Clinical trials: Pediatric Safety and Effectiveness
Pediatric Cancer, Solid Tumor Pediatric
Durvalumab and Tremelimumab for Pediatric Malignancies
The purpose of the study is to determine the recommended dose of durvalumab and tremelimumab (immunotherapy drugs) in pediatric patients with advanced solid and hematological cancers and expand in a second phase to test the…
- Conditions
- Pediatric Cancer, Solid Tumor Pediatric, Hematological Malignancies
- Trial name
- NCT03837899
- Testing
- Durvalumab / Tremelimumab Combination Therapy
- Where
- 19 sites in United States, France and 5 more
- Volunteers
- 50 people
- Sponsor
- AstraZeneca
Plaque Psoriasis
A Long-Term Study of Zasocitinib in Children and Teenagers With Plaque Psoriasis
The main aim of this study is to see how well the medicine zasocitinib works, how safe it is, and how children and teenagers aged 4 to under 18 with moderate-to-severe plaque psoriasis respond to it.
- Trial name
- NCT07250802
- Testing
- Zasocitinib
- Where
- 39 sites in United States, China and 5 more
- Volunteers
- 110 people
- Sponsor
- Takeda
Crohn's Disease
A Study to Assess Adverse Events, Change in Disease Activity, and How Intravenous and Subcutaneous Risankizumab Moves Through the Body of Pediatric Participants With Moderately to Severely Active Crohn's Disease
Crohn's Disease (CD) is a gastrointestinal disease that can cause chronic diarrhea with or without gross bleeding, abdominal pain, weight loss, and fever.
- Trial name
- RISE (NCT05995353)
- Testing
- Risankizumab
- Where
- 85 sites in United States, Belgium and 19 more
- Volunteers
- 110 people
- Sponsor
- AbbVie
Ulcerative Colitis
A Study to Learn More About How Risankizumab Works in Young Participants With Ulcerative Colitis
Ulcerative colitis (UC) is a type of inflammatory bowel disease that causes inflammation and bleeding from the lining of the rectum and colon (large intestine).
- Trial name
- MIGHTY (NCT07071519)
- Testing
- Risankizumab
- Where
- 73 sites in United States, Belgium and 14 more
- Volunteers
- 120 people
- Sponsor
- AbbVie
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
Pediatric Growth Hormone Deficiency
Safety and Efficacy Phase 2 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
This is a phase II, open label, active-controlled, randomized safety and dose finding study of different MOD-4023 dose levels compared to daily r-hGH therapy in pre-pubertal growth hormone deficient children.
- Trial name
- NCT01592500
- Testing
- MOD-4023, Somatropin
- Where
- 4 sites in Greece, Hungary and 1 more
- Volunteers
- 56 people
- Sponsor
- OPKO Health, Inc.
Mucopolysaccharidosis IVA, Morquio A Syndrome
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug…
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01515956
- Testing
- BMN 110
- Where
- 5 sites in United States, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Influenza
A Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Suraxavir Marboxil for Suspension
This study will evaluate the safety, efficacy and pharmacokinetics of Suraxavir Marboxil compared with oseltamivir in Pediatric Patients (2 to <12 Years) with Uncomplicated Influenza.
- Trial name
- NCT07113392
- Testing
- Suraxavir Marboxil, Oseltamivir
- Volunteers
- 144 people
- Sponsor
- Jiangxi Kvvit Pharmaceutical Co., Ltd.
Attention-Deficit/Hyperactivity Disorder
Open-label Study to Evaluate Long-term Safety and Efficacy of SPN-812 (Viloxazine Extended-release Capsule)
Open label extension, long-term multicenter study of safety and efficacy of SPN-812 in pediatric ADHD patients
- Trial name
- NCT02736656
- Testing
- SPN-812
- Where
- 16 sites in United States
- Volunteers
- 1,400 people
- Sponsor
- Supernus Pharmaceuticals, Inc.
Lupus Nephritis
The Pediatric Lupus Nephritis Mycophenolate Mofetil (PLUMM) Study
The study is a 1-year 2-part double-blinded placebo controlled 2-arm clinical trial. Treatment arms are (1) MMF dosed as per body-surface area (MMFBSA; 600mg/m2 body surface area per dose about every 12 hours) and (2)…
- Trial name
- PLUMM (NCT05538208)
- Testing
- Mycophenolate Mofetil
- Where
- 19 sites in United States
- Volunteers
- 105 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Dravet Syndrome
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.
