Clinical trials: Disorder, Pediatric
Venous Thromboembolism
Fragmin for the Treatment of Acute VTE in Pediatric Cancer Patients
Three month treatment of acute VTE with Fragmin in pediatric cancer patients
- Trial name
- NCT00952380
- Testing
- dalteparin
- Where
- 26 sites in United States, Norway and 3 more
- Volunteers
- 38 people
- Sponsor
- Pfizer
Homocystinuria Due to CBS Deficiency
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices
- Trial name
- NCT02998710
- Where
- 11 sites in United States, Ireland and 2 more
- Volunteers
- 110 people
- Sponsor
- Travere Therapeutics, Inc.
Migraine, Vascular Headaches
A Study of the Effectiveness and Safety of Topiramate for the Prevention of Migraine Attacks in Children
The primary purpose of this study is to evaluate the effectiveness and safety of topiramate compared with placebo in the prevention of migraine attacks in children (12 to 17 years of age).
- Trial name
- NCT00210535
- Volunteers
- 110 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Glycogen Storage Disease Type II-Pompe's Disease
A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who…
- Trial name
- Mini-COMET (NCT03019406)
- Testing
- Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829)
- Where
- 12 sites in United States, France and 3 more
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
B-Cell Pediatric ALL
Study in Pediatrics With Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma
The primary objective of this study is to evaluate the efficacy of moxetumomab pasudotox in pediatric participants with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) or B-cell lymphoblastic lymphoma.
- Trial name
- NCT02227108
- Testing
- Moxetumomab Pasudotox
- Where
- 21 sites in United States, Australia and 6 more
- Volunteers
- 37 people
- Sponsor
- MedImmune LLC
Venous Thromboembolism
EINSTEIN Junior: Oral Rivaroxaban in Children With Venous Thrombosis
The purpose of this study is to evaluate comparative efficacy and safety of rivaroxaban to standard of care in children with acute venous thromboembolism.
- Trial name
- EINSTEIN Jr (NCT02234843)
- Testing
- Rivaroxaban (Xarelto, BAY59-7939), Standard of Care
- Where
- 109 sites in United States, Argentina and 26 more
- Volunteers
- 500 people
- Sponsor
- Bayer
Acne
Epiduo Pediatric Acne Study
The purpose of this study is to evaluate the safety and efficacy of Epiduo® (adapalene and benzoyl peroxide) Gel 0.1%/2.5% administered once daily for up to 12 weeks in subjects 9 to 11 years of age with acne vulgaris.
- Trial name
- NCT01138735
- Testing
- adapalene/benzoyl peroxide, Topical Gel Vehicle
- Where
- 25 sites in United States, Canada
- Volunteers
- 285 people
- Sponsor
- Galderma R&D
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
Type 1 Diabetes Mellitus
New Glucose Sensor Pediatric
The purpose of this study is to assess performance of a new subcutaneous glucose sensor over a seven day sensor life when used with currently marketed Medtronic Diabetes devices.
- Trial name
- NCT01161043
- Testing
- Sensor wear
- Where
- 4 sites in United States
- Volunteers
- 83 people
- Sponsor
- Medtronic MiniMed, Inc.
Molluscum Contagiosum
A Trial of a Botanical Drug Containing East Indian Sandalwood Oil (EISO) for the Treatment of Molluscum Contagiosum
This trial will examine the safety, tolerability and efficacy of one strength of East Indian sandalwood oil (EISO) in a cream base compared to a placebo for treatment of molluscum contagiosum in pediatric subjects.
- Trial name
- NCT02024581
- Testing
- 10% East Indian sandalwood oil cream
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- ViroXis Corporation
Progressive Familial Intrahepatic Cholestasis (PFIC)
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
- Trial name
- NCT04185363
- Testing
- Maralixibat
- Where
- 28 sites in United States, Argentina and 14 more
- Volunteers
- 84 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
SCID
JSP191 Antibody Targeting Conditioning in SCID Patients
A Phase 1/2 study to evaluate the safety, tolerability, and efficacy of an antibody conditioning regimen, known as JSP191, in patients with Severe Combined Immune Deficiency undergoing blood stem cell transplantation
- Trial name
- NCT02963064
- Testing
- Humanized anti-CD117 Monoclonal Antibody (JSP191)
- Where
- 9 sites in United States
- Volunteers
- 23 people
- Sponsor
- Jasper Therapeutics, Inc.
