Clinical trials: Neurodegenerative Diseases
Wilson Disease
Early Access Program for ALXN1840 in Patients With Wilson Disease
This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD.
- Trial name
- NCT05686564
- Testing
- ALXN1840
- Sponsor
- Alexion Pharmaceuticals, Inc.
Amyotrophic Lateral Sclerosis
Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS
ALSUMMIT is a double-blind, randomized, placebo-controlled, multi-center, parallel, phase III clinical trial to evaluate and confirm the efficacy and long-term safety of repeated Lenzumestrocel (Neuronata-R® inj.) treatment.
- Trial name
- ALSummit (NCT04745299)
- Testing
- Lenzumestrocel, Riluzole
- Where
- 5 sites in South Korea
- Volunteers
- 123 people
- Sponsor
- Corestemchemon, Inc.
Alzheimer's Disease
Safety and Tolerability of AFFITOPE AD03
This is a randomized, controlled, parallel group, patient-blinded, single-center, phase I pilot study to assess tolerability and safety of repeated subcutaneous administration of a single-dose of AFFITOPE AD03 applied with or…
- Trial name
- MimoVax (NCT01309763)
- Testing
- AFFITOPE AD03, AFFITOPE AD03 + Alum
- Where
- Austria
- Volunteers
- 28 people
- Sponsor
- Affiris AG
Alzheimer Disease, Huntington Disease
Dimebon (PF-01913539)-Digoxin Drug-Drug Interaction Study In Healthy Subjects
This study has been designed to confirm, in healthy subjects, the lack of a clinically important pharmacokinetic interaction between Dimebon, at the proposed maximum commercial dose of 20 mg TID (administered every 8 hours), and…
- Trial name
- NCT00831506
- Testing
- digoxin, dimebon
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Pfizer
Spinal Muscular Atrophy
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
- Trial name
- STRONG (NCT03381729)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 11 sites in United States
- Volunteers
- 32 people
- Sponsor
- Novartis Gene Therapies
Alzheimer's Disease
Single Ascending Dose Study of BIIB037 in Participants With Alzheimer's Disease
The primary objective of the study is to evaluate the safety and tolerability of a range of BIIB037 doses administered as single intravenous (IV) infusions in participants with mild to moderate Alzheimer's Disease (AD).
- Trial name
- NCT01397539
- Testing
- BIIB037
- Where
- 3 sites in United States
- Volunteers
- 53 people
- Sponsor
- Biogen
Parkinson Disease
A Single-dose Study to Investigate the Tolerance and Pharmacokinetics of Finamine Tablets in China
Design：Randomization, double-blind, single-center, single-dose, dose-escalation , placebo and parallel control Objectives： 1. To investigate the tolerability and safety of Chinese healthy adult subjects after a single oral…
- Trial name
- NCT04162275
- Testing
- Finamine tablets
- Where
- China
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Yiling Pharmaceutical Inc.
Parkinson's Disease
Tolerability and Safety of Subcutaneous Administration of Two Doses of AFFITOPE® PD01A in Early Parkinson's Disease
This is a phase I study to assess the tolerability and safety of 4 injections of two different doses of AFFITOPE® PD01A formulated with an adjuvant to patients with early Parkinson's disease in male and female patients aged 45 to…
- Trial name
- NCT01568099
- Testing
- AFFITOPE® PD01A, Control
- Where
- Austria
- Volunteers
- 32 people
- Sponsor
- Affiris AG
Mild to Moderate Dementia Due to Alzheimer's Disease, With or Without Coexisting Cerebrovascular Disease
A Clinical Study Evaluating the Efficacy and Safety of RPh201 Treatment in Individuals With Alzheimer's Disease With or Without Coexisting Cerebrovascular Disease
This study is a randomized, double-blind, placebo-controlled, multicentre, Phase 2 study, with an optional open-label extension, to evaluate the safety, tolerability, and efficacy of RPh201 in subjects with mild to moderate AD…
- Trial name
- NCT03462121
- Testing
- RPh201
- Where
- 5 sites in Canada
- Volunteers
- 83 people
- Sponsor
- Regenera Pharma Ltd
Alzheimer Disease
A Biomarker-directed Study of XPro1595 in Patients With Alzheimer's
The purpose of this study is to evaluate safety and target engagement of XPro1595 in Alzheimer's patients with biomarkers of inflammation.
