Middletown, NJNCT05787574Now EnrollingIRB Ready

Primary Immune Deficiency Clinical Trial in Middletown, NJ

Access cutting-edge primary immune deficiency treatment through this phase 2 clinical trial at a research site in Middletown. Study-provided care at no cost to qualified participants.

Sponsored by Memorial Sloan Kettering Cancer Center

Quick Self-Assessment

See if you qualify for this Middletown location

Preparing your pre-screening questions…

Expert Care in Middletown

Access primary immune deficiency specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related primary immune deficiency treatment provided free

Apply for This Middletown Location

Check if you qualify for this primary immune deficiency clinical trial in Middletown, NJ

Secure & Confidential

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

Why Participate?

  • No-Cost Study Care

  • Local to Middletown

    Convenient for NJ residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Middletown site if eligible
  4. 4Begin participation

About This Primary Immune Deficiency Study in Middletown

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. "Fun

Sponsor: Memorial Sloan Kettering Cancer Center

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)12 - Patients with autoinflammatory disorders evidenced by cytokine or inflammation assays with at least 1.5x ULN of measured cytokines (defined in section 4) and/or an elevated ferritin or ESR \> 2 ULN
For inclusion on the emapalumab group, the lesion must be related 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).
Able to tolerate cytoreduction (based on adequate organ function as described below)
Patients of any age can enroll so long as they meet all other eligibility criteria
Adequate organ function is required, defined as follows:
Hepatic: Serum bilirubin ≤ 2 mg/dL, unless benign congenital hyperbilirubinemia. Patients with hyperbilirubinemia related to paroxysmal nocturnal hemoglobinuria or other hemolytic disorders related to their PIRD diagnosis are eligible.
Hepatic: AST, ALT, and alkaline phosphatase \< 2.5 times the upper limit of normal unless thought to be disease-related. Investigator will need to perform clinically indicated evaluations to assess if disease related or intrinsic liver disease. Additional testing may be done if clinically indicated, after the pre-transplant immune prophase and prior to start of conditioning as this will provide additional data to confirm disease related versus intrinsic liver dysfunction.
Renal: serum creatinine \<1.5x normal for age. If serum creatinine is outside the normal range, then CrCl \> 50 mL/min/1.73m\^2 (calculated or estimated) or GFR (mL/min/1.72m\^2) \>30% of predicted normal for age.
Cardiac: LVEF ≥ 50% by MUGA or resting echocardiogram.
Pulmonary: Pulmonary function testing (FEV1 and corrected DLCO) ≥ 50% predicted (pediatric patients unable to complete PFTs will need oxygen saturation as recorded by pulse oximetry of ≥92% on room air).
Adequate performance status:
Age ≥ 16 years: ECOG ≤ 1 or Karnofsky ≥ 70%
Age \< 16 years: Lansky 70%
Each patient must be willing to participate as a research subject and must sign an informed consent form or legal guardian with assent as appropriate. Donor Inclusion Criteria:
Related Donors:
8/8 or 7/8 HLA matched at A, B, C, and DRB1 loci, as tested by DNA analysis.
Haploidentical donors at A, B, C and DRB1 loci, as tested by DNA analysis
Unrelated Donors:
8/8 or 7/8 matched at A, B, C, and DRB1 loci, as tested by DNA analysis.
Able to provide informed consent for the donation process per institutional standards.
Meet standard criteria for donor collection (e.g. National Marrow Donor Program Guidelines or collecting center guidelines as approved by treating physician).

Exclusion Criteria

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.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Middletown?

Yes, this clinical trial (NCT05787574) has an active research site in Middletown, NJ that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

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

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

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

Primary Immune Deficiency Treatment Options in Middletown, NJ

If you're searching for primary immune deficiency treatment options in Middletown, NJ, this clinical trial (NCT05787574) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Middletown research site is actively enrolling participants for this phase 2 clinical trial. You'll receive care from experienced primary immune deficiency specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all primary immune deficiency clinical trials near you to find additional studies recruiting in your area.

More Multiple Myeloma Trials in Middletown, NJ

See all multiple myeloma clinical trials recruiting in Middletown — not just this study.

Browse Multiple Myeloma Trials in Middletown →

Browse More Trials by Condition

Multiple Myeloma TrialsInterstitial Cystitis TrialsAll Conditions →

Ready to Join in Middletown?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Middletown, NJ