Multiple SclerosisNCT02529839
Safety and Efficacy of an Immunoablative Nonmyeloablative Conditioning Protocol for Autologous Bone Marrow Transplantation (BMT) in Patients With Multiple Sclerosis (MS)
A study testing Autologous bone marrow transplantation, Fludarabine and Cyclophosphamide in people with Multiple Sclerosis, run by Hadassah Medical Organization.
Trial summary
The purpose of this study is to evaluate the safety and efficacy of an immunoablative nonmyeloablative conditioning protocol for autologous bone marrow transplantation in patients with Multiple Sclerosis. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- Autologous bone marrow transplantation, Fludarabine and Cyclophosphamide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- Oct 2015to Oct 2017
- Phase
- Interventional
- 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
- Consenting patients fulfilling the Poser's clinical criteria for definite MS
- Age: 18-65, males and females
- Relapsing and secondary progressive forms of MS with evidence of significant activity of MS (clinical and on the MRI).
- EDSS score of 2.0 to 7.0 (see table 1).
- Failure to at least one line of the currently available treatment, registered treatments (i.e. interferons, Copaxone, Tysabri, Gilenya, Tecfidera, immunosuppression) for MS. The lack of response to these treatments will be determined/defined by either an increase (deterioration) of one degree (or more) in the EDSS score, when baseline EDSS is less than 5.0 or 0.5 degree, when baseline EDSS is 5.0 or more, during the last year or the appearance of one major relapse of MS during the same period of time (under treatment), or evidence for new activity of MS (new T2 lesions or gadolinium enhancing lesions) during the last 12 months.
- Duration of disease: >2 years, except cases with rapid progression, i.e. annual relapse rate ≥2 per 2 years on a conventional treatment or malignant multiple sclerosis with very intense symptoms (types is in most cases deadly).
Who cannot join
- Patients suffering from significant cardiac, renal or hepatic failure or any other disease that may risk the patient or interfere with the ability to undergo high dose immunosuppression associated toxicities (according to the existing limitations for autologous transplantation).
- Patients with active infections.
- Patients with severe cognitive decline or inability to understand and sign the informed consent.
- Patients who were treated with investigational protocols during the last 3 months prior to the inclusion.
- Patients who received high dose immunosuppression with autologous stem cell rescue in the past with no effect.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Safety and Efficacy of an Immunoablative Nonmyeloablative Conditioning Protocol for Autologous Bone Marrow Transplantation in Patients With Multiple Sclerosis
The purpose of this study is to evaluate the safety and efficacy of an immunoablative nonmyeloablative conditioning protocol for autologous bone marrow transplantation in patients with Multiple Sclerosis. Patients meeting inclusion and exclusion criteria will start an immunoablative nonmyeloablative conditioning regimen followed by autologous bone marrow transplantation. Patients will be followed for one year by a neurologist to evaluate the course of the disease after treatment.
- Conditions
- Multiple Sclerosis
- Being tested
- Autologous bone marrow transplantation, Fludarabine, Cyclophosphamide, Alemtuzumab
- Keywords
- MS, BMT
- Medical subject headings
- Multiple Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous bone marrow transplantationProcedure or surgery
IMMUNOABLATIVE NONMYELOABLATIVE AUTOLOGOUS BONE MARROW TRANSPLANTATION on Day 0
FludarabineDrug
30mg/m2 on days -6 through -3
CyclophosphamideDrug
50mg/kg on days -5 through -4
AlemtuzumabDrug
3mg on day -3, 9mg on day -2, 12 mg on day -1
| Study group | Role | What they receive |
|---|---|---|
| Experimental | Experimental | Fludarabine 30mg/m2 for 4 days, Cyclophosphamide 50mg/kg for 2 days, Alemtuzumab administered subcutaneously 24mg total dose.
Autologous bone marrow transplantation Receives: Procedure: Autologous bone marrow transplantation; Drug: Fludarabine; Drug: Cyclophosphamide; Drug: Alemtuzumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment parameters of Neutrophils and Platelets
Absolute Neutrophil count >500 /microliter, Platelets>20,000/microliter
Measured: 1 year
Transplant related mortality by Day 100
Measured: Day 100
Secondary outcomes (5)
Changes in the Expanded Disability Status Scale (EDSS score, as compared to baseline)
Measured: 1 year
Overall survival (OS) at 1 year
Measured: 1 year
Progression-free survival (PFS) at 1 year
Measured: 1 year
Changes in MRI activity
T2 number
Measured: 1 year
Changes in MRI activity
Volume of gadolinium enhancing lesions
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2015 (estimated)
- Study startOct 2015
- Primary completionOct 2017 (estimated)
- Study completionOct 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple SclerosisStudies of the same condition, open and under way.
