Mitochondrial Dysfunction and Disease Progression
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|ClinicalTrials.gov Identifier: NCT02549703|
Recruitment Status : Completed
First Posted : September 15, 2015
Last Update Posted : February 20, 2019
While the last several years have seen great strides in the treatment of relapsing forms of MS, progressive MS, responsible for the majority of MS-related disability, lags far behind. Despite much research, the lack of understanding related to what causes patients' relentless decline in function results in an inability to develop targeted treatment strategies suitable for clinical trials. This grant has two main goals.
The first goal is to extend the investigators preliminary study on rat neurons treated with the CSF of MS patients to a larger number of Progressive patients in order to validate the initial findings and extend the study to include analysis of human neurons. The initiating PI (Dr. Casaccia) and the Partnering PI and Clinical Neurologist (Dr. Katz Sand) have recently identified components that are present in the CSF of progressive patients that impair the ability of rat neurons to produce energy. The partnering PI, Dr. Quinzii (Columbia University) together with collaborator Dr. Fossati (NY Stem Cells Foundation), have characterized human neurons generated from stem cells derived from skin biopsies of progressive patients and detected the presence of energetic deficits. The experimental plan will build on these results and test hypotheses of disease progression. The overall goal is to improve understanding on how to stop neurons from degenerating and stop clinical progression.
The second goal is to ask whether it is possible to define a progressive disease course on the basis of combined biochemical, functional and imaging measurements. The initiating PI will be responsible for the biochemical assessment of CSF and serum samples and, together with partnering PI Quinzii, will also provide functional bioassays measurements of mitochondrial bioenergetics impairment in patients. These data will be combined with clinical assessment and MRI evaluations conducted by the partnering PI Katz Sand and collaborator Inglese. A two year clinical and imaging follow up from the initial recruitment will allow to define whether the combined measurements can be used by clinical neurologists to define the disease course and better identify therapeutic options for patients.
The expectation is that the completion of the stated aims of research will allow an advancement of the current knowledge of the progressive form of MS and lead to potential new therapeutic targets.
|Condition or disease|
|Clinically Isolated Syndrome Relapsing-Remitting Multiple Sclerosis Secondary Progressive Multiple Sclerosis Primary Progressive Multiple Sclerosis|
|Study Type :||Observational|
|Actual Enrollment :||47 participants|
|Official Title:||Mitochondrial Dysfunction and Disease Progression|
|Actual Study Start Date :||September 2015|
|Actual Primary Completion Date :||September 27, 2018|
|Actual Study Completion Date :||September 27, 2018|
Relapsing Remitting Multiple Sclerosis
Patients with Relapsing Remitting Multiple Sclerosis/Clinically Isolated Syndrome
|Secondary Progressive Multiple Sclerosis|
|Primary Progressive Multiple Sclerosis|
- Spare respiratory capacity [ Time Frame: 2 years ]Mitochondrial bioenergetic measurements
- Oxygen consumption rate [ Time Frame: 2 years ]Mitochondrial bioenergetic measurements
- Multiple Sclerosis Functional Composite (MSFC) Score [ Time Frame: 1 year ]MS disease progression as measured by MSFC score which consists of the Timed 25-foot walk (T25FW) as a measure of ambulation, the Nine-hole peg test (9HPT) as a measure of arm and hand function.
- Multiple Sclerosis Functional Composite (MSFC) Score [ Time Frame: 2 years ]MS disease progression as measured by MSFC score which consists of the Timed 25-foot walk (T25FW) as a measure of ambulation, the Nine-hole peg test (9HPT) as a measure of arm and hand function.
- Expanded Disability Status Scale [ Time Frame: 2 years ]a formalized version of the neurological examination
- MS Impact Scale-29 (MSIS-29) [ Time Frame: 2 years ]a quality of life measure; an overall measure of functioning from the patient's perspective
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02549703
|United States, New York|
|Icahn School of Medicine|
|New York, New York, United States, 10029|
|Principal Investigator:||Ilana Katz Sand, MD||Icahn School of Medicine at Mount Sinai|