- Trial name
- NCT03943264
- Testing
- XPro1595
- Where
- 5 sites in Australia
- Volunteers
- 20 people
- Sponsor
- Inmune Bio, Inc.
Myotonic Dystrophy 1
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- FREEDOM-DM1 (NCT06204809)
- Testing
- PGN-EDODM1 for infusion
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Alzheimer's Disease, Dementia
Efficacy and Safety of Aricept in the Treatment of Severe Alzheimer's Disease
Donepezil hydrochloride (Aricept) has been approved to treat symptoms associated with mild to moderate Alzheimer's disease (AD).
- Trial name
- NCT00096473
- Testing
- Donepezil hydrochloride
- Where
- 42 sites in United States, Australia and 4 more
- Volunteers
- 229 people
- Sponsor
- Eisai Inc.
Friedreich's Ataxia
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
- Trial name
- NCT02445794
- Testing
- Low dose cohort, High dose cohort
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Biojiva LLC
Myasthenia Gravis, Generalized
A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis
The purpose of this study is to evaluate the pharmacodynamic (PD) effect, safety, and tolerability of Povetacicept in participants with generalized myasthenia gravis (gMG).
- Trial name
- ETNA (NCT07501702)
- Testing
- Povetacicept
- Where
- 31 sites in United States, Australia and 3 more
- Volunteers
- 34 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Spinocerebellar Degeneration
Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, phase III study to evaluate the efficacy and safety of KPS-0373 given once daily after breakfast in 142 patients with spinocerebellar…
- Trial name
- NCT07040137
- Testing
- KPS-0373
- Where
- Japan
- Volunteers
- 142 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Parkinson Disease
Mass Balance Recovery and Metabolite Identification of Carbon-14 BIA 28-6156
The study is designed to determine the absorption, distribution, metabolism and elimination (ADME) of BIA 28-6156 in humans, further explore the PK of BIA 28-6156, evaluate the extent of distribution of total radioactivity into…
- Trial name
- NCT05220072
- Testing
- Carbon-14 BIA 28-6156
- Where
- United Kingdom
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Bial R&D Investments, S.A.
Parkinson's Disease
A Clinical Study Investigating the Efficacy, Tolerability, and Safety of Continuous Subcutaneous ND0612 Infusion Given as Adjunct Treatment to Oral Levodopa in Patients With Parkinson's Disease With Motor Fluctuations
This is a multicenter, randomized, double blind, placebo controlled parallel group clinical study. Following a screening period of up to 28 days, eligible subjects will be randomized to receive adjunct treatment to oral LD/DDI…
- Trial name
- NCT02782481
- Testing
- ND0612
- Where
- Israel
- Sponsor
- NeuroDerm Ltd.
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
Tourette's Disorder
Safety and Tolerability Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current trial is to obtain long term efficacy, safety and tolerability data of once weekly aripiprazole in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01416441
- Testing
- Aripiprazole
- Where
- 80 sites in United States, Bulgaria and 8 more
- Volunteers
- 170 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Parkinson Disease
Evaluation of the Safety and Effectiveness of Bilateral Exablate Ablation of the Pallidothalamic Tract (PTT) for the Treatment of the Motor Complications of Parkinson's Disease (PD)
Evaluate the Safety and Effectiveness of Staged Bilateral Exablate Ablation of the Pallidothalamic Tract (PTT) for the Treatment of the Motor Complications of Parkinson's Disease (PD).
- Trial name
- NCT04728295
- Testing
- Exablate 4000
- Where
- 9 sites in United States, Spain and 1 more
- Volunteers
- 50 people
- Sponsor
- InSightec
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