Multiple SclerosisPhase 1NCT00014755
Phase I Pilot Study of Total-Body Irradiation, Anti-Thymocyte Globulin and Cyclophosphamide Followed By Syngeneic or Autologous Peripheral Blood Stem Cell Transplantation in Patients With Multiple Sclerosis
A Phase 1 study testing anti-thymocyte globulin, cyclophosphamide and filgrastim in people with Multiple Sclerosis, run by Fred Hutchinson Cancer Center at 6 sites in United States.
Trial summary
OBJECTIVES: I. Determine the toxicity of total-body irradiation, anti-thymocyte globulin, and cyclophosphamide followed by syngeneic or autologous peripheral blood stem cell (PBSC) transplantation in patients with multiple sclerosis. II. Determine the disease response of patients treated with this regimen. III. 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?
- anti-thymocyte globulin, cyclophosphamide and filgrastim
- Type of treatment
- Surgery or procedure
- How many people
- 35 participants
- Where
- 6 sitesUnited States
- Study dates
- Dec 1997to not given
- Phase
- Phase 1
- 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
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of rapidly progressive multiple sclerosis (MS) by Proser criteria and at high risk for a fatal outcome or severe disability with one of the following:
- Primary progressive disease
- Relapsing/remitting disease with 2 or more attacks in 2 years
- Secondary progressive disease
Extended disability status scale (EDSS) between 5.0 and 8.0 with deterioration in the EDSS of 1 or more points over the past year
More than 60 days since relapse of MS
No evidence of myelodysplasia
Sibling donor proven to be an identical twin by ABO typing, HLA typing, and VNTR analysis (for syngeneic transplantation)
--Prior/Concurrent Therapy--
Radiotherapy: No prior total-lymphoid irradiation
Other: No other concurrent investigational agents
--Patient Characteristics-- Hepatic: No hepatic impairment that would preclude high-dose immunosuppressive therapy
Renal: No renal impairment that would preclude high-dose immunosuppressive therapy
Cardiovascular: No cardiac impairment that would preclude high-dose immunosuppressive therapy
Pulmonary: No pulmonary impairment that would preclude high-dose immunosuppressive therapy
Other:
- No neurologic impairment that would preclude high-dose immunosuppressive therapy
- No active uncontrolled infection
- No active malignancy
- No other illness that would severely limit life expectancy
- No medical or psychiatric conditions that would preclude study
- No history of hypersensitivity to murine proteins or E. coli-derived proteins
- No demonstrated lack of compliance with prior medical care
- Able to undergo an MRI scan
- HIV negative
- Not pregnant or nursing
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
University of Colorado Cancer Center
Denver, Colorado, 80262
City of Hope National Medical Center
Duarte, California, 91010
Duke University Medical Center
Durham, North Carolina, 27710
University of Nebraska Medical Center
Omaha, Nebraska, 68198-3330
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Washington University Barnard Cancer Center
St Louis, Missouri, 63110
About this study
OBJECTIVES: I. Determine the toxicity of total-body irradiation, anti-thymocyte globulin, and cyclophosphamide followed by syngeneic or autologous peripheral blood stem cell (PBSC) transplantation in patients with multiple sclerosis.
II. Determine the disease response of patients treated with this regimen. III. Determine the safety and efficacy of filgrastim (G-CSF) for PBSC mobilization in this patient population.
Read the detailed description
PROTOCOL OUTLINE: This is a multicenter study. Patients receive oral prednisone on days 0-10. Beginning on day 1, patients undergoing autologous peripheral blood stem cell (PBSC) transplantation receive filgrastim (G-CSF) subcutaneously daily until leukapheresis is completed. Leukapheresis begins on approximately day 4 and continues until adequate CD34+ PBSC are collected.
PBSC are collected from syngeneic donors in a similar manner. Patients undergo total-body irradiation twice daily on days -5 and -4. Patients receive cyclophosphamide IV on days -3 and -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. Patients undergo autologous or syngeneic PBSC transplantation on day 0. Following PBSC transplantation, patients receive oral prednisone on days 7-30 and G-CSF IV daily beginning on day 0 and continuing until blood counts recover.
Patients are followed at 30, 80, and 90 days, monthly for 6 months, and then at 1 and 2 years.
