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New treatment option for Primary Immune Regulatory Disorder
Official title A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition
ClinicalTrials.gov ID: NCT05787574
What this study is testing
What is Emapalumab?
Emapalumab is an investigational medicine, being studied as a potential treatment for primary immune regulatory disorder.
Also referred to as Gamifant.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Adults
You may be able to join if
- 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 -...
- 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...
- Inclusion on the Fludarabine/dexamethasone group requires inflammation (as defined above) other than an IFNy pathway defect
- Able to tolerate cytoreduction (based on adequate organ function as described below)
You likely can't join if
- 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.
See the full 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)12 - Patients with autoinflammatory disorders evidenced by cytokine or...
- 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...
- Inclusion on the Fludarabine/dexamethasone group requires inflammation (as defined above) other than an IFNy pathway defect
- 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...
- 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...
- Renal: serum creatinine \ 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
- 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).
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- San Francisco, California, United States
- Atlanta, Georgia, United States
- Basking Ridge, New Jersey, United States
- Middletown, New Jersey, United States
- Montvale, New Jersey, United States
- Commack, New York, United States
- Harrison, New York, United States
- New York, New York, United States
- Rockville Centre, New York, United States
- Philadelphia, Pennsylvania, United States
- Houston, Texas, United States
- Milwaukee, Wisconsin, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, not specified. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Francisco, California, United States; Atlanta, Georgia, United States; Basking Ridge, New Jersey, United States; Middletown, New Jersey, United States; Montvale, New Jersey, United States; Commack, New York, United States and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.