SCIDNCT01652092
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
A study testing Alemtuzumab 0.3 mg, Cyclophosphamide and Busulfan in people with SCID and Omenn's Syndrome, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies. Read more about this study
- Who is it for?
- Ages Up to 50 years
- Conditions the study is for
- SCID, Omenn's Syndrome, Reticular Dysgenesis
- What is being tested?
- Alemtuzumab 0.3 mg, Cyclophosphamide and Busulfan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 57 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Sep 2012to Dec 2026
- Phase
- Interventional
- 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 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of immunodeficiency or histiocytic disorder including the following:
- Severe combined immunodeficiency (SCID - all variants)
- Second bone marrow transplant (BMT) for SCID (after graft rejection)
- Omenn's Syndrome
- Reticular dysgenesis
- Wiskott-Aldrich syndrome
Show all 28Show fewer
- Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)
- Hyper IgM Syndrome (CD40 Ligand Deficiency)
- Common variable immunodeficiency (CVID) with severe phenotype
- Chronic Granulomatous Disease (CGD)
- Other severe Combined Immune Deficiencies (CID)
- Hemophagocytic Lymphohistiocytosis (HLH)
- X-linked Lymphoproliferative Disease (XLP)
- Chediak-Higashi Syndrome (CHS)
- Griscelli Syndrome
- Langerhans Cell Histiocytosis (LCH)
- Acceptable stem cell sources include:
- HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow
- HLA identical or up to a 1 antigen mismatched unrelated BM donor
- Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards
- Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of >5 x 10^7 nucleated cells/kg as per current institutional guidelines
- Double unrelated umbilical cord blood units that are:
- up to 2 antigen mismatched to the patient
- up to 2 antigen mismatched to each other
- minimum cell dose of at least one single unit must be ≥ 3.5 x 10^7 nucleated cells/kg
- combined dose of both units must provide a total cell dose of ≥ 5 x 10^7 nucleated cells/kg
- Age: 0 to 50 years
- Adequate organ function and performance status.
Who cannot join
- pregnant or breastfeeding
- active, uncontrolled infection and/or HIV positive
- acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455
About this study
This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
Read the detailed description
Based on diagnosis and clinical history, a determination of the most appropriate regimen will be made based on the following prep plans:
Arm A: Fully Myeloablative Preparative Regimen, Arm B: Reduced Toxicity Ablative Preparative Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen
- Conditions
- SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency
- Being tested
- Alemtuzumab 0.3 mg, Cyclophosphamide, Busulfan, Stem Cell Transplantation, Fludarabine phosphate 40 mg
- Keywords
- immunodeficiency disorder, histiocytic disorder
- Medical subject headings
- Severe Combined Immunodeficiency, Reticular dysgenesis, Wiskott-Aldrich Syndrome, Common Variable Immunodeficiency, Granulomatous Disease, Chronic, Hyper-IgM Immunodeficiency Syndrome, Lymphoproliferative Disorders, Lymphohistiocytosis, Hemophagocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alemtuzumab 0.3 mgDrug
Also called Campath-1H
0.3 mg/kg intravenously (IV) on days -12 through -10
CyclophosphamideDrug
Also called Cytoxan
cyclophosphamide 50 mg/kg IV on days -9 through -6
BusulfanDrug
Also called Myerlan
busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2
Stem Cell TransplantationBiological
Unrelated donor bone marrow will be collected in the usual manner using established parameters determined by the National Marrow Donor Program. A minimum of 3 x 10^8 nucleated cells/kg recipient weight will be collected with a goal of ≥ 5 x 10^8 nucleated cells/kg recipient weight.
Umbilical cord blood selection will be per the current University of Minnesota Cord Blood Unit Selection algorithm. One or two units may be used to obtain the minimum cell dose. One of the UCB units selected for transplantation must contain ≥ 3.5 x 10^7 nucleated cells/kg recipient weight based on cell numbers at time of cryopreservation, and the total combined cell dose of both units must be > 5.0 x 10^7 nucleated cells/kg.
Fludarabine phosphate 40 mgDrug
Also called Fludara
40 mg/m^2 IV on days -5 through -2 (for children < 6 months and/or < 10 kg weight dose at 1.33 mg/kg)
MelphalanDrug
Also called Alkeran
140 mg/m^2 IV on day -3
Alemtuzumab 0.2 mgDrug
Also called Campath 1-H
0.2 mg/kg intravenously (IV) on days -14 through -10
BusulfanDrug
Also called Myerlan
busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6
Fludarabine phosphate 30 mgDrug
Also called Fludara
fludarabine 30 mg/m^2 IV on days -8 through -4
MESNADrug
Also called mercaptoethane sulfonate Na (Na being the symbol for sodium), Mesnex
administered as per the standard institutional protocol.
