Complete clinical response is defined as complete clinical response in the target organ and no clinical signs of active lupus as determined by a Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) score of ≤3; prednisone ≤10mg/day at 6 months and ≤5mg/day at 12 months or later.
Group
Value
95% CI
Autologous HSCT in SLE
54
36 – 60
Number of Participants With Adverse EventsSecondary· 18 months
Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module.
Group
Value
95% CI
Autologous HSCT in SLE
8
Anti-Nuclear AntibodySecondary· Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Anti-Nuclear antibody is a well accepted biological clinical laboratory marker of systemic lupus. Range of normal values is 0-0.9 EU.
Day -7
Group
Value
95% CI
Autologous HSCT in SLE
5.4
± 4.65
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
4.7
± 4.73
1 month
Group
Value
95% CI
Autologous HSCT in SLE
3.7
± 3.89
3 months
Group
Value
95% CI
Autologous HSCT in SLE
3.2
± 3.91
6 months
Group
Value
95% CI
Autologous HSCT in SLE
2.7
± 3.01
1 year
Group
Value
95% CI
Autologous HSCT in SLE
2.6
± 2.77
18 months
Group
Value
95% CI
Autologous HSCT in SLE
2.8
± 2.7
2 years
Group
Value
95% CI
Autologous HSCT in SLE
2.5
± 2.59
Extractable Nuclear Antigen (ENA)Secondary· Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Extractable nuclear antigen is a well accepted biological clinical laboratory marker of systemic lupus. Range of normal values is 0-19.
Day -7
Group
Value
95% CI
Autologous HSCT in SLE
66.9
± 74.48
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
64.8
± 70.61
1 month
Group
Value
95% CI
Autologous HSCT in SLE
61.5
± 73.72
3 months
Group
Value
95% CI
Autologous HSCT in SLE
58.5
± 67.03
6 months
Group
Value
95% CI
Autologous HSCT in SLE
51.3
± 57.22
1 year
Group
Value
95% CI
Autologous HSCT in SLE
57.2
± 58.07
18 months
Group
Value
95% CI
Autologous HSCT in SLE
60.6
± 60.76
2 years
Group
Value
95% CI
Autologous HSCT in SLE
50.6
± 49.04
Anti-Double Stranded Deoxyribonucleic Acid (DNA) AntibodySecondary· Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Anti-Double stranded deoxyribonucleic acid antibody is a well accepted biological clinical laboratory marker especially specific for systemic lupus. Range of normal values is 0-24 IU.
Day -7
Group
Value
95% CI
Autologous HSCT in SLE
17.3
± 24.92
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
8.8
± 16.8
1 month
Group
Value
95% CI
Autologous HSCT in SLE
0
± 0
3 months
Group
Value
95% CI
Autologous HSCT in SLE
0
± 0
6 months
Group
Value
95% CI
Autologous HSCT in SLE
0
± 0
1 year
Group
Value
95% CI
Autologous HSCT in SLE
0
± 0
18 months
Group
Value
95% CI
Autologous HSCT in SLE
0
± 0
2 years
Group
Value
95% CI
Autologous HSCT in SLE
0
± 0
Anti-Smith-Ribonuclear Protein AntibodySecondary· Day -7, day 0, 1 3, and 6 months, 1 year, 18 months and 2 years.
Anti-Smith-Ribonuclear protein antibody is a well accepted biological clinical laboratory marker of systemic lupus.Range of normal values is 0-19 EU.
Day -7
Group
Value
95% CI
Autologous HSCT in SLE
49
± 49.51
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
51.6
± 53.72
1 month
Group
Value
95% CI
Autologous HSCT in SLE
31.5
± 40.31
3 months
Group
Value
95% CI
Autologous HSCT in SLE
29.8
± 41.48
6 months
Group
Value
95% CI
Autologous HSCT in SLE
28.5
± 40.07
1 year
Group
Value
95% CI
Autologous HSCT in SLE
20
± 34.64
18 months
Group
Value
95% CI
Autologous HSCT in SLE
16.67
± 28.87
2 years
Group
Value
95% CI
Autologous HSCT in SLE
25.67
± 44.46
White Blood CellsSecondary· Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
The white blood cell test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 3.4-9.6 K/uL.
