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New treatment option for Primary T-cell Immunodeficiency Disorders
Official title Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies
ClinicalTrials.gov ID: NCT02579967
What this study is testing
What is Immunosuppression Only Conditioning -Closed with amendment L?
Immunosuppression Only Conditioning -Closed with amendment L is an investigational medicine, given as an once-weekly infusion into a vein, being studied as a potential treatment for primary t-cell immunodeficiency disorders.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 3 months
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 75. Healthy volunteers may be eligible.
You may be able to join if
- - RECIPIENT:
- Patients age \>= 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...
- Mutations should be confirmed in a CLIA-certified laboratory, if such testing is available.
You likely can't join if
- RECIPIENT:
- Patients who are receiving any other investigational agents, with the exception of virus-specific cytotoxic T-cells for the treatment of viral...
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (cyclophosphamide, busulfan, pentostatin...
- Active psychiatric disorder which may compromise compliance with the transplant protocol, or which does not allow for appropriate informed consent
- Active central nervous system (CNS) involvement by malignancy, except in cases of virus-associated malignancies with CNS involvement in which case...
- MAGT1 mutation and active need to take anti-platelet agents and/or therapeutic anti-coagulation that cannot be interrupted during aplasia.
See the full eligibility criteria
- - RECIPIENT:
- Patients age \>= 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...
- Mutations should be confirmed in a CLIA-certified laboratory, if such testing is available.
- Patients without a mutation must be deemed eligible and appropriate for allo BMT by the PI. Some patients may meet the clinical history criteria listed below, but will not be eligible if it is thought that their...
- Clinical history of at least two of the following:
- Life-threatening, organ-threatening, or severely disfiguring infection
- Protracted or recurrent infections requiring unusually long or repeated courses of antibiotics
- Infection with an opportunistic organism
- Chronic elevation in the blood (\>=2 documented elevations over a period of 6 months or longer) of a latent virus (EBV, CMV, HHV6, HHV8, etc.)
- Evidence of immune dysregulation, as manifested by autoimmune disease, atopy, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, granulomas, splenomegaly, or lymphadenopathy
- Patients with hemophagocytic lymphohistiocytosis or macrophage activation syndrome related to an underlying lymphoma with no other clinical history suggestive of a primary immunodeficiency will not be eligible
- Hypogammaglobulinemia, dysglobulinemia, or impaired response to vaccination
- Hematologic malignancy or lymphoproliferative disorder
- Tissue diagnosis should be confirmed by NCI Department of Pathology, if prior biopsies are available
- Virus-associated solid tumor malignancy or pre-cancerous lesion
- Tissue diagnosis should be confirmed by NCI Department of Pathology, if prior biopsies are available
- 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...
- Adequate end-organ function, as measured by:
- Left ventricular ejection fraction (LVEF) \>= 40% by 2D echocardiogram (ECHO) or MUGA, or left ventricular shortening fraction \>= 20% by ECHO for patients receiving RIC or RIC-MMF, or RIC-SHORT, or LVEF \>= 30% if the...
- Pulmonary function tests: DL(co) (corrected for hemoglobin) and FEV(1) \>= 40% of predicted for the RIC, RIC-MMF, and RIC-SHORT arms; or in pediatric patients, if unable to perform pulmonary function tests, there should...
- Bilirubin \<= 3.0 mg/dL (unless due to Gilbert s syndrome or hemolysis) for patients receiving RIC, RIC-MMF, RIC-SHORT; ALT and AST 10 x ULN for patients receiving RIC, RIC-MMF, RIC-SHORT. Patients who are above these...
- Estimated creatinine clearance of \>= 40 mL/min/1.73 m\^2, calculated using the Cockcroft-Gault equation for adults and Schwartz formula for pediatric patients, for patients with creatinine levels above the...
- Karnofsky or Lansky performance status of \>=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...
- 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...
- RECIPIENT:
- Patients who are receiving any other investigational agents, with the exception of virus-specific cytotoxic T-cells for the treatment of viral infection/reactivation prior to allo BMT.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (cyclophosphamide, busulfan, pentostatin, sirolimus, MMF, filgrastim or filgrastim biosimilar) used in the study
- Active psychiatric disorder which may compromise compliance with the transplant protocol, or which does not allow for appropriate informed consent
- Active central nervous system (CNS) involvement by malignancy, except in cases of virus-associated malignancies with CNS involvement in which case the patient may benefit from the transplant to control the malignancy.
- MAGT1 mutation and active need to take anti-platelet agents and/or therapeutic anti-coagulation that cannot be interrupted during aplasia.
- HIV positive or other acquired immunodeficiency that, as determined by the PI, interferes with the assessment of PID severity and/or the attribution of clinical manifestations of immunodeficiency to a PID.
- Lack of adequate central venous access potential Inclusion Criteria (Related Donor):
- Ages \>= 4
- Related donor deemed suitable and eligible and willing to donate per clinical evalations who are additionally willing to donate blood, urine, and marrow specimens for research. Related donors will be evaluated in...
- Ages \>= 18
- Unrelated donors will be evaluated in accordance with existing NMDP Standard Policies and Procedures, available at: http://bethematch.org/About-Us/Global-transplant-network/Standards/, except for the additional...
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
- Minneapolis, Minnesota, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 3 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years to 75 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States; Minneapolis, Minnesota, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.