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Stem Cell Transplantation

Stem Cell Transplant for Primary Immunodeficiency

N/A

Recruiting

Led By Christen Ebens, MD

Research Sponsored by Masonic Cancer Center, University of Minnesota

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age: 0 to 50 years

Diagnosis of immunodeficiency or histiocytic disorder including specific conditions such as Severe combined immunodeficiency (SCID), Omenn's Syndrome, Wiskott-Aldrich syndrome, Chronic Granulomatous Disease (CGD), Hemophagocytic Lymphohistiocytosis (HLH), and others

Must not have

Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy

Active, uncontrolled infection and/or HIV positive

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months and 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial provides guidance for the best way to treat patients with primary immune deficiencies using allogeneic HSCT.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects include reactions to the infusion of cells or drugs used in the process (like chills or fever), damage to organs from chemotherapy agents (such as heart or lung problems), risk of infection due to weakened immunity post-transplantation and graft-versus-host disease where transplanted cells attack your body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 50 years old or younger.

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I have been diagnosed with an immune system disorder like SCID or CGD.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have severe liver damage or hepatitis.

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I do not have an active, uncontrolled infection or HIV.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months and 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months and 1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Neutrophil Engraftment

Secondary study objectives

Disease-Free Survival

Incidence of Acute Graft-Versus-Host Disease

Incidence of Chimerism

+4 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: Arm D: No Preparative RegimenExperimental Treatment1 Intervention

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.

Group II: Arm C: Reduced Intensity ConditioningExperimental Treatment4 Interventions

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.

Group III: Arm B: Reduced Toxicity Ablative RegimenExperimental Treatment4 Interventions

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.

Group IV: Arm A: Fully Myeloablative regimenExperimental Treatment5 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Stem Cell Transplantation

2003

Completed Phase 3

~610

Melphalan

2008

Completed Phase 3

~1500