A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition

Description

The researchers are doing this study to find out whether emapalumab or a combination of fludarabine and dexamethasone are effective in preparing people with a primary immune regulatory disorder (PIRD) and/or an autoinflammatory condition to receive a stem cell transplant. The researchers will look at how well the study treatments reduce inflammation and aid in the engraftment process (the process of donated stem cells traveling to the bone marrow, where they begin to make new immune cells. "Funding Source - FDA OOPD"

Conditions

Primary Immune Regulatory Disorder, Autoimmune Lymphoproliferative, Immune System Diseases

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Study Overview

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Study Details

Study overview

The researchers are doing this study to find out whether emapalumab or a combination of fludarabine and dexamethasone are effective in preparing people with a primary immune regulatory disorder (PIRD) and/or an autoinflammatory condition to receive a stem cell transplant. The researchers will look at how well the study treatments reduce inflammation and aid in the engraftment process (the process of donated stem cells traveling to the bone marrow, where they begin to make new immune cells. "Funding Source - FDA OOPD"

Phase 2 Study BRIDGING PRE-TRANSPLANT INFLAMMATORY DAMPENING for PRIMARY IMMUNE REGULATORY DISORDERS (BRIDGE Trial)

A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition

Condition
Primary Immune Regulatory Disorder
Intervention / Treatment

-

Contacts and Locations

San Francisco

University of California, San Francisco (Data collection only), San Francisco, California, United States, 94143

Atlanta

Children's Healthcare of Atlanta (Data Collection Only), Atlanta, Georgia, United States, 30322

Basking Ridge

Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities), Basking Ridge, New Jersey, United States, 07920

Middletown

Memorial Sloan Kettering Monmouth (Limited Protocol Activities), Middletown, New Jersey, United States, 07748

Montvale

Memorial Sloan Kettering Bergen (Limited Protocol Activities), Montvale, New Jersey, United States, 07645

Commack

Memorial Sloan Kettering Suffolk - Commack (Limited Protocol Activities), Commack, New York, United States, 11725

Harrison

Memorial Sloan Kettering Westchester (Limited Protocol Activities), Harrison, New York, United States, 10604

New York

Memorial Sloan Kettering Cancer Center, New York, New York, United States, 10065

Rockville Centre

Memorial Sloan Kettering Nassau (Limited Protocol Activities), Rockville Centre, New York, United States, 11553

Houston

Texas Children's Hospital (Data Collection), Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Patients receiving first allo-HCT for the following immunologic conditions:
  • * Primary Immune Regulatory Disorder with or without a genetic lesion as defined by the Primary Immune Deficiency Treatment Consortium (PIDTC)
  • * Patients with autoinflammatory disorders evidenced by cytokine or inflammation assays with at least 1.5x ULN of measured cytokines and/or an elevated ferritin or ESR \> 2 ULN
  • * For inclusion on the emapalumab group, the lesion must be isolated to the IFNγ pathway (or mediators thereof) with an elevated CXCL9 \>1.5 ULN OR sIL2R \>1.5 ULN (or already controlled on immune modulation, provided that CXCL9 or sIL2R levels were elevated prior to initiation of immune modulation). Inclusion on the Fludarabine/dexamethasone group requires inflammation (as defined above) other than an isolated IFNγ pathway
  • * Able to tolerate cytoreduction (based on adequate organ function as described below)
  • * Patients of any age can enroll so long as they meet other
  • * Uncontrolled infection at the time of enrollment.
  • * Patients who have undergone previous allo-HCT.
  • * Patient seropositivity for HIV I/II and/or HTLV I/II.
  • * Females who are pregnant or breastfeeding.
  • * Patients unwilling to use contraception during the study period.
  • * Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Memorial Sloan Kettering Cancer Center,

Joseph Oved, MD, PRINCIPAL_INVESTIGATOR, Memorial Sloan Kettering Cancer Center

Study Record Dates

2027-03