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CompletedNCT01821781Updated Oct 27, 2026Results posted

Immune Disorder HSCT Protocol

A Phase 2 interventional study of Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan in Immune Deficiency Disorders, Severe Combined Immunodeficiency and Chronic Granulomatous Disease, sponsored by Washington University School of Medicine. Completed at 1 site in United States. Open to participants aged Up to 21 Years. Per ClinicalTrials.gov, last updated 2026-10-27.

Sponsored by Washington University School of Medicine · Phase 2, Interventional, and Treatment

Updated Oct 27, 2026Now CompletedResults posted+5 moreGo to Updates ↓
Phase
Phase 2
Study type
Interventional
Enrollment
28
Allocation
Not applicable
Ages
Up to 21 Years
Sex
All
01

Study summary

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.

02

Conditions studied

  • Immune Deficiency Disorders
  • Severe Combined Immunodeficiency
  • Chronic Granulomatous Disease
  • X-linked Agammaglobulinemia
  • Wiskott-Aldrich Syndrome
  • Hyper-IgM
  • DiGeorge Syndrome
  • Chediak-Higashi Syndrome
  • Common Variable Immune Deficiency
  • Immune Dysregulatory Disorders
  • Hemophagocytic Lymphohistiocytosis
  • IPEX
  • Autoimmune Lymphoproliferative Syndrome
  • X-linked Lymphoproliferative Syndrome

Keywords

  • Immune deficiency
  • Immune disorders
  • Immune dysregulatory
  • Reduced Intensity
  • Alemtuzumab
  • Campath
03

In context

Severe Combined Immunodeficiency

59 studies on the registry are indexed under Severe Combined Immunodeficiency; 14 are open to participants now.

This study's enrollment of 28 is above the median of 10 across 31 interventional studies indexed under Severe Combined Immunodeficiency.

Browse Severe Combined Immunodeficiency studies →

Lead sponsor

Washington University School of Medicine is the lead sponsor of 1,803 studies on the registry; 271 are open to participants now.

Of its 329 completed or terminated interventional studies of FDA-regulated products, 216 (66%) have results posted.

Browse Washington University School of Medicine studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • \</= 28 years of age
  • Performance status >/= 40
  • DLCO >/= 40%
  • >/=40% or LVSF >/=26%
  • Serum creatinine \< 2x
  • Liver enzymes \</= 5x ULN
  • Negative pregnancy test
  • Suitably matched donor (6/6 matched sib UCB, 8/8 matched sib BM or PBSC, 5-6/6 matched unrelated UCB, 7-8/8 matched unrelated BM, double cord)

Exclusion criteria

Exclusion Criteria:

  • Known diagnosis of I/II
  • Pregnant or breastfeeding
  • Uncontrolled invasive fungal or bacterial infections within 1 month prior to starting alemtuzumab
  • Uncontrolled viral infection within 1 week prior to starting alemtuzumab
Terms in these criteria, in plain language(3)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

LVEF (left ventricular ejection fraction)

The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Enrollment
28 participants (actual)

Study arms

  • Experimental
    Reduced Intensity Conditioning

    Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan

Interventions

  • DrugTransplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan

    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

06

What researchers measure

Primary outcomes

  1. Number of Participants With Donor Engraftment

    Number of participants that did not experience Treatment Failure at 1 year post transplant

    Time frame: 1 year post transplant

Secondary outcomes

  1. Number of Participants With Major Transplant Related Toxicities

    Time frame: 1 years post transplant

  2. Number of Days Until Participants Reached Neutrophil Engraftment

    Time frame: within 100 days post transplant

  3. Percent of Participants With Acute Graft Versus Host Disease (GVHD)

    Time frame: 180 days post transplant

  4. Number of Participants With Infectious Complications

    Time frame: 2 years post transplant

  5. Overall Participant Survival One Year Post-Transplant

    Time frame: 1 years post transplant

  6. Number of Days to Reach Platelet Engraftment

    Time frame: within 100 days post transplant

  7. Percentage of Participants That Developed Chronic GVHD

    Time frame: 2 years post transplant

  8. Overall Survival of Participants 3 Years Post-Transplant

    Time frame: 3 years

  9. Event-Free Survival of Infant Participants at 1 Year Post-Transplant

    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

  10. Event Free Survival in Participants One Year and Older at One Year Post-Transplant

    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.

