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.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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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

Other trials in United States

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)

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 groups in NCT00684255
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.

Aug 2007Jul 2008

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)

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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Systemic Lupus Erythematosus trials

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.

Detailed description

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

Results posted to the registry on 2 Dec 2009, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Reduced Intensity Regimen SSc	1	1

Primary outcome: Toxicity

Time frame: 1 year

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: 1 year. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Reduced Intensity Regimen SSc	0 of 1 (0%)	0 of 1 (0%)	Not reported

Limitations and caveats (from the sponsor): Study closed due to low accrual. Did not meet enrollment goals.

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.

Related trials for Systemic Lupus Erythematosus

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An Ascending Dose Study of DKB903 in Participants With Rheumatoid Arthritis (RA) and Participants With Systemic Lupus Erythematosus (SLE)
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RecruitingPhase 4
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RecruitingObservational study
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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?

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?

On the ClinicalTrials.gov record for NCT00684255.

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.