Systemic Lupus ErythematosusPhase 1NCT00684255
Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc)
A Phase 1 study testing Reduced Intensity Allogeneic Transplant, Fludarabine and Busulfan in people with Systemic Lupus Erythematosus and Systemic Sclerosis, run by New York Medical College at 1 site in United States.
Trial summary
The purpose of this study is to determine if a reduced intensity (RI) (non-myeloablative) chemoimmunotherapy followed by Allogeneic Stem Cell Transplantation AlloSCT (matched family donors and matched unrelated cord blood donors) will be well tolerated. Read more about this study
- Who is it for?
- Ages 7 to 50 years
- Conditions the study is for
- Systemic Lupus Erythematosus, Systemic Sclerosis
- What is being tested?
- Reduced Intensity Allogeneic Transplant, Fludarabine and Busulfan
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2007to Jul 2008
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 7 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diffuse Systemic Sclerosis and variants as per ACR criteria
- Medically refractory disease
- Adequate Organ Function - Pulmonary function
- Renal function, Cardiac function defined as:
- SGOT (AST) or SGPT (ALT) <5 x upper limit of normal
- Diagnosis of SLE - Medically refractory disease
Who cannot join
- Karnofsky/Lansky <60%
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University Medical Center
New York, New York, 10032
About this study
Official title: Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc)
The purpose of this study is to determine if a reduced intensity (RI) (non-myeloablative) chemoimmunotherapy followed by Allogeneic Stem Cell Transplantation AlloSCT (matched family donors and matched unrelated cord blood donors) will be well tolerated.
Read the detailed description
This is to test whether a reduced intensity will result in a high degree of mixed or complete donor chimerism and stabilization of autoimmune disease in a select group of patients with medically refractory SLE or SSc.
- Conditions
- Systemic Lupus Erythematosus, Systemic Sclerosis
- Being tested
- Reduced Intensity Allogeneic Transplant, Fludarabine, Busulfan, Campath
- Keywords
- Autoimmune Disease, Reduced Intensity Transplant
- Medical subject headings
- Lupus Erythematosus, Systemic, Scleroderma, Systemic, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Reduced Intensity Allogeneic TransplantProcedure or surgery
Eeduced intensity allogeneic stem cell transplantation with a fludarabine/busulfan/alemtuzumab conditioning regimen is anticipated to result in mixed and/or complete donor chimerism and potentially alter the natural history and outcome of patients with medically refractory Systemic Lupus Erythematosus (SLE) or Systemic Sclerosis (SSc).
FludarabineDrug
Fludarabine 30 mg/m2 Day -7, -6, -5, -4, -3, -2
BusulfanDrug
Busulfan 3.2 mg/kg Days _8, -7, -6, -5
CampathDrug
Also called Alemtuzumab
Campath: 2 mg/m2 Day -5; 6 mg/m2 Day -4, -3; 20 mg/m2 Day -2
| Study group | Role | What they receive |
|---|---|---|
| Reduced Intensity Regimen for Refractory SLE | Experimental | RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE). Receives: Procedure: Reduced Intensity Allogeneic Transplant; Drug: Fludarabine; Drug: Busulfan; Drug: Campath |
| Reduced Intensity Regimen for SSc | Experimental | RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc). Receives: Procedure: Reduced Intensity Allogeneic Transplant; Drug: Fludarabine; Drug: Busulfan; Drug: Campath |
What is being measured
The primary outcome is the main question the study is designed to answer.
Toxicity
Toxicity associated with reduced intensity regimen of fludarabine/busulfan and Campath followed by allogeneic stem cell transplant in patients with medically refractory Systemic Lupus Erythematosus (SLE) or SSc is measured.
Measured: 1 year
Secondary outcomes (3)
Chimerism
Percentage(%) of mixed and/or complete donor chimerism has been measured at different time points.
Measured: 1 year
Immune Reconstitution.
Peripheral blood for immune reconstitution for T-cell, B-cell and NK cells to be obtained for measurement of cell.
Measured: 1 year
Progression Free and Overall Survival.
Probability of progression free and overall survival will be measured.
