Clinical trials: Pediatric Disease
Infantile Spasms
Open-label Extension to Protocol 1042-0500
To allow open-label extension to patients who have completed Protocol 1042-0500
- Trial name
- NCT00442104
- Testing
- Ganaxolone
- Where
- 14 sites in United States
- Volunteers
- 54 people
- Sponsor
- Marinus Pharmaceuticals
Mydriasis, Dilation
Nyxol (0.75% Phentolamine Ophthalmic Solution) to Reverse Pharmacologically-Induced Mydriasis in Pediatric Subjects
The objectives of this study are: To evaluate the safety of Nyxol in pediatric subjects To evaluate the efficacy of Nyxol to expedite the reversal of pharmacologically induced mydriasis in pediatric subjects The Sponsor intends…
- Trial name
- MIRA-4 (NCT05223478)
- Testing
- Phentolamine Ophthalmic Solution 0.75%, Phentolamine Ophthalmic Solution Vehicle
- Where
- 2 sites in United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Opus Genetics, Inc
Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory
First-in-human Study of SAR443579 Infusion in Male and Female Children and Adult Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL), High Risk-myelodysplasia (HR-MDS), or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
This is an open-label, multicenter, Phase 1/Phase 2, dose escalation and dose expansion study to evaluate the safety, pharmacokinetics, pharmacodynamics and anti-leukemic activity of SAR443579 in various hematological…
- Conditions
- Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasia
- Trial name
- NCT05086315
- Testing
- SAR443579
- Where
- 23 sites in United States, Australia and 3 more
- Volunteers
- 101 people
- Sponsor
- Sanofi
Hepatitis C, Chronic
5 Year Long-term Follow up in Pediatric Participants Who Received PegIntron Plus Rebetol in P02538 Part I (P02538 Pt 2)
Study P02538 Part 2 is a 5-year long term follow-up (LTFU) study in pediatric participants who were treated with at least one dose of peginterferon alfa-2b (PEG-IFN) and ribavirin (RBV) and who completed the follow-up in the…
- Trial name
- NCT00761735
- Testing
- Peginterferon alfa-2b, Ribavirin
- Volunteers
- 94 people
- Sponsor
- Merck Sharp & Dohme LLC
Ulcerative Colitis
A Study Evaluating the Effects of Filgotinib in Children and Teenagers With Ulcerative Colitis
The aim of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of filgotinib as a treatment for UC in children and adolescents aged from 8 to less than 18 years.
- Trial name
- Galapeduca (NCT06865417)
- Testing
- Filgotinib
- Where
- 46 sites in Belgium, Croatia and 11 more
- Volunteers
- 80 people
- Sponsor
- Alfasigma S.p.A.
Primary Immunodeficiency
Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency
The purpose of this open-label study is to evaluate the pharmacokinetics, safety, and tolerability of subcutaneously (SC; under the skin) administered GAMUNEX-C compared to intravenously (IV; through the vein) administered…
- Trial name
- KIDS (NCT01465958)
- Testing
- GAMUNEX-C
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Grifols Therapeutics LLC
Transfusional Iron Overload, β-thalassemia Major
A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed.
- Conditions
- Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
- Trial name
- NCT00390858
- Testing
- Deferasirox
- Where
- 4 sites in France, Italy
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Allergic Rhinitis
Study of an Investigational Nasal Aerosol or Placebo in Children (Ages 4 to 11) With Perennial Allergic Rhinitis (PAR)
The purpose of this study is to evaluate the efficacy and safety of Beclomethasone dipropionate (BDP) nasal aerosol in subjects with Perennial Allergic Rhinitis (PAR).
- Trial name
- NCT01783548
- Testing
- BDP Nasal Aerosol
- Where
- 63 sites in United States
- Volunteers
- 547 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, 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
Fabry Disease
A Study of Fabrazyme in Pediatric Patients With Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme.