    Time frame: 1 year

07

Results

Posted Oct 27, 2026

Participant flow

Participant flow — Overall Study
MilestoneReduced Intensity Conditioning
Started28
Completed24
Not completed4

Outcome measures

PrimaryNumber of Participants With Donor Engraftment

Number of participants that did not experience Treatment Failure at 1 year post transplant

Time frame:
1 year post transplant
Reported as:
Count of participants · Participants
Number of Participants With Donor Engraftment
ParticipantsReduced Intensity Conditioning
Number of Participants With Donor Engraftment22
SecondaryNumber of Participants With Major Transplant Related Toxicities
Time frame:
1 years post transplant
Reported as:
Count of participants · Participants
Number of Participants With Major Transplant Related Toxicities
ParticipantsReduced Intensity Conditioning
hypertension2
acute kidney injury1
thrombotic microangiopathy1
hypokalemia2
hypocalcemia1
sepsis1
SecondaryNumber of Days Until Participants Reached Neutrophil Engraftment
Time frame:
within 100 days post transplant
Reported as:
Median · days
Number of Days Until Participants Reached Neutrophil Engraftment
daysReduced Intensity Conditioning
Number of Days Until Participants Reached Neutrophil Engraftment13 (11 to 29)
SecondaryPercent of Participants With Acute Graft Versus Host Disease (GVHD)
Time frame:
180 days post transplant
Reported as:
Number · percentage of participants
Percent of Participants With Acute Graft Versus Host Disease (GVHD)
percentage of participantsReduced Intensity Conditioning
Percent of Participants With Acute Graft Versus Host Disease (GVHD)10.7 (2.6 to 25.4)
SecondaryNumber of Participants With Infectious Complications
Time frame:
2 years post transplant
Reported as:
Count of participants · Participants
Number of Participants With Infectious Complications
ParticipantsReduced Intensity Conditioning
Cytomegalovirus (CMV)4
Epstein-Barr Virus (EBV)8
adenovirus (ADV)8
bacterial infections16
SecondaryOverall Participant Survival One Year Post-Transplant
Time frame:
1 years post transplant
Reported as:
Number · percentage of participants
Overall Participant Survival One Year Post-Transplant
percentage of participantsReduced Intensity Conditioning
Overall Participant Survival One Year Post-Transplant96.3 (76.5 to 99.5)
SecondaryNumber of Days to Reach Platelet Engraftment
Time frame:
within 100 days post transplant
Reported as:
Median · days
Number of Days to Reach Platelet Engraftment
daysReduced Intensity Conditioning
Number of Days to Reach Platelet Engraftment22 (16 to 85)
SecondaryPercentage of Participants That Developed Chronic GVHD
Time frame:
2 years post transplant
Reported as:
Number · percentage of participants
Percentage of Participants That Developed Chronic GVHD
percentage of participantsReduced Intensity Conditioning
Percentage of Participants That Developed Chronic GVHD12.9 (3.0 to 30.2)
SecondaryOverall Survival of Participants 3 Years Post-Transplant
Time frame:
3 years
Reported as:
Number · percentage of participants
Overall Survival of Participants 3 Years Post-Transplant
percentage of participantsReduced Intensity Conditioning
Overall Survival of Participants 3 Years Post-Transplant87.5 (66.0 to 95.8)
SecondaryEvent-Free Survival of Infant Participants at 1 Year Post-Transplant

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
Reported as:
Number · percentage of participants
Event-Free Survival of Infant Participants at 1 Year Post-Transplant
percentage of participantsReduced Intensity Conditioning
Event-Free Survival of Infant Participants at 1 Year Post-Transplant57.1 (17.2 to 83.7)
SecondaryEvent Free Survival in Participants One Year and Older at One Year Post-Transplant

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.

