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

Sponsored by Memorial Sloan Kettering Cancer Center

Quick Self-Assessment

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Current Status

Recruiting

Auto-verified from ClinicalTrials.gov

Verified Oct 29, 2025

Phase

Clinical Trial

Sponsor

Memorial Sloan Kettering Cancer Center

Enrollment Target

39

Start Date

Mar 2023

๐Ÿ“Š
1 of 39 participants interested
3% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

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.

Who Can Participate

Inclusion 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 IFNฮณ 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 IFNฮณ 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 inclus

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

๐Ÿ“‹

Study Type

INTERVENTIONAL

๐Ÿ‘ฅ

Target Enrollment

39 participants

โšง

Gender

ALL

๐Ÿข

Sponsor

Memorial Sloan Kettering Cancer Center

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

San Francisco

California

Location available
View San Francisco location page
RECRUITING

Atlanta

Georgia

Location available
View Atlanta location page
RECRUITING

Basking Ridge

New Jersey

Location available
View Basking Ridge location page
RECRUITING

Middletown

New Jersey

Location available
View Middletown location page
RECRUITING

Montvale

New Jersey

Location available
View Montvale location page
RECRUITING

Commack

New York

Location available
View Commack location page
RECRUITING

Harrison

New York

Location available
View Harrison location page
RECRUITING

New York

New York

Location available
View New York location page
RECRUITING

Rockville Centre

New York

Location available
View Rockville Centre location page

๐Ÿ“ And 3 more locations available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

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Looking for Primary Immune Deficiency Treatment in San Francisco?

Join others in California exploring innovative treatment options through clinical research

Primary Immune Deficiency Treatment Options in San Francisco, California

If you're searching for Primary Immune Deficiency treatment in San Francisco, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in San Francisco, Atlanta, Basking Ridge and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Primary Immune Deficiency. All study-related care is provided at no cost to participants.

Local Sites
3 locations in California
Now Enrolling
Up to 39 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Primary Immune Deficiency?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Primary Immune Deficiency

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Primary Immune Deficiency Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT05787574. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.