- Trial name
- NCT06872125
- Testing
- zorevunersen
- Where
- 61 sites in United States, China and 6 more
- Volunteers
- 170 people
- Sponsor
- Stoke Therapeutics, Inc
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
Steroid-Sensitive Nephrotic Syndrome
Efficacy and Safety of Rituximab in the First Episode of Pediatric Idiopathic Nephrotic Syndrome
The main objective is to demonstrate, from the initial episode of nephrotic syndrome (NS) in children with standard prednisolone treatment, once complete remission has occurred, that the use of Rituximab (a single intravenous…
- Trial name
- RTXFIRPedINS (NCT04783675)
- Testing
- Rituximab
- Where
- 8 sites in China
- Volunteers
- 44 people
- Sponsor
- Children's Hospital of Fudan University
B-cell Acute Lymphoblastic Leukemia
Study of Efficacy and Safety of CTL019 in Pediatric ALL Patients
This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of CTL019 in pediatric patients with r/r B-cell ALL.
- Trial name
- ELIANA (NCT02435849)
- Testing
- CTL019
- Where
- 23 sites in United States, Australia and 9 more
- Volunteers
- 80 people
- Sponsor
- Novartis Pharmaceuticals
Von Willebrand Disease (VWD)
A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants…
- Trial name
- NCT03879135
- Testing
- rVWF, rFVIII
- Where
- 33 sites in United States, Austria and 7 more
- Volunteers
- 38 people
- Sponsor
- Baxalta now part of Shire
Allergic Asthma Due to Dermatophagoides Farinae, Allergic Asthma Due to Dermatophagoides Pteronyssinus
Mite Asthma Pediatric Immunotherapy Trial
The trial aims to demonstrate efficacy of the House Dust Mite SLIT-tablet versus placebo as add-on treatment in children and adolescents (5-17 years) with House Dust Mite allergic asthma based on clinically relevant asthma…
- Conditions
- Allergic Asthma Due to Dermatophagoides Farinae, Allergic Asthma Due to Dermatophagoides Pteronyssinus, Allergic Rhinitis Due to House Dust Mite
- Trial name
- MAPIT (NCT03654976)
- Testing
- HDM SLIT-tablet
- Where
- 64 sites in United States, Bulgaria and 7 more
- Volunteers
- 533 people
- Sponsor
- ALK-Abelló A/S
Vitiligo, Pediatric ALL
Efficacy and Safety of 308-nm Excimer Lamp Combined With Tacrolimus vs Tacrolimus as Monotherapy in Treating Vitiligo on Children
Vitiligo is a auto immune that impact 2% of the global population, regardless from the phototype. Even though it affects patients in a physical way through loss of pigmentation, it is also impacting them on a mental/emotional…
- Conditions
- Vitiligo, Pediatric ALL, Dermatologic Disease, Autoimmune Diseases
- Trial name
- NCT06035614
- Testing
- exciplex, Tacrolimus ointment
- Where
- Jordan
- Volunteers
- 50 people
- Sponsor
- Clarteis
Multiple Sclerosis
Efficacy, Safety and Pharmacokinetics of Teriflunomide in Pediatric Patients With Relapsing Forms of Multiple Sclerosis
Primary Objective: To assess the effect of teriflunomide in comparison to placebo on disease activity measured by time to first clinical relapse after randomization in children and adolescents 10 to 17 years of age with relapsing…
- Trial name
- TERIKIDS (NCT02201108)
- Testing
- Teriflunomide
- Where
- 57 sites in United States, Belgium and 20 more
- Volunteers
- 166 people
- Sponsor
- Genzyme, a Sanofi Company
Locally Resectable Pediatrics Tumors
CEB-01 in Pediatrics With Locally Resectable Abdominal Tumors
The CEB-01 implant is a membrane containing SN-38, the active metabolite of irinotecan, an already authorized chemotherapeutic agent.
- Trial name
- NCT06986811
- Testing
- Standard surgery, CEB-01
- Where
- Spain
- Volunteers
- 80 people
- Sponsor
- CEBIOTEX
Sickle Cell Disease, Hydroxyurea Failure
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human…
- Conditions
- Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies
- Trial name
- NCT05329649
- Testing
- CTX001
- Where
- 7 sites in United States, Germany and 2 more
- Volunteers
- 13 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
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