Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Cancer Institute (NCI)
Study ID
NCT02579967
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 - 75 Years
Healthy Volunteers
Accepted

Interventions

  • Immunosuppression Only Conditioning -Closed with amendment L — DRUG
    Pentostatin 4 mg/m2/day IV on days -9 and -5, cyclophosphamide 5 mg/kg orally daily on days -9 through -2 (Closed with amendment L)
  • Reduced Intensity Conditioning — DRUG
    pentostatin 4 mg/m2/day IV on days -11 and -7, cyclophosphamide 3 mg/kg orally daily on days -11 through -4; busulfan IV, pharmokinetically dosed, on days -3 and -2.
  • Myeloablative Conditioning-Closed with amendment L — DRUG
    Pentostatin 4 mg/m2/day IV on days -13 and -9, low-dose cyclophosphamide orally daily on days -13 through -6; busulfan IV, pharmokinetically dosed, on days -5, -4, -3, and -2. (Closed with amendment L)
  • GVHD Prophylaxis — DRUG
    High-dose, post-transplantation cyclophosphamide (PTCy) 25-50 mg/kg on days +3 and +4, sirolimus 6 mg on days +5 through +90, and mycophenolate mofetil (MMF) on days +5 through 0, +18, +25, or +35 depending on treatment arm and cohort.
  • Allo BMT — PROCEDURE
    Allogeneic blood or marrow transplantation

Study Details

Background: Allogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems. Objective: To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with primary immunodeficiencies. Eligibility: Donors: Healthy people ages 4 or older Recipients: People ages 4-75 with a primary immunodeficiency that may be treated with allogeneic blood or marrow transplant Design: Participants will be screened with medical history, physical exam, and blood tests. Participants will have urine tests, EKG, and chest x-ray. Donors will have: Bone marrow harvest: With anesthesia, marrow is taken by a needle in the hipbone. OR Blood collection: They will have several drug injections over 5-7 days. Blood is taken by IV in one arm, circulates through a machine to remove stem cells, and returned by IV in the other arm. Possible vein assessment or pre-anesthesia evaluation Recipients will have: Lung test, heart tests, radiology scans, CT scans, and dental exam Possible tissue biopsies or lumbar puncture Bone marrow and a small piece of bone removed by needle in the hipbone. Chemotherapy 1-2 weeks before transplant day Donor stem cell donation through a catheter put into a vein in the chest or neck Several-week hospital stay. They will take medications and may need blood transfusions and additional procedures. After discharge, recipients will: Remain near the clinic for about 3 months. They will have weekly visits and may require hospital readmission. Have multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years.

Key Dates

Start date
Nov 19, 2015
Status verified
May 2026
Primary completion
Dec 31, 2031
Completion
Dec 31, 2036

Study Design

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

Arms

  • Experimental: 1/ IOC Arm-Closed with amendment L (07/05/2019)
    Immunosuppression Only Conditioning Arm
  • Experimental: 2/ RIC Arm - Closed with Amendment L (07/05/2019)
    Reduced Intensity Conditioning Arm
  • Experimental: 3/ MAC Arm-Closed with amendment L (07/05/2019)
    Myeloablative Conditioning Arm
  • Experimental: 4/ RIC-MMF Arm
    Reduced Intensity Conditioning with MMF duration de-escalation design
  • No Intervention: 5/ Donor Arm
    Donor
  • Experimental: 6/ RIC-SHORT Arm
    Reduced Intensity Conditioning with shortened duration and dose-reduced PTCy

Primary Outcome Measure

For the RIC-SHORT arm: To estimate the aGVHD-free, graft failure-free survival [ Time Frame: +180 after allo BMT ]

Central Contacts

Locations (2)

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
National Marrow Donor ProgramMinneapolisMinnesota55413-1753
John Miller, M.D.
[email protected]
763-406-8566

Find similar trials in Bethesda, MD

By research site
National Institutes of Health Clinical Center· Bethesda, MDNational Marrow Donor Program· Minneapolis, MN

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