Primary Immune Regulatory DisorderPhase 2NCT05787574
A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition
A Phase 2 study testing Emapalumab, Fludarabine and Dexamethasone and Allogeneic hematopoietic stem cell transplant (allo-HCT) in people with Primary Immune Regulatory Disorder and Autoimmune Lymphoproliferative, run by Joseph Oved at 13 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Immune Regulatory Disorder, Autoimmune Lymphoproliferative, Immune System Diseases
- What is being tested?
- Emapalumab, Fludarabine and Dexamethasone and Allogeneic hematopoietic stem cell transplant (allo-HCT)
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 13 sitesUnited States
- Study dates
- 15 Mar 2023to Mar 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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).
- 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
Show all 24Show fewer
- 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).
Who cannot join
- 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.
Trial locations and contacts
13 sites in 1 country. Contact the study team or a site near you to ask about taking part.
13 sites in 1 country
United States13 sites, 6 recruiting
- Recruiting
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Shanmuganathan Chandrakasan, MD, 404-785-1200
- Active, not recruiting
Memorial Sloan Kettering Basking Ridge
Basking Ridge, New Jersey, 07920
- Active, not recruiting
Memorial Sloan Kettering Suffolk - Commack
Commack, New York, 11725
- Active, not recruiting
Memorial Sloan Kettering Westchester
Harrison, New York, 10604
- Recruiting
Texas Children's Hospital
Houston, Texas, 77030
Caridad Martinez, MD, 832-822-4242
- Active, not recruiting
Memorial Sloan Kettering Monmouth
Middletown, New Jersey, 07748
- Recruiting
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Larisa Broglie, MD, 414-266-2420
- Active, not recruiting
Memorial Sloan Kettering Bergen
Montvale, New Jersey, 07645
- Recruiting
Columbia University Irving Medical Center
New York, New York, 10032
- Active, not recruiting
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Susan McClory, MD, PhD, 267-426-0762
- Active, not recruiting
Memorial Sloan Kettering Nassau
Rockville Centre, New York, 11553
- Recruiting
University of California, San Francisco
San Francisco, California, 94143
Christopher Dvorak, MD, 415-476-2188
About this study
Official title: Phase 2 Study Bridging Pre-Transplant Inflammatory Dampening for Primary Immune Regulatory Disorders (BRIDGE Trial)
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"
Read the detailed description
Defects in the immune cells can hinder the immune system's ability to fight infection. A stem cell transplant can replace non-working immune system cells with a donor's fully functioning immune cells, but if inflammation is present, the immune cells from the donor may not graft successfully. The proposed trial tests whether emapalumab or fludarabine and dexamethasone can help prepare the body to receive a stem cell transplant by reducing inflammation, so that the immune system will be able to produce fully functioning immune cells. Participants will receive emapalumab or a combination of fludarabine and dexamethasone over the course of four days in the hospital prior to a planned transplant procedure.
This study has two study groups:
- Participants in Group A have a high CXCL9, a specific cytokine, level and will receive emapalumab on Days -22, -15, -8, and -1.
- Participants in Group B have a generalized inflammation and will receive fludarabine and dexamethasone from Days -22 to -18 (5 days).
All study participants are eligible to receive one additional emapalumab 3mg/kg in the first 30 days post-transplant if they begin to have CXCL9 levels that are trending up. Participants will receive personalized rATG dosing. Participants will remain in the hospital according to the usual standard of care guidelines for stem cell transplant. Subject status and follow-up examination/data collection will occur on days 0, 7, 14, 21,30, 45, 60, 7, 100, 180, 270, and 365, then quarterly until 3 years post-transplant.
- Conditions
- Primary Immune Regulatory Disorder, Autoimmune Lymphoproliferative, Immune System Diseases
- Being tested
- Emapalumab, Fludarabine and Dexamethasone, Allogeneic hematopoietic stem cell transplant (allo-HCT)
- Keywords
- Stem Cell Transplant, Emapalumab, Fludarabine, Dexamethasone
- Medical subject headings
- Immune System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmapalumabDrug
Also called Gamifant
Emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1.
