Multiple SclerosisPhase 1/2NCT00497952
Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis
A Phase 1/2 study testing hematopoetic stem cell infusion in people with Multiple Sclerosis, run by Talaris Therapeutics Inc..
Trial summary
The goal of this research study is to establish chimerism with the goal to halt disease progression in patients with Multiple Sclerosis. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- hematopoetic stem cell infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jul 2007to Dec 2024
- Phase
- Phase 1/2
- 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 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Clinically definite MS according to the McDonald criteria
- Confirmed diagnosis of relapsing-remitting MS.
- Age between 18 and 55 years
- Extended Disability Status Score (EDSS) between 0 and 5.0
- Independently ambulatory (eligible for inclusion if subject was acutely non-ambulatory within the previous year and return of function is substantiated with EDSS score.)
- Relapse within the last year or sustained disability progression of 1.0 for six months
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- Treatment with high dose, high frequency Interferon-β therapy, or failure to tolerate Interferon-β therapy
- Diffusing capacity of the lung for carbon monoxide (DLCO)> 50% (unless cleared by physician)
- Ejection fraction (EF) > 40% (unless cleared by cardiologist)
- Required initial laboratory data (obtained within 30 days prior to transplant, unless otherwise specified)
- HIV-1,2 antigen and antibody negative
- HBsAg negative (chronic hepatitis B carriers without clinical evidence of liver disease can be considered on an individual basis if it is determined that the added risk is justified by the prognosis and lack of treatment alternatives)
- Hepatitis C antibody negative (positive antibody allowed if antigen (RNA)-negative and no clinical evidence of cirrhosis)
- Cytomegalovirus (CMV), hepatitis B, Human T-lymphotropic virus (HTLV)-1,2, Epstein-Barr virus (EBV), and Herpes antibody status known
- Pregnancy test negative (women of childbearing potential only)
- No life-threatening organ dysfunction.
- Uncontrolled or severe cardiovascular disease, including recent (<6 months) myocardial infarction, angina (symptomatic despite optimal medical management), life-threatening arrhythmia or hypertension
- Able to give informed consent
Who cannot join
- Women who are of child bearing potential must have a negative pregnancy test (serum pregnancy test - human chorionic gonadotrophin (HCG)) within 48 hours of initiating total body irradiation and agree to use reliable contraception for 1 year following transplant.
- Concomitant severe diseases (respiratory, renal, liver, cardiac failures, psychiatric disorders, neoplasms)
- Recurrent urinary, pulmonary infections.
- Active bacterial, viral, or fungal infection
- Active peptic ulcer disease
- Previous treatments with total lymphoid irradiation or total body irradiation
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- Interferon-neutralizing antibody positive with a titer greater than 20
- Relapse in the month preceding enrollment
- Poor compliance
About this study
The goal of this research study is to establish chimerism with the goal to halt disease progression in patients with Multiple Sclerosis.
Read the detailed description
While the cause of MS in not known, there is an autoimmune component that destroys nerve cells. Autoimmunity is a condition where an individual's immune system attacks his/her own cells. Bone marrow stem cell transplantation has been shown to halt autoimmunity. Stem cell transplant can be performed using the patient's own cells, or donor cells. The general consensus in the field is that donor transplant is most likely to halt disease progression. This study is designed to evaluate the safety of a donor transplant procedure as a therapy for relapsing remitting multiple sclerosis (RRMS).
Two factors limit the widespread application of traditional donor stem cell transplant: 1) preparing the patient for transplant (conditioning); and 2) graft-versus-host disease (GVHD). Traditional conditioning destroys the recipient's immune system and requires that the marrow transplant be successful because the patient is unable to fight off infection if the donor cells do not survive. GVHD occurs when donor immune cells recognize the recipient's cells as foreign tissue and attack them. Severe GVHD can result in death. This study utilizes a new approach to conditioning which leaves the patient's immune system intact. The transplant product is depleted of GVHD-producing cells but retains tolerance-promoting cells, called facilitating cells, which are intended to ensure the donor and recipient cells coexists peacefully. The toxicity of conditioning and transplantation is significantly reduced. The end result is a marrow system that contains recipient and donor cells, a state called mixed chimerism.
In this study, we will determine the appropriate cell dose to safely establish mixed chimerism following partial conditioning in patients with RRMS. The study takes a gradual approach to increasing the cell dose to achieve mixed chimerism. Each patient will receive a cell dose one unit above the dose received by the most recent safely transplanted patient. We believe this study will provide a breakthrough in the treatment of MS. The goal of this study is to evaluate the potential of safely establishing mixed chimerism to interrupt the autoimmune process and end the devastating effects of MS.
