Clinical trials: Pediatric Neurological Disorder
Tourette's Syndrome
A Study of the Effectiveness and Safety of Tetrabenazine MR in Pediatric Subjects With Tourette's Syndrome
The purpose of this clinical trial is to study the therapeutic effect of tetrabenazine MR in children with Tourette's Syndrome, as measured by the improvement in total tic score of the Yale Global Tic Severity Scale (YGTSS).
- Trial name
- TBZ-MR (NCT01133353)
- Testing
- Tetrabenazine MR
- Sponsor
- Bausch Health Americas, Inc.
Epilepsy
Pharmacokinetic Study With an Oral Suspension of Perampanel as Adjunctive Therapy in Pediatric Subjects With Epilepsy
The purpose of this study is to evaluate the pharmacokinetics (PK) of perampanel during the Maintenance Period of the Core Study following oral suspension administration given as an adjunctive therapy in pediatric participants…
- Trial name
- NCT02914314
- Testing
- perampanel
- Where
- 18 sites in United States, Latvia
- Volunteers
- 26 people
- Sponsor
- Eisai Inc.
CDKL5 Deficiency Disorder
Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in children and young adults with genetically confirmed CDKL5 gene mutation.
- Trial name
- Marigold (NCT03572933)
- Testing
- ganaxolone
- Where
- 36 sites in United States, Australia and 6 more
- Volunteers
- 101 people
- Sponsor
- Marinus Pharmaceuticals
Growth Hormone Deficiency
PK and PD Study of LUM-201 in Children With Idiopathic Growth Hormone Deficiency: (OraGrowtH212)
The goals of this single site trial are to study the pharmacokinetics (PK) and pharmacodynamics of LUM-201 and effects of LUM-201 administration on growth hormone release over time in children with idiopathic pediatric growth…
- Trial name
- OraGrowtH212 (NCT04806854)
- Testing
- LUM-201
- Where
- Chile
- Volunteers
- 24 people
- Sponsor
- Lumos Pharma
Seizures
Efficacy and Safety of Intramuscular Midazolam Compared to Buccal Midazolam in Pediatric Seizures
The goal of this study is to prove that intramuscular midazolam is more effective than buccal midazolam in cessation of seizure activity with comparable side effects.
- Trial name
- NCT02897856
- Testing
- Buccal midazolam, Intramuscular midazolam
- Where
- Qatar
- Volunteers
- 150 people
- Sponsor
- Hamad Medical Corporation
Narcolepsy With Cataplexy
A Multicenter Study of the Efficacy and Safety of Xyrem With an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects With Narcolepsy With Cataplexy
The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.
- Trial name
- NCT02221869
- Testing
- Xyrem
- Where
- 20 sites in United States, France and 2 more
- Volunteers
- 106 people
- Sponsor
- Jazz Pharmaceuticals
Glioblastoma
Adult Study: ABT-414 Alone or ABT-414 Plus Temozolomide vs. Lomustine or Temozolomide for Recurrent Glioblastoma Pediatric Study: Evaluation of ABT-414 in Children With High Grade Gliomas
This study was conducted to evaluate the efficacy and safety of depatuxizumab mafodotin (ABT-414) alone or with temozolomide versus temozolomide or lomustine alone in adult participants with recurrent glioblastoma.
- Trial name
- INTELLANCE-2 (NCT02343406)
- Testing
- Depatuxizumab mafodotin, Temozolomide, Lomustine
- Where
- 102 sites in United States, Australia and 19 more
- Volunteers
- 266 people
- Sponsor
- AbbVie
Partial Seizures
A Study to Assess the Efficacy and Safety of Adjunctive Zonisamide in Paediatric Partial Onset Seizures (CATZ Extension Study)
The purpose of this study is to assess the long-term safety and efficacy of zonisamide used as an adjunctive treatment in pediatric subjects treated with 1 or 2 other anti-epileptic drugs (AEDs).
- Trial name
- NCT01136954
- Testing
- Zonisamide
- Where
- Italy
- Volunteers
- 144 people
- Sponsor
- Eisai Limited
Hearing Loss, Conductive, Hearing Loss, Mixed
Expanded Indications in the Pediatric BONEBRIDGE Population
This study will assess safety and efficacy of the MED-EL BONEBRIDGE Bone Conduction Implant in children under 12 years old with conductive or mixed hearing loss.