- Conditions
- Multiple Sclerosis
- Being tested
- anti-thymocyte globulin, cyclophosphamide, filgrastim, prednisone, peripheral blood stem cell transplantation
- Keywords
- multiple sclerosis, neurologic and psychiatric disorders, rare disease
- Medical subject headings
- Multiple Sclerosis, Neurologic Manifestations, Mental Disorders, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinDrug
cyclophosphamideDrug
filgrastimDrug
prednisoneDrug
peripheral blood stem cell transplantationProcedure or surgery
irradiationProcedure or surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2001 (estimated)
- Study startDec 1997
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 Multiple SclerosisStudies of the same condition, open and under way.
- More trials run by Fred Hutchinson Cancer CenterThe 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 1
- Primary purpose
- Treatment
- Enrolment
- 35 participants
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Investigators
- Richard Nash, Study chair, Fred Hutchinson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00014755
- Sponsor's protocol ID
- 199/15796
- Other IDs
- FHCRC-1164.00
- Status last verified
- Apr 2001
- First submitted
- 10 Apr 2001
- Last updated
- 24 Jun 2005
- Results posted
- No
Publications
Bowen JD, Kraft GH, Wundes A, Guan Q, Maravilla KR, Gooley TA, McSweeney PA, Pavletic SZ, Openshaw H, Storb R, Wener M, McLaughlin BA, Henstorf GR, Nash RA. Autologous hematopoietic cell transplantation following high-dose immunosuppressive therapy for advanced multiple sclerosis: long-term results. Bone Marrow Transplant. 2012 Jul;47(7):946-51. doi: 10.1038/bmt.2011.208. Epub 2011 Nov 7.
PubMed 22056644
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Pilot Study of Total-Body Irradiation, Anti-Thymocyte Globulin and Cyclophosphamide Followed By Syngeneic or Autologous Peripheral Blood Stem Cell Transplantation in Patients With Multiple Sclerosis
- ClinicalTrials.gov ID
- NCT00014755
- Acronym / sponsor ID
- 199/15796
- Conditions
- Multiple Sclerosis
- Keywords
- multiple sclerosis, neurologic and psychiatric disorders, rare disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Study start
- Dec 1997
- First posted
- 11 Apr 2001 (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.
OBJECTIVES: I. Determine the toxicity of total-body irradiation, anti-thymocyte globulin, and cyclophosphamide followed by syngeneic or autologous peripheral blood stem cell (PBSC) transplantation in patients with multiple sclerosis.
II. Determine the disease response of patients treated with this regimen. III. Determine the safety and efficacy of filgrastim (G-CSF) for PBSC mobilization in this patient population.
Detailed description
PROTOCOL OUTLINE: This is a multicenter study. Patients receive oral prednisone on days 0-10. Beginning on day 1, patients undergoing autologous peripheral blood stem cell (PBSC) transplantation receive filgrastim (G-CSF) subcutaneously daily until leukapheresis is completed. Leukapheresis begins on approximately day 4 and continues until adequate CD34+ PBSC are collected.
PBSC are collected from syngeneic donors in a similar manner. Patients undergo total-body irradiation twice daily on days -5 and -4. Patients receive cyclophosphamide IV on days -3 and -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. Patients undergo autologous or syngeneic PBSC transplantation on day 0. Following PBSC transplantation, patients receive oral prednisone on days 7-30 and G-CSF IV daily beginning on day 0 and continuing until blood counts recover.
Patients are followed at 30, 80, and 90 days, monthly for 6 months, and then at 1 and 2 years.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
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 |
|---|---|---|
| anti-thymocyte globulin | Drug | – |
| cyclophosphamide | Drug | – |
| filgrastim | Drug | – |
| prednisone | Drug | – |
| peripheral blood stem cell transplantation | Procedure or surgery | – |
| irradiation | 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 24 Jun 2005.
- Derived from this study: PubMed 22056644Bowen JD, Kraft GH, Wundes A, Guan Q, Maravilla KR, Gooley TA, McSweeney PA, Pavletic SZ, Openshaw H, Storb R, Wener M, McLaughlin BA, Henstorf GR, Nash RA. Autologous hematopoietic cell transplantation following high-dose immunosuppressive therapy for advanced multiple sclerosis: long-term results. Bone Marrow Transplant. 2012 Jul;47(7):946-51. doi: 10.1038/bmt.2011.208. Epub 2011 Nov 7.
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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.
Richard Nash study chair
Fred Hutchinson Cancer Center
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing anti-thymocyte globulin, cyclophosphamide and filgrastim in people with Multiple Sclerosis, run by Fred Hutchinson Cancer Center at 6 sites 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?
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 24 Jun 2005. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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 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.
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?
On the ClinicalTrials.gov record for NCT00014755, 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 24 Jun 2005.