Clinical trials: Pediatric Safety and Effectiveness
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Vernal Keratoconjunctivitis
Double-Masked Trial of NOVA22007 (1mg/mL Ciclosporin/Cyclosporine) Versus Vehicle in Pediatric Patients With Active Severe Vernal Keratoconjunctivitis
The objective of this study is to compare the efficacy of two different dosing regimen of NOVA22007 (1mg/ml ciclosporin/cyclosporine) eye drops, emulsion versus placebo (vehicle of the formulation) administered four times a day…
- Trial name
- NCT01751126
- Testing
- NOVA22007 ''Ciclosporin''
- Where
- 46 sites in United States, Croatia and 9 more
- Volunteers
- 169 people
- Sponsor
- Santen SAS
Pediatric Glaucoma, Elevated IOP in Pediatric Patients
Study of Travoprost Ophthalmic Solution, 0.004% Compared to Timolol (0.5% or 0.25%) in Pediatric Glaucoma Patients
The purpose of this study is to evaluate the intraocular pressure (IOP)-lowering efficacy of Travoprost 0.004% POLYQUAD (PQ) ophthalmic solution compared to Timolol ophthalmic solution (0.5% or 0.25%) in pediatric glaucoma…
- Conditions
- Pediatric Glaucoma, Elevated IOP in Pediatric Patients, Ocular Hypertension in Pediatric Patients
- Trial name
- NCT01652664
- Testing
- Travoprost 0.004% PQ ophthalmic solution, Timolol, 0.5% or 0.25% ophthalmic solution, Travoprost Vehicle
- Volunteers
- 184 people
- Sponsor
- Alcon Research
Tourette Syndrome
Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a…
- Trial name
- NCT03325010
- Testing
- Valbenazine
- Where
- 35 sites in United States, Puerto Rico
- Volunteers
- 127 people
- Sponsor
- Neurocrine Biosciences
Neuromuscular Blockade
Efficacy, Safety, and Pharmacokinetics of Sugammadex for Reversal of Neuromuscular Blockade (NMB) in Pediatric Participants (MK-8616-089)
This trial will evaluate the efficacy, safety, and pharmacokinetics of sugammadex for the reversal of both moderate and deep neuromuscular blockade (NMB) induced by either rocuronium or vecuronium in pediatric participants.
- Trial name
- NCT03351608
- Testing
- Sugammadex 2 mg/kg, Sugammadex 4 mg/kg, Neostigmine + Glycopyrrolate
- Where
- 30 sites in United States, Austria and 6 more
- Volunteers
- 288 people
- Sponsor
- Merck Sharp & Dohme LLC
Hepatitis B, Chronic
A Study of Entecavir in Pediatric Patients With Chronic Hepatitis B Virus (HBV)-Infection
The purpose of this clinical study is to determine the appropriate doses of entecavir to use in children and adolescents. Safety, tolerability and efficacy will also be studied
- Trial name
- NCT00423891
- Testing
- Entecavir
- Where
- 19 sites in United States, Argentina and 6 more
- Volunteers
- 64 people
- Sponsor
- Bristol-Myers Squibb
Attention Deficit Hyperactivity Disorder (ADHD)
Treatment of Impulsive Aggression in Subjects With ADHD in Conjunction With Standard ADHD Treatment (CHIME 1)
The purpose of this study is to demonstrate the efficacy, safety, and tolerability of SPN-810 in the treatment of Impulsive Aggression (IA) in subjects with Attention-Deficit/Hyperactivity Disorder (ADHD) in conjunction with…
- Trial name
- NCT02618408
- Testing
- SPN-810 (18 mg), SPN-810 (36 mg)
- Where
- 28 sites in United States
- Volunteers
- 333 people
- Sponsor
- Supernus Pharmaceuticals, Inc.
Liver Transplantation, Infection
Efficacy and Safety of Basiliximab, Cyclosporine/Cyclosporine Microemulsion, and Steroids in Pediatric de Novo Liver Transplant Recipients Avoiding Intraoperative Steroids
Systemic infection is still a major concern in young children with liver transplantation. The approach of this study is to reduce the risk of systemic infections by avoiding intraoperative steroids (another class of…
- Trial name
- NCT00149890
- Testing
- Basiliximab, Cyclosporine/cyclosporine microemulsion, Steroid
- Where
- Germany
- Volunteers
- 77 people
- Sponsor
- Novartis
Acute Pain
A Study to Look at Tapentadol Oral Solution in Children and Adolescents in Pain
The purpose of the study was to evaluate the efficacy of tapentadol oral solution, based on the total amount of supplemental opioid analgesic used over 12 hours or 24 hours after initiation of investigational medicinal product…
- Trial name
- NCT02081391
- Testing
- Tapentadol oral solution 4 mg/mL, Tapentadol oral solution 20 mg/mL
- Where
- 43 sites in United States, Bulgaria and 8 more
- Volunteers
- 216 people
- Sponsor
- Grünenthal GmbH
Plaque Psoriasis
Plaque Psoriasis Study in Pediatric Subjects
The purpose of this study is to compare the safety, efficacy and calcium metabolism of up to 8 weeks of treatment with calcitriol 3 mcg/g ointment versus its vehicle, when used twice daily, without occlusion, to treat children…
- Trial name
- NCT02186665
- Testing
- calcitriol ointment
- Where
- 34 sites in United States, Belgium and 5 more
- Volunteers
- 19 people
- Sponsor
- Galderma R&D
Chronic Sinusitis
Safety and Efficacy of Balloon Sinuplasty in Pediatric Sinusitis
A non-randomized, multi-center, prospective, clinical study intended to evaluate the safety and efficacy of balloon sinuplasty devices in pediatric patients with longstanding sinusitis following failed medical management.
