Clinical trials: Childhood
Gaucher Disease
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests.
- Trial name
- PEDS (NCT04721366)
- Testing
- Standard of Care
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Takeda
Somatostatin Receptor Positive, NETs
Phase I Trial to Determine the Dose and Evaluate the PK and Safety of Lutetium Lu 177 Edotreotide Therapy in Pediatric Participants With SSTR-positive Tumors
The purpose of the study is to determine the appropriate pediatric dosage and evaluate the pharmacokinetics (PK) and safety of Lutetium Lu 177 Edotreotide Targeted Radiopharmaceutical Therapy (RPT) as a monotherapy or following…
- Conditions
- Somatostatin Receptor Positive, NETs, Lymphoma, Solid Tumor
- Trial name
- KinLET (NCT06441331)
- Testing
- Lutetium Lu 177-Edotreotide, Amino Acid Solution
- Where
- 9 sites in United States, France and 1 more
- Volunteers
- 20 people
- Sponsor
- ITM Solucin GmbH
Spinal Muscular Atrophy Type II
Exoskeleton Impact on the Quality of Life on Patients With Spinal Muscular Atrophy
The purpose of this study is to evaluate the impact of the use of a pediatric exoskeleton on the quality of life of children, specifically in the psychological and care dimensions.
- Trial name
- NCT05416034
- Testing
- Use of the ATLAS 2025 exoskeleton at home
- Where
- Spain
- Volunteers
- 3 people
- Sponsor
- MarsiBionics
Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma
MORAb-004 in Treating Young Patients With Recurrent or Refractory Solid Tumors or Lymphoma
This phase I trial studies the side effects and best dose of MORAb-004 in treating young patients with recurrent or refractory solid tumors or lymphoma.
- Conditions
- Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Childhood Burkitt Lymphoma
- Trial name
- NCT01748721
- Testing
- anti-endosialin/TEM1 monoclonal antibody MORAb-004, pharmacological study, laboratory biomarker analysis
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Morphotek
Childhood CNS Tumor
SGT-53 in Children With Recurrent or Progressive CNS Malignancies
An early phase 1 for pediatric patients with recurrent or progressive CNS malignancies
- Trial name
- NCT03554707
- Testing
- SGT-53, Radiation, Irinotecan
- Where
- United States
- Sponsor
- SynerGene Therapeutics, Inc.
Atopic Dermatitis, Infant, Newborn
Moisturizer Mediated Prevention of Symptoms of Atopic Dermatitis in Early Childhood
In this study, it will be investigated if symptoms of atopic dermatitis of children with high familial risk will be delayed beyond the 6th or even 12th month of life by applying the SanaCutan Basiscreme.
- Trial name
- MOPAD (NCT04398758)
- Testing
- SanaCutan Basiscreme
- Where
- 10 sites in Germany
- Volunteers
- 360 people, healthy volunteers welcome
- Sponsor
- Infectopharm Arzneimittel GmbH
Pediatric Tumor of CNS, Pediatric Tumor of Brain
A Study of the Safety, Dosing, and Delivery of NEO100 in Patients With Pediatric Brain Tumors
This is an open label, Phase 1b safety, dose-finding, brain tumor delivery, and pharmacokinetics study of intranasal NEO100 in patients with pediatric-type diffuse high grade gliomas.
- Conditions
- Pediatric Tumor of CNS, Pediatric Tumor of Brain, Diffuse Midline Glioma, H3 K27M-Mutant, Pediatric Tumor of Brain Stem
- Trial name
- NCT06357377
- Testing
- NEO100
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Neonc Technologies, Inc.
Infections, Rotavirus
To Test 2 Doses of GSK Biologicals' Oral Live Attenuated Human Rotavirus (HRV) Vaccine in Healthy Infants in Co-administration With Specific Childhood Vaccines
The main objectives of this study is to determine vaccine efficacy against any rotavirus (RV) gastroenteritis (GE) during the first efficacy period.
