Clinical trials: Pediatric Neurological Disorder
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Pediatric Onset Multiple Sclerosis (POMS)
Impact of Physical Activity on Cognitive Outcomes in Youth With Pediatric-Onset Multiple Sclerosis (POMS)
The purpose of this study is to to determine how implementing a home-based virtual reality video (VR) game exercise program in young people with Multiple Sclerosis(MS) can improve disability outcomes by measuring its impact on…
- Trial name
- NCT03933020
- Testing
- VR active video game intervention, Educational session, Standard Management of Physical Activity
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- The University of Texas Health Science Center, Houston
Fibromyalgia
Coping Skills Training for Adolescents With Fibromyalgia
Juvenile fibromyalgia is a chronic pain condition that can cause considerable suffering and difficulty in an adolescent's day-to-day activities.
- Trial name
- NCT00086047
- Testing
- Coping Skills Training, Education
- Where
- 3 sites in United States
- Volunteers
- 114 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Central Nervous System Tumor, Pediatric, Leukemia
Etoposide, Cyclophosphamide, Thalidomide, Celecoxib, and Fenofibrate in Relapsed or Progressive Cancer
RATIONALE: Drugs used in chemotherapy, such as etoposide and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Conditions
- Central Nervous System Tumor, Pediatric, Leukemia, Lymphoma, Neuroblastoma
- Trial name
- NCT00357500
- Testing
- celecoxib, cyclophosphamide, etoposide
- Where
- 10 sites in United States
- Volunteers
- 101 people
- Sponsor
- Dana-Farber Cancer Institute
Tourette Syndrome
Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome
This is an open-label, rollover study to collect long-term safety, tolerability, and investigator- and participant-reported pharmacodynamic (PD) data after chronic administration of NBI-98854 in pediatric participants with…
- Trial name
- NCT03732534
- Testing
- NBI-98854
- Where
- 10 sites in United States, Puerto Rico
- Volunteers
- 6 people
- Sponsor
- Neurocrine Biosciences
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
Head Injury
Shared Decision Making in Parents of Children With Head Trauma: Head CT Choice
The investigators will test the impact of a decision aid, Head CT Choice, to determine if its use improves parents' knowledge and engagement in decision making and safely decreases healthcare utilization in children presenting to…
- Trial name
- NCT02063087
- Testing
- Head CT Decision Aid
- Where
- 7 sites in United States
- Volunteers
- 971 people
- Sponsor
- Mayo Clinic
Duchenne Muscular Dystrophy
Phase I/II Study of SRP-4053 in DMD Patients
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.
- Trial name
- NCT02310906
- Testing
- SRP-4053
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Sarepta Therapeutics, Inc.
Tonsillitis, Pain
A Comparison of Postoperative Tramadol Versus Acetaminophen With Codeine in Children Undergoing Tonsillectomy
Tonsillectomy is the most common pediatric surgical procedure performed in the US. The postoperative period can be particularly painful.
- Trial name
- NCT01267136
- Testing
- Codeine with acetaminophen, Tramadol suspension
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- Children's Hospitals and Clinics of Minnesota
Migraine Disorders
A Pilot Clinical Trial of a New Neuromodulation Device for Acute Attacks of Migraine in Children and Adolescents
Migraine is a neurological disease characterized by severe and recurrent headaches. Children and adolescents with migraine often present to the emergency department (ED) with acute attacks, where migraine accounts for up to ~30%…
- Trial name
- NCT05102591
- Testing
- Ketorolac, Metoclopramide, Active Remote Electrical Neuromodulation Device
- Where
- Canada
- Volunteers
- 22 people
- Sponsor
- University of Calgary
Hearing Loss, Unilateral
Cochlear Implantation in Pediatric Cases of Unilateral Hearing Loss
This feasibility study evaluates whether children with unilateral, moderate to profound sensorineural hearing loss experience an improvement in speech perception, hearing in noise, localization, and quality of life with a…
- Trial name
- CIPUHL (NCT02963974)
- Testing
- Cochlear implant
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Relapsed Solid Tumor, Refractory Solid Tumor
REGN2810 in Pediatric Patients With Relapsed, Refractory Solid, or Central Nervous System (CNS) Tumors and Safety and Efficacy of REGN2810 in Combination With Radiotherapy in Pediatric Patients With Newly Diagnosed or Recurrent Glioma
Phase 1: To confirm the safety and anticipated recommended phase 2 dose (RP2D) of REGN2810 (cemiplimab) for children with recurrent or refractory solid or Central Nervous System (CNS) tumors To characterize the pharmacokinetics…
- Conditions
- Relapsed Solid Tumor, Refractory Solid Tumor, Relapsed Central Nervous System Tumor, Refractory Central Nervous System Tumor
- Trial name
- NCT03690869
- Testing
- cemiplimab (monotherapy), cemiplimab (maintenance), Conventional or hypofractionated
- Where
- 20 sites in United States
- Volunteers
- 57 people
- Sponsor
- Regeneron Pharmaceuticals
Tourette Syndrome
Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
This is a Phase 2, double-blind, placebo-controlled, randomized withdrawal study to evaluate the safety and maintenance of efficacy of an optimized once-daily (qd) dose of NBI-98854 in pediatric subjects with TS.
