RecruitingPhase 2NCT02579967

Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies

Sponsor

National Cancer Institute (NCI)

Enrollment

354 participants

Start Date

Nov 19, 2015

Study Type

INTERVENTIONAL

Conditions

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

Summary

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.

Eligibility

Min Age: 4 YearsMax Age: 75 Years

Plain Language Summary

Simplified for easier understanding

This NIH trial offers bone marrow (stem cell) transplantation to patients with primary immunodeficiency disorders (PIDs) — inherited conditions where the immune system does not work properly. A donor's healthy stem cells can potentially replace the faulty immune system and improve or cure the condition. You may be eligible if: - You are between 4 and 75 years old - You have a confirmed PID (by genetic testing or clinical history) severe enough to warrant a transplant - You have had at least two serious clinical manifestations of immune deficiency (life-threatening infections, opportunistic infections, autoimmune disease, blood cancers, etc.) - A suitable donor is available (related or unrelated) - Your heart, lungs, kidneys, and liver function meet minimum thresholds You may NOT be eligible if: - You are currently in another investigational study - You have active psychiatric illness preventing compliance - You have HIV or another acquired immunodeficiency - You have active CNS involvement by malignancy (except virus-related) - You lack adequate venous access - You are pregnant or breastfeeding Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGImmunosuppression Only Conditioning -Closed with amendment L

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)

DRUGReduced Intensity Conditioning

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.

DRUGMyeloablative Conditioning-Closed with amendment L

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)

DRUGGVHD Prophylaxis

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.

PROCEDUREAllo BMT

Allogeneic blood or marrow transplantation