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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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years post stem cell transplant

Awards & highlights

BOLT+BMT Trial Summary

This trial will test whether a combination of two transplants can improve quality of life for people with primary immunodeficiency and end-stage lung disease.

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.Check my eligibility

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.See study design

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.

BOLT+BMT Trial 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.

BOLT+BMT Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Efficacy: B-cell chimerism

Efficacy: BOS score

Efficacy: Myeloid chimerism

+5 more

Secondary outcome measures

Ability to withdraw immunosuppression

Acute cellular rejection

Acute graft-versus-host disease (GVHD)

+11 more

BOLT+BMT 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 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Paul SzabolcsLead Sponsor

7 Previous Clinical Trials

216 Total Patients Enrolled

Paul Szabolcs, MDPrincipal InvestigatorDivision of BMT and Cellular Therapy, Children's Hospital of Pittsburgh of UPMC

7 Previous Clinical Trials

213 Total Patients Enrolled

Media Library

CD3/CD19 negative allogeneic hematopoietic stem cells (Hematopoietic Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT01852370 — Phase 1 & 2

Severe Combined Immunodeficiency Research Study Groups: BOLT+BMT

Severe Combined Immunodeficiency Clinical Trial 2023: CD3/CD19 negative allogeneic hematopoietic stem cells Highlights & Side Effects. Trial Name: NCT01852370 — Phase 1 & 2

CD3/CD19 negative allogeneic hematopoietic stem cells (Hematopoietic Stem Cell Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01852370 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the age ceiling for this trial set at 85 years or lower?

"To be considered for this experiment, the applicant must fall between 5 and 45 years old. There are 449 medical studies available to minors and 1085 suitable trials for seniors."

Answered by AI

What are the ambitions of this medical trial?

"This trial intends to assess the efficacy of T-cell chimerism through two years post stem cell transplant. Secondary objectives include evaluating time until immunosuppression withdrawal, measuring rituximab-related adverse events, and counting allograft failures in patients who underwent a lung or combined lung+stem cell transplant."

Answered by AI

Are there any open enrollment opportunities for this investigation?

"According to information on clinicaltrials.gov, the recruitment effort for this experiment is currently closed; it was first posted in June of 2013 and last modified in November 2022. Nevertheless, there are over 1,500 other studies actively enrolling patients at present."

Answered by AI

Is it feasible for me to join this research endeavor?

"This trial is recruiting 16 individuals with wiskott-aldrich syndrome within the ages of 5 and 45 to participate. Eligibility requirements are based on these criteria."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

Paul Szabolcs 2013. "Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01852370.

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