Fludarabine and DexamethasoneDrug
Fludarabine and dexamethasone for 5 days in a row on Days -22 through -18.
Allogeneic hematopoietic stem cell transplant (allo-HCT)Procedure or surgery
Also called stem cell transplant
Participants in both groups will receive their standard-of-care stem cell transplant on Day 0.
| Study group | Role | What they receive |
|---|---|---|
| Group A: Emapalumab (for isolated Interferongamma mediated disease) | Experimental | Participants in this group will receive emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1. Receives: Drug: Emapalumab; Procedure: Allogeneic hematopoietic stem cell transplant (allo-HCT) |
| Group B: Fludarabine and Dexamethasone (for generalized autoinflammation) | Experimental | Participants in this group will receive fludarabine and dexamethasone for 5 days in a row on Days -22 through -18. Receives: Drug: Fludarabine and Dexamethasone; Procedure: Allogeneic hematopoietic stem cell transplant (allo-HCT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment
Engraftment is defined as the first of three days of absolute neutrophil count >500,000/µL and the first of seven days of platelets >20,000/µL in the absence of transfusional support.
Measured: Day 100 post-alloHCT
Secondary outcomes (18)
Overall Survival (OS)
Overall Survival (OS) is defined as the duration of time between allo-HCT and death due to any cause.
Measured: 1 year
Event Free Survival (EFS)
Event-Free Survival (EFS) is the length of time from the start of a treatment or study until a patient experiences a defined negative "event" (such as disease recurrence, progression, or death).
Measured: Up to 2 years post-alloHCT
Non-Relapse Mortality (TRM)
Participants who die for any reason after disease recurrence are considered to have experienced disease related mortality, and will be counted as competing events. Patients alive will be censored at the day of last follow up.
Measured: 1 year
GVHD free/relapse free survival (GRFS)
Duration of time between transplantation and development of grade III-IV GVHD, chronic GVHD requiring systemic therapy, disease relapse, or death, whichever occurs first.
Measured: 1 year
Chronic graft vs host disease/relapse free survival (CRFS)
Duration of time between transplantation and development of moderate or severe chronic GVHD (NIH consensus criteria), or death, whichever occurs first.
Measured: 1 year
All-cause mortality at 1 & 2 years
Measured: 1 year and 2 years post-alloHCT
Incidence of viral reactivations
Incidence of viral reactivations including: Cytomegalovirus (CMV), Adenovirus, Epstein-Barr Virus (EBV), Human Herpes-6 (HHV6), BK-virus defined as viral reactivations necessitating use of anti-viral treatment.
Measured: 1 year
Incidence of Acute graft versus host disease (GVHD) at Day 100, 180, and 1-year follow-up
Measured: Day 100, 180, and 1 year post-alloHCT
Incidence of Chronic GVHD at Day 180 and 1-year follow-up
Measured: Day 180 and 1 year post-alloHCT
Time to neutrophil engraftment
Neutrophil engraftment is defined as the first of 3 consecutive days of absolute neutrophil count (ANC) ≥ 500 K/µL.
Measured: 100 days
Time to platelet engraftment
Platelet engraftment is defined as the first of 7 consecutive days with a platelet count exceeding 20,000/µL without transfusion support.
Measured: 100 days
Incidence of primary engraftment failure
Primary engraftment failure is defined as failure to achieve neutrophil engraftment by Day 30 after transplantation.
Measured: Day 30 post-alloHCT
Incidence of secondary engraftment failure
Secondary engraftment failure is defined as <500/µL circulating neutrophils at any time after primary engraftment that is not attributed to disease recurrence or drug therapy.
Measured: 1 year post-alloHCT
Donor Chimerism
The percentage of donor contribution to hematopoiesis (chimerism) will be determined using short tandem repeat (STR) based analysis of the peripheral blood (Chimerism - all lineages on Day 30, 100, 180, and 365) and bone marrow (all samples obtained post-alloHCT).
