Clinically Isolated SyndromeNCT02549703
Mitochondrial Dysfunction and Disease Progression
An observational study of Clinically Isolated Syndrome and Relapsing-Remitting Multiple Sclerosis, run by Icahn School of Medicine at Mount Sinai at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Clinically Isolated Syndrome, Relapsing-Remitting Multiple Sclerosis, Secondary Progressive Multiple Sclerosis
- How many people
- 47 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2015to 27 Sep 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Multiple sclerosis patients willing and able to undergo the assessments required for this study.
Who may be able to join
- male and female subjects age 18 or older
- diagnosis of one of the following:
- RRMS according to McDonald 2010 criteria or a diagnosis of CIS with clinical symptoms and MRI consistent with MS
- PPMS according to McDonald 2010 criteria
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read more
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.
- Conditions
- Clinically Isolated Syndrome, Relapsing-Remitting Multiple Sclerosis, Secondary Progressive Multiple Sclerosis, Primary Progressive Multiple Sclerosis
- Keywords
- multiple sclerosis, progressive multiple sclerosis, mitochondria, neurodegeneration
- Medical subject headings
- Multiple Sclerosis, Relapsing-Remitting, Multiple Sclerosis, Chronic Progressive, Multiple Sclerosis, Nerve Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Relapsing Remitting Multiple Sclerosis | Patients with Relapsing Remitting Multiple Sclerosis/Clinically Isolated Syndrome | |
| Secondary Progressive Multiple Sclerosis | Not described | |
| Primary Progressive Multiple Sclerosis | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Spare respiratory capacity
Mitochondrial bioenergetic measurements
Measured: 2 years
Oxygen consumption rate
Mitochondrial bioenergetic measurements
Measured: 2 years
Secondary outcomes (4)
Multiple Sclerosis Functional Composite (MSFC) Score
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.
Measured: 1 year
Multiple Sclerosis Functional Composite (MSFC) Score
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.
Measured: 2 years
Expanded Disability Status Scale
a formalized version of the neurological examination
Measured: 2 years
MS Impact Scale-29 (MSIS-29)
a quality of life measure; an overall measure of functioning from the patient's perspective
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2015 (estimated)
- Study startSep 2015
- Primary completion27 Sep 2018
- Study completion27 Sep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Clinically Isolated SyndromeStudies of the same condition, open and under way.
- More trials run by Icahn School of Medicine at Mount SinaiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Sponsor type
- University, hospital or other
- Collaborators
- Columbia University, New York Stem Cell Foundation Research Institute
- Responsible party
- Sponsor
- Investigators
- Ilana Katz Sand, MD, Principal investigator, Icahn School of Medicine at Mount Sinai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02549703
- Sponsor's protocol ID
- GCO 14-1495
- Other IDs
- CDMRP-MS140072
- Status last verified
- Feb 2019
- First submitted
- 9 Sep 2015
- Last updated
- 20 Feb 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mitochondrial Dysfunction and Disease Progression
- ClinicalTrials.gov ID
- NCT02549703
- Acronym / sponsor ID
- GCO 14-1495
- Conditions
- Clinically Isolated Syndrome, Relapsing-Remitting Multiple Sclerosis, Secondary Progressive Multiple Sclerosis, Primary Progressive Multiple Sclerosis
- Keywords
- multiple sclerosis, progressive multiple sclerosis, mitochondria, neurodegeneration
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Icahn School of Medicine at Mount SinaiUniversity, hospital or other
- Collaborators
- Columbia University, New York Stem Cell Foundation Research Institute
- Study start
- Sep 2015 (actual)
- Primary completion
- 27 Sep 2018 (actual)
- Study completion
- 27 Sep 2018 (actual)
- First posted
- 15 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Relapsing Remitting Multiple Sclerosis | – | –Patients with Relapsing Remitting Multiple Sclerosis/Clinically Isolated Syndrome |
| Secondary Progressive Multiple Sclerosis | – | – |
| Primary Progressive Multiple Sclerosis | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Multiple sclerosis patients willing and able to undergo the assessments required for this study.
Key inclusion criteria (7 listed)
- male and female subjects age 18 or older
- diagnosis of one of the following:
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Spare respiratory capacity
Time frame: 2 years
Mitochondrial bioenergetic measurements
Oxygen consumption rate
Time frame: 2 years
Mitochondrial bioenergetic measurements
4 secondary outcomes
Multiple Sclerosis Functional Composite (MSFC) Score
Time frame: 1 year
Multiple Sclerosis Functional Composite (MSFC) Score
Time frame: 2 years
Expanded Disability Status Scale
Time frame: 2 years
MS Impact Scale-29 (MSIS-29)
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ilana Katz Sand, MD principal investigator
Icahn School of Medicine at Mount Sinai
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Clinically Isolated Syndrome and Relapsing-Remitting Multiple Sclerosis, run by Icahn School of Medicine at Mount Sinai at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Spare respiratory capacity (time frame: 2 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2019.