Clinical trials: Degenerative Neurologic Disease
- Condition Degenerative Neurologic Disease
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Unverricht-Lundborg Disease
Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease in Adolescents and Adults
The study will compare the efficacy and safety of brivaracetam with placebo in patients with Unverricht-Lundborg disease.
- Trial name
- NCT00357669
- Testing
- Brivaracetam 25 mg, Brivaracetam 50 mg
- Where
- 13 sites in Finland, France and 5 more
- Volunteers
- 50 people
- Sponsor
- UCB Pharma SA
Alzheimers Disease
A Postmarketing Study of LEQEMBI in United Kingdom (UK) Participants With Alzheimer's Disease (AD)
The primary purpose of the study is to describe the incidence and severity of known adverse events (AEs), including Amyloid-Related Imaging Abnormalities Oedema/Effusion (ARIA-E), Amyloid-Related Imaging…
- Trial name
- NCT07781345
- Testing
- No Intervention
- Where
- 3 sites in United Kingdom
- Volunteers
- 400 people
- Sponsor
- Eisai Limited
Neurofibromatosis
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
The purpose of this tria is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel to patients with neurofibromatosis type I.
- Trial name
- NCT04461886
- Testing
- NPC-12G gel
- Where
- 4 sites in Japan
- Volunteers
- 100 people
- Sponsor
- Nobelpharma
Alzheimer Disease
Memesto Wearable Device for Persons With Dementia
An estimated 70% of the 7.2+ million people in the U.S. with Alzheimer's Disease and Alzheimer's Disease-Related Dementias experience agitation, characterized by poorly organized and purposeless psychomotor activity that…
- Trial name
- NCT05153161
- Testing
- Memesto
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Edgewater Safety Systems, Inc.
Retinitis Pigmentosa (RP), Usher Syndrome Type 2
Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
The purpose of this Phase 2 Open-label study is to evaluate the long-term safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in participants with Retinitis Pigmentosa due to mutations in exon 13…
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- CAPELLA (NCT07796646)
- Testing
- No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen
- Volunteers
- 81 people
- Sponsor
- Laboratoires Thea
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
Tourette Syndrome
Aripiprazole Oral Solution in the Treatment of Children and Adolescents With Tourette's Syndrome
This study is a multicenter, randomized, double-blind, placebo-controlled trial designed to assess the safety, efficacy, tolerability and steady-state plasma trough concentration of flexible-dosed aripiprazole once-daily…
- Trial name
- NCT03487783
- Testing
- Aripiprazole Oral Solution
- Where
- China
- Volunteers
- 121 people
- Sponsor
- Otsuka Beijing Research Institute
Muscular Atrophy, Spinal
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.
- Trial name
- NCT04256265
- Testing
- Risdiplam
- Where
- 29 sites in United States
- Sponsor
- Genentech, Inc.
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Tuberous Sclerosis Complex (TSC)
Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC)
1. To assess treatment efficacy with YCJ-01 by changes in the number of epileptic seizures in patients with refractory epilepsy secondary to Tuberous Sclerosis Complex. 2.
- Trial name
- Spectrum (NCT07403266)
- Testing
- YCJ-01 Full Spectrum cannbis Extract
- Where
- Spain
- Volunteers
- 84 people
- Sponsor
- Oils4Cure
Parkinson Disease
Special Survey on Parkinson's Disease (PD) Patients Without Concomitant Use of L-Dopa
The survey is conducted to collect safety and effectiveness information in Parkinson's Disease patients treated with Pramipexole without concomitant L-Dopa supplementation in the daily clinical settings in Japan.
- Trial name
- NCT00613301
- Where
- 119 sites in Japan
- Volunteers
- 416 people
- Sponsor
- Boehringer Ingelheim
Muscular Atrophy, Spinal
A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- ONWARD (NCT04729907)
- Testing
- Nusinersen
- Where
- 39 sites in United States, Brazil and 14 more
- Volunteers
- 115 people
- Sponsor
- Biogen
Hallucinations, Psychoses
Treatment of Hallucinosis/Psychosis in Parkinson's Disease by an Investigational Drug
The primary objective is to demonstrate that the investigational new drug, ACP-103, is well tolerated by, and will not worsen parkinsonism in, patients with Parkinson's disease and psychosis.
- Conditions
- Hallucinations, Psychoses, Parkinson's Disease
- Trial name
- NCT00087542
- Testing
- ACP-103
- Where
- 13 sites in United States
- Volunteers
- 60 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)
This is a study of the safety and tolerability of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA)-related…
- Trial name
- NCT04980872
- Testing
- Miransertib
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 60 people
- Sponsor
- Merck Sharp & Dohme 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
Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant
Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation
The purpose of this study is to characterize the disease progression of confirmed OPA1 mutation-associated autosomal dominant optic atrophy (ADOA) by evaluating the changes in ocular structural and functional outcomes.
- Conditions
- Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
- Trial name
- NCT06140329
- Where
- 7 sites in United States, Australia and 4 more
- Volunteers
- 1 people
- Sponsor
- PYC Therapeutics
Tourette Syndrome
Evaluation of Safety and Tolerability of Long-term TEV-50717 (Deutetrabenazine) for Treatment of Tourette Syndrome in Children and Adolescents
This is an otherwise open-label, single-arm study that includes a 2-week, double-blind, placebo controlled, randomized drug withdrawal period followed by a 3 week blinded maintenance or re-titration, and then a maintenance…
- Trial name
- ARTISTS (NCT03567291)
- Testing
- TEV-50717
- Where
- 76 sites in United States, Argentina and 13 more
- Volunteers
- 228 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Alexander Disease
Zilganersen Expanded Access Program for Individuals With Alexander Disease
The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).
- Trial name
- NCT07487389
- Testing
- zilganersen
- Sponsor
- Ionis Pharmaceuticals, Inc.
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Myotonic Dystrophy 1
Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy.
- Trial name
- NCT02858908
- Testing
- Tideglusib
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- AMO Pharma Limited