- Trial name
- NCT05615649
- Testing
- MED-EL BONEBRIDGE Bone Conduction Implant
- Where
- 6 sites in United States
- Volunteers
- 36 people
- Sponsor
- Med-El Corporation
Epilepsy, Partial
A Study Of The Safety And Efficacy Of Levetiracetam (Keppra®) (Ucb L059) In Children With Epilepsy
Open-label study to allow pediatric patients who have participated in prior Levetiracetam (Keppra®) studies to continue their treatment with adequate monitoring and standardized follow-up care until Levetiracetam (Keppra®) is…
- Trial name
- NCT00150709
- Testing
- Levetiracetam
- Volunteers
- 223 people
- Sponsor
- UCB Pharma
Muscular Dystrophy, Duchenne
Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically…
- Trial name
- MoveDMD® (NCT02439216)
- Testing
- Edasalonexent
- Where
- 5 sites in United States
- Volunteers
- 31 people
- Sponsor
- Catabasis Pharmaceuticals
Epilepsy
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Intravenous Brivaracetam in Subjects >= 1 Month to < 16 Years of Age With Epilepsy
The purpose of the study is to evaluate the pharmacokinetics (PK), safety, and tolerability of brivaracetam (BRV) administered intravenously (iv) in subjects >= 1 month to < 16 years of age with epilepsy.
- Trial name
- NCT03405714
- Testing
- Brivaracetam
- Where
- 17 sites in United States, Czechia and 5 more
- Volunteers
- 50 people
- Sponsor
- UCB Biopharma S.P.R.L.
Duchenne Muscular Dystrophy
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
- Trial name
- ENVOL (NCT06128564)
- Testing
- delandistrogene moxeparvovec
- Where
- 7 sites in Belgium, France and 4 more
- Volunteers
- 13 people
- Sponsor
- Hoffmann-La Roche
Pediatric Growth Hormone Deficiency
Versartis Long-Term Safety Study of Somavaratan
Protocol 13VR3 is is a multi-center, open-label study assessing long-term somavaratan administration. Patients will be monitored for safety throughout their participation in the study.
- Trial name
- VISTA (NCT02068521)
- Testing
- somavaratan
- Volunteers
- 385 people
- Sponsor
- Versartis Inc.
Danon Disease
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2…
- Trial name
- NCT03882437
- Testing
- RP-A501
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
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
Spina Bifida
Establishing the Preliminary Utility of a Novel Pediatric Manual Mobile Standing Wheelchair
To establish the preliminary utility of a novel pediatric manual mobile standing wheelchair (PedMMSWC).
- Trial name
- NCT07290556
- Testing
- PedMMSWC Wheelchair User Testing, PedMMSWC Simulated Home, School, Community Environment
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Optimal Mobility, Inc
Fibromyalgia
Adolescent Fibromyalgia Study
This study will compare pregabalin with placebo over a 15 week period in adolescents with fibromyalgia aged 12-17 years to evaluate the safety and efficacy of pregabalin.
- Trial name
- NCT01020474
- Testing
- pregabalin (Lyrica)
- Where
- 36 sites in United States, Czechia and 2 more
- Volunteers
- 107 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Neuromyelitis Optica Spectrum Disorder
Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
A Phase 2, open-label, multicenter study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab in eligible pediatric participants 2 to < 18 years of age with recently active neuromyelitis optica…
- Trial name
- NCT05549258
- Testing
- Inebilizumab
- Where
- 19 sites in United States, Argentina and 9 more
- Volunteers
- 15 people
- Sponsor
- Amgen
Metachromatic Leukodystrophy (MLD)
A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain
The main aim of this study is to learn about metachromatic leukodystrophy (MLD) in children and teenagers in Spain. This includes checking the number of new MLD cases and the frequency of children with MLD in 2022 in Spain.
- Trial name
- mECHromatic (NCT05755568)
- Testing
- No Intervention
- Where
- 10 sites in Spain
- Sponsor
- Takeda