Time frame:
1 year
Reported as:
Number · percentage of participants
Event Free Survival in Participants One Year and Older at One Year Post-Transplant
percentage of participantsReduced Intensity Conditioning
Event Free Survival in Participants One Year and Older at One Year Post-Transplant90.5 (67.0 to 97.5)
Post-hocTreatment Failure (TF) in Infants at One Year Post-Transplant

Treatment failure (TF) is defined as loss of donor engraftment in the affected cell lineage or recurrence of inborn error of immunity (IEI).

Time frame:
1 year
Reported as:
Number · percentage of participants
Treatment Failure (TF) in Infants at One Year Post-Transplant
percentage of participantsReduced Intensity Conditioning
Treatment Failure (TF) in Infants at One Year Post-Transplant42.9 (7.6 to 75.7)
Post-hocTreatment Failure Rate in Older Children At 3 Years Post-Transplant

Treatment failure (TF) is defined as loss of donor engraftment in the affected cell lineage or recurrence of inborn error of immunity (IEI).

Time frame:
3 years
Reported as:
Number · percentage of participants
Treatment Failure Rate in Older Children At 3 Years Post-Transplant
percentage of participantsReduced Intensity Conditioning
Treatment Failure Rate in Older Children At 3 Years Post-Transplant9.5 (1.5 to 26.6)
Post-hocInfants With Treatment Failure That Were Successfully Salvaged With Second Allo-HSCT
Time frame:
3 years
Reported as:
Count of participants · Participants
Infants With Treatment Failure That Were Successfully Salvaged With Second Allo-HSCT
ParticipantsReduced Intensity Conditioning
Infants With Treatment Failure That Were Successfully Salvaged With Second Allo-HSCT4
Post-hocPatients With Neutrophil Recovery Within 30 Days Post-Transplant
Time frame:
day 30
Reported as:
Count of participants · Participants
Patients With Neutrophil Recovery Within 30 Days Post-Transplant
ParticipantsReduced Intensity Conditioning
Patients With Neutrophil Recovery Within 30 Days Post-Transplant28
Post-hocPatients' Myeloid Donor Engraftment Levels at 30 Days Post-Transplant
Time frame:
30 days
Reported as:
Count of participants · Participants
Patients' Myeloid Donor Engraftment Levels at 30 Days Post-Transplant
ParticipantsReduced Intensity Conditioning
over 90%27
53%1
Post-hocT-Cell Chimerism of Participants 30 Days Post-Transplant

Chimerism of patients with sufficient lymphocyte counts at day 30

Time frame:
30 days
Reported as:
Count of participants · Participants
T-Cell Chimerism of Participants 30 Days Post-Transplant
ParticipantsReduced Intensity Conditioning
complete donor T cell chimerism (95%+)10
mixed T cell chimerism11
no donor T cell chimerism1
Post-hocCD19 B Cell Counts at 3 Months Post-Transplant
Time frame:
3 months
Reported as:
Median · cells per microliter
CD19 B Cell Counts at 3 Months Post-Transplant
cells per microliterReduced Intensity Conditioning
CD19 B Cell Counts at 3 Months Post-Transplant81 (0 to 1890)
Post-hocCD5616 NK Cell Counts at 3 Months Post-Transplant
Time frame:
3 months
Reported as:
Median · cells per microliter
CD5616 NK Cell Counts at 3 Months Post-Transplant
cells per microliterReduced Intensity Conditioning
CD5616 NK Cell Counts at 3 Months Post-Transplant135 (48 to 1188)
Post-hocPercentage of Infant Participants (<1 Year) With Event-Free Survival
Time frame:
1 year post-transplant
Reported as:
Number · percentage of participants
Percentage of Infant Participants (<1 Year) With Event-Free Survival
percentage of participantsReduced Intensity Conditioning
Percentage of Infant Participants (<1 Year) With Event-Free Survival57.1 (17.2 to 83.7)