| Study group | Role | What they receive |
|---|---|---|
| Arm A: Fully Myeloablative regimen | Other | For use in patients with diseases including Wiskott-Aldrich syndrome, MHC Class II deficiency, hypomorphic SCID, etc. Receives Alemtuzumab 0.3 mg/kg intravenously (IV) on days -12 through -10, cyclophosphamide 50 mg/kg IV plus MESNA on days -9 through -6, busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2 and stem cell infusion on day 0. Receives: Drug: Alemtuzumab 0.3 mg; Drug: Cyclophosphamide; Drug: Busulfan; Biological: Stem Cell Transplantation; Drug: MESNA |
| Arm B: Reduced Toxicity Ablative Regimen | Other | For use in patients with diseases including SCID, CGD, CHS and other CID. Receives Alemtuzumab 0.3 mg/kg intravenously (IV) on days -12 through -10, busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6, fludarabine phosphate 40 mg/m^2 IV on days -5 through -2 and stem cell infusion on day 0. Receives: Drug: Alemtuzumab 0.3 mg; Biological: Stem Cell Transplantation; Drug: Fludarabine phosphate 40 mg; Drug: Busulfan |
| Arm C: Reduced Intensity Conditioning | Other | For use in patients with diseases including HLH. Receives Alemtuzumab 0.2 mg/kg intravenously (IV) on days -14 through -10, fludarabine phosphate 30 mg/m^2 IV on days -8 through -4, melphalan 140 mg/m^2 IV on day -3 and stem cell infusion on day 0. Receives: Biological: Stem Cell Transplantation; Drug: Melphalan; Drug: Alemtuzumab 0.2 mg; Drug: Fludarabine phosphate 30 mg |
| Arm D: No Preparative Regimen | Other | For use in patients with complete SCID phenotype with no evidence of maternal engraftment or residual immune function who will be receiving their stem cell transplantation from a genotypically matched donor. Receives: Biological: Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neutrophil Engraftment
Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm3 (0.5 x 109/L) or greater.
Measured: Day 42
Secondary outcomes (7)
Incidence of Graft Failure
Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets.
Measured: Day 100
Incidence of Chimerism
a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease.
Measured: Day 100, 6 Months, 1 Year
Incidence of Acute Graft-Versus-Host Disease
Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Measured: Day 100
Incidence of Chronic Graft-Versus-Host Disease
Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
Measured: 6 Months and 1 Year
Incidence of Transplant-Related Mortality
In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.
Measured: 6 Months
Disease-Free Survival
the length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works.
Measured: 6 Months
Overall Survival
Overall survival will be defined as time from enrollment to date of death or censored at the date of last documented contact for patients still alive.
Measured: 6 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2012 (estimated)
- Study start4 Sep 2012
- Primary completion30 Dec 2025
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SCIDStudies of the same condition, open and under way.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Meera Srikanthan, MD, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01652092
- Sponsor's protocol ID
- 2012OC055
- Other IDs
- MT2012-10C
- Status last verified
- Jun 2026
- First submitted
- 25 Jul 2012
- Last updated
- 17 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
- ClinicalTrials.gov ID
- NCT01652092
- Acronym / sponsor ID
- 2012OC055
- Conditions
- SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency
- Keywords
- immunodeficiency disorder, histiocytic disorder
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 4 Sep 2012 (actual)
- Primary completion
- 30 Dec 2025 (actual)
- Study completion
- Dec 2026 (estimated)
- First posted
- 27 Jul 2012 (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 is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
Detailed description
Based on diagnosis and clinical history, a determination of the most appropriate regimen will be made based on the following prep plans:
Arm A: Fully Myeloablative Preparative Regimen, Arm B: Reduced Toxicity Ablative Preparative Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Arm A: Fully Myeloablative regimen | Other | Drug: Alemtuzumab 0.3 mg, Drug: Cyclophosphamide, Drug: Busulfan, Biological: Stem Cell Transplantation, Drug: MESNAFor use in patients with diseases including Wiskott-Aldrich syndrome, MHC Class II deficiency, hypomorphic SCID, etc. Receives Alemtuzumab 0.3 mg/kg intravenously (IV) on days -12 through -10, cyclophosphamide 50 mg/kg IV plus MESNA on days -9 through -6, busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2 and stem cell infusion on day 0. |
| Arm B: Reduced Toxicity Ablative Regimen | Other | Drug: Alemtuzumab 0.3 mg, Biological: Stem Cell Transplantation, Drug: Fludarabine phosphate 40 mg, Drug: BusulfanFor use in patients with diseases including SCID, CGD, CHS and other CID. Receives Alemtuzumab 0.3 mg/kg intravenously (IV) on days -12 through -10, busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6, fludarabine phosphate 40 mg/m^2 IV on days -5 through -2 and stem cell infusion on day 0. |
| Arm C: Reduced Intensity Conditioning | Other | Biological: Stem Cell Transplantation, Drug: Melphalan, Drug: Alemtuzumab 0.2 mg, Drug: Fludarabine phosphate 30 mgFor use in patients with diseases including HLH. Receives Alemtuzumab 0.2 mg/kg intravenously (IV) on days -14 through -10, fludarabine phosphate 30 mg/m^2 IV on days -8 through -4, melphalan 140 mg/m^2 IV on day -3 and stem cell infusion on day 0. |
| Arm D: No Preparative Regimen | Other | Biological: Stem Cell TransplantationFor use in patients with complete SCID phenotype with no evidence of maternal engraftment or residual immune function who will be receiving their stem cell transplantation from a genotypically matched donor. |
Who may qualify
- Ages
- Up to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (28 listed)
- Diagnosis of immunodeficiency or histiocytic disorder including the following:
- Acceptable stem cell sources include:
- Age: 0 to 50 years
- Adequate organ function and performance status.
