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Surgery

Lung + Bone Marrow Transplants for Primary Immunodeficiency (BOLT-BMT Trial)

Phase 1 & 2

Waitlist Available

Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)

Eligibility Criteria Checklist

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

Must have

Subject fulfills criteria for United Network of Organ Sharing (UNOS) listing

Subject must have evidence of an underlying primary immunodeficiency for which Bone Marrow Transplant (BMT) is clinically indicated. Examples of such diseases include, but are not limited to: Severe Combined Immunodeficiency (SCID), Combined immunodeficiency with defects in T-cell-mediated immunity, including Omenn syndrome and DiGeorge Syndrome, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD), Hyper Immunoglobulin E (IgE) Syndrome or Job's Syndrome, CD40 or CD40L deficiency, Wiskott-Aldrich Syndrome, Mendelian Susceptibility to Mycobacterial Disease, GATA2-associated Immunodeficiency

Timeline

Screening 3 weeks

Treatment Varies

Follow Up within 6 months post lung transplant (bolt)

Awards & highlights

BOLT-BMT Trial Summary

This trial is testing whether a combination of two transplant procedures is safe and effective for people with primary immunodeficiency and end-stage lung disease.

Who is the study for?

This trial is for individuals aged 10-45 with primary immunodeficiency and severe lung disease needing a lung transplant. They must meet organ sharing criteria, have certain normal blood tests, agree to birth control methods post-transplant, and be able to consent. Excluded are those with cancer, HIV/HTLV, pregnancy/lactation, allergy to stem cell product ingredients or uncontrolled infections.Check my eligibility

What is being tested?

The study is testing the safety and effectiveness of a new treatment approach combining bilateral orthotopic lung transplantation (BOLT) with bone marrow transplant (BMT) that uses partially-matched cadaveric donors whose cells lack CD3+/CD19+ markers. The goal is to enroll 8 participants who will undergo both procedures.See study design

What are the potential side effects?

Potential side effects may include reactions related to the immune system due to the transplants such as graft-versus-host disease, infection risks from immunosuppression needed after BMT, infertility risks associated with BMT procedure and possible complications from surgery like bleeding or clotting.

BOLT-BMT Trial Eligibility Criteria

Inclusion Criteria

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

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I meet the criteria to be on the organ transplant list.

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I have a primary immunodeficiency 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 ~ within 6 months post lung transplant (bolt)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~within 6 months post lung transplant (bolt)

This trial's timeline: 3 weeks for screening, Varies for treatment, and within 6 months post lung transplant (bolt) 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: Count of Participants B-cell chimerism

Efficacy: Count of Participants with Absence of Severe Allograft Dysfunction

Efficacy: Count of Participants with Myeloid Chimerism

+4 more

Secondary outcome measures

Count of Participants Able to Initiate Withdrawal of Immunosuppression

Count of Participants Able to Proceed to BMT

Count of Participants Who Achieve Tolerance

+11 more

Other outcome measures

EXPLORATORY: Change in Immunologic Markers

EXPLORATORY: Change in Markers of Immune Reconstitution

EXPLORATORY: Count of Participants with Mixed Chimerism EXPLORATORY: Count of Participants with Mixed Chimerism

BOLT-BMT Trial Design

1Treatment groups

Experimental Treatment

Group I: CD3/CD19 neg allogeneic BMTExperimental Treatment1 Intervention

All participants will receive a double lung transplant followed by a bone marrow (hematopoietic stem cells) transplant. The lungs and allogeneic hematopoietic stem cells will be 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

CD3/CD19 neg allogeneic BMT

2017

Completed Phase 2

~10

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

PPDIndustry Sponsor

159 Previous Clinical Trials

36,910 Total Patients Enrolled

University of PittsburghOTHER

1,719 Previous Clinical Trials

16,342,336 Total Patients Enrolled

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor

3,269 Previous Clinical Trials

5,481,464 Total Patients Enrolled

Media Library

Bilateral Orthotopic Lung Transplant (Surgery) Clinical Trial Eligibility Overview. Trial Name: NCT03330795 — Phase 1 & 2

Primary Immunodeficiency Research Study Groups: CD3/CD19 neg allogeneic BMT

Primary Immunodeficiency Clinical Trial 2023: Bilateral Orthotopic Lung Transplant Highlights & Side Effects. Trial Name: NCT03330795 — Phase 1 & 2

Bilateral Orthotopic Lung Transplant (Surgery) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03330795 — Phase 1 & 2

Frequently Asked Questions

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

Who is eligible to participate in this research endeavor?

"This trial is in search of 5 patients aged 10-45 with pid, who also meet the United Network of Organ Sharing (UNOS) listing criteria. Furthermore, combined immunodeficiency involving T-cell mediated immunity—including Omenn and DiGeorge Syndrome, as well as CD40 or CD40L deficiency—is necessary for eligibility."

Answered by AI

What aims are being sought through this clinical trial's data collection?

"The objectives of this trial, to be measured over a period of two years after the Bone Marrow Transplant (BMT), are assessing Safety: Engraftment Failure. Secondary aims include examining Lymphocyte Count for T-cell Lymphopenias, which is determining if participants reach age adjustedly low normal lymphocyte count 1 year following BMT; calculating how many patients achieve tolerance by withdrawing from systemic immunosuppression without increasing cGVHD score and stable or improving PFTs; and counting the number of individuals who can proceed to BMT within 6 months after their lung transplant."

Answered by AI

Does this clinical trial extend to geriatric patients?

"This study is strictly limited to patients aged 10-45."

Answered by AI

Are there any openings for prospective participants in this experiment?

"According to clinicaltrials.gov, this study is not actively seeking participants at the present moment. Initially posted on December 1st 2017 and last updated October 6th 2021, it appears that recruitment has been completed for now although there are still 85 other trials recruiting patients."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant

2017. "Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03330795.