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

Part of paid clinical trials in San Francisco, California.

Sponsor
Joseph Oved
Study ID
NCT05787574
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Autoimmune Lymphoproliferative
  • Immune System Diseases
  • Primary Immune Regulatory Disorder

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Emapalumab — DRUG
    Emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1.
  • Fludarabine and Dexamethasone — DRUG
    Fludarabine and dexamethasone for 5 days in a row on Days -22 through -18.
  • Allogeneic hematopoietic stem cell transplant (allo-HCT) — PROCEDURE
    Participants in both groups will receive their standard-of-care stem cell transplant on Day 0.

Study Details

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"

Key Dates

Start date
Mar 15, 2023
Status verified
May 2026
Primary completion
Mar 31, 2027
Completion
Mar 31, 2027

Study Design

Enrollment
39 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • 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.
  • 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.

Primary Outcome Measure

Engraftment [ Time Frame: Day 100 post-alloHCT ]

Central Contacts

Locations (13)

FacilityCityStateZIPSite coordinators
University of California, San FranciscoSan FranciscoCalifornia94143
Christopher Dvorak, MD
415-476-2188
Children's Healthcare of AtlantaAtlantaGeorgia30322
Shanmuganathan Chandrakasan, MD
404-785-1200
Memorial Sloan Kettering Basking RidgeBasking RidgeNew Jersey07920-
Memorial Sloan Kettering MonmouthMiddletownNew Jersey07748-
Memorial Sloan Kettering BergenMontvaleNew Jersey07645-
Memorial Sloan Kettering Suffolk - CommackCommackNew York11725-
Memorial Sloan Kettering WestchesterHarrisonNew York10604-
Columbia University Irving Medical CenterNew YorkNew York10032-
Memorial Sloan Kettering Cancer CenterNew YorkNew York10065-
Memorial Sloan Kettering NassauRockville CentreNew York11553-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Susan McClory, MD, PhD
267-426-0762
Texas Children's HospitalHoustonTexas77030
Caridad Martinez, MD
832-822-4242
Children's Hospital of WisconsinMilwaukeeWisconsin53226
Larisa Broglie, MD
414-266-2420

Find similar trials in San Francisco, CA

By research site
University of California, San Francisco· San Francisco, CAChildren's Healthcare of Atlanta· Atlanta, GAMemorial Sloan Kettering Basking Ridge· Basking Ridge, NJMemorial Sloan Kettering Monmouth· Middletown, NJMemorial Sloan Kettering Bergen· Montvale, NJMemorial Sloan Kettering Suffolk - Commack· Commack, NY

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