Clinical trials: Neurodegenerative Diseases
- Condition Neurodegenerative Diseases
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
This observational registry collects existing medical and routine care data from people with amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and other motor system disorders.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy, Spinal Cord Diseases, Central Nervous System Diseases
- Trial name
- NCT07900672
- Testing
- Standard of Care (Investigator Choice)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Vanishing White Matter Disease
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants.
- Trial name
- NCT05757141
- Testing
- Fosigotifator
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Calico Life Sciences LLC
Chronic Disease, HIV/AIDS
Observational Pharmaco-Epidemiology Research & Analysis
Greater advances are needed in two separate but related areas in healthcare: 1) the Clinical Decision Support Systems that complement the EHR use in support of routine patient care, population management and disease management…
- Conditions
- Chronic Disease, HIV/AIDS, Liver Diseases, Inflammatory Bowel Diseases
- Trial name
- OPERA (NCT03813862)
- Testing
- Non-interventional (observational only)
- Where
- United States
- Volunteers
- 2,000,000 people, healthy volunteers welcome
- Sponsor
- Epividian
Parkinson Disease
Compassionate Use for BIA 28-6156 in Patients With Parkinson's Disease (PD) Who Have a Pathogenic Variant in the GBA1 Gene (GBA-PD) and Completed the ACTIVATE Study
This Program is designed to provide continued access BIA 28-6156, free and compassionately for an eligible patient previously enrolled in the BIA 28-6156-201 (ACTIVATE) clinical study and that had completed the study.
- Trial name
- NCT07522606
- Testing
- BIA 28-6156
- Where
- 36 sites in United States, Italy and 3 more
- Sponsor
- Bial R&D Investments, S.A.
Tourette Syndrome
A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder
The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to [>=] 6 and less than [<] 12 years of age), adolescents (>=12 and <18 years of…
- Trial name
- NCT06021522
- Testing
- Ecopipam
- Where
- 67 sites in United States, Bulgaria and 6 more
- Volunteers
- 150 people
- Sponsor
- Emalex Biosciences Inc.
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- REVERSE (NCT02652780)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 37 people
- Sponsor
- GenSight Biologics
Leber Hereditary Optic Neuropathy
REALITY LHON Registry
This study is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on retrospective subjects' medical chart abstractions and cross-sectional administration of patient-reported…
- Trial name
- REALITY (NCT03295071)
- Testing
- Patient-reported outcomes (PROs)
- Where
- 11 sites in United States, France and 3 more
- Volunteers
- 44 people
- Sponsor
- GenSight Biologics
Leber Hereditary Optic Neuropathy
RESCUE and REVERSE Long-term Follow-up
The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in…
- Trial name
- RESTORE (NCT03406104)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 62 people
- Sponsor
- GenSight Biologics
Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
EAP_GS010_single Patient
Expanded Access Use for a single patient of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected with G11778A ND4 Leber Hereditary Optic Neuropathy
- Trial name
- NCT03672968
- Testing
- GS010
- Sponsor
- GenSight Biologics
Leber Hereditary Optic Disease
Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients
The goal of this Clinical trial is to assess the safety and efficacy of GS010 at two dose levels on visual acuity and retinal mitochondrial activity in patients affected with ND4 Leber Hereditary Optic Neuropathy (LHON)
- Trial name
- REVISE (NCT07303296)
- Testing
- GS010 High dose, GS010 Low dose
- Where
- France
- Volunteers
- 14 people
- Sponsor
- GenSight Biologics
Gastrointestinal Disease, Inflammatory Bowel Disease (IBD)
Elemental 028 Extra Case Studies
Elemental 028 Extra is a nutritionally complete ACBS approved amino acid-based (elemental) feed designed to provide adequate daily amounts of both macronutrients and micronutrients when used as a sole source of nutrition and to…
- Conditions
- Gastrointestinal Disease, Inflammatory Bowel Disease (IBD), Short Bowel, Malabsorption
- Trial name
- E028E (NCT06877923)
- Testing
- Elemental 028 Extra
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Nutricia UK Ltd
Dementia, Alzheimer Disease
Assessing a Novel Virtual Environment That Assists With Activities of Daily Living
This Phase 1 study looks at how a new videogame-based program can help residents and care partners prepare for ADLs in a fun way with minimal risk, potentially reducing escalating problems associated with ADL completions.
