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This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.
Locally preserved from ClinicalTrials.govOpen ClinicalTrials.govlast source update 2026-10-27
What this record can show
Results are reported by the registry submitter and preserved exactly. They are not independent verification or a treatment recommendation.
Registry facts
Linked local records
Local links use governed condition terms or exact source-reported intervention names. They are navigation candidates, not efficacy claims.
Interventions
Between days -23 and -15: alemtuzumab test dose, 3mg IV or SQ Day -14: alemtuzumab, 10mg IV or SQ Day -13: alemtuzumab, 15mg IV or SQ Day -12: alemtuzumab, 20mg IV or SQ Days -8 to -4: fludarabine, 30mg/m2 IV Day -4: thiotepa 4mg/kg IV q 12 hours Day -3: melphalan, 140mg/m2 IV Day 0: stem cell infusion Day +7: G-CSF
Source-reported results
Results are reported by the registry submitter and preserved exactly. They are not independent verification or a treatment recommendation.
Number of participants that did not experience Treatment Failure at 1 year post transplant
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 22 | - |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 2 | - |
| Reduced Intensity Conditioning | 28 | 1 | - |
| Reduced Intensity Conditioning | 28 | 1 | - |
| Reduced Intensity Conditioning | 28 | 2 | - |
| Reduced Intensity Conditioning | 28 | 1 | - |
| Reduced Intensity Conditioning | 28 | 1 | - |
Eligibility
primary outcomes
Time frame: 1 year post transplant
Number of participants that did not experience Treatment Failure at 1 year post transplant
secondary outcomes
Time frame: 1 years post transplant
Time frame: within 100 days post transplant
Time frame: 180 days post transplant
Time frame: 2 years post transplant
Time frame: 1 years post transplant
Time frame: within 100 days post transplant
Time frame: 2 years post transplant
Time frame: 3 years
Time frame: 1 year
In cancer, event-free survival is the length of time after primary treatment for a cancer ends that the patient remains free of certain complications or events that the treatment was intended to prevent or delay. These events may include the return of the cancer or the onset of certain symptoms, such as bone pain from cancer that has spread to the bone. In a clinical trial, measuring the event-free survival is one way to see how well a new treatment works. Also called EFS.
Time frame: 1 year
Event Free Survival: In cancer, the length of time after primary treatment for a cancer ends that the patient remains free of certain complications or events that the treatment was intended to prevent or delay. These events may include the return of the cancer or the onset of certain symptoms, such as bone pain from cancer that has spread to the bone. In a clinical trial, measuring the event-free survival is one way to see how well a new treatment works. Also called EFS.
Publications
No exact PMID-linked public article is currently readable locally.
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 13 | 11 to 29 |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 10.7 | 2.6 to 25.4 |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 4 | - |
| Reduced Intensity Conditioning | 28 | 8 | - |
| Reduced Intensity Conditioning | 28 | 8 | - |
| Reduced Intensity Conditioning | 28 | 16 | - |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 96.3 | 76.5 to 99.5 |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 22 | 16 to 85 |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 12.9 | 3.0 to 30.2 |
Population: This analysis includes the participants that were being followed at the 3-year timepoint. The median follow-up was 36 months (range 8 to 133 months).
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 16 | 87.5 | 66.0 to 95.8 |
In cancer, event-free survival is the length of time after primary treatment for a cancer ends that the patient remains free of certain complications or events that the treatment was intended to prevent or delay. These events may include the return of the cancer or the onset of certain symptoms, such as bone pain from cancer that has spread to the bone. In a clinical trial, measuring the event-free survival is one way to see how well a new treatment works. Also called EFS.
Population: Infants were defined as those less than a year old
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 7 | 57.1 | 17.2 to 83.7 |
Event Free Survival: In cancer, the length of time after primary treatment for a cancer ends that the patient remains free of certain complications or events that the treatment was intended to prevent or delay. These events may include the return of the cancer or the onset of certain symptoms, such as bone pain from cancer that has spread to the bone. In a clinical trial, measuring the event-free survival is one way to see how well a new treatment works. Also called EFS.
Population: Older children were considered those one year or older
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 21 | 90.5 | 67.0 to 97.5 |
Treatment failure (TF) is defined as loss of donor engraftment in the affected cell lineage or recurrence of inborn error of immunity (IEI).
Population: Infants were participants less than one year of age
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 7 | 42.9 | 7.6 to 75.7 |
Treatment failure (TF) is defined as loss of donor engraftment in the affected cell lineage or recurrence of inborn error of immunity (IEI).
Population: Participants one year and older
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 20 | 9.5 | 1.5 to 26.6 |
Population: Infants defined as less than one year of age
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 5 | 4 | - |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 28 | - |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning · over 90% | 28 | 27 | - |
| Reduced Intensity Conditioning · 53% | 28 | 1 | - |
Chimerism of patients with sufficient lymphocyte counts at day 30
Population: Chimerism of patients with sufficient lymphocyte counts at day 30
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning · complete donor T cell chimerism (95%+) | 22 | 10 | - |
| Reduced Intensity Conditioning · mixed T cell chimerism | 22 | 11 | - |
| Reduced Intensity Conditioning · no donor T cell chimerism | 22 | 1 | - |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 81 | 0 to 1890 |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 28 | 135 | 48 to 1188 |
| Group | N | Value | Spread / interval |
|---|---|---|---|
| Reduced Intensity Conditioning | 7 | 57.1 | 17.2 to 83.7 |
| Milestone | Reduced Intensity Conditioning |
|---|---|
| STARTED | 28 |
| COMPLETED | 24 |
| NOT COMPLETED | 4 |
| Event | System | Groups: affected / at risk |
|---|---|---|
| Hypertension | Cardiac disorders | Reduced Intensity Conditioning: 2 / 28 |
| Hypokalemia | Blood and lymphatic system disorders | Reduced Intensity Conditioning: 2 / 28 |
Organisations & named parties
Organisation links are temporarily unavailable.