Measured: Day 30, 100, 180, and 1 year post-alloHCT
Incidence of grade ≥ 3 non-hematologic adverse events
Incidence of grade ≥ 3 non-hematologic adverse events as defined by CTCAE version 5.0 at Day 100, 180, and 1 year post allo-HCT.
Measured: Day 100, 180, and 1 year post-alloHCT
Lymphocyte Reconstitution
CD4+ Immune Reconstitution (CD4+IR) is defined at CD4+ > 50u/L at two consecutive measures within 100 days post-alloHCT. Lymphocyte subsets (includes CD3, CD4, CD8, CD19, CD56+16, CD45RA, CD45) will be documented by flow cytometry.
Measured: Day 30, 60, 100, 180, and 1 year and 2 years post-alloHCT
Quality of Life (PROMIS score)
Patient-reported outcomes will be self-reported using the PROMIS Profile Measure Scores. Scores are reported as standardized T-scores based on a U.S. general population average of 50 and a standard deviation of 10.
Measured: up to 3 years post-alloHCT
Percentage of patients who are eligible to proceed to transplant after receiving an immune suppression prophase
Measured: 30 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2023
- Study start15 Mar 2023
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune Regulatory DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- This is a two-stage phase 2 study.
- Masking
- None (open label)
- Enrolment
- 39 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Joseph Oved
- Sponsor type
- University, hospital or other
- Collaborators
- Sobi, Inc.
- Responsible party
- Sponsor-investigator, Joseph Oved, Columbia University
- Investigators
- Joseph Oved, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05787574
- Sponsor's protocol ID
- AAAV8620
- Other IDs
- R01FD007829
- Status last verified
- May 2026
- First submitted
- 15 Mar 2023
- Last updated
- 2 Jun 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study Bridging Pre-Transplant Inflammatory Dampening for Primary Immune Regulatory Disorders (BRIDGE Trial)
- ClinicalTrials.gov ID
- NCT05787574
- Acronym / sponsor ID
- AAAV8620
- Conditions
- Primary Immune Regulatory Disorder, Autoimmune Lymphoproliferative, Immune System Diseases
- Keywords
- Stem Cell Transplant, Emapalumab, Fludarabine, Dexamethasone
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 39 participants (estimated)
- Lead sponsor
- Joseph OvedUniversity, hospital or other
- Collaborators
- Sobi, Inc.
- Study start
- 15 Mar 2023 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 28 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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"
Detailed description
Defects in the immune cells can hinder the immune system's ability to fight infection. A stem cell transplant can replace non-working immune system cells with a donor's fully functioning immune cells, but if inflammation is present, the immune cells from the donor may not graft successfully. The proposed trial tests whether emapalumab or fludarabine and dexamethasone can help prepare the body to receive a stem cell transplant by reducing inflammation, so that the immune system will be able to produce fully functioning immune cells. Participants will receive emapalumab or a combination of fludarabine and dexamethasone over the course of four days in the hospital prior to a planned transplant procedure.
This study has two study groups:
- Participants in Group A have a high CXCL9, a specific cytokine, level and will receive emapalumab on Days -22, -15, -8, and -1.
- Participants in Group B have a generalized inflammation and will receive fludarabine and dexamethasone from Days -22 to -18 (5 days).
All study participants are eligible to receive one additional emapalumab 3mg/kg in the first 30 days post-transplant if they begin to have CXCL9 levels that are trending up. Participants will receive personalized rATG dosing. Participants will remain in the hospital according to the usual standard of care guidelines for stem cell transplant. Subject status and follow-up examination/data collection will occur on days 0, 7, 14, 21,30, 45, 60, 7, 100, 180, 270, and 365, then quarterly until 3 years post-transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This is a two-stage phase 2 study.