- Trial name
- NCT00074958
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 7 sites in United States, France and 2 more
- Volunteers
- 16 people
- Sponsor
- Genzyme, a Sanofi Company
Refractory or Relapsed Solid Tumors, Lymphoma
Phase I/II Study of Avelumab in Pediatric Cancer Participants
This is a multi-center, open-label, international study to evaluate the dose, safety and tolerability, antitumor activity, pharmacokinetic and pharmacodynamics of avelumab in pediatric subjects 0 to less than 18 years of age with…
- Trial name
- NCT03451825
- Testing
- Avelumab
- Where
- 11 sites in United States, Belgium and 3 more
- Volunteers
- 21 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Lyme Borreliosis
Phase 2 Study Of VLA15, A Vaccine Candidate Against Lyme Borreliosis, In A Healthy Pediatric And Adult Study Population
VLA15-221 is a Phase 2 study, which will be conducted in two parts: Main Study Phase (Part A) and Booster Phase (Part B). The study will compare the safety and immunogenicity of two different primary immunization schedules…
- Trial name
- NCT04801420
- Testing
- VLA15
- Where
- 19 sites in United States
- Volunteers
- 625 people, healthy volunteers welcome
- Sponsor
- Pfizer
Rhinitis, Allergic, Perennial
A Study Of GW685698X For The Treatment Of Perennial Allergic Rhinitis In Pediatrics
The purpose of this study is to assess the effect of an aqueous nasal spray investigational compound GW685698X compared to placebo on the hypothalamic pituitary adrenocortical (HPA) axis system in children 2 to 11 years of age…
- Trial name
- NCT00116883
- Testing
- GW685698X aqueous nasal spray
- Where
- 12 sites in United States
- Volunteers
- 90 people
- Sponsor
- GlaxoSmithKline
Liver Neoplasms, Adenoma
Liver MRI With Primovist/Eovist in Pediatric Subjects Who Are Suspected or Have Focal Liver Lesions.
Medical records are reviewed to obtain information about the use of a MRI diagnostic imaging agent (contrast agent) called Primovist/Eovist in children older than 2 months and less than 18 years.
- Conditions
- Liver Neoplasms, Adenoma, Carcinoma, Liver Abscess
- Trial name
- NCT01043523
- Testing
- Gadoxetic Acid Disodium (Eovist, BAY86-4873)
- Where
- 9 sites in United States, Italy and 3 more
- Volunteers
- 52 people
- Sponsor
- Bayer
Venous Thrombosis, Pediatrics
Rivaroxaban Pharmacokinetics/Pharmacodynamics (PK/PD) Study in Pediatric Subjects
The first study with rivaroxaban in pediatric subjects is a Phase I study, where the pharmacokinetic/pharmacodynamic (PK/PD) profile of rivaroxaban will be investigated to confirm that the exposure is comparable to adults.
- Trial name
- NCT01145859
- Testing
- Rivaroxaban (Xarelto, BAY59-7939)
- Where
- 36 sites in United States, Australia and 8 more
- Volunteers
- 59 people
- Sponsor
- Bayer
B-cell Acute Lymphoblastic Leukemia
Study of Tisagenlecleucel in Chinese Pediatric and Young Adult Subjects With Relapsed or Refractory B-cell ALL
This is a single arm, multi-center, phase II study to evaluate the efficacy and safety of tisagenlecleucel in Chinese pediatric and young adult subjects with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL)
- Trial name
- NCT04156659
- Testing
- Tisagenlecleucel
- Sponsor
- Novartis Pharmaceuticals
Neuromyelitis Optica Spectrum Disorder
Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
- Trial name
- NCT05346354
- Testing
- Ravulizumab
- Where
- 16 sites in United States, Canada and 5 more
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
- Trial name
- NCT04105166
- Testing
- RP-L301
- Where
- 3 sites in United States, Spain
- Volunteers
- 4 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Spinal Muscular Atrophy
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
The aim of this retrospective medical chart review is to describe the clinical outcomes, clinical characteristics, and demographics of patients with spinal muscular atrophy (SMA) type 1 treated with onasemnogene abeparvovec (OA)…
- Trial name
- SMA-REAL (NCT07737756)
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals
Obstructive Sleep Apnea
Pediatric Nasal Mask Usability Study
The purpose of the study is to demonstrate the effectiveness of a pediatric mask in treating obstructive sleep apnea (OSA) in a pediatric population.
- Trial name
- NCT01154322
- Testing
- Pixi pediatric mask
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- ResMed
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.