- Conditions
- Dementia, Alzheimer Disease, Neurodegenerative Diseases
- Trial name
- NCT05418296
- Testing
- A novel digital health device
- Where
- 2 sites in United States
- Volunteers
- 38 people
- Sponsor
- mandy
Alzheimer's Disease, Behavioral Variant Frontotemporal Dementia
Open-Label Study of Leuco-methylthioninium Bis(Hydromethanesulfonate) (LMTM) in Subjects With Alzheimer's Disease or Behavioral Variant Frontotemporal Dementia (bvFTD)
The purpose of this study is to provide subjects who have completed participation in a Phase 2 or Phase 3 trial of LMTM continued access to therapy and to evaluate the long-term safety of LMTM.
- Trial name
- NCT02245568
- Testing
- LMTM
- Where
- 135 sites in United States, Australia and 15 more
- Volunteers
- 913 people
- Sponsor
- TauRx Therapeutics Ltd
Degenerative Conditions, Neurologic
The Fixation Study
To perform a clinical evaluation of safety and performance for the SeaSpine Non- Cervical Pedicle Screw Systems
- Trial name
- NCT04300777
- Testing
- SeaSpine Non-Cervical Pedicle Screw Systems
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- SeaSpine, Inc.
Leigh Syndrome
A Study of the Natural Course of SURF1 Deficiency
The purpose of the study is to prospectively and systematically collect standardized clinical information, to describe important features of the disease course of SURF1 deficiency.
- Trial name
- NCT05277363
- Testing
- None (Observational study)
- Where
- United States
- Sponsor
- Taysha Gene Therapies, Inc.
Alzheimer's Disease
Safety, Tolerability and Abeta-specific Antibody Response of Repeated i.m. Injections of Adjuvanted CAD106 in Mild Alzheimer Patients
This study will assess the safety, tolerability and Abeta-specific antibody response of repeated intra-muscular injections of adjuvanted CAD106 in patients with mild Alzheimer's Disease.
- Trial name
- NCT01097096
- Testing
- CAD106, Alum, MF59
- Where
- 31 sites in United States, Belgium and 8 more
- Volunteers
- 177 people
- Sponsor
- Novartis Pharmaceuticals
Alzheimer Disease
To Investigate the Safety and Tolerability of Repeated Subcutaneous Injections of CAD106 in Alzheimer's Patients
This study will investigate the Safety and Tolerability of Repeated Subcutaneous Injections of CAD106 in Alzheimer's Patients.
- Trial name
- NCT01023685
- Testing
- CAD106
- Where
- 8 sites in United States
- Volunteers
- 24 people
- Sponsor
- Novartis
Alzheimer's Disease
An Open-Label Crenezumab Study in Participants With Alzheimer's Disease
In the BN40031 OLE study, a dose of crenezumab of 60 mg/kg intravenous (IV) every 4 weeks (Q4W) will be offered to all participants who complete Study BN29552 or BN29553 and who meet eligibility criteria in order to evaluate…
- Trial name
- CREAD OLE (NCT03491150)
- Testing
- Crenezumab
- Where
- 66 sites in United States, Australia and 14 more
- Volunteers
- 149 people
- Sponsor
- Hoffmann-La Roche
Neurodegenerative Diseases, Osteoarthritis
Safety and Clinical Outcomes Study: SVF Deployment for Orthopedic, Neurologic, Urologic, and Cardio-pulmonary Conditions
To evaluate for any adverse effects that may be related to the administration and reception of autologous adipose derived stromal vascular fraction (SVF).
- Conditions
- Neurodegenerative Diseases, Osteoarthritis, Erectile Dysfunction, Autoimmune Diseases
- Trial name
- NCT01953523
- Testing
- Administration of autologous adipose derived SVF
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Elliot Lander