Progressive Multiple SclerosisPhase 2ACTiMuSNCT01815632
Assessment of Bone Marrow-derived Cellular Therapy in Progressive Multiple Sclerosis (ACTiMuS)
A Phase 2 study testing Early infusion of autologous marrow and Late infusion of autologous marrow in people with Progressive Multiple Sclerosis, run by North Bristol NHS Trust at 1 site in United Kingdom.
Trial summary
Multiple sclerosis - MS - affects 1.3m people worldwide, costing the European Union economy €9 billion/year, through both direct and indirect consequences of progressive disability. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Progressive Multiple Sclerosis
- What is being tested?
- Early infusion of autologous marrow and Late infusion of autologous marrow
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jan 2014to Oct 2019
- Phase
- Phase 2
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of clinically-definite MS as defined by the McDonald criteria
- Aged 18 - 65 years.
- EDSS of 4.0 to 6 inclusive
- Disease duration >5 years
- Disease progression (increase in physical disability, not due to major relapse) in preceding year
- Signed, written informed consent
- Willing and able to comply with study visits according to protocol for the full study period
Who cannot join
- Pregnancy, breastfeeding or lactation
- History of autologous/allogenic bone marrow transplantation or peripheral blood stem cell transplant
- Bone marrow insufficiency
- History of lymphoproliferative disease or previous total lymphoid irradiation
- Immune deficiency
- Current or recent (<5 years) malignancy
Show all 20Show fewer
- Chronic or frequent drug-resistant bacterial infections or presence of active infection requiring antimicrobial treatment
- Frequent and/or serious viral infection
- Systemic or invasive fungal disease within 2 years of entry to study
- Significant renal, hepatic, cardiac or respiratory dysfunction
- Contraindication to anaesthesia
- Bleeding or clotting diathesis
- Current or recent (within preceding 12 months) immunomodulatory therapy other than corticosteroid therapy
- Treatment with corticosteroids within the preceding three months
- Significant relapse within preceding 6 months
- Predominantly relapsing-remitting disease over preceding 12 months
- Radiation exposure in the past year other than chest / dental x-rays
- Previous claustrophobia
- The presence of any implanted metal or other contraindication to MRI
- Participation in another experimental study or treatment within the preceding 24 months
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site, 1 recruiting
- Recruiting
Southmead Hospital
Bristol, BS10 5NB
Neil J Scolding, FRCP PhD
Claire M Rice, MA MRCP PhD
About this study
Multiple sclerosis - MS - affects 1.3m people worldwide, costing the European Union economy €9 billion/year, through both direct and indirect consequences of progressive disability. Despite the usual relapsing-remitting presentation, over 80% of patients develop progressive disability; 40% require a wheelchair within 10 years of diagnosis. At present, there are no treatments that reverse, halt or even slow progressive disability in MS.
The investigators recently completed one of the first feasibility/safety trials in the world of reparative bone marrow cell therapy in 6 patients with longstanding MS (www.nature.com/clpt/journal/v87/n6/full/clpt201044a.html). Safety was confirmed, and intensive repeated tests on the patients measuring nerve conduction in various pathways in the brain and in the spinal cord showed statistically significant improvements at 12 months in every patient. While highly preliminary and involving only a very small number of patients, these results at least raise the possibility of a significant (though very partial) underlying repair effect within the damaged nervous system.
The investigators believe this urgently requires further testing - both to accelerate benefit for patients, and to begin improving therapeutic efficacy. The investigators therefore propose a programme of translational and clinical stem cell research, aiming (1) to continue translation with a phase two controlled trial of bone marrow cells in patients with longstanding MS; and (2) to explore in parallel the potential mechanisms of action, by studying bone marrow cells from treated patients and control subjects, aiming to establish which of the various relevant bone marrow subpopulations contribute to efficacy, and which particular reparative mechanism(s) are important. The investigators hope these studies will not only confirm the therapeutic benefit of this approach, but also provide the basis for improving the magnitude and impact of this novel and exciting treatment modality.
Read the detailed description
The primary objective is to determine whether autologous bone marrow (BM) (ie taken from the patients themselves rather than from a BM donor) cell therapy is truly beneficial in chronic multiple sclerosis - as our small, uncontrolled phase 1 trial suggested (www.nature.com/clpt/journal/v87/n6/full/clpt201044a.html).