Day -7
Group
Value
95% CI
Autologous HSCT in SLE
6.89
± 3.07
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
0.47
± 0.31
1 month
Group
Value
95% CI
Autologous HSCT in SLE
10.32
± 6.6
3 months
Group
Value
95% CI
Autologous HSCT in SLE
3.84
± 1.4
6 months
Group
Value
95% CI
Autologous HSCT in SLE
4.21
± 1.38
1 year
Group
Value
95% CI
Autologous HSCT in SLE
5.81
± 1.01
18 months
Group
Value
95% CI
Autologous HSCT in SLE
5.24
± 0.82
2 years
Group
Value
95% CI
Autologous HSCT in SLE
7.17
± 3.93
Absolute Neutrophil CountSecondary· Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
The absolute neutrophil count test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 1.29-7.5 K/uL.
Day -7
Group
Value
95% CI
Autologous HSCT in SLE
5.66
± 2.88
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
0.45
± 0.3
1 month
Group
Value
95% CI
Autologous HSCT in SLE
9.11
± 6.67
3 months
Group
Value
95% CI
Autologous HSCT in SLE
2.72
± 0.93
6 months
Group
Value
95% CI
Autologous HSCT in SLE
2.78
± 1.38
1 year
Group
Value
95% CI
Autologous HSCT in SLE
3.44
± 0.97
18 months
Group
Value
95% CI
Autologous HSCT in SLE
2.72
± 1.04
2 years
Group
Value
95% CI
Autologous HSCT in SLE
4.04
± 1.7
Absolute Lymphocyte CountSecondary· Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
The absolute lymphocyte count test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 0.45-4.9 K/uL.
Day -7
Group
Value
95% CI
Autologous HSCT in SLE
0.54
± 0.4
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
0.0065
± 0.0064
1 month
Group
Value
95% CI
Autologous HSCT in SLE
0.42
± 0.18
3 months
Group
Value
95% CI
Autologous HSCT in SLE
0.53
± 0.46
6 months
Group
Value
95% CI
Autologous HSCT in SLE
0.82
± 0.31
1 year
Group
Value
95% CI
Autologous HSCT in SLE
1.75
± 0.64
18 months
Group
Value
95% CI
Autologous HSCT in SLE
1.8
± 0.61
2 years
Group
Value
95% CI
Autologous HSCT in SLE
1.8
± 0.84
Platelet CountSecondary· Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
The platelet count test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 162-380 K/uL.
Day -7
Group
Value
95% CI
Autologous HSCT in SLE
251
± 62.9
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
113
± 49
1 month
Group
Value
95% CI
Autologous HSCT in SLE
166
± 61.6
3 months
Group
Value
95% CI
Autologous HSCT in SLE
187
± 95.4
6 months
Group
Value
95% CI
Autologous HSCT in SLE
170
± 64
1 year
Group
Value
95% CI
Autologous HSCT in SLE
226
± 47.9
18 months
Group
Value
95% CI
Autologous HSCT in SLE
210
± 43.5
2 years
Group
Value
95% CI
Autologous HSCT in SLE
308
± 251.5
Cluster of Differentiation 3 (CD3) + CellsSecondary· Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.
The CD3+Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 650-2108 uL.
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
2.99
± 3.25
1 month
Group
Value
95% CI
Autologous HSCT in SLE
239.47
± 210.74
3 months
Group
Value
95% CI
Autologous HSCT in SLE
435.97
± 335.69
6 months
Group
Value
95% CI
Autologous HSCT in SLE
699.47
± 256.67
1 year
Group
Value
95% CI
Autologous HSCT in SLE
1493.29
± 605.26
2 years
Group
Value
95% CI
Autologous HSCT in SLE
1678.76
± 888.41
Cluster of Differentiation 4 (CD4) + CellsSecondary· Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.
The CD4 + Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 358-1259 uL.
