Clinical trials: Pediatric Disease
Short Bowel Syndrome
A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older
The main aims of the study are to check for side effects from teduglutide. Participants will receive a daily injection of teduglutide just under the skin (subcutaneous) for 24 weeks. Then they are followed up for another 4 weeks.
- Trial name
- NCT05027308
- Testing
- Teduglutide
- Where
- 6 sites in Japan
- Volunteers
- 3 people
- Sponsor
- Takeda
Partial Epilepsy
Safety and Efficacy Study of Cenobamate in Pediatric Subjects 2-17 Years of Age With Partial-onset (Focal) Seizures
Primary objective: To evaluate the safety and tolerability of cenobamate in pediatric subjects 2-17 years of age with partial-onset (focal) seizures
- Trial name
- NCT05067634
- Testing
- Xcopri
- Where
- 35 sites in United States, Australia and 5 more
- Volunteers
- 154 people
- Sponsor
- SK Life Science, Inc.
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Hay Fever, Seasonal Allergic Rhinitis
Safety and Efficacy of Ciclesonide in Pediatric Patients (6 to 11 Years of Age) With Seasonal Allergic Rhinitis (BY9010/M1-417)
The aim of the study is to determine the efficacy and safety of ciclesonide nasal spray in children with seasonal allergic rhinitis. Ciclesonide will be administered intranasally at two dose levels once daily.
- Trial name
- NCT00305487
- Testing
- Ciclesonide nasal spray
- Where
- 71 sites in United States
- Volunteers
- 660 people
- Sponsor
- AstraZeneca
Primary Immunodeficiency, Agammaglobulinemia
Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)
The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).
- Conditions
- Primary Immunodeficiency, Agammaglobulinemia, Hypogammaglobulinemia, Antibody Deficiency
- Trial name
- NCT01581593
- Testing
- Kedrion IVIG 10%
- Where
- 15 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Kedrion S.p.A.
Bladder, Neurogenic
Study to Evaluate the Efficacy and Safety of Tamsulosin in Children With Neurogenic Bladder
Aim of this study is to evaluate the efficacy and safety of a range of doses of tamsulosin hydrochloride as treatment in children with an elevated detrusor leak point pressure associated with a known neurological deficit
- Trial name
- NCT00796614
- Testing
- tamsulosin hydrochloride
- Where
- 52 sites in United States, Belgium and 11 more
- Volunteers
- 231 people
- Sponsor
- Boehringer Ingelheim
Familial Hypercholesterolemia - Homozygous
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).
- Trial name
- ORION-13 (NCT04659863)
- Testing
- Inclisiran
- Where
- 11 sites in United States, Canada and 6 more
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
Migraine
A Study of Lasmiditan (LY573144) in Children Aged 6 to 17 With Migraine
The purpose of the study is the measure the levels of lasmiditan in the body of children aged 6 to 17 with migraine. The study also will also examine the safety and tolerability of lasmiditan in children aged 6 to 17 with…
- Trial name
- NCT03988088
- Testing
- Lasmiditan
- Where
- 11 sites in United States, Japan and 1 more
- Volunteers
- 18 people
- Sponsor
- Eli Lilly and Company
Patent Ductus Arteriosus
Post Marketing Clinical Follow Up Study to Evaluate Efficacy and Safety of the Occlutech PDA Occluder in Patients With Patent Ductus Arteriosus Defects
This retrospective and prospective, multicenter, international post marketing follow up study evaluates the safety and efficacy of the Occlutech® PDA Occluder, delivered using the Occlutech Occlusions Pusher (OOP), in subjects…
- Trial name
- NCT05264753
- Testing
- Occlutech® PDA Occluder
- Where
- 15 sites in Canada, France and 7 more
- Volunteers
- 255 people
- Sponsor
- Occlutech International AB
Danon Disease
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2…
- Trial name
- NCT03882437
- Testing
- RP-A501
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Rett Syndrome
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)
The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome.