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 posted26 May 2008 (estimated)
- Study startAug 2007
- Primary completionJul 2008
- Study completionJul 2008
Why it stopped: inactive
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Systemic Lupus ErythematosusStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mitchell Cairo, New York Medical College
- Investigators
- Mitchell Cairo, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00684255
- Sponsor's protocol ID
- AAAB1324
- Other IDs
- CHNY-01-511
- Status last verified
- Feb 2014
- First submitted
- 22 May 2008
- Last updated
- 1 Apr 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc)
- ClinicalTrials.gov ID
- NCT00684255
- Acronym / sponsor ID
- AAAB1324
- Conditions
- Systemic Lupus Erythematosus, Systemic Sclerosis
- Keywords
- Autoimmune Disease, Reduced Intensity Transplant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: inactive
- Enrolment
- 1 participants (actual)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Study start
- Aug 2007
- Primary completion
- Jul 2008 (actual)
- Study completion
- Jul 2008 (actual)
- First posted
- 26 May 2008 (estimated)
- Results
- Posted 2 Dec 2009
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 determine if a reduced intensity (RI) (non-myeloablative) chemoimmunotherapy followed by Allogeneic Stem Cell Transplantation AlloSCT (matched family donors and matched unrelated cord blood donors) will be well tolerated.
Detailed description
This is to test whether a reduced intensity will result in a high degree of mixed or complete donor chimerism and stabilization of autoimmune disease in a select group of patients with medically refractory SLE or SSc.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Reduced Intensity Regimen for Refractory SLE | Experimental | Procedure: Reduced Intensity Allogeneic Transplant, Drug: Fludarabine, Drug: Busulfan, Drug: CampathRI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE). |
| Reduced Intensity Regimen for SSc | Experimental | Procedure: Reduced Intensity Allogeneic Transplant, Drug: Fludarabine, Drug: Busulfan, Drug: CampathRI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc). |
Who may qualify
- Ages
- 7 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Diffuse Systemic Sclerosis and variants as per ACR criteria
- Medically refractory disease
- Adequate Organ Function - Pulmonary function
- Renal function, Cardiac function defined as:
- SGOT (AST) or SGPT (ALT) <5 x upper limit of normal
Key exclusion criteria (1 listed)
- Karnofsky/Lansky <60%
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 |
|---|---|---|
| Reduced Intensity Allogeneic TransplantEeduced intensity allogeneic stem cell transplantation with a fludarabine/busulfan/alemtuzumab conditioning regimen is anticipated to result in mixed and/or complete donor chimerism and potentially alter the natural history and outcome of patients with medically refractory Systemic Lupus Erythematosus (SLE) or Systemic Sclerosis (SSc). | Procedure or surgery | Reduced Intensity Regimen for Refractory SLE, Reduced Intensity Regimen for SSc |
| FludarabineFludarabine 30 mg/m2 Day -7, -6, -5, -4, -3, -2 | Drug | Reduced Intensity Regimen for Refractory SLE, Reduced Intensity Regimen for SSc |
| BusulfanBusulfan 3.2 mg/kg Days _8, -7, -6, -5 | Drug | Reduced Intensity Regimen for Refractory SLE, Reduced Intensity Regimen for SSc |
| CampathAlso: AlemtuzumabCampath: 2 mg/m2 Day -5; 6 mg/m2 Day -4, -3; 20 mg/m2 Day -2 | Drug | Reduced Intensity Regimen for Refractory SLE, Reduced Intensity Regimen for SSc |
Primary outcome
Toxicity
Time frame: 1 year
Toxicity associated with reduced intensity regimen of fludarabine/busulfan and Campath followed by allogeneic stem cell transplant in patients with medically refractory Systemic Lupus Erythematosus (SLE) or SSc is measured.
3 secondary outcomes
Chimerism
Time frame: 1 year
Immune Reconstitution.
Time frame: 1 year
Progression Free and Overall Survival.
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- (Click on "Morgan Stanley Children's Hospital" and then "Clinical Services" and then "Blood \& Marrow Transplantation")Link provided by the sponsor.
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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.
Mitchell Cairo, MD principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Reduced Intensity Allogeneic Transplant, Fludarabine and Busulfan in people with Systemic Lupus Erythematosus and Systemic Sclerosis, run by New York Medical College 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 6 inclusion and 1 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: stopped early, as of the sponsor's last update on 1 Apr 2014. 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 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?
Yes. Results are posted in the registry (first posted 2 Dec 2009). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Toxicity (time frame: 1 year). 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 1 Apr 2014.