Hepatitis C
An Open-Label Study of the Effect of Telaprevir in Combination With Peginterferon Alfa-2b and Ribavirin in Pediatric Subjects Infected With Hepatitis C Virus
The purpose of this study is to assess the safety, efficacy, and pharmacokinetics in a carefully monitored cohort of pediatric subjects infected with hepatitis C virus (HCV) on a telaprevir-based regimen in Part A and with dose…
- Trial name
- NCT01701063
- Testing
- Telaprevir, Peginterferon alfa-2b, Ribavirin
- Where
- 23 sites in United States, Belgium and 4 more
- Volunteers
- 42 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Venous Thromboembolism (VTE), Pulmonary Embolism
Hokusai Study in Pediatric Patients With Confirmed Venous Thromboembolism (VTE)
This is an event driven Phase 3, prospective, randomized, open-label, blinded endpoint evaluation (PROBE) parallel group study in subjects with confirmed VTE.
- Conditions
- Venous Thromboembolism (VTE), Pulmonary Embolism, Deep Vein Thrombosis (DVT)
- Trial name
- NCT02798471
- Testing
- Edoxaban, Standard of Care
- Where
- 140 sites in United States, Argentina and 32 more
- Volunteers
- 290 people
- Sponsor
- Daiichi Sankyo
Esophageal Atresia, Digestive System Abnormalities
Flourish™ Pediatric Esophageal Atresia
This study is continued evaluation of the safety and probable benefit of the Flourish Pediatric Esophageal Atresia device through the Humanitarian Device Exemption (HDE) pathway.
- Conditions
- Esophageal Atresia, Digestive System Abnormalities, Esophageal Disorders Congenital
- Trial name
- NCT03615495
- Testing
- Flourish Pediatric Esphogeal Atresia device
- Where
- 12 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Cook Research Incorporated
Refractory Epilepsy
Trial to Evaluate the Safety and Effectiveness of Vagus Nerve Stimulation for Children With Refractory Epilepsy
Children with refractory epilepsy who are candidates for a treatment with vagus nerve stimulation will be prospectively randomized into 2 arms.
- Trial name
- NCT03062514
- Testing
- PINS Vagus Nerve Stimualtor
- Where
- China
- Volunteers
- 84 people
- Sponsor
- Beijing Pins Medical Co., Ltd
Cholestatic Liver Disease
MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia.
- Trial name
- MERGE (NCT04168385)
- Testing
- Maralixibat
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 52 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Influenza
Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants From Birth to < 1 Year With Influenza-Like Symptoms
This study will evaluate the safety, pharmacokinetics and efficacy of baloxavir marboxil in healthy pediatric participants from birth to <1 year with influenza like symptoms
- Trial name
- NCT03653364
- Testing
- Baloxavir Marboxil
- Where
- 27 sites in United States, Bulgaria and 7 more
- Volunteers
- 49 people
- Sponsor
- Hoffmann-La Roche
Asthma
Pharmacokinetics and PharmacoDynamics of GW685698 in Paedeatric Asthmatic Patients
This study will investigate the effect of dosing with flutucasone furoate in asthmatic subjects aged 5-11 years of age. A randomized, two-way crossover, with placebo control, over a 14 day treatment period, it will investigate…
- Trial name
- NCT01332292
- Testing
- Fluticasone furoate
- Where
- 5 sites in United States
- Volunteers
- 27 people
- Sponsor
- GlaxoSmithKline
Cerebral Adrenoleukodystrophy
A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)
- Trial name
- NEXUS (NCT04528706)
- Testing
- MIN-102
- Where
- 5 sites in Argentina, France and 2 more
- Volunteers
- 13 people
- Sponsor
- Minoryx Therapeutics, S.L.