Clinical trials: Neurologic Disorder
- Condition Neurologic Disorder
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Duchenne Muscular Dystrophy
HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies.
- Trial name
- NCT02525302
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Akashi Therapeutics
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Choroidal Neovascularization (CNV)
Assess the Efficacy/Safety of Intravitreal Ranibizumab in Patients With Vision Loss Due to Choroidal Neovascularization.
This study was conducted to evaluate the efficacy and safety of 0.5 mg ranibizumab in adult and adolescent patients with visual impairment due to choridal neovascularization (CNV).
- Trial name
- NCT01840410
- Testing
- Ranibizumab
- Where
- 59 sites in Australia, Canada and 19 more
- Volunteers
- 183 people
- Sponsor
- Novartis Pharmaceuticals
Periventricular Leukomalacia, White Matter Disease
Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia
The goal of this study is to explore a new treatment that may help repair brain damage in individuals with periventricular leukomalacia (PVL), a condition that affects white matter in the brain.
- Conditions
- Periventricular Leukomalacia, White Matter Disease, Hypoxic-Ischemic Encephalopathy, Demyelinating Diseases
- Trial name
- NCT06985303
- Testing
- Investigational Combination Product
- Sponsor
- MGAM LLC
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
MELAS Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of…
- Trial name
- NCT06644534
- Testing
- TTI-0102
- Where
- 2 sites in France, Netherlands
- Volunteers
- 9 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Poliomyelitis
Sequential Vaccination of Poliomyelitis Vaccine (Vero Cells), Inactivated, Sabin Strains From Different Manufacturers
To evaluate the immunogenicity and safety of sequential vaccination with Sinovac sIPV among infants who have received two doses of Biological Products Co., Ltd. sIPV.
- Trial name
- NCT06346834
- Testing
- sIPV
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Soft Tissue Anesthesia (Numbness)
Safety and Efficacy Study of NV-101 in Dental Patients
The purpose of this study was: to determine if NV-101 accelerates recovery from numbness compared to placebo to evaluate safety of NV-101
- Trial name
- NCT00154167
- Testing
- NV-101 (phentolamine mesylate solution)
- Where
- United States
- Volunteers
- 122 people, healthy volunteers welcome
- Sponsor
- Novalar Pharmaceuticals, Inc.
Encephalitis
SouthEast Asia Encephalitis Project
Encephalitis, an acute inflammation of the central nervous system associated with neurologic dysfunction is of public health concern worldwide, because of its high mortality and neurological sequelae rates.
- Trial name
- SEAe (NCT04089436)
- Testing
- CSF sampling, Blood sampling, Nasopharyngeal and rectal sampling
- Where
- 7 sites in Burma, Cambodia and 2 more
- Volunteers
- 800 people
- Sponsor
- Institut Pasteur
Epilepsy
An Open-Label Study to Determine Safety , Tolerability, and Efficacy of Oral Lacosamide in Children With Epilepsy
SP848 is an open-label study to evaluate long-term safety, tolerability, and efficacy in children with epilepsy treated with Lacosamide (LCM) oral solution (syrup) or LCM tablets as adjunctive therapy.
- Trial name
- NCT00938912
- Testing
- Lacosamide
- Where
- 69 sites in United States, Belgium and 9 more
- Volunteers
- 366 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Friedreich Ataxia, Friedreich's Ataxia
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
- Trial name
- FORWARD-FA (NCT07778836)
- Testing
- Nomlabofusp
- Where
- 4 sites in United States
- Volunteers
- 150 people
- Sponsor
- Larimar Therapeutics, Inc.
Fabry Disease
Treatment Protocol of Replagal for Patients With Fabry Disease
The study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week.
- Trial name
- NCT01031173
- Testing
- agalsidase alfa
- Where
- 25 sites in United States
- Sponsor
- Shire
Migraine
Machine Learning Assessment of Next-Day Migraine Likelihood
Development of a machine learning (ML) algorithm for assessment of next-day migraine likelihood, drawing on self-reported migraine-related information, and geographic location, collected via the Nerivio app- a mobile application…
- Trial name
- NCT07227194
- Where
- United States
- Volunteers
- 53,065 people
- Sponsor
- Theranica
Bardet-Biedl Syndrome, POMC Deficiency
Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway
A trial to compare the weekly and daily formulations of setmelanotide in participants with genetic defects in the melanocortin-4 receptor pathway.
- Trial name
- NCT05194124
- Testing
- Setmelanotide 2 mg, Setmelanotide 2.5 mg, Setmelanotide 3 mg
- Where
- 7 sites in United States, Canada and 4 more
- Volunteers
- 19 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Genetic Disorder, Prader-Willi Syndrome
Retrospective Observational Study on Efficacy and Safety of Norditropin® in Children With Prader-Willi Syndrome
This study is conducted in Europe. The aim of this observational study is to collect data from children with Prader-Willi Syndrome, who have been treated off-label with Norditropin® for more than 12 months to seek approval for…
- Trial name
- NCT00705172
- Testing
- somatropin
- Where
- 3 sites in Denmark, Germany and 1 more
- Volunteers
- 41 people
- Sponsor
- Novo Nordisk A/S
Wilson Disease
Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
This study is being conducted to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease (WD).
- Trial name
- NCT05047523
- Testing
- ALXN1840, Standard of Care
- Where
- 20 sites in Australia, France and 5 more
- Volunteers
- 40 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Congenital Myotonic Dystrophy
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who…
- Trial name
- REACH CDM X (NCT05004129)
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 2 more
- Volunteers
- 76 people
- Sponsor
- AMO Pharma Limited
Nutritional and Metabolic Diseases
Efficacy and Safety of Stannsoporfin in Neonates
This is a multi-center, randomized, sham injection-controlled (placebo) masked trial of a single intramuscular injection of Stannsoporfin compared to "sham" (placebo) in healthy term and near-term newborns admitted to the…
- Trial name
- NCT02685137
- Testing
- stannsoporfin
- Where
- United States
- Volunteers
- 185 people
- Sponsor
- InfaCare Pharmaceuticals Corporation, a Mallinckrodt Company
Spinal Muscular Atrophy (SMA)
A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy
This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection…
- Trial name
- NCT07617779
- Testing
- NKG001
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Nikegen Pharmaceutical (Hangzhou) Company Limited
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