Multiple SclerosisPhase 2NCT00040482
High Dose Chemo/Radiotherapy and Hematopoietic Stem Cell Transplant for Patients With Multiple Sclerosis
A Phase 2 study testing Cyclophosphamide, ATG and MESNA in people with Multiple Sclerosis, run by Baylor College of Medicine at 1 site in United States.
Trial summary
Multiple Sclerosis is a disease that may be caused by the immune system reacting against the nervous system. It is possible, that by changing the immune system we can modify the progression of this disease. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- Cyclophosphamide, ATG and MESNA
- Type of treatment
- Surgery or procedure
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 1999to Aug 2005
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- The diagnosis of primary or secondary progressive multiple sclerosis or relapsing-remitting multiple sclerosis. Patients with relapsing/remitting MS should demonstrate sustained accumulated disability based upon accepted standard. Relapsing/remitting MS is characterized by unpredictable recurrent attacks of neurologic dysfunction followed by complete, partial or no recovery.
- By the Expanded Disability Status Scale (EDSS) the measure of disability equals or exceeds 5.0 but does not exceed 7.5.
- For primary or secondary progressive MS, the progression of disease using EDSS criteria during the proceeding 12 months equals or exceeds 1.0.
- Symptoms and manifestations of MS activity have not responded to conventional treatment, e.g. high dose prednisone, beta interferon, conventional dose cytoxan, glatiramer acetate etc.
- The patient is able to undergo collection of sufficient numbers of HSCs to meet protocol requirements, i.e. 3X10e6 CD34+ cells.
- Males and females <60 years old.
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- Patient must have a life expectance of >6 weeks.
- Patients should not have received prior lymphoid irradiation.
- Patients should not have a history of hypersensitivity to murine proteins or E.coli derived proteins.
- Patients should not have a history of lack of compliance with medical care or any medical or psychiatric conditions which would compromise their ability to comply with the protocol.
- Patients should not have evidence of myelodysplasia or an active malignancy.
- Patients should not be receiving concurrent beta interferon therapy or high dose prednisone.
- The patient does not exhibit significant organ toxicity from any cause. Significant organ toxicity includes:- Creatinine greater than twice normal; glomerular filtration rate less than 40 ml/min. - FEV1 or FVC less than 75% predicted; DLCO less than 50% predicted.- Significant cardiac dysfunction defined as life endangering arrythmias or a shortened ejection fraction(less than 26%).- Hepatic transaminases greater than two times normal; total bilirubin greater than 3.0 mg/dl.
- The patients are able to give informed consent.
- Both male and female patients must agree to use effective contraception for a minimum of 12 months post transplant. Effective contraception includes total abstinence, oral contraceptives, an intrauterine device, contraceptive implants under the skin (Norplants), contraceptive injections, or contraceptive foam with a condom. In addition, the male partner should use a condom.
Who cannot join
- Active infection.
- Unable to tolerate an MRI.
- HIV positive patient.
- Pregnant and lactating women
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Intensive Immunosuppression Followed by Rescue With CD34 Selected, T Cell Depleted, Leukopheresis Products in Patients With Multiple Sclerosis
Multiple Sclerosis is a disease that may be caused by the immune system reacting against the nervous system. It is possible, that by changing the immune system we can modify the progression of this disease. In this study, we will try to learn whether treatment with a bone marrow transplant (BMT) can help patients with multiple sclerosis. We will also try to learn what the side effects are of this treatment in patients with multiple sclerosis.
Read the detailed description
To participate in this study, patients will need to have a central line (a thin plastic catheter or tube that is placed during surgery into one of the large veins in the neck or chest). Central lines are used to give intravenous medications or to draw blood. Before the transplant we will test the patients blood for viruses which can cause problems after the transplant. These viruses include Hepatitis B, which causes liver damage, cytomegalovirus, which causes lung disease and HIV which causes AIDS. If a patient is positive for the AIDS virus, they will not be able to undertake the transplant. In addition to these blood tests patients will also have an MRI (where pictures are created using magnetic rather than x-ray energy) of the brain and other evaluations that are standard for any patient before having a transplant.
Before the transplant patients will receive daily G-CSF (Neupogen). This medicine will help to stimulate the production of white blood cells (WBC) that will be used for the bone marrow transplant. In addition, 6 tablespoons of blood will be collected to look at how the immune system is functioning before and after transplant. After the white blood cells have reached a certain level, patients will undergo leukapheresis. Leukapheresis is a procedure where blood is removed from a patients arm, pumped into a machine where the white blood cells are separated from most of the other cells, then returned to the patient through the same needle or through a needle in the other arm. After collection of the white blood cells, we will use a device in the lab to select out certain types of white blood cells (CD34+).
After leukapheresis, patients will receive an antibody from horses called Atgam (ATG) to help destroy the immune system and also a drug called cyclophosphamide. After this, radiation treatment will be given to the entire body. This will be done 2 times a day for 3 days. This treatment will kill most of the blood-forming cells in the bone marrow. We will then give the CD34+ white blood cells that were collected during leukapheresis.
