30 Participants Needed

Stem Cell Transplant for Primary Immunodeficiency

AR
Christen Ebens, MD profile photo
Overseen ByChristen Ebens, MD
Age: < 65
Sex: Any
Trial Phase: Academic
Sponsor: Masonic Cancer Center, University of Minnesota
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 6 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is stem cell transplant generally safe for treating primary immunodeficiency?

Stem cell transplants, specifically allogeneic hematopoietic stem cell transplants, have been shown to be generally safe and effective for treating primary immunodeficiency in both children and adults. While there are risks such as graft-versus-host disease (a condition where the donor cells attack the recipient's body) and graft failure, survival rates are high, and safety has improved over the years with advancements in treatment protocols.12345

How is the treatment Allogeneic Hematopoietic Stem Cell Transplant unique for primary immunodeficiency?

Allogeneic Hematopoietic Stem Cell Transplant is unique because it can potentially cure primary immunodeficiencies by replacing the patient's faulty immune system with healthy stem cells from a donor. This treatment is especially important when no matched donor is available, as it allows for the use of partially matched (haploidentical) donors, expanding the pool of potential donors.16789

What data supports the effectiveness of the treatment Allogeneic Hematopoietic Stem Cell Transplant for Primary Immunodeficiency?

Research shows that allogeneic hematopoietic stem cell transplant (a procedure where healthy stem cells are transferred from a donor to a patient) can cure many primary immunodeficiency disorders, with an 80% success rate when using a matched sibling donor. This treatment has significantly improved the prognosis for patients with these conditions.110111213

Who Is on the Research Team?

Christen L Ebens - Associate Professor ...

Christen Ebens, MD

Principal Investigator

Masonic Cancer Center, University of Minnesota

Are You a Good Fit for This Trial?

This trial is for people aged 0-50 with primary immune deficiencies or histiocytic disorders, such as SCID, Omenn's Syndrome, Wiskott-Aldrich syndrome. Participants need a suitable stem cell donor and good organ function. Those with acute hepatitis, severe liver issues, active infections including HIV, or who are pregnant/breastfeeding cannot join.

Inclusion Criteria

I am 50 years old or younger.
I have been diagnosed with an immune system disorder like SCID or CGD.
I have a suitable stem cell donor or cord blood unit that matches me.
See 1 more

Exclusion Criteria

Pregnant or breastfeeding
I have severe liver damage or hepatitis.
I do not have an active, uncontrolled infection or HIV.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Preparative Regimen

Participants receive a preparative regimen based on their condition, which may include myeloablative, reduced toxicity, or reduced intensity conditioning, followed by stem cell infusion on day 0.

2 weeks

Engraftment Monitoring

Participants are monitored for neutrophil engraftment, defined as the first day of three consecutive days where the neutrophil count is 500 cells/mm3 or greater.

6 weeks

Follow-up

Participants are monitored for safety and effectiveness, including incidence of acute and chronic Graft-Versus-Host Disease, overall survival, and transplant-related mortality.

6 months

Long-term Follow-up

Participants are monitored for long-term outcomes such as disease-free survival and incidence of chronic Graft-Versus-Host Disease.

1 year

What Are the Treatments Tested in This Trial?

Interventions

  • Allogeneic Hematopoetic Stem Cell Transplant
Trial Overview The trial tests a standard care approach using allogeneic hematopoietic stem cell transplant (HSCT) combined with drugs like Alemtuzumab at different doses, Melphalan, Busulfan, Cyclophosphamide and Fludarabine phosphate to treat various primary immune deficiencies.
How Is the Trial Designed?
4Treatment groups
Experimental Treatment
Group I: Arm D: No Preparative RegimenExperimental Treatment1 Intervention
Group II: Arm C: Reduced Intensity ConditioningExperimental Treatment4 Interventions
Group III: Arm B: Reduced Toxicity Ablative RegimenExperimental Treatment4 Interventions
Group IV: Arm A: Fully Myeloablative regimenExperimental Treatment5 Interventions