The investigators also aim to answer the following questions:
- Do BM mesenchymal stem cells from patients with MS differ in the range or extent of reparative and neuroprotective properties from those from control individuals?
- What reparative and neuroprotective properties do BM stem cell subpopulations other than mesenchymal stem cells possess, and do these differ between MS patients and controls?
- Can BM stem cell subpopulations be isolated from blood samples from MS patients following treatment, and, if so, for how long?
Bone marrow contains stem cells capable of replacing all the cells in the blood. Recently, bone marrow has been found to contain cells capable of replacing cells in tissues and organs other than blood. In addition, infusion of bone marrow-derived cells has been shown to have significant effects on the immune system and to promote the survival of central nervous system cells under toxic conditions. These properties are of considerable interest to those working to develop cell-based therapies for neurodegenerative disease.
The potential of such cells to aid repair in multiple sclerosis (MS) has been examined in experimental models of MS. Myelin is the substance that insulates neurons within the central nervous system and is attacked in MS. Infusion of adult bone marrow cells into a vein aids myelin repair (remyelination) in these models of MS. Recently, the investigators performed a small pilot study to examine the effects of harvesting bone marrow from MS patients and infusing this back into each patient's vein. This was well tolerated and no significant adverse events were encountered. Electrophysiological studies appeared to show some improvement but, given the small numbers of participants in the phase 1 trial, no definitive conclusion can be made regarding this.
The current study seeks to address the question of whether genuine improvements in neurophysiological tests are seen in MS patients with progressive disease following infusion of bone marrow.
A prospective, randomised, double-blind, placebo-controlled, stepped wedge design will be employed at a single centre (Bristol, UK). Eighty patients with progressive MS will be recruited; 60 will have secondary progressive disease (SPMS) but a subset (n=20) will have primary progressive disease (PPMS). Participants will be randomised to either early (immediate) or late (1 year) intravenous infusion of autologous, unfractionated bone marrow. The placebo intervention is infusion of autologous blood. The primary outcome measure is global evoked potential derived from multimodal evoked potentials. Secondary outcome measures include adverse event reporting, clinical (EDSS and MSFC) and self-assessment (MSIS-29) rating scales, optical coherence tomography (OCT) as well as brain and spine MRI. Participants will be followed up for a further year following the final intervention. Outcomes will be analyzed on an intention-to-treat basis.
- Conditions
- Progressive Multiple Sclerosis
- Being tested
- Early infusion of autologous marrow, Late infusion of autologous marrow
- Keywords
- multiple sclerosis, progressive MS, stem cell, cell therapy, neurodegeneration, CNS repair
- Medical subject headings
- 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.
Early infusion of autologous marrowOther
Intravenous infusion of autologous bone marrow without prior myeloablation on the day of bone marrow harvest and infusion of autologous blood at one year post-bone marrow harvest
Late infusion of autologous marrowOther
Infusion of blood on day of bone marrow harvest with intravenous infusion of autologous bone marrow without prior myeloablation one year post-bone marrow harvest.
| Study group | Role | What they receive |
|---|---|---|
| Early infusion of autologous marrow | Experimental | Intravenous infusion of autologous bone marrow (without myeloablation) on the day of bone marrow harvest. Infusion of autologous blood at one year post-harvest Receives: Other: Early infusion of autologous marrow |
| Late infusion of autologous marrow | Experimental | Intravenous infusion of autologous blood on the day of bone marrow harvest. Infusion of autologous bone marrow (without myeloablation) at one year post-harvest Receives: Other: Late infusion of autologous marrow |
What is being measured
The primary outcome is the main question the study is designed to answer.
Global evoked potential (GEP): mean change from time of marrow infusion to end of study
Multimodal evoked potentials will be examined at 0, 6, 12, 18 and 24 months. Evoked potential abnormalities will be quantified according to a 4-point graded ordinal score modified from Leocani et al (JNNP 2006, 77:1030-1035) (0=normal; 1=increased latency; 2=increased latency and abnormal amplitude; 3=absent).
