lymphoblastic Leukemia, B-cell Acute Lymphoblastic Leukemia
- Trial name
- AMELIA (NCT03289455)
- Testing
- AUTO3 (CD19/22 CAR T cells
- Where
- 3 sites in United Kingdom
- Volunteers
- 23 people
- Sponsor
- Autolus Limited
Arthritis, Juvenile Idiopathic
Study Evaluating Etanercept in 3 Subtypes of Childhood Arthritis
This study will evaluate the effect of etanercept on the clinical benefit, safety, and physical functioning (ability to function in daily life) in children and adolescent subjects with 3 subtypes of childhood arthritis.
- Trial name
- CLIPPER (NCT00962741)
- Testing
- Etanercept
- Where
- 42 sites in Australia, Belgium and 17 more
- Volunteers
- 127 people
- Sponsor
- Pfizer
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 provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study
- Conditions
- Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease
- Trial name
- NCT01576367
- Testing
- ACZ885
- Where
- 12 sites in Belgium, Canada and 5 more
- Volunteers
- 17 people
- Sponsor
- Novartis Pharmaceuticals