Immune Deficiency DisordersPhase 2NCT01821781
Immune Disorder HSCT Protocol
A Phase 2 study testing Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan in people with Immune Deficiency Disorders and Severe Combined Immunodeficiency, run by Washington University School of Medicine at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease
- What is being tested?
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Apr 2013to Apr 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- </= 28 years of age
- Performance status >/= 40
- DLCO >/= 40%
- LVEF >/=40% or LVSF >/=26%
- Serum creatinine < 2x ULN
- 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)
Who cannot join
- Known diagnosis of HIV 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime
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.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM
- Being tested
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Keywords
- Immune deficiency, Immune disorders, Immune dysregulatory, Reduced Intensity, Alemtuzumab, Campath
- Medical subject headings
- Severe Combined Immunodeficiency, Granulomatous Disease, Chronic, Bruton type agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM Immunodeficiency Syndrome, DiGeorge Syndrome, Chediak-Higashi Syndrome, Common Variable Immunodeficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanDrug
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
| Study group | Role | What they receive |
|---|---|---|
| Preparative | Experimental | Not described Receives: Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with donor engraftment
Measured: 1 year post transplant
Secondary outcomes (9)
Major Transplant Related Toxicities
Measured: 1 years post transplant
Time to neutrophil recovery
Measured: within 100 days post transplant
Number of patient with acute GVHD
Measured: 180 days post transplant
Number of participants with infectious complications
Measured: 2 years post transplant
Time to immune reconstitution
Measured: 2 years post transplant
Overall survival
Measured: 2 years post transplant
Time to platelet recovery
Measured: within 100 days post transplant
Number of patients with chronic GVHD
Measured: 2 years post transplant
Disease free survival
Measured: 2 years post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2013 (estimated)
- Study start29 Apr 2013
- Primary completion25 Apr 2025
- Study completionApr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Immune Deficiency DisordersStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01821781
- Sponsor's protocol ID
- 201301135
- Status last verified
- Feb 2026
- First submitted
- 19 Mar 2013
- Last updated
- 19 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
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 at a glance
From the registry record, last updated by the sponsor on 19 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime
- ClinicalTrials.gov ID
- NCT01821781
- Acronym / sponsor ID
- 201301135
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM
- Keywords
- Immune deficiency, Immune disorders, Immune dysregulatory, Reduced Intensity, Alemtuzumab, Campath
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Study start
- 29 Apr 2013 (actual)
- Primary completion
- 25 Apr 2025 (actual)
- Study completion
- Apr 2026 (estimated)
- First posted
- 1 Apr 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Preparative | Experimental | Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- </= 28 years of age
- Performance status >/= 40
- DLCO >/= 40%
- LVEF >/=40% or LVSF >/=26%
- Serum creatinine < 2x ULN
Key exclusion criteria (4 listed)
- Known diagnosis of HIV 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanBetween 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 | Drug | Preparative |
Primary outcome
Number of participants with donor engraftment
Time frame: 1 year post transplant
9 secondary outcomes
Major Transplant Related Toxicities
Time frame: 1 years post transplant
Time to neutrophil recovery
Time frame: within 100 days post transplant
Number of patient with acute GVHD
Time frame: 180 days post transplant
Number of participants with infectious complications
Time frame: 2 years post transplant
Time to immune reconstitution
Time frame: 2 years post transplant
Overall survival
Time frame: 2 years post transplant
Time to platelet recovery
Time frame: within 100 days post transplant
Number of patients with chronic GVHD
Time frame: 2 years post transplant
Disease free survival
Time frame: 2 years post transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Feb 2026.
- Derived from this study: PubMed 31599480Bhatt 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan in people with Immune Deficiency Disorders and Severe Combined Immunodeficiency, run by Washington University School of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 19 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with donor engraftment (time frame: 1 year post transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01821781, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Feb 2026.