The recording of the evoked potentials shall be in accordance with the Guidelines of the International Federation of Clinical Neurophysiology and analysis will be performed using standard methods. Electrophysiological responses shall be considered abnormal if they exceed 2.5 standard deviations of the normal values or cannot be detected.
Measured: Entry and every 6 months for 2 years
Secondary outcomes (6)
Safety
Evaluation of number and nature of adverse events
Measured: Continuous throughout study period (2 years)
Expanded disability status scale
Time to EDSS progression of at least one point from a baseline EDSS of 4.0, 4.5 or 5.0 or at least 0.5 points from a baseline EDSS ≥5.5
Measured: At entry then 6 weeks, 6 months and 1 year after each infusion
Multiple sclerosis impact scale (MSIS-29)
Mean change from baseline to end of study on the patient-based MSIS-29 scale
Measured: At entry then 6 weeks, 6 months and 1 year after each infusion
Multiple sclerosis functional composite (MSFC)
Mean change on MSFC score from baseline to final visit
Measured: At entry then 6 weeks, 6 months and 1 year after each infusion
MRI head and cord
The secondary MRI outcome measures will relate to 1) lesion load, 2) atrophy measures both of the brain and of cross-sectional area of the spinal cord, and changes in mean diffusivity.
Exploratory analysis of the resting-state fMRI data will investigate correlations between network patterns and 'strength' of networks connectivity from the resting-state fMRI with classifications revealed by the various evoked potential studies
Measured: At entry, 1 year and 2 years post-harvest
Optical coherence tomography
Measurement of retinal nerve fibre layer thickness and macular volume
Measured: Entry, 1 year and 2 years post-harvest
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2013 (estimated)
- Study startJan 2014
- Primary completionOct 2019 (estimated)
- Study completionOct 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Progressive Multiple SclerosisStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- North Bristol NHS Trust
- Sponsor type
- University, hospital or other
- Collaborators
- Bristol Royal Hospital for Children, University of Bristol, University of Nottingham, NHS Blood and Transplant, Kenneth and Claudia Silverman Family Foundation, Multiple Sclerosis Trust, Medical Research Council, Rosetrees Trust, Catholic Bishops of England and Wales, Friends of Frenchay
- Responsible party
- Sponsor
- Investigators
- Neil J Scolding, FRCP PhD, Principal investigator, University of Bristol & North Bristol NHS Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01815632
- Sponsor's protocol ID
- ACTiMuS
- Other IDs
- ISRCTN27232902
- Acronym
- ACTiMuS
- Status last verified
- Jun 2018
- First submitted
- 19 Mar 2013
- Last updated
- 27 Jun 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Rice CM, Marks DI, Ben-Shlomo Y, Evangelou N, Morgan PS, Metcalfe C, Walsh P, Kane NM, Guttridge MG, Miflin G, Blackmore S, Sarkar P, Redondo J, Owen D, Cottrell DA, Wilkins A, Scolding NJ. Assessment of bone marrow-derived Cellular Therapy in progressive Multiple Sclerosis (ACTiMuS): study protocol for a randomised controlled trial. Trials. 2015 Oct 14;16:463. doi: 10.1186/s13063-015-0953-1.
PubMed 26467901
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Bone Marrow-derived Cellular Therapy in Progressive Multiple Sclerosis (ACTiMuS)
- ClinicalTrials.gov ID
- NCT01815632
- Acronym / sponsor ID
- ACTiMuS / ACTiMuS
- Conditions
- Progressive Multiple Sclerosis
- Keywords
- multiple sclerosis, progressive MS, stem cell, cell therapy, neurodegeneration, CNS repair
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- North Bristol NHS TrustUniversity, hospital or other
- Collaborators
- Bristol Royal Hospital for Children, University of Bristol, University of Nottingham, NHS Blood and Transplant, Kenneth and Claudia Silverman Family Foundation, Multiple Sclerosis Trust, Medical Research Council, Rosetrees Trust, Catholic Bishops of England and Wales, Friends of Frenchay
- Study start
- Jan 2014
- Primary completion
- Oct 2019 (estimated)
- Study completion
- Oct 2019 (estimated)
- First posted
- 21 Mar 2013 (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.
