Clinical trials: ALL, Childhood
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Growth Disorder, Growth Hormone Deficiency in Children
Validation of Two Measures for Growth Hormone Deficiency in Children, the Treatment Related Impact Measure of Childhood Growth Hormone Deficiency (TRIM-CGHD) and the Treatment Burden Measure of Childhood Growth Hormone Deficiency (TB-CGHD)
This study is conducted in Europe and the United States of America (USA). The aim of the study is to validate two measures for growth hormone deficiency in children, the Treatment Related Impact Measure of Childhood Growth…
- Trial name
- NCT02580032
- Testing
- No treatment given
- Where
- 28 sites in United States, United Kingdom
- Volunteers
- 252 people
- Sponsor
- Novo Nordisk A/S
Melanoma Stage III, B-cell Non Hodgkin Lymphoma
Long-Term Follow-up of Patients Treated With Miltenyi Cell and Gene Therapies
The goal of this Long-Term Follow-Up Trial is to assess long-term safety and efficacy of Miltenyi CAR T treatment.
- Conditions
- Melanoma Stage III, B-cell Non Hodgkin Lymphoma, Pediatric ALL, Childhood Non-Hodgkin Lymphoma
- Trial name
- LONGSAFE (NCT06508775)
- Testing
- MB-CART19.1, MB-CART20.1, MB-CART2019.1
- Where
- 7 sites in Germany
- Volunteers
- 40 people
- Sponsor
- Miltenyi Biomedicine GmbH
Childhood Asthma
Asthma Control Study
The "Digital Health Tool for Parental Management of Childhood Asthma -- Impact on Asthma Control Test Scores" Study is a 16 week, prospective, intent-to-treat, 2-arm randomized controlled trial that aims to evaluate the impact of…
- Trial name
- NCT03304067
- Testing
- Tueo Program
- Where
- United States
- Volunteers
- 262 people
- Sponsor
- Evidation Health
Obesity
Cities for Better Health Childhood Obesity Prevention Initiative
This research project is part of the 'Cities for Better Health Childhood Obesity Prevention Initiative' (CBH COPI). The objective of the CBH COPI initiative is to develop, implement, and evaluate the effectiveness of a package of…
- Trial name
- CBH COPI (NCT06855563)
- Testing
- Community-based intervention packages
- Where
- 6 sites in Australia, Brazil and 4 more
- Volunteers
- 36,000 people
- Sponsor
- Novo Nordisk A/S
SMA II
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
- Trial name
- NCT05901987
- Testing
- GC101
- Where
- 5 sites in China
- Volunteers
- 33 people
- Sponsor
- GeneCradle Inc
Meningococcal Disease
Persistence of Immunogenicity of MenACWY Conjugate Vaccine 5 Years After Childhood Vaccination, and Immune Response to a Booster Dose
In this extension study, V59P20E1, the sponsor was planning to assess 5-year antibody persistence in subjects who received one or two doses of MenACWY conjugate vaccine at 2 to 10 years of age, and to evaluate response to a…
- Trial name
- NCT01823536
- Testing
- Meningococcal (groups A, C, W, and Y) oligosaccharide diphtheria CRM-197 conjugate vaccine
- Where
- 16 sites in United States, Ukraine
- Volunteers
- 465 people, healthy volunteers welcome
- Sponsor
- Novartis Vaccines
Atopic Dermatitis
Efficacy and Steroid Sparing Potential Study of DGLA Cream in Early Childhood Patients With Moderate to Severe Atopic Dermatitis
This study will investigate the steroid sparing potential of DS107E to vehicle in patients with moderate to severe atopic dermatitis.
- Trial name
- NCT03676933
- Testing
- DS107E, Vehicle, Hydrocortisone Butyrate 0.1% Cream
- Where
- Ireland
- Volunteers
- 5 people
- Sponsor
- DS Biopharma
Childhood Obesity
Community-based Program to Treat Childhood Obesity
This study will evaluate the effectiveness of a comprehensive community based program on reducing excess weight in children and teens. This study will recruit 150 participants between the ages of 6-12 and 13-17.
