Clinical trials: Disorder, Pediatric
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
Iron Deficiency Anemia
A Study to Evaluate Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects
This is a Phase 3, randomized, open-label, multicenter, study in male and female pediatric subjects (2 years to <18 years of age) with IDA, or felt by their clinician to be at risk of developing IDA.
- Trial name
- NCT03893045
- Testing
- ferumoxytol, Iron sucrose
- Where
- 15 sites in United States, Lithuania and 1 more
- Volunteers
- 75 people
- Sponsor
- AMAG Pharmaceuticals, Inc.
Insomnia Disorder
Evaluating the Effects of Tasimelteon vs. Placebo in Treating Pediatric Insomnia
This is a multicenter, double-blind, randomized study to evaluate the efficacy and safety of a daily single oral dose of tasimelteon and matching placebo in male and female pediatric participants with insomnia disorder.
- Trial name
- NCT06953869
- Testing
- Tasimelteon Oral Suspension
- Where
- 35 sites in United States, Germany and 2 more
- Volunteers
- 420 people
- Sponsor
- Vanda Pharmaceuticals
Drug Resistant Epilepsy, Autism Spectrum Disorder
Effectiveness and Impact on the Quality of Life of Ketogenic Diet in Pediatric Patients
The goal of this observational study is to learn about the clinical and nutritional effectiveness of ketogenic diet (KD) in pediatric patients with genetic, neurological or metabolic conditions requiring KD.
- Conditions
- Drug Resistant Epilepsy, Autism Spectrum Disorder, Chronic Migraine, Brain Tumor, Pediatric
- Trial name
- NCT06309251
- Testing
- ketogenic diet (as per clinical practice)
- Where
- 3 sites in Italy
- Volunteers
- 100 people
- Sponsor
- Danone Nutricia SpA Società Benefit
Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplant
A Study Using Real-world Data to Examine Outcomes in Children and Adults Who Develop a Blood Clotting Disorder Called Thrombotic Microangiopathy (TMA) After Undergoing a Hematopoietic Stem Cell Transplant (HSCT).
The purpose of this study was to assess OS outcomes in pediatric (≥ 28 days of age at the time of HSCT-TMA diagnosis) and adult participants diagnosed with HSCT-TMA(referred to as TMA from hereon) within 52 weeks after HSCT and…
- Trial name
- ALX-TMA-502 (NCT07732361)
- Where
- 43 sites in United States, Belgium and 8 more
- Volunteers
- 307 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Episodic Migraine
A Study to Assess the Adverse Events and Change in Disease Activity of Oral Atogepant Tablets in Pediatric Participants (6-17 Years of Age) With Episodic Migraine
A migraine is a moderate to severe headache on one side of the head. A migraine attack is a headache that may be accompanied by throbbing, nausea, vomiting, sensitivity to light and sound, or other symptoms.
- Trial name
- NCT05711394
- Testing
- Atogepant
- Where
- 98 sites in United States, Belgium and 14 more
- Volunteers
- 450 people
- Sponsor
- AbbVie
Hemophilia B
BAX 326 Pediatric Study
The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.
- Trial name
- NCT01488994
- Testing
- BAX326
- Where
- 11 sites in India, Poland and 4 more
- Volunteers
- 23 people
- Sponsor
- Baxalta now part of Shire
Bipolar I Disorder, Schizophrenia
Long-term Safety Extension Study of OLZ/SAM in Pediatric Subjects
To evaluate the long-term safety and tolerability of OLZ/SAM in pediatric subjects with schizophrenia or Bipolar I disorder
- Trial name
- NCT04987229
- Testing
- OLZ/SAM
- Where
- 35 sites in United States, Argentina and 3 more
- Volunteers
- 236 people
- Sponsor
- Alkermes, Inc.
Acute Treatment of Migraine
A Study to Learn About the Study Medicine Called Zavegepant (PF-07930207) in Children With a History of Migraine
The purpose of the study is to learn about safety and how the body processes the study medicine called Zavegepant (PF-07930207) in children with a history of migraine.
- Trial name
- NCT06995729
- Testing
- Zavegepant
- Where
- 14 sites in United States
- Volunteers
- 16 people
- Sponsor
- Pfizer
Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)
Intermediate-size Expanded Access Program (EAP), Mesenchymal Stromal Cells (MSC) for Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)
The objectives of this intermediate-size expanded access protocol are to assess the safety and efficacy of remestemcel-L in participants with MIS-C associated with COVID-19.
