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Cell Therapy

Allogeneic Blood or Bone Marrow Transplant for T-Cell Immunodeficiency Disorders

Phase 2
Recruiting
Led By Dimana Dimitrova, M.D.
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age greater than or equal to 4 years
T-cell infiltration of liver, spleen, lymph nodes, marrow, lungs, gut, or other organs by T cells, as evidenced by laboratory, radiographic, and/or anatomic pathology evaluation, resulting in organ dysfunction and/or organomegaly
Must not have
MagT1 mutation and active need to take anti-platelet agents and/or therapeutic anti- coagulation that cannot be interrupted during aplasia
Prohibitive allergy to a study drug or to compounds of similar chemical or biologic composition of the agents (e-ATG, steroids, cyclophosphamide, busulfan, pentostatin, tacrolimus, MMF, G-CSF) used in the study.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up +28, +42, +60, +100, +180, and 1 year after hct
Awards & highlights
No Placebo-Only Group

Summary

This trial is studying allogeneic blood or bone marrow transplant to see if it is safe and effective in treating people with T-cell problems.

Who is the study for?
This trial is for people aged 4 and older with T-cell disorders, such as immune deficiencies or lymphoproliferative diseases. Participants must have certain medical conditions like abnormal T-cell function or specific genetic mutations affecting the immune system. They need to be in a stable condition to undergo transplant procedures and agree to use contraception if of childbearing potential.
What is being tested?
The study tests whether transferring healthy blood stem cells from one person (donor) to another (recipient) can effectively treat various T-cell related health issues. It involves screening, preparation including chemotherapy, receiving donor stem cells through a catheter, and follow-up visits.
What are the potential side effects?
Potential side effects include reactions to medications used during conditioning like chemotherapy, risk of infection due to immunosuppression, organ inflammation from graft-versus-host disease (GVHD), complications from bone marrow collection in donors, and general anesthesia risks.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am at least 4 years old.
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My organs are enlarged or not working well due to T-cell infiltration.
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I have a latent herpesvirus infection in my T cells.
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I have a T-cell disorder due to an immune system problem.
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My liver function tests are within the specific limits for the treatment I am receiving.
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I have or had hemophagocytic lymphohistiocytosis.
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My blood or bone marrow has a specific type of immune cell called large granular lymphocytes.
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My lung function tests meet the required levels, or I don't need extra oxygen and can breathe normally.
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My kidneys work well enough, with a creatinine clearance rate of 50 mL/min or higher.
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I have a blood disorder affecting one or more cell types needing transfusions or support.
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I have an ongoing active Epstein-Barr virus infection.
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I have a genetic mutation that affects my immune system.
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I have ADA2 deficiency causing low white blood cells, anemia, or low platelets needing regular treatment.
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I have had long or repeated fevers due to an immune system problem.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a MagT1 mutation and need continuous blood-thinning medication.
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I am not allergic to any of the medications used in this study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~+28, +42, +60, +100, +180, and 1 year after hct
This trial's timeline: 3 weeks for screening, Varies for treatment, and +28, +42, +60, +100, +180, and 1 year after hct for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To estimate the percentage of recipients with >50% donor T cell chimerism and graft-failure free survival
Secondary study objectives
Event-free survival
Incidence of Acute Graftversus-host disease
Graft-vs-Host Disease
+5 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Active Control
Group I: 2/IOC ArmExperimental Treatment3 Interventions
Immunosuppression Only Conditioning Arm
Group II: 1/RIC ArmExperimental Treatment3 Interventions
Reduced Intensity Conditioning Arm
Group III: 3/donor armActive Control1 Intervention
Healthy Donor- Donors for recipients in arm 1 or arm 2
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
GVHD Prophylaxis
2002
Completed Phase 1
~20
Reduced Intensity Conditioning
2008
Completed Phase 1
~30

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor
13,958 Previous Clinical Trials
41,112,403 Total Patients Enrolled
Dimana Dimitrova, M.D.Principal InvestigatorNational Cancer Institute (NCI)
1 Previous Clinical Trials
254 Total Patients Enrolled

Media Library

Allogeneic HSC (Cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03663933 — Phase 2
Immune Disorders Research Study Groups: 1/RIC Arm, 2/IOC Arm, 3/donor arm
Immune Disorders Clinical Trial 2023: Allogeneic HSC Highlights & Side Effects. Trial Name: NCT03663933 — Phase 2
Allogeneic HSC (Cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03663933 — Phase 2
~34 spots leftby Jul 2026
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