Clinical trials: ALL, Childhood
Childhood Absence Epilepsy
Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence Seizures
The primary purpose of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in pediatric participants with treatment-resistant childhood absence seizures.
- Trial name
- NCT03355300
- Testing
- Cannabidiol Oral Solution
- Where
- 9 sites in United States
- Volunteers
- 11 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Diphtheria, Pertussis
Study to Assess if Quinvaxem Can be Interchanged With Other Pentavalent Vaccines During Standard Childhood Vaccination
This is a study to show that vaccination with 1 dose of Tritanrix HB+Hib followed by Quinvaxem vaccine as the 2nd and 3rd dose is not inferior to vaccination with Quinvaxem for all 3 doses, with respect to protection against all…
- Conditions
- Diphtheria, Pertussis, Tetanus, Hepatitis B
- Trial name
- NCT01357720
- Testing
- Quinvaxem, Quinvaxem/Tritanrix
- Where
- Philippines
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Crucell Holland BV
Attention Deficit/Hyperactivity Disorder
Efficacy and Safety Study of Methylphenidate Hydrochloride Extended Release in Adults With Childhood-onset Attention Deficit/Hyperactivity Disorder (ADHD)
This study will evaluate efficacy and safety of methylphenidate hydrochloride extended release compared to placebo in adult patients with childhood-onset attention deficit/hyperactivity disorder (ADHD).
- Trial name
- NCT01259492
- Testing
- Ritalin LA 20 mg, Ritalin LA 30 mg
- Where
- 68 sites in United States, Belgium and 7 more
- Volunteers
- 725 people
- Sponsor
- Novartis Pharmaceuticals
Heterozygous Familial Hypercholesterolemia
Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.
- Trial name
- HAUSER-RCT (NCT02392559)
- Testing
- Evolocumab
- Where
- 66 sites in United States, Australia and 24 more
- Volunteers
- 158 people
- Sponsor
- Amgen
EBV-induced Lymphomas, EBV-associated Malignancies
Biological Therapy in Treating Patients at High-Risk or With Lymphoma, Lymphoproliferative Disease, or Malignancies
The purpose of this phase I/II trial is to study the side effects and best dose of biological therapy to treat patients at high-risk or with Epstein-Barr virus-associated lymphoma or lymphoproliferative disease.
- Conditions
- EBV-induced Lymphomas, EBV-associated Malignancies, Transplant Patients With EBV Viremia at High Risk of Developing a Recurrent EBV Lymphoma
- Trial name
- NCT00002663
- Testing
- Epstein-Barr virus-specific cytotoxic T lymphocytes (EBV-CTLs)
- Where
- United States
- Volunteers
- 58 people
- Sponsor
- Atara Biotherapeutics
Spinal Muscular Atrophy Type 3
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
- Trial name
- SMA3 (NCT03819660)
- Testing
- Amifampridine Phosphate 10 MG Oral Tablet
- Where
- Italy
- Volunteers
- 13 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Sarcoma
A Study of Avastin (Bevacizumab) in Combination With Standard Chemotherapy in Children and Adolescents With Sarcoma.
This open-label two-arm study will assess the safety and efficacy of a combination of bevacizumab + standard chemotherapy with standard chemotherapy alone as active comparator in childhood and adolescent patients with metastatic…
- Trial name
- NCT00643565
- Testing
- Standard chemotherapy, bevacizumab [Avastin]
- Where
- 61 sites in Belgium, Brazil and 12 more
- Volunteers
- 154 people
- Sponsor
- Hoffmann-La Roche
B Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia
CD19 /22 CAR T Cells (AUTO3) for the Treatment of B Cell Acute Lymphoblastic Leukemia (ALL)
The purpose of this study is to test the safety and efficacy of AUTO3, a CAR T cell treatment targeting CD19 and CD22 in paediatric or young adult patients with relapsed or refractory B cell acute lymphoblastic leukaemia.
