Clinical trials: Disorder, Pediatric
Multiple Sclerosis
Retrospective Cohort Study of Rebif® Use in Pediatric Multiple Sclerosis (MS) Subjects (REPLAY)
The aim of this retrospective study is to review and describe safety, tolerability and efficacy of Rebif® (subcutaneous interferon [IFN]-beta-1a) in children and adolescents, using information already recorded in medical records.
- Trial name
- REPLAY (NCT01207648)
- Testing
- Rebif®
- Where
- 18 sites in United States, Argentina and 6 more
- Volunteers
- 307 people
- Sponsor
- EMD Serono
Hereditary Angioedema
Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat
The sebetralstat Early Access Program (EAP) provides early access to the investigational medicinal product (IMP) sebetralstat to eligible and approved Hereditary Angioedema (HAE) pediatric (ages 2-11) post-trial and naïve…
- Trial name
- NCT07216378
- Testing
- Sebetralstat
- Where
- 6 sites in United States
- Sponsor
- KalVista Pharmaceuticals, Ltd.
B-Cell Acute Lymphoblastic Leukemia
Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients
This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD…
- Trial name
- CASSIOPEIA (NCT03876769)
- Testing
- CTL019
- Where
- 43 sites in United States, Belgium and 9 more
- Volunteers
- 121 people
- Sponsor
- Novartis Pharmaceuticals
Seasonal Allergic Rhinitis (SAR)
Efficacy and Safety of GSP 301 Nasal Spray in Children (Aged 6 to Under 12 Years) With Seasonal Allergic Rhinitis (SAR)
Study to evaluate the efficacy, safety and tolerability of GSP 301 nasal spray (NS) compared with placebo NS in pediatric subjects (aged 6 to under 12 years) with Seasonal Allergic Rhinitis (SAR).
- Trial name
- GSP 301-305 (NCT03463031)
- Testing
- GSP 301 NS
- Where
- 32 sites in United States
- Volunteers
- 446 people
- Sponsor
- Glenmark Specialty S.A.
Familial Hypercholesterolemia
Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)
The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.
- Trial name
- HAUSER-OLE (NCT02624869)
- Testing
- Evolocumab
- Where
- 46 sites in United States, Australia and 21 more
- Volunteers
- 163 people
- Sponsor
- Amgen
Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP
A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Patients With Immune (Idiopathic) Thrombocytopenia Purpura
This is an extension study designed to assess the safety and durability of platelet count increases with romiplostim treatment of thrombocytopenic patients with immune (Idiopathic) thrombocytopenia purpura.
- Trial name
- NCT01071954
- Testing
- Romiplostim
- Where
- 31 sites in United States, Australia and 2 more
- Volunteers
- 66 people
- Sponsor
- Amgen
Allergies
Evaluation of Treatment Satisfaction in Children With an Allergy and Who Received an Antihistamine
This is a non-interventional retrospective data collection study from children aged from 2 to 12 years old diagnosed with an allergy and who received a treatment consisting of an H1-antihistamine.
- Trial name
- NCT00453583
- Where
- United States
- Volunteers
- 4,581 people
- Sponsor
- UCB Pharma
Acne Vulgaris
An Evaluation of the Adrenal Suppression Potential and PK of CB-03-01 Cream in Pediatric Patients With Acne Vulgaris
This study is designed to determine the hypothalamic-pituitary-adrenal (HPA) axis suppression potential and pharmacokinetic (PK) properties of CB-03-01 Cream, 1%, applied every twelve hours for two weeks, in pediatric patients 9…
- Trial name
- NCT02720627
- Testing
- cortexolone 17α-propionate
- Where
- 10 sites in United States, Poland
- Volunteers
- 27 people
- Sponsor
- Cassiopea SpA
Heart Failure
Open-label Extension Study to Evaluate Long-term Safety of Sacubitril/Valsartan in Pediatric Patients With HF
The purpose of this study was to evaluate long-term safety and tolerability and to provide post-trial access to sacubitril/valsartan to eligible participants who successfully completed CLCZ696B2319 (PANORAMA-HF) core study Part 2…
- Trial name
- NCT03785405
- Testing
- sacubitril/valsartan
- Where
- 79 sites in United States, Argentina and 25 more
- Volunteers
- 216 people
- Sponsor
- Novartis Pharmaceuticals
Acute Lymphocytic Leukemia
CD19-targeted CAR T Cells for Patients With Relapsed or Refractory in B-cell Acute Lymphoblastic Leukemia
This is a phase I, open-label, single-arm study conducted in China to evaluate the safety, tolerability, PK, and determine the recommended phase II dose (RP2D) and/or maximum tolerated dose (MTD) (if applicable) of JWCAR029 in…
- Trial name
- NCT05727683
- Testing
- CD19-targeted Chimeric Antigen Receptor (CAR) T Cells
- Where
- China
- Volunteers
- 33 people
- Sponsor
- Shanghai Ming Ju Biotechnology Co., Ltd.
