Trial Outcomes & Findings for Nonmyeloablative Conditioning and Transplantation for Patients With Refractory Systemic Lupus Erythematosus (SLE) (NCT NCT02080195)

NCT ID: NCT02080195

Last Updated: 2019-10-02

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

1 participants

Primary outcome timeframe

1 year

Results posted on

2019-10-02

Participant Flow

Participant milestones

Participant milestones
Measure	Nonmyeloablative Conditioning and BMT 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 Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Nonmyeloablative Conditioning and BMT n=1 Participants 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
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

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.

Outcome measures

Outcome measures
Measure	Nonmyeloablative Conditioning and BMT n=1 Participants 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
The Feasibility of the Conditioning Regimen and Post Transplantation Cyclophosphamide in Refractory SLE Patients With Donors Having Various Degrees of Matching	1 Participants

SECONDARY outcome

Timeframe: 1 year

Population: 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.

Change in Responder Index for Systemic Lupus Erythematosis (RIFLE) assessment. This is a qualitative assessment of organ function. The 12 month response will be assessed as: complete= complete or partial resolution in more than one organ, partial= complete or partial resolution in at least one organ, the same= no change or no worsening in any organ, worse= worsening in any organ

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Number of patients alive and alive without relapse, respectively.

Outcome measures

Outcome measures
Measure	Nonmyeloablative Conditioning and BMT n=1 Participants 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
Survival Overall Survival	1 Participants
Survival Event Free Survival	1 Participants

SECONDARY outcome

Timeframe: 60 days

Number of participants with primary and/or secondary graft failure.

Outcome measures

Outcome measures
Measure	Nonmyeloablative Conditioning and BMT n=1 Participants 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
Graft Failure Primary graft failure	0 Participants
Graft Failure Secondary graft failure	0 Participants

SECONDARY outcome

Timeframe: Up to 2 years

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+).

Outcome measures

Outcome measures
Measure	Nonmyeloablative Conditioning and BMT n=1 Participants 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
Acute Graft Versus Host Disease (GVHD) Grades II-IV acute GVHD	0 Participants
Acute Graft Versus Host Disease (GVHD) Grades III-IV acute GVHD	0 Participants

SECONDARY outcome

Timeframe: Up to 2 years

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). Mild chronic GVHD involves only 1 or 2 organs or sites (except the lung), with no clinically significant functional impairment (maximum of score 1 in all affected organs or sites). Moderate chronic GVHD involves 1) at least 1 organ or site with clinically significant but no major disability (maximum score of 2 in any affected organ or site) OR 2) 3 or more organs or sites with no clinically significant functional impairment (maximum score of 1 in all affected organs or sites). Severe chronic GVHD indicates major disability caused by chronic GVHD (score of 3 in any organ or site).

Outcome measures

Outcome measures
Measure	Nonmyeloablative Conditioning and BMT n=1 Participants 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
Chronic Graft Versus Host Disease (GVHD)	0 Participants

Adverse Events

Nonmyeloablative Conditioning and BMT

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Javier Bolaños Meade, MD

The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Phone: 410-614-6398

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place