Blood will be collected for immune reconstitution studies monthly for 3 months and at 6, 9, and 12 months after transplant to look at how the immune system is functioning. The amount of blood taken will be no more than 90ml (6 tablespoons). After the first year of treatment patients will continue to have 90ml (6 tablespoons) of blood taken for immune reconstitution studies every 6 months for 2 more years.
- Conditions
- Multiple Sclerosis
- Being tested
- Cyclophosphamide, ATG, MESNA, Radiation therapy
- Medical subject headings
- Multiple Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
ATGDrug
MESNADrug
Radiation therapyProcedure or surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2002 (estimated)
- Study startApr 1999
- Study completionAug 2005
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 Baylor College of MedicineThe 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
- Responsible party
- Principal investigator, Robert Krance, Baylor College of Medicine
- Investigators
- Malcolm K. Brenner, MD, Study chair, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00040482
- Sponsor's protocol ID
- H7156
- Other IDs
- Multiple Sclerosis
- Status last verified
- Jan 2020
- First submitted
- 26 Jun 2002
- Last updated
- 18 Jan 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intensive Immunosuppression Followed by Rescue With CD34 Selected, T Cell Depleted, Leukopheresis Products in Patients With Multiple Sclerosis
- ClinicalTrials.gov ID
- NCT00040482
- Acronym / sponsor ID
- H7156
- Conditions
- Multiple Sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
- Study start
- Apr 1999
- Study completion
- Aug 2005
- First posted
- 28 Jun 2002 (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 is a disease that may be caused by the immune system reacting against the nervous system. It is possible, that by changing the immune system we can modify the progression of this disease. In this study, we will try to learn whether treatment with a bone marrow transplant (BMT) can help patients with multiple sclerosis. We will also try to learn what the side effects are of this treatment in patients with multiple sclerosis.
Detailed description
To participate in this study, patients will need to have a central line (a thin plastic catheter or tube that is placed during surgery into one of the large veins in the neck or chest). Central lines are used to give intravenous medications or to draw blood. Before the transplant we will test the patients blood for viruses which can cause problems after the transplant. These viruses include Hepatitis B, which causes liver damage, cytomegalovirus, which causes lung disease and HIV which causes AIDS. If a patient is positive for the AIDS virus, they will not be able to undertake the transplant. In addition to these blood tests patients will also have an MRI (where pictures are created using magnetic rather than x-ray energy) of the brain and other evaluations that are standard for any patient before having a transplant.
Before the transplant patients will receive daily G-CSF (Neupogen). This medicine will help to stimulate the production of white blood cells (WBC) that will be used for the bone marrow transplant. In addition, 6 tablespoons of blood will be collected to look at how the immune system is functioning before and after transplant. After the white blood cells have reached a certain level, patients will undergo leukapheresis. Leukapheresis is a procedure where blood is removed from a patients arm, pumped into a machine where the white blood cells are separated from most of the other cells, then returned to the patient through the same needle or through a needle in the other arm. After collection of the white blood cells, we will use a device in the lab to select out certain types of white blood cells (CD34+).
After leukapheresis, patients will receive an antibody from horses called Atgam (ATG) to help destroy the immune system and also a drug called cyclophosphamide. After this, radiation treatment will be given to the entire body. This will be done 2 times a day for 3 days. This treatment will kill most of the blood-forming cells in the bone marrow. We will then give the CD34+ white blood cells that were collected during leukapheresis.
Blood will be collected for immune reconstitution studies monthly for 3 months and at 6, 9, and 12 months after transplant to look at how the immune system is functioning. The amount of blood taken will be no more than 90ml (6 tablespoons). After the first year of treatment patients will continue to have 90ml (6 tablespoons) of blood taken for immune reconstitution studies every 6 months for 2 more years.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (15 listed)
- The diagnosis of primary or secondary progressive multiple sclerosis or relapsing-remitting multiple sclerosis. Patients with relapsing/remitting MS should demonstrate sustained accumulated disability based upon accepted standard. Relapsing/remitting MS is characterized by unpredictable recurrent attacks of neurologic dysfunction followed by complete, partial or no recovery.
- By the Expanded Disability Status Scale (EDSS) the measure of disability equals or exceeds 5.0 but does not exceed 7.5.
- For primary or secondary progressive MS, the progression of disease using EDSS criteria during the proceeding 12 months equals or exceeds 1.0.
- Symptoms and manifestations of MS activity have not responded to conventional treatment, e.g. high dose prednisone, beta interferon, conventional dose cytoxan, glatiramer acetate etc.
- The patient is able to undergo collection of sufficient numbers of HSCs to meet protocol requirements, i.e. 3X10e6 CD34+ cells.
Key exclusion criteria (4 listed)
- Active infection.
- Unable to tolerate an MRI.
- HIV positive patient.
- Pregnant and lactating women
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 |
|---|---|---|
| Cyclophosphamide | Drug | – |
| ATG | Drug | – |
| MESNA | Drug | – |
| Radiation therapy | Procedure or surgery | – |
Primary outcome
Not reported in the source record.
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 18 Jan 2020.
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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.
Malcolm K. Brenner, MD study chair
Baylor College of Medicine
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 Cyclophosphamide, ATG and MESNA in people with Multiple Sclerosis, run by Baylor College of Medicine 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 15 inclusion and 4 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 18 Jan 2020. 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.
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?
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.
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 18 Jan 2020.