Asthma
Chronic-Dose Safety and Efficacy Study of Albuterol-HFA-BAI in Pediatric Asthmatics
This study is designed to evaluate the repeat-dose safety and effectiveness of a bronchodilator inhaler relative to placebo (inactive drug inhaler) in children aged 4-11 years with asthma.
- Trial name
- NCT00308685
- Testing
- Albuterol
- Where
- 14 sites in United States
- Volunteers
- 95 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Acute Myeloid Leukemia
Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the Blood
Quizartinib is an experimental drug. It is not approved for regular use. It can only be used in medical research. Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able to join this study if it…
- Trial name
- NCT03793478
- Testing
- Quizartinib, Fludarabine, Cytarabine
- Where
- 27 sites in United States, Belgium and 8 more
- Volunteers
- 65 people
- Sponsor
- Daiichi Sankyo
Jansky-Bielschowsky Disease, Batten Disease
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 144 weeks, in patients with CLN2.
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
- Trial name
- NCT02678689
- Testing
- BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 14 people
- Sponsor
- BioMarin Pharmaceutical
Adolescent Lupus Nephritis, Pediatric Lupus Nephritis
Voclosporin in Adolescent and Pediatric Subjects With Lupus Nephritis
The purpose of this study is to assess the efficacy and safety of voclosporin compared to placebo in achieving renal response following 24 weeks of therapy in adolescent and pediatric subjects with active lupus nephritis (LN).
- Trial name
- VOCAL (NCT05288855)
- Testing
- Voclosporin
- Where
- 6 sites in United States, Colombia and 3 more
- Volunteers
- 9 people
- Sponsor
- Aurinia Pharmaceuticals Inc.
Molluscum Contagiosum
A Study of VBP-245 in Pediatric Patients With Molluscum Contagiosum
A Multicenter, Randomized, Double-Blind, Vehicle-Controlled Phase II Study to Evaluate the Efficacy, Tolerability, and Safety of Topical VBP-245 in Pediatric Subjects for the Treatment of Molluscum Contagiosum (VBP-245-MCV).
- Trial name
- NCT03077750
- Testing
- VBP-245 Topical Gel, Vehicle
- Where
- 3 sites in United States
- Volunteers
- 110 people
- Sponsor
- Veloce BioPharma LLC
T1DM - Type 1 Diabetes Mellitus
The True Vie I3 Continuous Glucose Monitoring System in Pediatric Patients With Type 1 Diabetes Mellitus
The purpose of the investigation is to evaluate the performance of the I3 CGM in Pediatric Patients with Type 1 Diabetes Mellitus (T1DM) according to the FDA's special controls for iCGM.
- Trial name
- NCT05908448
- Testing
- 13 CGM (continuous glucose monitor)
- Where
- United States
- Volunteers
- 134 people
- Sponsor
- Sinocare
Lysosomal Acid Lipase Deficiency
Lysosomal Acid Lipase Deficiency in Risk Groups
A multicenter real-world observational study of the prevalence, diagnostic pathways, and clinical characteristics of lysosomal acid lipase deficiency in pediatric and adolescent risk groups in the Russian Federation (HELIOS)
- Trial name
- HELIOS (NCT07455864)
- Where
- 2 sites in Russia
- Volunteers
- 25 people
- Sponsor
- AstraZeneca
Non Alcoholic Steatohepatitis
Elafibranor, PK and Safety in Children and Adolescents 8 to 17 Years of Age With Non Alcoholic Steatohepatitis (NASH)
The study was being conducted in order to assess the pharmacokinetics and the safety of elafibranor following once daily administration of two dose levels of elafibranor (80 milligrams [mg] and 120mg) during 3 months in children…
- Trial name
- NCT03883607
- Testing
- Elafibranor 80mg, Elafibranor 120mg
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Genfit
Hodgkin Lymphoma, Adult, Hodgkin Disease Recurrent
Phase 2 Study Evaluating Autologous CD30.CAR-T Cells in Patients With Relapsed/Refractory Hodgkin Lymphoma (CHARIOT)
This is a two-part, Phase 2, multicenter, open-label, single arm study to evaluate the safety and efficacy of autologous CD30.CAR-T in adult and pediatric subjects with relapsed or refractory CD30+ classical Hodgkin Lymphoma.
- Conditions
- Hodgkin Lymphoma, Adult, Hodgkin Disease Recurrent, Hodgkin Disease Refractory, Hodgkin Disease, Pediatric
- Trial name
- NCT04268706
- Testing
- CD30.CAR-T, Fludarabine, Bendamustine
- Where
- 5 sites in United States
- Volunteers
- 97 people
- Sponsor
- Tessa Therapeutics
Thrombotic Microangiopathies, Hematopoietic Stem Cell Transplantation
Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA
The purpose of this study is to evaluate the safety and efficacy of narsoplimab in pediatric patients with thrombotic microangiopathies (TMA) following hematopoietic stem cell transplant (HSCT).
- Trial name
- NCT05855083
- Testing
- Biological: narsoplimab
- Where
- 16 sites in United States, Germany and 3 more
- Volunteers
- 18 people
- Sponsor
- Omeros Corporation
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