- Conditions
- Multiple Sclerosis
- Being tested
- hematopoetic stem cell infusion
- Keywords
- Relapsing-remitting multiple sclerosis, Marrow/Stem cell transplant
- Medical subject headings
- Multiple Sclerosis, Multiple Sclerosis, Relapsing-Remitting
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hematopoetic stem cell infusionBiological
Enriched hematopoietic stem cell infusion
| Study group | Role | What they receive |
|---|---|---|
| Multiple Sclerosis Patients | Experimental | Recipients treated with a hematopoetic stem cell infusion from a living donor Receives: Biological: hematopoetic stem cell infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Stem cell engraftment
Measured: One month to three years
Secondary outcomes (1)
Disease remission
Measured: 3 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2007 (estimated)
- Study startJul 2007
- Primary completionDec 2024 (estimated)
- Study completionDec 2024 (estimated)
Why it stopped: all clinical development programs terminated by sponsor
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.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Talaris Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Suzanne T Ildstad, MD, Study director, Talaris Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00497952
- Sponsor's protocol ID
- ICT-13080-122206
- Status last verified
- Oct 2023
- First submitted
- 6 Jul 2007
- Last updated
- 12 Oct 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis
- ClinicalTrials.gov ID
- NCT00497952
- Acronym / sponsor ID
- ICT-13080-122206
- Conditions
- Multiple Sclerosis
- Keywords
- Relapsing-remitting multiple sclerosis, Marrow/Stem cell transplant
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: all clinical development programs terminated by sponsor
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Talaris Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- Jul 2007
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 9 Jul 2007 (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 goal of this research study is to establish chimerism with the goal to halt disease progression in patients with Multiple Sclerosis.
Detailed description
While the cause of MS in not known, there is an autoimmune component that destroys nerve cells. Autoimmunity is a condition where an individual's immune system attacks his/her own cells. Bone marrow stem cell transplantation has been shown to halt autoimmunity. Stem cell transplant can be performed using the patient's own cells, or donor cells. The general consensus in the field is that donor transplant is most likely to halt disease progression. This study is designed to evaluate the safety of a donor transplant procedure as a therapy for relapsing remitting multiple sclerosis (RRMS).
Two factors limit the widespread application of traditional donor stem cell transplant: 1) preparing the patient for transplant (conditioning); and 2) graft-versus-host disease (GVHD). Traditional conditioning destroys the recipient's immune system and requires that the marrow transplant be successful because the patient is unable to fight off infection if the donor cells do not survive. GVHD occurs when donor immune cells recognize the recipient's cells as foreign tissue and attack them. Severe GVHD can result in death. This study utilizes a new approach to conditioning which leaves the patient's immune system intact. The transplant product is depleted of GVHD-producing cells but retains tolerance-promoting cells, called facilitating cells, which are intended to ensure the donor and recipient cells coexists peacefully. The toxicity of conditioning and transplantation is significantly reduced. The end result is a marrow system that contains recipient and donor cells, a state called mixed chimerism.
In this study, we will determine the appropriate cell dose to safely establish mixed chimerism following partial conditioning in patients with RRMS. The study takes a gradual approach to increasing the cell dose to achieve mixed chimerism. Each patient will receive a cell dose one unit above the dose received by the most recent safely transplanted patient. We believe this study will provide a breakthrough in the treatment of MS. The goal of this study is to evaluate the potential of safely establishing mixed chimerism to interrupt the autoimmune process and end the devastating effects of MS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Multiple Sclerosis Patients | Experimental | Biological: hematopoetic stem cell infusionRecipients treated with a hematopoetic stem cell infusion from a living donor |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (18 listed)
- Clinically definite MS according to the McDonald criteria
- Confirmed diagnosis of relapsing-remitting MS.
- Age between 18 and 55 years
- Extended Disability Status Score (EDSS) between 0 and 5.0
- Independently ambulatory (eligible for inclusion if subject was acutely non-ambulatory within the previous year and return of function is substantiated with EDSS score.)
Key exclusion criteria (9 listed)
- Women who are of child bearing potential must have a negative pregnancy test (serum pregnancy test - human chorionic gonadotrophin (HCG)) within 48 hours of initiating total body irradiation and agree to use reliable contraception for 1 year following transplant.
- Concomitant severe diseases (respiratory, renal, liver, cardiac failures, psychiatric disorders, neoplasms)
- Recurrent urinary, pulmonary infections.
- Active bacterial, viral, or fungal infection
- Active peptic ulcer disease
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 |
|---|---|---|
| hematopoetic stem cell infusionEnriched hematopoietic stem cell infusion | Biological | Multiple Sclerosis Patients |
Primary outcome
Stem cell engraftment
Time frame: One month to three years
1 secondary outcome
Disease remission
Time frame: 3 Years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2023.
- Individual participant dataSharing planned: Undecided
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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.
Suzanne T Ildstad, MD study director
Talaris Therapeutics Inc.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing hematopoetic stem cell infusion in people with Multiple Sclerosis, run by Talaris Therapeutics Inc..
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 18 inclusion and 9 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: withdrawn, as of the sponsor's last update on 12 Oct 2023. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Stem cell engraftment (time frame: One month to three years). 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 12 Oct 2023.