- Trial name
- NCT01374646
- Testing
- JOIN Intervention
- Where
- United States
- Volunteers
- 155 people
- Sponsor
- UnitedHealth Group
Pediatric Epileptic Syndrome, Partial-onset Seizures
A Study to Evaluate Efficacy and Safety of Perampanel Administered as an Adjunctive Therapy in Pediatric Participants With Childhood Epilepsy
The purpose of the study is to evaluate the efficacy of perampanel as measured by the 50 percent (%) responder rate during the maintenance period of the core study for seizure frequency in participants with pediatric epileptic…
- Trial name
- NCT04015141
- Testing
- Perampanel Oral Suspension, Perampanel Tablet
- Where
- 35 sites in United States, Belgium and 4 more
- Volunteers
- 100 people
- Sponsor
- Eisai Inc.
Childhood Obesity
Leveraging Interactive Digital Technology to Increase Access to Family-Based Behavioral Treatment for Childhood Obesity
The primary goal of this SBIR Direct to Phase II is to expand upon the existing training platform to create an "all-in-one" digital product, FBT 2.0, that offers an integrated suite of intervention components, including (a)…
- Trial name
- FBT 2 (NCT06728800)
- Testing
- FBT 2.0, Delayed-Start Control (AAP educational materials)
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- 3-C Institute for Social Development
Pediatric Obesity, Childhood Obesity
A Pharmacokinetic Study Comparing VI-0521 With Placebo in Obese Adolescents
The primary objective of this study is to describe the pharmacokinetic profiles of VI-0521 in obese adolescents.
- Trial name
- NCT02714062
- Testing
- VI-0521 Mid Dose, VI-0521 Top Dose
- Where
- 4 sites in United States
- Volunteers
- 42 people
- Sponsor
- VIVUS LLC
Systemic Infection
Pilot Evaluation of the FilmArray® Childhood Systemic Infection (CSI) Panel
The purpose of this study is to collect data to support decisions made by BioFire regarding assay development, panel composition, and intended use for the final FilmArray CSI reagent pouch.
- Trial name
- NCT03064100
- Testing
- Observational Study
- Where
- 3 sites in United States
- Volunteers
- 1,500 people
- Sponsor
- BioFire Diagnostics, LLC
Systemic Lupus Erythematosus
A Study of Telitacicept in Subjects With Childhood-onset Systemic Lupus Erythematosus
This is a multi-center, open-label, phase 1 study.
- Trial name
- NCT05687526
- Testing
- Telitacicept
- Where
- 12 sites in China
- Volunteers
- 16 people
- Sponsor
- RemeGen Co., Ltd.
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.
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
Lung Diseases, Interstitial (in Pediatric Populations), Childhood Interstitial Lung Disease (chILD)
A Treatment Protocol to Support the Care of Children and Adolescents With Fibrosing Interstitial Lung Disease (ILD)
This Expanded Access Program (EAP) is intended to facilitate the availability of OFEV® (nintedanib) to children and adolescents with chronic fibrosing interstitial lung disease (ILD) on top of current standard of care treatment…
- Trial name
- NCT05624281
- Testing
- nintedanib
- Sponsor
- Boehringer Ingelheim
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
Relapsed B-cell Acute Lymphoblastic Leukemia, Childhood, Refractory B-cell Acute Lymphoblastic Leukemia, Childhood
A Clinical Study of CAR-T Cells Treatment for Children With CD19+/CD22+ R/R ALL and Lymphoma
This is a single arm, open-label, uni-center, phase I study . In this study, Children withCD19+/CD22+ R/R B-cell acute lymphoblastic leukemia or lymphoma will be treated with CAR-T19/CAR-T22 Immunotherapy to determine the safety…
- Conditions
- Relapsed B-cell Acute Lymphoblastic Leukemia, Childhood, Refractory B-cell Acute Lymphoblastic Leukemia, Childhood, Relapsed/Refractory B-cell Lymphoma, Childhood
- Trial name
- NCT04204161
- Testing
- CAR-T19/CAR-T22
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Shenzhen BinDeBio Ltd.
Cataract
Evaluate the Safety of DEXYCU for the Treatment of Inflammation Following Ocular Surgery for Childhood Cataract
A Phase 3/4, Prospective, Randomized, Active Treatment-Controlled, Parallel-Design, Multicenter Study to Evaluate the Safety of DEXYCUfor the Treatment of Inflammation Following Ocular Surgery for Childhood Cataract
- Trial name
- NCT05191706
- Testing
- Dexamethasone, Prednisolone Acetate Ophthalmic
- Where
- 9 sites in United States
- Volunteers
- 17 people
- Sponsor
- EyePoint Pharmaceuticals, Inc.