An interventional study of Alemtuzumab 0.3 mg and Cyclophosphamide in SCID, Omenn's Syndrome and Reticular Dysgenesis, sponsored by Masonic Cancer Center, University of Minnesota. Active, not recruiting at 1 site in United States. Open to participants aged Up to 50 Years. Per ClinicalTrials.gov, last updated 2026-06-17.
Sponsored by Masonic Cancer Center, University of Minnesota · Not applicable, Interventional, and Treatment
This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
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
59 studies on the registry are indexed under Severe Combined Immunodeficiency; 14 are open to participants now.
This study's enrollment of 57 is above the median of 10 across 31 interventional studies indexed under Severe Combined Immunodeficiency.
Browse Severe Combined Immunodeficiency studies →Masonic Cancer Center, University of Minnesota is the lead sponsor of 286 studies on the registry; 35 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Diagnosis of immunodeficiency or histiocytic disorder including the following:
Acceptable stem cell sources include:
Double unrelated umbilical cord blood units that are:
Exclusion Criteria
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.
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in 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.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
CD4 count
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Drug: Alemtuzumab 0.3 mg · Drug: Cyclophosphamide · Drug: Busulfan · Biological: Stem Cell Transplantation · Drug: MESNA
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.
Drug: Alemtuzumab 0.3 mg · Biological: Stem Cell Transplantation · Drug: Fludarabine phosphate 40 mg · Drug: Busulfan
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.
Biological: Stem Cell Transplantation · Drug: Melphalan · Drug: Alemtuzumab 0.2 mg · Drug: Fludarabine phosphate 30 mg
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.
Biological: Stem Cell Transplantation
0.3 mg/kg intravenously (IV) on days -12 through -10
Also known as: Campath-1H
cyclophosphamide 50 mg/kg IV on days -9 through -6
Also known as: Cytoxan
busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2
Also known as: Myerlan
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.
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)
Also known as: Fludara
140 mg/m\^2 IV on day -3
Also known as: Alkeran
0.2 mg/kg intravenously (IV) on days -14 through -10
Also known as: Campath 1-H
busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6
Also known as: Myerlan
fludarabine 30 mg/m\^2 IV on days -8 through -4
Also known as: Fludara
administered as per the standard institutional protocol.
Also known as: mercaptoethane sulfonate Na (Na being the symbol for sodium), Mesnex
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.
Time frame: Day 42
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 6 Months
This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drugs tested here: Alemtuzumab · Cyclophosphamide · Busulfan · Fludarabine phosphate · Melphalan · Mesna
Severe Combined Immunodeficiency →
Masonic Cancer Center, University of Minnesota →