- Trial name
- NCT00140686
- Testing
- Rotavirus (vaccine)
- Where
- 101 sites in Czechia, Finland and 4 more
- Volunteers
- 3,994 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Allergy, Infections
A Follow-up Study With Children Who Participated in the TEMPO Study in Their First Year of Life
This is a follow-up study of a randomised clinical trial, called TEMPO (a double-blind randomized clinical trial investigating infant formula and human breast milk consumption), in which infants participated in their first year…
- Trial name
- NCT05449756
- Testing
- Self-administered questionnaires
- Where
- Netherlands
- Volunteers
- 705 people, healthy volunteers welcome
- Sponsor
- Nutricia Research
B Cell Chronic Lymphocytic Leukemia
Efficacy/Safety of Frontline Alemtuzumab (Campath, MabCampath) vs Chlorambucil in Patients With Progressive B-Cell Lymphocytic Leukemia
This is a Phase III, open-label, multicenter, randomized, comparative study of Campath versus chlorambucil as front line therapy in patients with progressive B-Cell Lymphocytic Leukemia (B-CLL).
- Trial name
- NCT00046683
- Testing
- alemtuzumab
- Where
- 20 sites in United States
- Volunteers
- 284 people
- Sponsor
- Genzyme, a Sanofi Company
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Childhood Asthma
Comparison of Combination of Beclomethasone Dipropionate and Formoterol Fumarate Versus Single Components Assessed by Knemometry and Urinary Cortisol Measurements in Asthmatic Children
This is a single centre, double-blind, double-dummy, randomised, single-centre, 2-way cross-over study in asthmatic children already treated with inhaled corticosteroids.
- Trial name
- NCT01450774
- Testing
- CHF 1535 50/6µg, beclomethasone dipropionate 50µg + formoterol fumarate 6µg
- Where
- Denmark
- Volunteers
- 72 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Attention Deficit/Hyperactivity Disorder
Open-label Extension Evaluating Methylphenidate Hydrochloride Extended Release in Adults With Attention Deficit/Hyperactivity Disorder
This study will evaluate the long-term safety of methylphenidate hydrochloride extended release in adults with attention deficit/hyperactivity disorder
- Trial name
- NCT01338818
- Testing
- Ritalin LA (methylphenidate hydrochloride extended release)
- Where
- 48 sites in United States, Belgium and 4 more
- Volunteers
- 299 people
- Sponsor
- Novartis Pharmaceuticals
Hyperopia and Myopia
Eye Imaging for the Study of Childhood Myopia
Myopia (nearsightedness) is increasing worldwide and is becoming a serious public health problem. Studies estimate that by 2050, if no action is taken, almost half of the world's population will be myopic.
- Trial name
- SELENA (NCT07335146)
- Testing
- Ophtalmic measurements to estimate geometrical distances, MRI, Ophtalmic measurements to measure wavefront aberrations
- Where
- France
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Essilor International
Childhood Obesity
Evaluation of a Commercial Gaming Impact in a Childhood Obesity Intervention
Recently, researchers have been interested in the use of game play, mechanics and psychology for health-related engagement and behavior change.
- Trial name
- NCT01757925
- Testing
- weight managment program, Active Gaming
- Where
- 11 sites in United States
- Volunteers
- 77 people, healthy volunteers welcome
- Sponsor
- UnitedHealth Group
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
Leukemia, Myelodysplastic Syndromes
Chemotherapy in Treating Patients Who Have Hematologic Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of DX-8951f in treating patients who have hematologic…
- Trial name
- NCT00004047
- Testing
- exatecan mesylate
- Where
- United States
- Sponsor
- Daiichi Sankyo
Cerebral Palsy, SMA II
Evaluation of the Exoskeleton ATLAS 2030 as Robot-assisted Physical Therapy to Children With Neuromuscular Diseases
This study aims to evaluate the safety, usability and acceptability of a motorized mobility assistance exoskeleton. The protocol explores the use of the motorized device during static and dynamic rehabilitation sessions focused…
- Conditions
- Cerebral Palsy, SMA II, Neuromuscular Diseases
- Trial name
- NCT04837157
- Testing
- Exoskeleton rehabilitation
- Where
- Spain
- Volunteers
- 6 people
- Sponsor
- MarsiBionics
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
Unspecified Childhood Solid Tumor, Protocol Specific
Carboplatin and Irinotecan in Treating Children With Refractory Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of combining carboplatin and irinotecan in treating…
- Trial name
- NCT00024284
- Testing
- carboplatin, irinotecan hydrochloride
- Where
- 7 sites in United States, Canada
- Sponsor
- Bristol-Myers Squibb