Allogeneic Hematopoetic Stem Cell Transplant is already approved in European Union, United States, Canada, Japan, China, Switzerland for the following indications:

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Approved in European Union as Allogeneic Hematopoietic Stem Cell Transplant for:
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Approved in United States as Allogeneic Hematopoietic Stem Cell Transplant for:
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Approved in Canada as Allogeneic Hematopoietic Stem Cell Transplant for:
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Approved in Japan as Allogeneic Hematopoietic Stem Cell Transplant for:
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Approved in China as Allogeneic Hematopoietic Stem Cell Transplant for:
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Approved in Switzerland as Allogeneic Hematopoietic Stem Cell Transplant for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Masonic Cancer Center, University of Minnesota

Lead Sponsor

Trials
285
Recruited
15,700+

Published Research Related to This Trial

In a study of seven pediatric patients with various primary immunodeficiencies who underwent eight hematopoietic stem cell transplants, the status of patients before the transplant was identified as the most critical factor influencing outcomes, highlighting the importance of early diagnosis and timely referral for treatment.
Out of the seven patients, four survived and are doing well after the transplant, while complications such as graft failure and regimen-related toxicity were significant challenges, indicating that while the procedure can be effective, careful management of pre-existing conditions is essential for improving survival rates.
Allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiencies: Hospital Israelita Albert Einstein experience.Fernandes, JF., Kerbauy, FR., Ribeiro, AA., et al.[2016]
In a study of 20 patients with primary immunodeficiency diseases, hematopoietic stem cell transplants resulted in a 65% overall survival rate, indicating a promising outcome for this treatment.
The median engraftment times for neutrophils and platelets were 14.25 days and 24.7 days, respectively, with graft-versus-host disease occurring in 30% of patients, highlighting both the efficacy and potential complications of the procedure.
Hematopoietic Stem Cell Transplant for Primary Immunodeficiency Diseases: A Single-Center Experience.Patiroglu, T., Akar, HH., Unal, E., et al.[2018]
Allogeneic hematopoietic stem cell transplantation (HSCT) is an established curative treatment for primary immunodeficiencies, particularly effective when applied early in the disease course to prevent severe complications from infections.
Recent analyses of large patient cohorts indicate that HSCT has led to significant improvements in outcomes and prognosis for patients with disorders like severe combined immunodeficiency (SCID).
Stem cell transplantation for treatment of primary immunodeficiency disorders.Mรผller, SM., Friedrich, W.[2007]

Citations

Allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiencies: Hospital Israelita Albert Einstein experience. [2016]
Hematopoietic Stem Cell Transplant for Primary Immunodeficiency Diseases: A Single-Center Experience. [2018]
Stem cell transplantation for treatment of primary immunodeficiency disorders. [2007]
Stem cell transplantation for primary immunodeficiencies: King Faisal Specialist Hospital experience from 1993 to 2006. [2008]
Allogeneic stem cell transplantation for treatment of immunodeficiency. [2018]
Curative allogeneic hematopoietic stem cell transplantation following reduced toxicity conditioning in adults with primary immunodeficiency. [2022]
Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience. [2023]
Successful outcome following allogeneic hematopoietic stem cell transplantation in adults with primary immunodeficiency. [2022]
Ethical considerations of using a single minor donor for three bone marrow harvests for three HLA-matched siblings with primary immunodeficiency. [2019]
Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. [2019]
11.United Statespubmed.ncbi.nlm.nih.gov
T-cell receptor &#945;&#946;+ and CD19+ cell-depleted haploidentical and mismatched hematopoietic stem cell transplantation in primary immune deficiency. [2022]
[Hematopoietic stem cell transplantation in children with primary immunodeficiencies]. [2009]
13.United Statespubmed.ncbi.nlm.nih.gov
Kinetics of T-cell development of umbilical cord blood transplantation in severe T-cell immunodeficiency disorders. [2019]
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