- Conditions
- B Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia, Refractory Childhood Acute Lymphoblastic Leukemia, B-cell Acute Lymphoblastic Leukemia
- Trial name
- AMELIA (NCT03289455)
- Testing
- AUTO3 (CD19/22 CAR T cells
- Where
- 3 sites in United Kingdom
- Volunteers
- 23 people
- Sponsor
- Autolus Limited
Idiopathic Thrombocytopenic Purpura
Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body.
The purpose of this study is to investigate the efficacy, safety and tolerability of eltrombopag in children with previously treated chronic immune thrombocytopenia who are between 1 and 17 years of age. This is a 2 part study.
- Trial name
- PETIT2 (NCT01520909)
- Testing
- Eltrombopag
- Where
- 51 sites in United States, Argentina and 11 more
- Volunteers
- 92 people
- Sponsor
- GlaxoSmithKline
Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome
Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)
- Conditions
- Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease
- Trial name
- NCT01302860
- Testing
- ACZ885
- Where
- 12 sites in Belgium, Canada and 5 more
- Volunteers
- 17 people
- Sponsor
- Novartis Pharmaceuticals
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
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.
Childhood-Onset Fluency Disorder
A 10-week Efficacy Study of NOE-105 in Childhood Onset Fluency Disorder (Orpheus)
This study is designed to evaluate the effectiveness of NOE-105 on speech fluency without the known antipsychotic-induced side effects of commonly used treatments for childhood onset fluency disorder (COFD).
- Trial name
- NCT05583955
- Testing
- NOE-105
- Where
- 9 sites in United States, Australia
- Volunteers
- 75 people
- Sponsor
- Noema Pharma AG
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
Spinal Muscular Atrophy Type I
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic…
- Trial name
- NCT03837184
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 4 sites in Japan, South Korea and 1 more
- Volunteers
- 2 people
- Sponsor
- Novartis Gene Therapies
Juvenile Idiopahtic Arthritis
Extension Study Evaluating Etanercept in 3 Subtypes of Childhood Arthritis
This is a 8-year extension study in pediatric subjects who have been diagnosed with one of 3 subtypes of Juvenile Idiopathic Arthritis (JIA) [extended oligoarticular JIA, enthestitis related arthritis (ERA), or psoriatic…
- Trial name
- CLIPPER2 (NCT01421069)
- Testing
- etanercept
- Where
- 35 sites in Australia, Belgium and 17 more
- Volunteers
- 109 people
- Sponsor
- Pfizer
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
Varicella, Chickenpox Vaccines
Evaluate Vaccine Against Chickenpox and a Combined Vaccine Against 4 Viral Childhood Diseases: Measles, Mumps, Rubella and Chickenpox
An observer-blind study to evaluate GlaxoSmithKline Biologicals' live attenuated varicella vaccine and GlaxoSmithKline Biologicals' combined measles-mumps-rubella-varicella vaccine in the prevention of varicella disease in…
- Trial name
- NCT00226499
- Testing
- Priorix-tetra™, Priorix™, Varilrix™
- Where
- 104 sites in Czechia, Greece and 8 more
- Volunteers
- 5,803 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Infections, Rotavirus
Vaccine Efficacy Against Rotavirus Diarrhea; Vaccine Given With Routine Childhood Vaccinations in Healthy African Infants
The primary objective of this study is to determine if the GSK Biologicals' human rotavirus (HRV) vaccine (pooled HRV groups) given concomitantly with routine expanded program on immunisation (EPI) vaccinations can prevent severe…
- Trial name
- NCT00241644
- Testing
- Rotarix™
- Where
- 13 sites in Malawi, South Africa
- Volunteers
- 2,089 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome
Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study
- Conditions
- Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease
- Trial name
- NCT01576367
- Testing
- ACZ885
- Where
- 12 sites in Belgium, Canada and 5 more
- Volunteers
- 17 people
- Sponsor
- Novartis Pharmaceuticals