| Arm | Type | Receives |
|---|---|---|
| Group A: Emapalumab (for isolated Interferongamma mediated disease) | Experimental | Drug: Emapalumab, Procedure: Allogeneic hematopoietic stem cell transplant (allo-HCT)Participants in this group will receive emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1. |
| Group B: Fludarabine and Dexamethasone (for generalized autoinflammation) | Experimental | Drug: Fludarabine and Dexamethasone, Procedure: Allogeneic hematopoietic stem cell transplant (allo-HCT)Participants in this group will receive fludarabine and dexamethasone for 5 days in a row on Days -22 through -18. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (24 listed)
- Patients receiving first allo-HCT for the following immunologic conditions:
- 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:
- Adequate performance status:
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EmapalumabAlso: GamifantEmapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1. | Drug | Group A: Emapalumab (for isolated Interferongamma mediated disease) |
| Fludarabine and DexamethasoneFludarabine and dexamethasone for 5 days in a row on Days -22 through -18. | Drug | Group B: Fludarabine and Dexamethasone (for generalized autoinflammation) |
| Allogeneic hematopoietic stem cell transplant (allo-HCT)Also: stem cell transplantParticipants in both groups will receive their standard-of-care stem cell transplant on Day 0. | Procedure or surgery | Group A: Emapalumab (for isolated Interferongamma mediated disease), Group B: Fludarabine and Dexamethasone (for generalized autoinflammation) |
Primary outcome
Engraftment
Time frame: Day 100 post-alloHCT
Engraftment is defined as the first of three days of absolute neutrophil count >500,000/µL and the first of seven days of platelets >20,000/µL in the absence of transfusional support.
18 secondary outcomes
Overall Survival (OS)
Time frame: 1 year
Event Free Survival (EFS)
Time frame: Up to 2 years post-alloHCT
Non-Relapse Mortality (TRM)
Time frame: 1 year
GVHD free/relapse free survival (GRFS)
Time frame: 1 year
Chronic graft vs host disease/relapse free survival (CRFS)
Time frame: 1 year
All-cause mortality at 1 & 2 years
Time frame: 1 year and 2 years post-alloHCT
Incidence of viral reactivations
Time frame: 1 year
Incidence of Acute graft versus host disease (GVHD) at Day 100, 180, and 1-year follow-up
Time frame: Day 100, 180, and 1 year post-alloHCT
Incidence of Chronic GVHD at Day 180 and 1-year follow-up
Time frame: Day 180 and 1 year post-alloHCT
Time to neutrophil engraftment
Time frame: 100 days
Time to platelet engraftment
Time frame: 100 days
Incidence of primary engraftment failure
Time frame: Day 30 post-alloHCT
Incidence of secondary engraftment failure
Time frame: 1 year post-alloHCT
Donor Chimerism
Time frame: Day 30, 100, 180, and 1 year post-alloHCT
Incidence of grade ≥ 3 non-hematologic adverse events
Time frame: Day 100, 180, and 1 year post-alloHCT
Lymphocyte Reconstitution
Time frame: Day 30, 60, 100, 180, and 1 year and 2 years post-alloHCT
Quality of Life (PROMIS score)
Time frame: up to 3 years post-alloHCT
Percentage of patients who are eligible to proceed to transplant after receiving an immune suppression prophase
Time frame: 30 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2026.
- Individual participant dataSharing planned: Yes. 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.
Related trials for Primary Immune Regulatory Disorder
RecruitingObservational study
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
Recruiting
Suppressive Functions of Regulatory T Cells in Migraine
Chronic Migraine
Not yet recruitingPhase 1/2
Upadacitinib in Treatment of JAK/STAT Pathway Disorders With Activating Mutations
JAK1 GOF, STAT1 GOF
RecruitingPhase 2
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder
RecruitingPhase 1/2
Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
Leukemia, High Risk Acute Lymphoblastic Leukemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Joseph Oved, MD principal investigator
Columbia University
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Emapalumab, Fludarabine and Dexamethasone and Allogeneic hematopoietic stem cell transplant (allo-HCT) in people with Primary Immune Regulatory Disorder and Autoimmune Lymphoproliferative, run by Joseph Oved at 13 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 24 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 2 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Joseph Oved, MD (212 305 9770, jho2001@cumc.columbia.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Engraftment (time frame: Day 100 post-alloHCT). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jun 2026.