Adverse events

Collected over Adverse events were assessed up to 2 years. All-Cause Mortality was assessed up to 3 years.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Reduced Intensity Conditioning4/28 (14.3%)3/28 (10.7%)0/28 (0%)
Most frequent serious events
Most frequent serious events
EventReduced Intensity Conditioning
HypertensionCardiac disorders2/28
HypokalemiaBlood and lymphatic system disorders2/28

Baseline characteristics

Age, Continuous
Age, Continuous(years)Reduced Intensity Conditioning
Median3 ± 1
Sex: Female, Male
Sex: Female, Male(Participants)Reduced Intensity Conditioning
Female10
Male18
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Reduced Intensity Conditioning
Hispanic or Latino3
Not Hispanic or Latino23
Unknown or Not Reported2
Region of Enrollment
Region of Enrollment(participants)Reduced Intensity Conditioning
United States28
Donor Source
Donor Source(Participants)Reduced Intensity Conditioning
Matched Unrelated Donor (MUD)16
Mismatched Unrelated Donor (MMUD)8
Matched Sibling Donor (MSD)4
Diagnosis
Diagnosis(Participants)Reduced Intensity Conditioning
hemophagocytic lymphohistiocytosis (HLH)8
chronic granulomatous disease (CGD)6
severe combined immune deficiency (SCID)5
Other7
DOCK8 deficiency2
Graft Source
Graft Source(Participants)Reduced Intensity Conditioning
bone marrow (BM)21
peripheral blood stem cells (PBSC)5
umbilical cord blood (UCB)2
08

Study locations

1 site
  • Washington University
    St Louis, Missouri 63110, United States
09

References and documents

Publications

  • Bhatt ST, Schulz G, Hente M, Slater A, Murray L, Shenoy S, Bednarski JJ. A single-center experience using alemtuzumab, fludarabine, melphalan, and thiotepa as conditioning for transplantation in pediatric patients with chronic granulomatous disease. Pediatr Blood Cancer. 2020 Jan;67(1):e28030. doi: 10.1002/pbc.28030. Epub 2019 Oct 10. PubMed 31599480 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedDate before it movedWhere the date was before it moved

Ran from Apr 2013 to primary completion in May 2025, 144 months.

Results were first posted in Oct 2026, 1 year 5 months after primary completion.

Primary completion moved once since we started tracking on Sep 25, 2026: Apr 2025 → May 2025, on Oct 27, 2026.

Earlier changes are on the record history on ClinicalTrials.gov ↗

How these figures are worked out, and which studies are left out of them.