Day 0
Group
Value
95% CI
Autologous HSCT in SLE
2.58
± 2.98
1 month
Group
Value
95% CI
Autologous HSCT in SLE
103.37
± 117.43
3 months
Group
Value
95% CI
Autologous HSCT in SLE
112.8
± 101.94
6 months
Group
Value
95% CI
Autologous HSCT in SLE
316.25
± 193.01
1 year
Group
Value
95% CI
Autologous HSCT in SLE
702.87
± 362.75
2 years
Group
Value
95% CI
Autologous HSCT in SLE
958.03
± 735.04
Adverse events — posted to ClinicalTrials.gov
Time frame: 18 months.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Autologous HSCT in SLE
Serious: 8/9 (89%)
Deaths: 2/9
Serious adverse events (16 terms)
Reaction
System
Autologous HSCT in SLE
hypoxia
Respiratory, thoracic and mediastinal disorders
—
Death Not Associated with CTCAE term: Death NOS
General disorders
—
allergic reaction/hypersensitivity (including drug fever)
This study will examine a new approach to treating patients with severe systemic lupus erythematosus (SLE) that involves collecting stem cells (cells produced by the bone marrow that develop into blood cells) from the patient, completely shutting down the patient's immune system, and then giving back the patient's stem cells. SLE is a chronic, inflammatory disorder of the immune system that can affect many organs. It is called an autoimmune disease because the patient's lymphocytes (white blood cells that normally protect against invading organisms), go out of control and attack the body's own tissues.
Patients between 15 and 40 years of age with severe SLE affecting a major organ that is resistant to standard treatment may be eligible for this study. Candidates are screened with a medical history and physical examination, blood and urine tests, skin tuberculin test, and radiology studies to evaluate the extent of disease. They have endocrinology, nutrition, dental, and social work consultations, ultrasound or MUGA (multi-gated acquisition scan) scan heart imaging, electrocardiogram and lung function tests, bone marrow biopsy, and lymph node aspirate. Depending on which organs are affected, patients may have additional tests, such as lumbar puncture (spinal tap), kidney or lung biopsy, MRI (magnetic resonance imaging) of the brain and spinal cord, and PET (positron emission tomography) scan. They also complete quality of life questionnaires and have disability functional testing and neurocognitive (thinking) assessments.
Participants have a central venous line (plastic tube) inserted into a neck or chest vein for administering stem cells and medicines and for drawing blood. They undergo seven apheresis procedures during the course of the study to collect stem cells for transplant and for research. For apheresis, whole blood is collected through a needle in an arm vein and directed to a cell-separating machine where the white cells are extracted and the rest of the blood is returned to the patient through the same needle.
Patients are primed with three medications (methylprednisolone, rituximab, and cyclophosphamide) through the central line to help control the disease. In addition, a medication called G-CSF (growth colony stimulating factor) is injected under the skin for several days to boost production of stem cells. After enough stem cells have been collected for transplantation (infusion through the central line), patients are admitted to the hospital for an 8-day conditioning regimen followed by transplantation. The conditioning treatment consists of rituximab, fludarabine, and cyclophosphamide to eliminate all the white blood cells from the blood and bone marrow. The stem cells are then infused and the patient is closely monitored by a team of physicians and nurses. When the stem cells have engrafted, the bone marrow has recovered, and the patient feels well enough - usually 2 to 3 weeks after transplant - the patient is discharged from the hospital. Prednisone tapering begins as soon as feasibly possible, but no later then 28 days after transplant.
Patients return to the National Institutes of Health (NIH) Clinical Center for frequent follow-up visits during the first 2 to 3 months following transplant. The time between visits is then extended to once every 3 months the first year, then every 6 months the second year, and then at least yearly for 5 years after the transplant. These visits include a physical examination, blood and urine tests, lumbar puncture (if there is central nervous system involvement), other appropriate biopsies and tests as needed to monitor the patient's health, short apheresis procedures to collect blood for research purposes, and quality of life questionnaires. Some select procedures will be optional. Bone marrow biopsies and lymph node aspirates are done at beginning and at 6, 12, and 24 months after transplant. PET scans are done at 1, 6, 12, and 24 months.
...
Publications & conference data
7 peer-reviewed publications reference this trial (live from Europe PMC):
NCT06904066 — Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients W
· Phase 1
· recruiting
NCT02181478 — Intra-Osseous Co-Transplant of UCB and hMSC
· EARLY_PHASE1
· completed
NCT02248597 — Donor Stem Cell Transplant Followed by Cyclophosphamide in Treating Patients With Hematological Diseases
· Phase 2
· completed
NCT02129582 — Targeted Marrow Irradiation, Fludarabine Phosphate, and Busulfan Before Donor Progenitor Cell Transplant in Treating Pat
· Phase 1
· completed
NCT02070406 — Gene-Modified T Cells, Vaccine Therapy, and Ipilimumab in Treating Patients With Locally Advanced or Metastatic Malignan
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Cancer Institute (NCI)
Last refreshed: 5 January 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00076752.