- Trial name
- NCT06152237
- Testing
- TSHA-102
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 6 people
- Sponsor
- Taysha Gene Therapies, Inc.
Glaucoma, Ocular Hypertension
A Study to Evaluate Pharmacokinetics of Tafluprost Ophthalmic Solution (0.0015%) in Pediatric Patients With Glaucoma or Ocular Hypertension
The purpose of the study is to examine how preservative free tafluprost ophthalmic solution (0.0015%) is distributed in blood circulation after ocular administration in children who have glaucoma or elevated intraocular pressure.
- Trial name
- NCT02102750
- Testing
- Preservative free tafluprost opthalmic solution
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 17 people
- Sponsor
- Santen Oy
Chronic Kidney Disease; Iron Deficiency Anemia
A Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)
Primary Objectives: To evaluate the safety (compared to iron sucrose) and efficacy of ferumoxytol in pediatric CKD subjects with iron deficiency anemia (IDA) or who are at risk of development of IDA Secondary Objective: To…
- Trial name
- NCT03619850
- Testing
- Ferumoxytol, Iron sucrose
- Where
- 21 sites in United States, Hungary and 3 more
- Volunteers
- 129 people
- Sponsor
- AMAG Pharmaceuticals, Inc.
Cough
Evaluation of the Efficacy and Tolerability of ABO/MEG-B-09 in Children With Acute Cough
The present clinical trial will examine the use of ABO/MEG-B-09 syrup in children with acute cough as compared to a placebo syrup.
- Trial name
- ABOMEG (NCT01259674)
- Testing
- AboMeg-B-09
- Where
- Italy
- Volunteers
- 102 people
- Sponsor
- Aboca Spa Societa' Agricola
Lennox-Gastaut Syndrome
Study of Rufinamide in Pediatric Subjects 1 to Less Than 4 Years of Age With Lennox-Gastaut Syndrome Inadequately Controlled With Other Anti-epileptic Drugs
This study was designed to evaluate the cognitive effect, safety, and pharmacokinetics (PK) of rufinamide on Lennox-Gastaut Syndrome (LGS) inadequately controlled in pediatric participants already taking other anti-epileptic…
- Trial name
- NCT01405053
- Testing
- Rufinamide, Any other approved Antiepileptic Drug
- Where
- 47 sites in United States, Canada and 5 more
- Volunteers
- 37 people
- Sponsor
- Eisai Inc.
Pediatric Central Nervous System Tumor
Study of Tozuleristide and the Canvas Imaging System in Pediatric Subjects With CNS Tumors Undergoing Surgery
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
- NCT03579602
- Testing
- tozuleristide, Canvas System
- Where
- 9 sites in United States
- Volunteers
- 118 people
- Sponsor
- Blaze Bioscience Inc.
Pulmonary Arterial Hypertension
A Pharmacokinetics Study for Pediatric Participants With Pulmonary Arterial Hypertension
The purpose of this study is to see how much study drug is in the blood of children with pulmonary arterial hypertension (PAH) after dosing to establish the correct dose for further clinical research.
- Trial name
- NCT01484431
- Testing
- Tadalafil- Tablet or Oral suspension
- Where
- 16 sites in United States, Canada and 4 more
- Volunteers
- 20 people
- Sponsor
- Eli Lilly and Company
Hearing Loss
MyPhonak App Junior Pediatric Usability and Benefit Investigation
Participants age 8-17 with bilateral hearing loss will be fit with Phonak BTE (Behind-The-Ear) hearing aids and given access to the myPhonak Junior hearing app.
- Trial name
- NCT05055453
- Testing
- MyPhonak Junior App, Phonak Sky hearing aid
- Where
- Germany
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Sonova AG
Nasal Congestion
Efficacy and Safety of Pediatric Drugs in Nasal Congestion
The purpose of this study is to evaluate the efficacy of the Naridrin in improving nasal congestion.
- Trial name
- NCT02601235
- Testing
- Naridrin, Afrin
- Where
- Brazil
- Volunteers
- 292 people
- Sponsor
- EMS
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
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