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Nonmyeloablative Conditioning and Transplantation for Patients With Refractory Systemic Lupus Erythematosus (SLE)

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ClinicalTrials.gov Identifier: NCT02080195
Recruitment Status : Terminated (Study was unable to accrue subjects)
First Posted : March 6, 2014
Results First Posted : October 2, 2018
Last Update Posted : October 2, 2018
Sponsor:
Collaborator:
Maryland Stem Cell Research Fund
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Lupus Erythematosus
Graft-versus-host Disease
Interventions Drug: Cyclophosphamide
Drug: Fludarabine
Drug: Tacrolimus
Drug: Mycophenolate Mofetil
Drug: Rabbit antithymocyte globulin
Radiation: Total body irradiation
Biological: Allogeneic bone marrow transplant
Enrollment 1
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Nonmyeloablative Conditioning and BMT
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Nonmyeloablative Conditioning and Bone Marrow Transplant in Systemic Lupus Erythematosus patients

Cyclophosphamide: Cyclophosphamide (alkylating agent) conditioning regimen and post transplantation cyclophosphamide with matched, partially matched, haplo-identical or unrelated donors

Sodium-2-mercapto ethane sulphonate: Antineoplastic detoxifying agent

Fludarabine monophosphate: Purine antimetabolite

Tacrolimus: Calcineurin inhibitor

Mofetil: Immunosuppressant

Rabbit antithymocyte globulin: Selective immunosuppressant

Period Title: Overall Study
Started 1
Completed 1
Not Completed 0
Arm/Group Title Nonmyeloablative Conditioning and BMT
Hide Arm/Group Description

Nonmyeloablative Conditioning and Bone Marrow Transplant in Systemic Lupus Erythematosus patients

Cyclophosphamide: Cyclophosphamide (alkylating agent) conditioning regimen and post transplantation cyclophosphamide with matched, partially matched, haplo-identical or unrelated donors

Sodium-2-mercapto ethane sulphonate: Antineoplastic detoxifying agent

Fludarabine monophosphate: Purine antimetabolite

Tacrolimus: Calcineurin inhibitor

Mofetil: Immunosuppressant

Rabbit antithymocyte globulin: Selective immunosuppressant

Overall Number of Baseline Participants 1
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
<=18 years
0
   0.0%
Between 18 and 65 years
1
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
Female
1
 100.0%
Male
0
   0.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
1
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
1
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 1 participants
1
1.Primary Outcome
Title The Feasibility of the Conditioning Regimen and Post Transplantation Cyclophosphamide in Refractory SLE Patients With Donors Having Various Degrees of Matching
Hide Description Number of participants who were alive at 1 year after transplant and who had not suffered graft rejection, acute or chronic GVHD, or Grade 3 or higher (CTCAE V4.0) adverse events.
Time Frame 1 year
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nonmyeloablative Conditioning and BMT
Hide Arm/Group Description:

Nonmyeloablative Conditioning and Bone Marrow Transplant in Systemic Lupus Erythematosus patients

Cyclophosphamide: Cyclophosphamide (alkylating agent) conditioning regimen and post transplantation cyclophosphamide with matched, partially matched, haplo-identical or unrelated donors

Sodium-2-mercapto ethane sulphonate: Antineoplastic detoxifying agent

Fludarabine monophosphate: Purine antimetabolite

Tacrolimus: Calcineurin inhibitor

Mofetil: Immunosuppressant

Rabbit antithymocyte globulin: Selective immunosuppressant

Overall Number of Participants Analyzed 1
Measure Type: Count of Participants
Unit of Measure: Participants
1
 100.0%
2.Secondary Outcome
Title RIFLE Score
Hide Description Change in Responder Index for Systemic Lupus Erythematosis (RIFLE) assessment. This is a qualitative scale measuring organ function. The count below is the number of individuals that improved within 1 year.
Time Frame 1 year
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The only enrolled participant on this study was never assessed for a follow-up RIFLE scale, so no data was collected for this outcome measure.
Arm/Group Title Nonmyeloablative Conditioning and BMT
Hide Arm/Group Description:

Nonmyeloablative Conditioning and Bone Marrow Transplant in Systemic Lupus Erythematosus patients

Cyclophosphamide: Cyclophosphamide (alkylating agent) conditioning regimen and post transplantation cyclophosphamide with matched, partially matched, haplo-identical or unrelated donors

Sodium-2-mercapto ethane sulphonate: Antineoplastic detoxifying agent

Fludarabine monophosphate: Purine antimetabolite

Tacrolimus: Calcineurin inhibitor

Mofetil: Immunosuppressant

Rabbit antithymocyte globulin: Selective immunosuppressant

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Survival
Hide Description Number of patients alive and alive without relapse, respectively.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nonmyeloablative Conditioning and BMT
Hide Arm/Group Description:

Nonmyeloablative Conditioning and Bone Marrow Transplant in Systemic Lupus Erythematosus patients

Cyclophosphamide: Cyclophosphamide (alkylating agent) conditioning regimen and post transplantation cyclophosphamide with matched, partially matched, haplo-identical or unrelated donors