Registered
Mar 19, 2013
First posted
Apr 1, 2013
StartActual
Apr 29, 2013
Primary completionActual
May 8, 2025was Apr 2025
CompletionActual
Apr 23, 2026
Results first posted
Oct 27, 2026
Last update
Oct 27, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Status
Active, not recruiting→Completed
changed Oct 27, 2026
Results
Results posted
posted Oct 27, 2026
Primary completion
Apr 25, 2025→May 8, 2025
Oct 27, 2026
Enrollment
20 (estimated)→28 (actual)
Oct 27, 2026
Interventions
Arm changed Preparative→Reduced Intensity Conditioning
Oct 27, 2026
Primary outcomes
1 edited
What changed in the outcomes
  • EditedNumber of participants with donor engraftmentParticipants With Donor EngraftmentTime frame: 1 year post transplant
Oct 27, 2026
Documents
Oct 27, 2026
Secondary outcomes
2 edited, 8 added, 7 removed
What changed in the outcomes
  • EditedNumber of Participants With Major Transplant Related ToxicitiesTime frame: 1 years post transplant
  • EditedNumber of patient with acute GVHDParticipants With Infectious ComplicationsTime frame: 180 days post transplant → 2 years post transplant
  • AddedNumber of Days Until Participants Reached Neutrophil EngraftmentTime frame: within 100 days post transplant
  • AddedPercent of Participants With Acute Graft Versus Host Disease (GVHD)Time frame: 180 days post transplant
  • AddedOverall Participant Survival One Year Post-TransplantTime frame: 1 years post transplant
  • AddedNumber of Days to Reach Platelet EngraftmentTime frame: within 100 days post transplant
  • AddedPercentage of Participants That Developed Chronic GVHDTime frame: 2 years post transplant
  • AddedOverall Survival of Participants 3 Years Post-TransplantTime frame: 3 years
  • AddedEvent-Free Survival of Infant Participants at 1 Year Post-TransplantTime frame: 1 year
  • AddedEvent Free Survival in Participants One Year and Older at One Year Post-TransplantTime frame: 1 year
  • RemovedTime to neutrophil recoveryTime frame: within 100 days post transplant
  • RemovedNumber of participants with infectious complicationsTime frame: 2 years post transplant
  • RemovedTime to immune reconstitutionTime frame: 2 years post transplant
  • RemovedOverall survivalTime frame: 2 years post transplant
  • RemovedTime to platelet recoveryTime frame: within 100 days post transplant
  • RemovedNumber of patients with chronic GVHDTime frame: 2 years post transplant
  • RemovedDisease free survivalTime frame: 2 years post transplant
Oct 27, 2026
Show all 1 update
  1. Oct 27, 2026
    Active, not recruiting→Completed
    Results posted
    Documents Protocol and statistical analysis plan ↗ posted
    Primary completion Apr 25, 2025→May 8, 2025
    Enrollment 20 (estimated)→28 (actual)
    Primary outcomes 1 edited
    What changed in the outcomes
    • EditedNumber of participants with donor engraftmentParticipants With Donor EngraftmentTime frame: 1 year post transplant
    Secondary outcomes 2 edited, 8 added, 7 removed
    What changed in the outcomes
    • EditedNumber of Participants With Major Transplant Related ToxicitiesTime frame: 1 years post transplant
    • EditedNumber of patient with acute GVHDParticipants With Infectious ComplicationsTime frame: 180 days post transplant → 2 years post transplant
    • AddedNumber of Days Until Participants Reached Neutrophil EngraftmentTime frame: within 100 days post transplant
    • AddedPercent of Participants With Acute Graft Versus Host Disease (GVHD)Time frame: 180 days post transplant
    • AddedOverall Participant Survival One Year Post-TransplantTime frame: 1 years post transplant
    • AddedNumber of Days to Reach Platelet EngraftmentTime frame: within 100 days post transplant
    • AddedPercentage of Participants That Developed Chronic GVHDTime frame: 2 years post transplant
    • AddedOverall Survival of Participants 3 Years Post-TransplantTime frame: 3 years
    • AddedEvent-Free Survival of Infant Participants at 1 Year Post-TransplantTime frame: 1 year
    • AddedEvent Free Survival in Participants One Year and Older at One Year Post-TransplantTime frame: 1 year
    • RemovedTime to neutrophil recoveryTime frame: within 100 days post transplant
    • RemovedNumber of participants with infectious complicationsTime frame: 2 years post transplant
    • RemovedTime to immune reconstitutionTime frame: 2 years post transplant
    • RemovedOverall survivalTime frame: 2 years post transplant
    • RemovedTime to platelet recoveryTime frame: within 100 days post transplant
    • RemovedNumber of patients with chronic GVHDTime frame: 2 years post transplant
    • RemovedDisease free survivalTime frame: 2 years post transplant
    Interventions Arm changed Preparative→Reduced Intensity Conditioning
    + 4 other changes: verification date, design details, index terms and registry notes

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

11

Registry details

Key details

Study ID
NCT01821781
Lead sponsor
Washington University School of Medicine
Responsible party
Sponsor
First posted
Apr 1, 2013
Start date
Apr 29, 2013
Primary completion
May 8, 2025
Completion
Apr 23, 2026
Results posted
Oct 27, 2026
Last update
Oct 27, 2026

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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