- Trial name
- INTACT (NCT00939471)
- Testing
- Relieva™ Balloon Sinuplasty™ System
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Integra LifeSciences Corporation
Iron Deficiency Anemia
Multicenter Randomized Active-controlled Study to Investigate Efficacy & Safety of IV FCM in Pediatric Patients With IDA
The primary objective of this study is to demonstrate the efficacy and safety of intravenous ferric carboxymaltose (FCM), compared to oral iron, in pediatric participants who have iron deficiency anemia.
- Trial name
- NCT03523117
- Testing
- Ferric carboxymaltose, Ferrous Sulfate
- Where
- 16 sites in United States
- Volunteers
- 79 people
- Sponsor
- American Regent, Inc.
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Lymphoma, Non-Hodgkin
A Study to Evaluate the Safety and Efficacy of JCAR017 in Pediatric Subjects With Relapsed/Refractory (r/r) B-cell Acute Lymphoblastic Leukemia (B-ALL) and B-cell Non-Hodgkin Lymphoma (B-NHL)
This is a Phase 1/2, open-label, single arm, multicohort study to evaluate the safety and efficacy of JCAR017 in pediatric subjects aged ≤ 25 years with CD19+ r/r B-ALL and B-NHL.
- Trial name
- NCT03743246
- Testing
- JCAR017, Lymphodepleting, Fludarabine
- Where
- 19 sites in United States, France and 4 more
- Volunteers
- 21 people
- Sponsor
- Celgene
Asthma
Efficacy and Safety of Ciclesonide (CIC) Administered Twice Daily in Pediatric Patients With Asthma.
Primary objective: To demonstrate the efficacy of ciclesonide, compared to placebo, at 80 μg twice daily (BID) or 40 μg BID for 12 weeks in patients with persistent asthma.
- Trial name
- NCT00392288
- Testing
- Ciclesonide
- Where
- 6 sites in United States, Hungary and 4 more
- Volunteers
- 528 people
- Sponsor
- AstraZeneca
Hypertension
An Extension Study to Evaluate the Long Term Safety, Tolerability and Efficacy of Aliskiren Compared to Enalapril in Pediatric Hypertensive Patients 6-17 Years of Age
The purpose of this study is to evaluate in a randomized, double-blind fashion, the long-term safety, tolerability and efficacy profile of aliskiren compared to the active comparator enalapril in children, 6 - 17 years old with…
- Trial name
- NCT01151410
- Testing
- Aliskiren, Enalapril
- Where
- 36 sites in United States, Guatemala and 5 more
- Volunteers
- 208 people
- Sponsor
- Novartis Pharmaceuticals
Cytomegalovirus (CMV) Infection
Letermovir Treatment in Pediatric Participants Following Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) (MK-8228-030)
The primary objective of this study is to evaluate the pharmacokinetics (PK) of letermovir (LET) in pediatric participants. Participants will be enrolled in the following 3 age groups: Age Group 1: From 12 to <18 years of age…
- Trial name
- NCT03940586
- Testing
- Letermovir oral granules, Letermovir tablet, Letermovir intravenous
- Where
- 40 sites in United States, Australia and 9 more
- Volunteers
- 65 people
- Sponsor
- Merck Sharp & Dohme LLC
Post Operative Pain
Effectiveness, Safety, and Tolerability Study of Oxymorphone Immediate Release (IR) Oral Liquid in Post Surgical Pediatric Subjects
The purpose of this study is to evaluate the effectiveness, tolerability, and safety of oxymorphone immediate release (IR) oral liquid as an analgesic for acute postoperative pain in pediatric subjects.
- Trial name
- NCT01210352
- Testing
- oxymorphone HCl
- Where
- 8 sites in United States
- Volunteers
- 61 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Chemotherapy Induced Nausea and Vomiting
A Study of the Safety and Efficacy of Aprepitant for the Prevention of Chemotherapy-Induced Nausea and Vomiting (CINV) in Pediatric Participants (MK-0869-208)
This study will compare the safety and efficacy of a three-day oral aprepitant regimen (aprepitant plus ondansetron) to ondansetron alone in the prevention of chemotherapy-induced nausea and vomiting (CINV) in the 120 hours…
- Trial name
- NCT01362530
- Testing
- Aprepitant 125 mg, Aprepitant 80 mg, Aprepitant powder for suspension (PFS)
- Volunteers
- 307 people
- Sponsor
- Merck Sharp & Dohme LLC
Schizophrenia, Autism
Pediatric Open-Label Extension Study
This is an open-label, 104-week, multicenter, extension study designed to evaluate the long-term safety, tolerability and effectiveness of flexibly dosed lurasidone (20, 40, 60 or 80 mg/day) in pediatric subjects who have…
- Conditions
- Schizophrenia, Autism, Bipolar Depression
- Trial name
- NCT01914393
- Testing
- Lurasidone 20, 40, 60, 80 mg, flexibly dosed
- Where
- 88 sites in United States, Bulgaria and 13 more
- Volunteers
- 702 people
- Sponsor
- Sumitomo Pharma America, Inc.
Hereditary Angioedema
Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II
KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old with HAE Type I or II.
- Trial name
- KONFIDENT-KID (NCT06467084)
- Testing
- KVD900 150 mg, KVD900 300 mg, KVD900 600 mg
- Where
- 24 sites in United States, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.