What side effects are associated with this type of intervention?

The most common treatments for Severe Combined Immunodeficiency (SCID) include lung transplantation and hematopoietic stem cell transplantation (HSCT). Lung transplantation can lead to side effects such as infection, rejection of the transplanted organ, and complications from immunosuppressive medications, including increased risk of malignancies and kidney damage. Hematopoietic stem cell transplantation (HSCT) may cause graft-versus-host disease (GVHD), infections due to immunosuppression, and organ damage, particularly to the liver and lungs. Both treatments require careful monitoring and management to mitigate these risks.

What prior FDA approvals exist?

The FDA has approved several treatments for Severe Combined Immunodeficiency (SCID), primarily focusing on hematopoietic stem cell transplantation (HSCT) as a curative approach. HSCT involves the transplantation of stem cells to reconstitute the immune system and has been a cornerstone in SCID treatment. While there are no specific FDA approvals for the combination of Bilateral Orthotopic Lung Transplantation (BOLT) followed by Cadaveric Partially-Matched HSCT, the use of HSCT alone has been well-documented and approved for SCID. This approach aims to restore immune function in patients with this severe immunodeficiency.

What other types of research exist?

Promising novel alternatives to BOLT followed by Cadaveric Partially-Matched HSCT for SCID patients include: 1) Gene therapy, which aims to correct the genetic defect causing SCID, potentially eliminating the need for HSCT. 2) Ex vivo lung perfusion and reconditioning, which can improve the quality and availability of donor lungs, potentially reducing the need for BOLT. 3) Use of haploidentical family member donors or umbilical cord blood for HSCT, which may offer similar outcomes to traditional donor sources with potentially fewer complications.

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Hematopoietic Stem Cell Transplantation

Stem Cell Transplant for Immunodeficiency (BOLT+BMT Trial)

Phase 1 & 2

Waitlist Available

Led By Paul Szabolcs, MD

Research Sponsored by Paul Szabolcs

Eligibility Criteria Checklist

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

Must have

GFR ≥ 50 mL/min/1.73 m2.

Male or female, 5 through 45 years old, inclusive, at the time of informed consent.

Must not have

Uncontrolled pulmonary infection, as determined by radiographic findings and/or significant clinical deterioration.

Females who are pregnant or who are lactating.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years post stem cell transplant

Awards & highlights

No Placebo-Only Group

Summary

This study is evaluating whether a lung transplant followed by a stem cell transplant is safe and effective for people with primary immunodeficiency.

Who is the study for?

This trial is for people aged 5-45 with primary immunodeficiency and severe lung disease needing a lung transplant. They must have normal liver function, heart function, kidney function, not be pregnant or HIV positive, and agree to birth control post-transplant.

What is being tested?

The study tests if getting new lungs (BOLT) followed by stem cell transplant from a partially-matched donor is safe/effective for immune deficiency diseases. Participants receive CD3/CD19 negative allogeneic hematopoietic stem cells.

What are the potential side effects?

Potential side effects may include reactions to the stem cell product due to DMSO or other ingredients used in manufacturing, infertility risks post bone marrow transplant (BMT), and complications from underlying conditions.

Eligibility Criteria

Inclusion Criteria

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

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My kidney function is good.

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I am between 5 and 45 years old.

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I have a genetic immune disorder that requires a bone marrow transplant.

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I am eligible for a double lung transplant due to end-stage lung disease.

Exclusion Criteria

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

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I do not have an uncontrolled lung infection.

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I am not pregnant or breastfeeding.

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I have cancer.

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I have a condition that does not require a stem cell transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years post stem cell transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years post stem cell transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years post stem cell transplant for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Efficacy: B-cell chimerism

Efficacy: BOS score

Efficacy: Myeloid chimerism

+5 more

Secondary study objectives

Ability to withdraw immunosuppression

Acute cellular rejection

Acute graft-versus-host disease (GVHD)

+11 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: BOLT+BMTExperimental Treatment1 Intervention

All patients will receive a double lung transplant followed by a hematopoietic stem cell transplant. The lungs and stem cells are from the same partially HLA-matched cadaveric donor. Prior to transplantation, the marrow will be negatively selected for CD3/CD19 using a CliniMACS® depletion device.

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Severe Combined Immunodeficiency (SCID) treatments primarily focus on restoring immune function through Hematopoietic Stem Cell Transplantation (HSCT), where healthy donor stem cells are transplanted to reconstitute the patient's immune system. This is vital for SCID patients as it provides them with functional immune cells to combat infections. The trial involving Bilateral Orthotopic Lung Transplantation followed by Cadaveric Partially-Matched HSCT is particularly important as it not only replaces diseased lungs but also reconstitutes the immune system, addressing both immediate and long-term health needs of SCID patients.