- Trial name
- NCT04456439
- Testing
- Remestemcel-L, Hydrocortisone, Diphenhydramine
- Sponsor
- Mesoblast International Sàrl
Colitis, Ulcerative
A Study of Vedolizumab in Children and Teenagers With Moderate to Severe Ulcerative Colitis (UC)
Vedolizumab is a medicine that helps to reduce inflammation and pain in the digestive system. In this study, children and teenagers with moderate to severe ulcerative colitis will be treated with vedolizumab.
- Trial name
- NCT04779307
- Testing
- Vedolizumab
- Where
- 65 sites in United States, Australia and 12 more
- Volunteers
- 121 people
- Sponsor
- Takeda
Hypertension, Chronic Kidney Disease
Pharmacokinetics Following Single-dose of Valsartan in Japanese Pediatric Patients
This study will assess the pharmacokinetics and safety following single dose of valsartan in Japanese pediatric patients with hypertension, chronic kidney disease, or nephrotic syndrome.
- Conditions
- Hypertension, Chronic Kidney Disease, Nephrotic Syndrome
- Trial name
- NCT01447485
- Testing
- Valsartan (VAL489)
- Where
- 2 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Novartis Pharmaceuticals
Acid Sphingomyelinase Deficiency
A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France
This was an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632…
- Trial name
- NCT06949358
- Testing
- Olipudase alfa
- Where
- 2 sites in France
- Volunteers
- 3 people
- Sponsor
- Sanofi
Migraine
A Study to Test if Fremanezumab is Effective in Preventing Chronic Migraine in Participants 6 to 17 Years of Age
The primary objective of the study is to evaluate the effectiveness of fremanezumab as compared to placebo for the preventive treatment of chronic migraine (CM).
- Trial name
- NCT04464707
- Testing
- Fremanezumab
- Where
- 89 sites in United States, Canada and 7 more
- Volunteers
- 292 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Pneumonia, Bacterial
A Trial to Evaluate the Safety and Reactogenicity of an Investigational Pneumococcal Vaccine in Toddlers 12 to 15 Months of Age Receiving a Single Booster Dose
The main purpose of this study is to assess the safety and reactogenicity of a single dose of the new pneumococcal vaccine (called Pn-MAPS30plus) in toddlers who have previously completed a two-dose primary vaccination series…
- Trial name
- NCT07406334
- Testing
- Pn-MAPS30plus, PCV20
- Where
- 2 sites in South Africa
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Malaria
Surveillance of Effectiveness/Safety of Artemether-lumefantrine in Patients With Malaria
The purpose of this study is to describe the pediatric and adult patients (U.S. and foreign residents) diagnosed with malaria and treated with artemether-lumefantrine with regard to their demographics, including evaluation of…
- Trial name
- NCT01228344
- Testing
- Artemether-lumefantrine
- Where
- United States
- Volunteers
- 324 people
- Sponsor
- Novartis Pharmaceuticals
Seasonal Allergic Rhinitis
A Study Comparing Montelukast With Placebo in Children With Seasonal Allergic Rhinitis (0476-219)(COMPLETED)
This study will evaluate the effect of montelukast compared to placebo in children during the spring allergic rhinitis season.
- Trial name
- NCT00968149
- Testing
- montelukast sodium
- Volunteers
- 413 people
- Sponsor
- Organon and Co
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
Epilepsy
Pediatric Epilepsy Study
This study will evaluate the safety and effectiveness of oxcarbazepine (Trileptal) as monotherapy in the treatment of partial seizures in pediatric patients 1 month to 3 years of age.
- Trial name
- NCT00050947
- Testing
- oxcarbazepine
- Where
- 30 sites in United States, Brazil and 2 more
- Volunteers
- 94 people
- Sponsor
- Novartis Pharmaceuticals
Eczema, Atopic Dermatitis
A Study of B244 Delivered as a Topical Spray to Assess Safety in Pediatric Subjects With Atopic Dermatitis
An open-label, multicenter, Phase Ib study of B244 delivered as a topical spray to assess safety in pediatric subjects aged 2 to 17 years with atopic dermatitis Condition or disease Intervention/treatment Phase Atopic Dermatitis…
- Trial name
- NCT03775434
- Testing
- Experimental: B244
- Where
- 6 sites in United States
- Volunteers
- 28 people
- Sponsor
- AOBiome LLC