- Trial name
- NCT03530293
- Testing
- Valbenazine
- Where
- 49 sites in United States, Puerto Rico
- Volunteers
- 81 people
- Sponsor
- Neurocrine Biosciences
Obstructive Sleep Apnea Syndrome
Steroids for Pediatric Apnea Research in Kids
This double-blind, randomized controlled trial will evaluate the use of nasal corticosteroids for the treatment of the childhood obstructive sleep apnea syndrome (OSAS).
- Trial name
- SPARK (NCT02180672)
- Testing
- Nasal Fluticasone
- Where
- United States
- Volunteers
- 211 people
- Sponsor
- Children's Hospital of Philadelphia
Hypofibrinogenemia, Afibrinogenemia
Human Fibrinogen Concentrate in Pediatric Cardiac Surgery
The goal of the study is to determine whether the use of Human Fibrinogen Concentrate (RiaSTAP) will decrease blood loss and the need for component blood therapy in neonates and infants undergoing cardiopulmonary bypass.
- Conditions
- Hypofibrinogenemia, Afibrinogenemia, Bleeding Disorders
- Trial name
- RiaSTAP (NCT02822599)
- Testing
- RiaStAP, Saline
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Central Nervous System Tumor, Pediatric
Chemotherapy Combined With Radiation Therapy for Newly Diagnosed CNS AT/RT
RATIONALE: Drugs used in chemotherapy work in different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells.
- Trial name
- NCT00084838
- Testing
- filgrastim, cisplatin, cyclophosphamide
- Where
- 12 sites in United States
- Volunteers
- 25 people
- Sponsor
- Dana-Farber Cancer Institute
Meningitis, Meningococcal
Immunogenicity and Safety of a Meningococcal Conjugate Vaccine Given Concomitantly With Other Vaccines in Toddlers
This Phase III, open-label, randomized, parallel-group, active-controlled, multicenter study was conducted to assess the immunogenicity and safety of a single dose of Meningococcal Polysaccharide (Serogroups A, C, Y and W)…
- Trial name
- NCT03205371
- Testing
- MenACYW conjugate vaccine, MMR, Varicella
- Where
- 37 sites in Mexico, Russia and 2 more
- Volunteers
- 1,183 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Pediatric Cancer, CNS Tumor, Childhood
Preventative Skin Care for Children Undergoing Targeted CNS Tumor Therapy
This research study is examining a preventive skin care regimen for children diagnosed with a brain tumor and receiving anti-cancer therapy with a MEK, Pan-RAF, or BRAF inhibitor.
- Conditions
- Pediatric Cancer, CNS Tumor, Childhood, Skin Inflammation
- Trial name
- NCT04479514
- Testing
- Warm Baths or Showers, Moisturizer, SPF 30 or Higher Suncreen
- Where
- 2 sites in United States
- Volunteers
- 14 people
- Sponsor
- Dana-Farber Cancer Institute
Pediatric Traumatic Brain Injury
Overcoming Membrane Transporters to Improve CNS Drug Delivery - Improving Brain Antioxidants After Traumatic Brain Injury
The overall purpose of this research study is to investigate the safety of pharmacological therapies that may potentially improve pediatric outcomes after traumatic brain injury.
- Trial name
- Pro-NAC (NCT01322009)
- Testing
- Probenecid and N-acetyl cysteine
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of Pittsburgh
Spinal Muscular Atrophy
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche