Clinical trials: Neuro-Degenerative Disease
Friedreich Ataxia
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological…
- Trial name
- UNIFAI (NCT06016946)
- Where
- 34 sites in United States, Australia and 15 more
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Friedreich's Ataxia Research Alliance
Friedreich Ataxia
Characterization of the Interruptions of the GAA Expansion and Study of Their Influence on the Severity of Friedreich's Ataxia
Friedreich's ataxia (FA) is the most frequent recessive genetic ataxia with an estimated prevalence of 1/50 000. The first symptoms appear around the age of 10 years with a progressive course and the need for an armchair 10- 15…
- Trial name
- INTREP-AF (NCT04346238)
- Testing
- Reuse of existing data from patients' medical records
- Where
- France
- Volunteers
- 120 people
- Sponsor
- University Hospital, Montpellier
Neuro-Degenerative Disease
Contribution of New Generation Oxford Nanopore-type High-throughput Sequencing in the Diagnostic Strategy of Neurogenetic Diseases.
Since 2012, NGS sequencing of long fragments or long reads has developed in various fields of research and today presents itself as a very promising alternative solution in the analysis of repeat amplifications.
- Trial name
- NEURONGS3 (NCT04621422)
- Testing
- Subject carrying an amplification of nucleotide repeats in one of the following 9 genes FMR1, DMPK, ZNF9, SCA2, JPH3, HD, FXN, C9ORF72, RFC1
- Volunteers
- 60 people
- Sponsor
- University Hospital, Bordeaux
Nutritional Deficiency, Neuro-Degenerative Disease
Nutritional Prognostic Factors in ALS
Background. Several nutritional factors have been evaluated as prognostic factors for survival in ALS patients at earlier stages of the disease [body mass index (BMI), body composition expressed as fat free mass (FFM), fat mass…
- Conditions
- Nutritional Deficiency, Neuro-Degenerative Disease, Gastrostomy
- Trial name
- PEG-ALS (NCT03487536)
- Testing
- Nutritional assessment
- Where
- Italy
- Volunteers
- 47 people
- Sponsor
- University of Bari
Friedreich Ataxia
Neurology Measures in FA Children
The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA.
- Trial name
- NCT03418740
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Children's Hospital of Philadelphia
Brain (Nervous System) Cancers, Brain Arterial Disease
A Vision-Language Foundation Model for Brain Disease Diagnosis From Multimodal Data
The goal of this observational study is to develop an innovative, comprehensive, and explainable AI vision-language foundation model (VLM) to advance the diagnosis and interpretation of brain diseases using multi-modal data.
- Conditions
- Brain (Nervous System) Cancers, Brain Arterial Disease, Neuro-Degenerative Disease, Brain Tumors
- Trial name
- NCT07126821
- Testing
- No Interventions
- Where
- China
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Xiangya Hospital of Central South University
Langerhan's Cell Histiocytosis, Juvenile Xanthogranuloma
Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders
This is a research study of a drug called cobimetinib in children and adults diagnosed with Langerhans cell histiocytosis (LCH), and other histiocytic disorders that has returned or does not respond to treatment.
- Conditions
- Langerhan's Cell Histiocytosis, Juvenile Xanthogranuloma, Erdheim-Chester Disease, Rosai Dorfman Disease
- Trial name
- NACHO-COBI (NCT04079179)
- Testing
- Cobimetinib
- Where
- 12 sites in United States
- Volunteers
- 90 people
- Sponsor
- Carl Allen
Peripheral Neuropathy, Neuro-Degenerative Disease
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
This is a multicenter, longitudinal, prospective observational natural history study of subjects with a molecularly confirmed diagnosis of CMT4J.
- Conditions
- Peripheral Neuropathy, Neuro-Degenerative Disease, Neuromuscular Diseases
- Trial name
- CMT4J (NCT06151600)
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- Elpida Therapeutics SPC
Encephalopathy, Birth Defect
Clinical Utility of Pediatric Whole Exome Sequencing
The investigator aims to examine the clinical utility of WES, including assessment of a variety of clinical outcomes in undiagnosed pediatric cases.
- Conditions
- Encephalopathy, Birth Defect, Intellectual Disability, Multiple Congenital Anomaly
- Trial name
- NCT03525431
- Testing
- Whole Exome Sequencing
- Where
- 4 sites in United States
- Volunteers
- 529 people
- Sponsor
- University of California, San Francisco
Neuro-Degenerative Disease
Efficiency of E-learning and Role-playing for the Training of Nursing Home Caregivers in the Support of Agitation in Neurodegenerative Diseases
This study will investigate the effects of "I-Learn cognition and behaviour" training in the care in agitation in patients with neurodegenerative diseases.
- Trial name
- NCT04937127
- Testing
- I-LEARN training, usual care
- Where
- 7 sites in France
- Volunteers
- 170 people
- Sponsor
- University Hospital, Tours
Friedreich Ataxia, Neuro-Degenerative Disease
FA Clinical Outcome Measures
This multicenter natural history study aims to expand the network of clinical research centers in FA, and to provide a framework for facilitating therapeutic interventions.
- Trial name
- FA-COMS (NCT03090789)
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Friedreich's Ataxia Research Alliance
Diabetes, Endocrine System Diseases
Evaluating Innovations in Transition From Pediatric to Adult Care - The Transition Navigator Trial
The Transition Navigator Trial (TNT) is a pragmatic randomized controlled trial evaluating the effectiveness of usual care plus a patient navigator service versus usual care plus newsletters and other educational materials, to…
- Conditions
- Diabetes, Endocrine System Diseases, Gastro-Intestinal Disorder, Neuro-Degenerative Disease
- Trial name
- TNT (NCT03342495)
- Testing
- Patient Navigator, Usual Care
- Where
- 3 sites in Canada
- Volunteers
- 337 people
- Sponsor
- University of Calgary
Cancer, Pregnancy Related
Distribution of Biospecimens From Biorepositories/Biobanks for Research Use
This protocol seeks to assist biorepositories/biobanks in distributing their stored specimens and data to researchers that will actually utilize them to advance medicine and technology.
- Conditions
- Cancer, Pregnancy Related, Gastro-Intestinal Disorder, Cardio-Respiratory Distress
- Trial name
- NCT03884400
- Testing
- Specimens obtained from surgical, phlebotomy or other non-invasive procedure
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- iSpecimen Inc