- More trials run by Hadassah Medical OrganizationThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hadassah Medical Organization
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Igor Resnick, Prof., Principal investigator, Bone Marrow Transplantation, Cancer Immunotherapy & Immunobiology Research Center, Hadassah University Hospital, Ein Kerem, Jerusalem, Israel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02529839
- Sponsor's protocol ID
- 0105-15-HMO-CTIL
- Status last verified
- Apr 2015
- First submitted
- 10 Jun 2015
- Last updated
- 20 Aug 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of an Immunoablative Nonmyeloablative Conditioning Protocol for Autologous Bone Marrow Transplantation in Patients With Multiple Sclerosis
- ClinicalTrials.gov ID
- NCT02529839
- Acronym / sponsor ID
- 0105-15-HMO-CTIL
- Conditions
- Multiple Sclerosis
- Keywords
- MS, BMT
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Hadassah Medical OrganizationUniversity, hospital or other
- Study start
- Oct 2015
- Primary completion
- Oct 2017 (estimated)
- Study completion
- Oct 2017 (estimated)
- First posted
- 20 Aug 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.
The purpose of this study is to evaluate the safety and efficacy of an immunoablative nonmyeloablative conditioning protocol for autologous bone marrow transplantation in patients with Multiple Sclerosis. Patients meeting inclusion and exclusion criteria will start an immunoablative nonmyeloablative conditioning regimen followed by autologous bone marrow transplantation. Patients will be followed for one year by a neurologist to evaluate the course of the disease after treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental | Experimental | Procedure: Autologous bone marrow transplantation, Drug: Fludarabine, Drug: Cyclophosphamide, Drug: AlemtuzumabFludarabine 30mg/m2 for 4 days, Cyclophosphamide 50mg/kg for 2 days, Alemtuzumab administered subcutaneously 24mg total dose. Autologous bone marrow transplantation |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Consenting patients fulfilling the Poser's clinical criteria for definite MS
- Age: 18-65, males and females
- Relapsing and secondary progressive forms of MS with evidence of significant activity of MS (clinical and on the MRI).
- EDSS score of 2.0 to 7.0 (see table 1).
- Failure to at least one line of the currently available treatment, registered treatments (i.e. interferons, Copaxone, Tysabri, Gilenya, Tecfidera, immunosuppression) for MS. The lack of response to these treatments will be determined/defined by either an increase (deterioration) of one degree (or more) in the EDSS score, when baseline EDSS is less than 5.0 or 0.5 degree, when baseline EDSS is 5.0 or more, during the last year or the appearance of one major relapse of MS during the same period of time (under treatment), or evidence for new activity of MS (new T2 lesions or gadolinium enhancing lesions) during the last 12 months.
Key exclusion criteria (5 listed)
- Patients suffering from significant cardiac, renal or hepatic failure or any other disease that may risk the patient or interfere with the ability to undergo high dose immunosuppression associated toxicities (according to the existing limitations for autologous transplantation).
- Patients with active infections.
- Patients with severe cognitive decline or inability to understand and sign the informed consent.
- Patients who were treated with investigational protocols during the last 3 months prior to the inclusion.
- Patients who received high dose immunosuppression with autologous stem cell rescue in the past with no effect.
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 |
|---|---|---|
| Autologous bone marrow transplantationIMMUNOABLATIVE NONMYELOABLATIVE AUTOLOGOUS BONE MARROW TRANSPLANTATION on Day 0 | Procedure or surgery | Experimental |
| Fludarabine30mg/m2 on days -6 through -3 | Drug | Experimental |
| Cyclophosphamide50mg/kg on days -5 through -4 | Drug | Experimental |
| Alemtuzumab3mg on day -3, 9mg on day -2, 12 mg on day -1 | Drug | Experimental |
Primary outcome
Engraftment parameters of Neutrophils and Platelets
Time frame: 1 year
Absolute Neutrophil count >500 /microliter, Platelets>20,000/microliter
Transplant related mortality by Day 100
Time frame: Day 100
5 secondary outcomes
Changes in the Expanded Disability Status Scale (EDSS score, as compared to baseline)
Time frame: 1 year
Overall survival (OS) at 1 year
Time frame: 1 year
Progression-free survival (PFS) at 1 year
Time frame: 1 year
Changes in MRI activity
Time frame: 1 year
Changes in MRI activity
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2015.
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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.
Igor Resnick, Prof. principal investigator
Bone Marrow Transplantation, Cancer Immunotherapy & Immunobiology Research Center, Hadassah University Hospital, Ein Kerem, Jerusalem, Israel
Questions about this trial
What is this clinical trial studying?
A study testing Autologous bone marrow transplantation, Fludarabine and Cyclophosphamide in people with Multiple Sclerosis, run by Hadassah Medical Organization.
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 6 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: status not confirmed, as of the sponsor's last update on 20 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Igor Resnick, Prof. (972-50-787-4663, gashka.resnick@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Engraftment parameters of Neutrophils and Platelets (time frame: 1 year). 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 Aug 2015.