Sodium-2-mercapto ethane sulphonate: Antineoplastic detoxifying agent

Fludarabine monophosphate: Purine antimetabolite

Tacrolimus: Calcineurin inhibitor

Mofetil: Immunosuppressant

Rabbit antithymocyte globulin: Selective immunosuppressant

Overall Number of Participants Analyzed 1
Measure Type: Count of Participants
Unit of Measure: Participants
Overall Survival
1
 100.0%
Event Free Survival
1
 100.0%
4.Secondary Outcome
Title Graft Failure
Hide Description Number of participants with primary and/or secondary graft failure.
Time Frame 60 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nonmyeloablative Conditioning and BMT
Hide Arm/Group Description:

Nonmyeloablative Conditioning and Bone Marrow Transplant in Systemic Lupus Erythematosus patients

Cyclophosphamide: Cyclophosphamide (alkylating agent) conditioning regimen and post transplantation cyclophosphamide with matched, partially matched, haplo-identical or unrelated donors

Sodium-2-mercapto ethane sulphonate: Antineoplastic detoxifying agent

Fludarabine monophosphate: Purine antimetabolite

Tacrolimus: Calcineurin inhibitor

Mofetil: Immunosuppressant

Rabbit antithymocyte globulin: Selective immunosuppressant

Overall Number of Participants Analyzed 1
Measure Type: Count of Participants
Unit of Measure: Participants
Primary graft failure
0
   0.0%
Secondary graft failure
0
   0.0%
5.Secondary Outcome
Title Acute Graft Versus Host Disease (GVHD)
Hide Description Percentage of participants who developed grades II-IV and grades III-IV acute GVHD. Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade I (skin involvement stages 1+ to 2+, with no liver or GI involvement), Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+).
Time Frame Up to 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nonmyeloablative Conditioning and BMT
Hide Arm/Group Description:

Nonmyeloablative Conditioning and Bone Marrow Transplant in Systemic Lupus Erythematosus patients

Cyclophosphamide: Cyclophosphamide (alkylating agent) conditioning regimen and post transplantation cyclophosphamide with matched, partially matched, haplo-identical or unrelated donors

Sodium-2-mercapto ethane sulphonate: Antineoplastic detoxifying agent

Fludarabine monophosphate: Purine antimetabolite

Tacrolimus: Calcineurin inhibitor

Mofetil: Immunosuppressant

Rabbit antithymocyte globulin: Selective immunosuppressant

Overall Number of Participants Analyzed 1
Measure Type: Count of Participants
Unit of Measure: Participants
Grades II-IV acute GVHD
0
   0.0%
Grades III-IV acute GVHD
0
   0.0%
6.Secondary Outcome
Title Chronic Graft Versus Host Disease (GVHD)
Hide Description Percentage of participants who developed chronic GVHD as defined by the NIH consensus criteria. This system gives scores from 0 to 3 for Karnofsky performance score, skin, mouth, eyes, gastrointestinal, liver, lungs, joints, and genitals, as well as an overall severity (mild, moderate, or severe). Higher scores indicate more severe disease. Scores are not totalled or added up.
Time Frame Up to 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nonmyeloablative Conditioning and BMT
Hide Arm/Group Description:

Nonmyeloablative Conditioning and Bone Marrow Transplant in Systemic Lupus Erythematosus patients

Cyclophosphamide: Cyclophosphamide (alkylating agent) conditioning regimen and post transplantation cyclophosphamide with matched, partially matched, haplo-identical or unrelated donors

Sodium-2-mercapto ethane sulphonate: Antineoplastic detoxifying agent

Fludarabine monophosphate: Purine antimetabolite

Tacrolimus: Calcineurin inhibitor

Mofetil: Immunosuppressant

Rabbit antithymocyte globulin: Selective immunosuppressant

Overall Number of Participants Analyzed 1
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
Time Frame Adverse events were assessed weekly through at least Day 60.
Adverse Event Reporting Description Reportable serious adverse events per protocol include: any mortality within the first 100 days after bone marrow transplant (BMT), any graft failures (defined as <5% donor chimerism) associated with failure of neutrophil recovery to >500/mm³ by day ~60 after transplantation, and all unexpected events as deemed significant by the PIs. Other events were neither tracked nor reported for this study.
 
Arm/Group Title Nonmyeloablative Conditioning and BMT
Hide Arm/Group Description

Nonmyeloablative Conditioning and Bone Marrow Transplant in Systemic Lupus Erythematosus patients

Cyclophosphamide: Cyclophosphamide (alkylating agent) conditioning regimen and post transplantation cyclophosphamide with matched, partially matched, haplo-identical or unrelated donors

Sodium-2-mercapto ethane sulphonate: Antineoplastic detoxifying agent

Fludarabine monophosphate: Purine antimetabolite

Tacrolimus: Calcineurin inhibitor

Mofetil: Immunosuppressant

Rabbit antithymocyte globulin: Selective immunosuppressant

All-Cause Mortality
Nonmyeloablative Conditioning and BMT
Affected / at Risk (%)
Total   0/1 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Nonmyeloablative Conditioning and BMT
Affected / at Risk (%)
Total   0/1 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Nonmyeloablative Conditioning and BMT
Affected / at Risk (%)
Total   0/1 (0.00%) 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Javier Bolaños Meade, MD
Organization: The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Phone: 410-614-6398
Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier: NCT02080195     History of Changes
Other Study ID Numbers: J13134
NA_00082453 ( Other Identifier: Johns Hopkins IRB )
First Submitted: February 28, 2014
First Posted: March 6, 2014
Results First Submitted: September 6, 2018
Results First Posted: October 2, 2018
Last Update Posted: October 2, 2018