Status and phase
Conditions
Treatments
About
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....
Full description
Background:
Objectives:
-To estimate the acute graft-versus-host disease (aGVHD)-free, graft failure-free survival at day +180 after allo BMT, analyzed separately by conditioning arm/cohort
Eligibility:
Patients age greater than or equal to 4 through 75 years
PID deemed to be of sufficient past severity to warrant allo BMT, by meeting the two criteria below:
PID as defined by identified genetic defect or, in the absence of a mutation, patients with an immune defect potentially amenable to allo BMT who meet the clinical history criteria below may be eligible
Clinical history of at least two of the following:
At least one 7-8/8 (9-10/10) HLA-matched related or unrelated donor, or an HLA-haploidentical related donor
Adequate end-organ function
Consensus opinion by the investigative team that the patient has the potential to benefit from transplant despite existing, non-hematopoietic organ dysfunction
Not pregnant or breastfeeding
HIV negative
Disease status: patients with malignancy should be referred in remission for evaluation, except in the case of virus-associated malignancy who may be referred at any time
Design:
The study will have two arms that vary in mycophenolate mofetil (MMF) duration.
RIC and RIC-MMF arms: pentostatin 4 mg/m2/day IV on days -11 and -7, low-dose cyclophosphamide orally daily on days -11 through -4; busulfan IV, pharmacokinetically dosed, on days -3 and -2.
RIC-SHORT arm: pentostatin 4 mg/m2/day IV on days -9 and -5, low-dose cyclophosphamide orally daily on days -9 through -2; busulfan IV, pharmacokinetically dosed, on days -3 and -2.
Bone marrow is the preferred graft source. Peripheral blood stem cells are permitted on RIC-MMF arm but not on RIC-SHORT arm.
GVHD prophylaxis:
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
INCLUSION CRITERIA - RECIPIENT:
Patients age greater than or equal to 4 through 75 years
PID deemed to be of sufficient past severity to warrant allo BMT, by meeting the two criteria below:
PID as defined by identified genetic defect or, in the absence of a PID-associated genetic mutation, patients with an immune defect potentially amenable to allo BMT who meet the clinical history criteria below may be eligible upon discussion with the PI
Clinical history of at least two of the following:
Availability of at least one 7-8/8 (9-10/10) HLA-matched related (excluding an identical twin) or unrelated donor, or an HLA-haploidentical related donor
Consensus among the PI, key AIs, and consultants (as necessary) that correction of the patient s immune system through BMT has the potential to improve the patient s health, quality of life, and/or life expectancy, after taking into consideration the patient s existing non-hematopoietic, potentially irreversible organ dysfunction
Adequate end-organ function, as measured by:
Karnofsky or Lansky performance status of greater than or equal to 60% or ECOG performance status of 2 or less
Ability of subject or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document
Not pregnant or breastfeeding. As therapeutic agents used in this trial may be harmful to a fetus, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for at least one year post-allo BMT. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in the study, she should inform her treating physician immediately.
Disease status: Patients with malignancy are to be referred in remission for evaluation, except in cases of virus-associated malignancy who may be referred at any time. Should a patient have progressive disease or a donor becomes unavailable after enrollment, the patient will be referred back to his/her primary hematologist-oncologist for treatment. If this course of action is not in the best interest of the patient according to the clinical judgment of the PI, then the patient may receive standard treatment for the malignant disease under the current study, although this should only occur as a bridge to transplant. If under either of these settings, it becomes apparent that the patient will not be able to proceed to transplant, then he/she must come off the study. Patients receiving standard therapy will be told about the therapy, associated risks, potential benefits, alternatives to the proposed therapy, and the availability of receiving the same treatment elsewhere, outside of a research protocol.
EXCLUSION CRITERIA - RECIPIENT:
Inclusion Criteria (Related Donor):
Exclusion Criteria (Related Donor):
None
INCLUSION CRITERIA - UNRELATED DONOR:
EXCLUSION CRITERIA - UNRELATED DONOR:
-Unrelated donors: failure to qualify as a National Marrow Donor Program (NMDP) donor per current NMDP Standards, available at: http://bethematch.org/About-Us/Global-transplant-network/Standards/.
Primary purpose
Allocation
Interventional model
Masking
254 participants in 6 patient groups
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Central trial contact
Dimana Dimitrova, M.D.
Data sourced from clinicaltrials.gov
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