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Trial record 55 of 75 for:    multiple sclerosis stem cell

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
Sponsor:
Collaborators:
Columbia University
The New York Stem Cell Foundation
Information provided by (Responsible Party):
Icahn School of Medicine at Mount Sinai

Brief Summary:

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

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Study Type : Observational
Actual Enrollment : 47 participants
Observational Model: Cohort
Time Perspective: Prospective
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

Resource links provided by the National Library of Medicine


Group/Cohort
Relapsing Remitting Multiple Sclerosis
Patients with Relapsing Remitting Multiple Sclerosis/Clinically Isolated Syndrome
Secondary Progressive Multiple Sclerosis
Primary Progressive Multiple Sclerosis



Primary Outcome Measures :
  1. Spare respiratory capacity [ Time Frame: 2 years ]
    Mitochondrial bioenergetic measurements

  2. Oxygen consumption rate [ Time Frame: 2 years ]
    Mitochondrial bioenergetic measurements


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. Expanded Disability Status Scale [ Time Frame: 2 years ]
    a formalized version of the neurological examination

  4. 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
Blood and CSF samples will be stored indefinitely at the Casaccia lab. Skin samples will be stored indefinitely at the New York Stem Cell Foundation.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Multiple sclerosis patients willing and able to undergo the assessments required for this study.
Criteria

Inclusion criteria:

  • male and female subjects age 18 or older
  • diagnosis of one of the following:

    1. RRMS according to McDonald 2010 criteria or a diagnosis of CIS with clinical symptoms and MRI consistent with MS
    2. PPMS according to McDonald 2010 criteria
    3. SPMS defined as at least six months of progressive decline following an initial relapsing disease course
  • able and willing to undergo clinical evaluation, MRI, lumbar puncture, and skin biopsy and to return for follow up assessments at the end of year 1 and year 2
  • able and willing to provide informed consent.

Exclusion criteria:

  • pregnancy
  • inability to undergo lumbar puncture, due to anticoagulant therapy that cannot be held for the day of the procedure or results of screening laboratory testing or the presence of another medical condition that would render the procedure unsafe, as determined by the investigator
  • inability to undergo MRI, due to the presence of metallic implants incompatible with MRI or any other reason
  • presence of other severe medical conditions likely to influence study results or that raise the likelihood of harm to the patient as a result of study participation, as determined by the investigator (e.g. the presence of a brain mass, which could influence the CSF results and also might make lumbar puncture unsafe)
  • inability to complete the protocol for any reason

Information from the National Library of Medicine

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


Locations
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United States, New York
Icahn School of Medicine
New York, New York, United States, 10029
Sponsors and Collaborators
Icahn School of Medicine at Mount Sinai
Columbia University
The New York Stem Cell Foundation
Investigators
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Principal Investigator: Ilana Katz Sand, MD Icahn School of Medicine at Mount Sinai

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Responsible Party: Icahn School of Medicine at Mount Sinai
ClinicalTrials.gov Identifier: NCT02549703     History of Changes
Other Study ID Numbers: GCO 14-1495
CDMRP-MS140072 ( Other Grant/Funding Number: US Department of Defense )
First Posted: September 15, 2015    Key Record Dates
Last Update Posted: February 20, 2019
Last Verified: February 2019
Keywords provided by Icahn School of Medicine at Mount Sinai:
multiple sclerosis
progressive multiple sclerosis
mitochondria
neurodegeneration
Additional relevant MeSH terms:
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Multiple Sclerosis
Multiple Sclerosis, Relapsing-Remitting
Multiple Sclerosis, Chronic Progressive
Sclerosis
Pathologic Processes
Disease Progression
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Disease Attributes