Multiple sclerosis - MS - affects 1.3m people worldwide, costing the European Union economy €9 billion/year, through both direct and indirect consequences of progressive disability. Despite the usual relapsing-remitting presentation, over 80% of patients develop progressive disability; 40% require a wheelchair within 10 years of diagnosis. At present, there are no treatments that reverse, halt or even slow progressive disability in MS.
The investigators recently completed one of the first feasibility/safety trials in the world of reparative bone marrow cell therapy in 6 patients with longstanding MS (www.nature.com/clpt/journal/v87/n6/full/clpt201044a.html). Safety was confirmed, and intensive repeated tests on the patients measuring nerve conduction in various pathways in the brain and in the spinal cord showed statistically significant improvements at 12 months in every patient. While highly preliminary and involving only a very small number of patients, these results at least raise the possibility of a significant (though very partial) underlying repair effect within the damaged nervous system.
The investigators believe this urgently requires further testing - both to accelerate benefit for patients, and to begin improving therapeutic efficacy. The investigators therefore propose a programme of translational and clinical stem cell research, aiming (1) to continue translation with a phase two controlled trial of bone marrow cells in patients with longstanding MS; and (2) to explore in parallel the potential mechanisms of action, by studying bone marrow cells from treated patients and control subjects, aiming to establish which of the various relevant bone marrow subpopulations contribute to efficacy, and which particular reparative mechanism(s) are important. The investigators hope these studies will not only confirm the therapeutic benefit of this approach, but also provide the basis for improving the magnitude and impact of this novel and exciting treatment modality.
Detailed description
The primary objective is to determine whether autologous bone marrow (BM) (ie taken from the patients themselves rather than from a BM donor) cell therapy is truly beneficial in chronic multiple sclerosis - as our small, uncontrolled phase 1 trial suggested (www.nature.com/clpt/journal/v87/n6/full/clpt201044a.html).
The investigators also aim to answer the following questions:
- Do BM mesenchymal stem cells from patients with MS differ in the range or extent of reparative and neuroprotective properties from those from control individuals?
- What reparative and neuroprotective properties do BM stem cell subpopulations other than mesenchymal stem cells possess, and do these differ between MS patients and controls?
- Can BM stem cell subpopulations be isolated from blood samples from MS patients following treatment, and, if so, for how long?
Bone marrow contains stem cells capable of replacing all the cells in the blood. Recently, bone marrow has been found to contain cells capable of replacing cells in tissues and organs other than blood. In addition, infusion of bone marrow-derived cells has been shown to have significant effects on the immune system and to promote the survival of central nervous system cells under toxic conditions. These properties are of considerable interest to those working to develop cell-based therapies for neurodegenerative disease.
The potential of such cells to aid repair in multiple sclerosis (MS) has been examined in experimental models of MS. Myelin is the substance that insulates neurons within the central nervous system and is attacked in MS. Infusion of adult bone marrow cells into a vein aids myelin repair (remyelination) in these models of MS. Recently, the investigators performed a small pilot study to examine the effects of harvesting bone marrow from MS patients and infusing this back into each patient's vein. This was well tolerated and no significant adverse events were encountered. Electrophysiological studies appeared to show some improvement but, given the small numbers of participants in the phase 1 trial, no definitive conclusion can be made regarding this.
The current study seeks to address the question of whether genuine improvements in neurophysiological tests are seen in MS patients with progressive disease following infusion of bone marrow.
A prospective, randomised, double-blind, placebo-controlled, stepped wedge design will be employed at a single centre (Bristol, UK). Eighty patients with progressive MS will be recruited; 60 will have secondary progressive disease (SPMS) but a subset (n=20) will have primary progressive disease (PPMS). Participants will be randomised to either early (immediate) or late (1 year) intravenous infusion of autologous, unfractionated bone marrow. The placebo intervention is infusion of autologous blood. The primary outcome measure is global evoked potential derived from multimodal evoked potentials. Secondary outcome measures include adverse event reporting, clinical (EDSS and MSFC) and self-assessment (MSIS-29) rating scales, optical coherence tomography (OCT) as well as brain and spine MRI. Participants will be followed up for a further year following the final intervention. Outcomes will be analyzed on an intention-to-treat basis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Early infusion of autologous marrow | Experimental | Other: Early infusion of autologous marrowIntravenous infusion of autologous bone marrow (without myeloablation) on the day of bone marrow harvest. Infusion of autologous blood at one year post-harvest |
| Late infusion of autologous marrow | Experimental | Other: Late infusion of autologous marrowIntravenous infusion of autologous blood on the day of bone marrow harvest. Infusion of autologous bone marrow (without myeloablation) at one year post-harvest |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of clinically-definite MS as defined by the McDonald criteria
- Aged 18 - 65 years.