Key exclusion criteria (3 listed)
- pregnant or breastfeeding
- active, uncontrolled infection and/or HIV positive
- acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
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 |
|---|---|---|
| Alemtuzumab 0.3 mgAlso: Campath-1H0.3 mg/kg intravenously (IV) on days -12 through -10 | Drug | Arm A: Fully Myeloablative regimen, Arm B: Reduced Toxicity Ablative Regimen |
| CyclophosphamideAlso: Cytoxancyclophosphamide 50 mg/kg IV on days -9 through -6 | Drug | Arm A: Fully Myeloablative regimen |
| BusulfanAlso: Myerlanbusulfan 0.8 or 1.1 mg/kg IV on days -5 through -2 | Drug | Arm A: Fully Myeloablative regimen |
| Stem Cell TransplantationUnrelated donor bone marrow will be collected in the usual manner using established parameters determined by the National Marrow Donor Program. A minimum of 3 x 10^8 nucleated cells/kg recipient weight will be collected with a goal of ≥ 5 x 10^8 nucleated cells/kg recipient weight. Umbilical cord blood selection will be per the current University of Minnesota Cord Blood Unit Selection algorithm. One or two units may be used to obtain the minimum cell dose. One of the UCB units selected for transplantation must contain ≥ 3.5 x 10^7 nucleated cells/kg recipient weight based on cell numbers at time of cryopreservation, and the total combined cell dose of both units must be > 5.0 x 10^7 nucleated cells/kg. | Biological | Arm A: Fully Myeloablative regimen, Arm B: Reduced Toxicity Ablative Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen |
| Fludarabine phosphate 40 mgAlso: Fludara40 mg/m^2 IV on days -5 through -2 (for children < 6 months and/or < 10 kg weight dose at 1.33 mg/kg) | Drug | Arm B: Reduced Toxicity Ablative Regimen |
| MelphalanAlso: Alkeran140 mg/m^2 IV on day -3 | Drug | Arm C: Reduced Intensity Conditioning |
| Alemtuzumab 0.2 mgAlso: Campath 1-H0.2 mg/kg intravenously (IV) on days -14 through -10 | Drug | Arm C: Reduced Intensity Conditioning |
| BusulfanAlso: Myerlanbusulfan 0.8 or 1.1 mg/kg IV on days -9 through -6 | Drug | Arm B: Reduced Toxicity Ablative Regimen |
| Fludarabine phosphate 30 mgAlso: Fludarafludarabine 30 mg/m^2 IV on days -8 through -4 | Drug | Arm C: Reduced Intensity Conditioning |
| MESNAAlso: mercaptoethane sulfonate Na (Na being the symbol for sodium), Mesnexadministered as per the standard institutional protocol. | Drug | Arm A: Fully Myeloablative regimen |
Primary outcome
Neutrophil Engraftment
Time frame: Day 42
Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm3 (0.5 x 109/L) or greater.
7 secondary outcomes
Incidence of Graft Failure
Time frame: Day 100
Incidence of Chimerism
Time frame: Day 100, 6 Months, 1 Year
Incidence of Acute Graft-Versus-Host Disease
Time frame: Day 100
Incidence of Chronic Graft-Versus-Host Disease
Time frame: 6 Months and 1 Year
Incidence of Transplant-Related Mortality
Time frame: 6 Months
Disease-Free Survival
Time frame: 6 Months
Overall Survival
Time frame: 6 Months
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 17 Jun 2026.
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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.
Meera Srikanthan, MD principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Alemtuzumab 0.3 mg, Cyclophosphamide and Busulfan in people with SCID and Omenn's Syndrome, run by Masonic Cancer Center, University of Minnesota 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 28 inclusion and 3 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 17 Jun 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.
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?
Neutrophil Engraftment (time frame: Day 42). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Jun 2026.