Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Cancer Institute (NCI)
Study ID
NCT04339777
Phase
PHASE2
Status
Recruiting
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Conditions

  • Autoimmune Lymphoproliferative
  • Common Variable Immunodeficiency
  • Immune System Diseases
  • Lymphoproliferative Disorders
  • Primary T-cell Immunodeficiency Disorders

Eligibility Criteria

Sex
ALL
Age
4 Years - 69 Years
Healthy Volunteers
Not accepted

Interventions

  • Busulfan test dose — DRUG
    0.8 mg/kg IV infusion over 2 hours
  • Fludarabine — DRUG
    40 mg/m2 IV infusion over 30 min once daily for 4 days
  • Busulfan — DRUG
    AUC Targeted Dose based on busulfan test dose PKs, IV infusion over 3 hours once daily (3.2 mg/kg IV per day will be the default dose) per the below time frame: For 10/10 Matched Related and Unrelated Donor Recipients For the High Intensity Arm, the busulfan dose will be given for 4 days (-6, -5, -4, and -3). For Intermediate Intensity Arm, the busulfan dose will be given for 3 days (-6,-5, and -4). For Low Intensity Arm, the busulfan dose will be given for 2 days on days (-6 and -5). 9/10 HLA Matched Related or Unrelated Donor Recipients For the High Intensity Arm, the busulfan dose will be given for 3 days (-6, -5, and -4). For the the Intermediate Intensity Arm, the busulfan dose will be given for 2 days (-6 and -5). For the Low Intensity Arm, the busulfan dose will be given for 1 day on day (-6).
  • Alemtuzumab — DRUG
    Alemtuzumab will be given if there is evidence of immune dysregulation 10 mg/m2 SC divided over three days (-14, -13, and -12)
  • Total body Irradiation — RADIATION
    200 cGy Transplant Day -1 (Only for 9/10 HLA Matched Related or Unrelated Donor Recipients )
  • Allogeneic HSCT — PROCEDURE
    Stem cell transplant
  • Tacrolimus (Tacro) — DRUG
    Tacrolimus 0.02 mg/kg IV continuous infusion over 24 hours starting on day +5
  • Mycophenolate mofetil (MMF) — DRUG
    Mycophenolate mofetil 15 mg/kg IV over 2 hours three times a day starting on day +5 will continue until Approximately+35 (+/- two days)
  • Cyclophosphamide (Cytoxan) — DRUG
    Cyclophosphamide: 50 mg/kg IV once daily over 2 hours on days +3 and +4, dosed according to ideal body weight

Study Details

Background: During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies. But transplant has many risks and complications. Objective: To see if stem cell transplant can be successfully performed in people with primary immunodeficiency disease and cure them. Eligibility: People ages 4-69 for whom a primary immunodeficiency (PID) or Primary Immune Regulatory Disorder (PIRD), has caused significant health problems and either standard management has not worked or there are no standard management options, along with their donors Design: Donors will be screened under protocol 01-C-0129. They will donate blood or bone marrow. Participants will be screened with: Medical history Physical exam Blood, urine, and heart tests CT or PET scans Before transplant, participants will have dental and eye exams. They will have a bone marrow biopsy. For this, a needle will be inserted through the skin into the pelvis to remove marrow. Participants will be hospitalized before their transplant. They will have a central catheter put into a vein in their chest or neck. They will get medications through the catheter to prevent complications. Participants will get stem cells through the catheter. They will stay in the hospital for at least 4 weeks. They will give blood, urine, bone marrow, and stool samples. They may need blood transfusions. They may need more scans. They will take more medications. Participants will have visits on days 30, 60, 100, 180, and 360, and 24 months after the transplant. Then they will have visits once a year for about 5 years

Key Dates

Start date
Sep 22, 2020
Status verified
Apr 2026
Primary completion
Oct 1, 2026
Completion
Nov 30, 2027

Study Design

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

Arms

  • Active Comparator: Arm A
    Low Intensity, Intermediate Intensity and High Intensity Conditioning with or without alemtuzumab
  • Active Comparator: Arm B
    Intermediate Intensity Conditioning with or without Alemtuzumab

Primary Outcome Measure

Sustained donor engraftment [ Time Frame: baseline to day +100 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892
For more information at the NIH Clinical Center contact National Cancer Institute Referral Office
888-624-1937

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By research site
National Institutes of Health Clinical Center· Bethesda, MD

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