- EDSS of 4.0 to 6 inclusive
- Disease duration >5 years
- Disease progression (increase in physical disability, not due to major relapse) in preceding year
Key exclusion criteria (20 listed)
- Pregnancy, breastfeeding or lactation
- History of autologous/allogenic bone marrow transplantation or peripheral blood stem cell transplant
- Bone marrow insufficiency
- History of lymphoproliferative disease or previous total lymphoid irradiation
- Immune deficiency
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Early infusion of autologous marrowIntravenous infusion of autologous bone marrow without prior myeloablation on the day of bone marrow harvest and infusion of autologous blood at one year post-bone marrow harvest | Other | Early infusion of autologous marrow |
| Late infusion of autologous marrowInfusion of blood on day of bone marrow harvest with intravenous infusion of autologous bone marrow without prior myeloablation one year post-bone marrow harvest. | Other | Late infusion of autologous marrow |
Primary outcome
Global evoked potential (GEP): mean change from time of marrow infusion to end of study
Time frame: Entry and every 6 months for 2 years
Multimodal evoked potentials will be examined at 0, 6, 12, 18 and 24 months. Evoked potential abnormalities will be quantified according to a 4-point graded ordinal score modified from Leocani et al (JNNP 2006, 77:1030-1035) (0=normal; 1=increased latency; 2=increased latency and abnormal amplitude; 3=absent). The recording of the evoked potentials shall be in accordance with the Guidelines of the International Federation of Clinical Neurophysiology and analysis will be performed using standard methods. Electrophysiological responses shall be considered abnormal if they exceed 2.5 standard deviations of the normal values or cannot be detected.
6 secondary outcomes
Safety
Time frame: Continuous throughout study period (2 years)
Expanded disability status scale
Time frame: At entry then 6 weeks, 6 months and 1 year after each infusion
Multiple sclerosis impact scale (MSIS-29)
Time frame: At entry then 6 weeks, 6 months and 1 year after each infusion
Multiple sclerosis functional composite (MSFC)
Time frame: At entry then 6 weeks, 6 months and 1 year after each infusion
MRI head and cord
Time frame: At entry, 1 year and 2 years post-harvest
Optical coherence tomography
Time frame: Entry, 1 year and 2 years post-harvest
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jun 2018.
- Derived from this study: PubMed 26467901Rice CM, Marks DI, Ben-Shlomo Y, Evangelou N, Morgan PS, Metcalfe C, Walsh P, Kane NM, Guttridge MG, Miflin G, Blackmore S, Sarkar P, Redondo J, Owen D, Cottrell DA, Wilkins A, Scolding NJ. Assessment of bone marrow-derived Cellular Therapy in progressive Multiple Sclerosis (ACTiMuS): study protocol for a randomised controlled trial. Trials. 2015 Oct 14;16:463. doi: 10.1186/s13063-015-0953-1.
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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.
Neil J Scolding, FRCP PhD principal investigator
University of Bristol & North Bristol NHS Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Early infusion of autologous marrow and Late infusion of autologous marrow in people with Progressive Multiple Sclerosis, run by North Bristol NHS Trust at 1 site in United Kingdom.
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 20 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: status not confirmed, as of the sponsor's last update on 27 Jun 2018. 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 Kingdom. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Claire M Rice, MA MRCP PhD (00 44 117 4146695, claire.rice@nbt.nhs.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Global evoked potential (GEP): mean change from time of marrow infusion to end of study (time frame: Entry and every 6 months for 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01815632, which also lists 1 linked publication.
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 27 Jun 2018.