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Trial record 15 of 65 for:    Recruiting, Not yet recruiting Studies | autoinflammatory
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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05787574
Recruitment Status : Recruiting
First Posted : March 28, 2023
Last Update Posted : January 30, 2024
Sponsor:
Collaborator:
Sobi, Inc.
Information provided by (Responsible Party):
Memorial Sloan Kettering Cancer Center

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Primary Immune Regulatory Disorder Autoimmune Lymphoproliferative Immune System Diseases Drug: Emapalumab Drug: Fludarabine and Dexamethasone Procedure: Stem Cell Transplant Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 39 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: This is a two-stage phase 2 study.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Study BRIDGING PRE-TRANSPLANT INFLAMMATORY DAMPENING for PRIMARY IMMUNE REGULATORY DISORDERS (BRIDGE Trial)
Actual Study Start Date : March 15, 2023
Estimated Primary Completion Date : March 2027
Estimated Study Completion Date : March 2027

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Group A: Emapalumab (for isolated Interferongamma mediated disease)
Participants in this group will receive emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1.
 Drug: Emapalumab
Emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1.

Procedure: Stem Cell Transplant
Participants in both groups will receive their standard-of-care stem cell transplant on Day 0.
Experimental: Group B: Fludarabine and Dexamethasone (for generalized autoinflammation)
Participants in this group will receive fludarabine and dexamethasone for 5 days in a row on Days -22 through -18.
 Drug: Fludarabine and Dexamethasone
Fludarabine and dexamethasone for 5 days in a row on Days -22 through -18.

Procedure: Stem Cell Transplant
Participants in both groups will receive their standard-of-care stem cell transplant on Day 0.


Outcome Measures
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Primary Outcome Measures :
  1. Engraftment [ Time Frame: 1 year ]
    is defined as the first of three days of absolute neutrophil count >500k/µL and the first of seven days of platelets >20,000/µL in the absence of transfusional support.


Secondary Outcome Measures :
  1. Determine Overall Survival (OS) [ Time Frame: 5 years ]
    the duration of time between HCT and death due to any cause


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 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 inclusion 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/1.73m2 (calculated or estimated) or GFR (mL/min/1.72m2) >30% of predicted normal for age.
    • Normal GFR by 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.

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.

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:

    o 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).
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05787574


Contacts
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Contact: Joseph Oved, MD 646-888-3314 ovedj@mskcc.org
Contact: Roni Tamari, MD 646-608-3738

Locations
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United States, New Jersey
Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities) Recruiting
Basking Ridge, New Jersey, United States, 07920
Contact: Joseph Oved, MD    646-888-3314      
Memorial Sloan Kettering Monmouth (Limited Protocol Activities) Recruiting
Middletown, New Jersey, United States, 07748
Contact: Joseph Oved, MD    646-888-3314      
Memorial Sloan Kettering Bergen (Limited Protocol Activities) Recruiting
Montvale, New Jersey, United States, 07645
Contact: Joseph Oved, MD    646-888-3314      
United States, New York
Memorial Sloan Kettering Suffolk - Commack (Limited Protocol Activities) Recruiting
Commack, New York, United States, 11725
Contact: Joseph Oved, MD    646-888-3314      
Memorial Sloan Kettering Westchester (Limited Protocol Activities) Recruiting
Harrison, New York, United States, 10604
Contact: Joseph Oved, MD    646-888-3314      
Memorial Sloan Kettering Cancer Center Recruiting
New York, New York, United States, 10065
Contact: Joseph Oved, MD    646-888-3314      
Contact: Jaap Jan Boelens, MD, PhD    212-639-3643      
Memorial Sloan Kettering Nassau (Limited Protocol Activities) Recruiting
Rockville Centre, New York, United States, 11553
Contact: Joseph Oved, MD    646-888-3314      
Sponsors and Collaborators
Memorial Sloan Kettering Cancer Center
Sobi, Inc.
Investigators
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Principal Investigator: Joseph Oved, MD Memorial Sloan Kettering Cancer Center
More Information
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Additional Information:

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Responsible Party: Memorial Sloan Kettering Cancer Center
ClinicalTrials.gov Identifier: NCT05787574    
Other Study ID Numbers: 23-040
First Posted: March 28, 2023    Key Record Dates
Last Update Posted: January 30, 2024
Last Verified: January 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Memorial Sloan Kettering Cancer Center:
Stem Cell Transplant
Emapalumab
Fludarabine
Dexamethasone
23-040
Additional relevant MeSH terms:
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Immune System Diseases
Dexamethasone
Fludarabine
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
 Physiological Effects of Drugs
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents