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New treatment option for Lymphoproliferative Disorders
Official title Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
ClinicalTrials.gov ID: NCT04339777
What this study is testing
What is Busulfan test dose?
Busulfan test dose is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for lymphoproliferative 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: 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.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 5 years
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 69
You may be able to join if
- Age \>= 4 years and \ =12 kilograms
- Mutation in a known monogenic (IEI) gene performed by a CLIA certified laboratory, who have failed standard medical management, or when no standard...
- Availability of an 8/8, 7/8, or 6/8 HLA-matched related or unrelated donor (if the mismatch is at DQ this will be considered an 8/8 matched donor)...
- Adequate end-organ function, as measured by: --Left ventricular ejection fraction \> 40%, preferably by 2-D echocardiogram (ECHO) obtained within 60...
- Creatinine: Adult patients: \ = 30 ml/min; Pediatric patients (\ 30 mL/min/1.73m\^2.
You likely can't join if
- Patients who are receiving any other investigational agents (with the exception of virus-specific therapy e.g. cytotoxic T-cells for the treatment of...
- Patients with known brain metastases should be excluded from this clinical trial because of their poor prognosis and because they often develop...
- HIV-positive patients are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy...
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (steroids, cyclophosphamide, busulfan...
- Active psychiatric disorder which is deemed by the PI to have significant risk of compromising compliance with the transplant protocol or which does...
- Pregnant women are excluded from this study because the study agents have the potential for teratogenic or abortifacient effects. Because there is an...
See the full eligibility criteria
- Age \>= 4 years and \ =12 kilograms
- Mutation in a known monogenic (IEI) gene performed by a CLIA certified laboratory, who have failed standard medical management, or when no standard medical management is available. OR Patients without a known IEI...
- Availability of an 8/8, 7/8, or 6/8 HLA-matched related or unrelated donor (if the mismatch is at DQ this will be considered an 8/8 matched donor), or a haploidentical related donor. Karnofsky or Lansky performance...
- Adequate end-organ function, as measured by: --Left ventricular ejection fraction \> 40%, preferably by 2-D echocardiogram (ECHO) obtained within 60 days prior to enrollment.
- Creatinine: Adult patients: \ = 30 ml/min; Pediatric patients (\ 30 mL/min/1.73m\^2.
- Serum conjugated bilirubin \ 30% and DLCO \>30%. Children who are unable to have DLCO assessed due to age, are still eligible if no evidence of dyspnea at rest and no need for supplemental oxygen.
- Ability of subject or parent/guardian to understand and the willingness to sign a written informed consent document. For people \<18 years old, their legal guardian must give informed consent. Pediatric patients will...
- 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...
- Willingness to remain in the NIH hospital or, if discharged, stay close to the NIH, for a minimum of 100 days after transplant or longer, if there are complications. If outpatient in the first 100 days after transplant...
- Patients who are receiving any other investigational agents (with the exception of virus-specific therapy e.g. cytotoxic T-cells for the treatment of viral infection/reactivation prior to allo HCT).
- Patients with known brain metastases should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of...
- HIV-positive patients are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in patients receiving...
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (steroids, cyclophosphamide, busulfan, tacrolimus, sirolimus, MMF, G-CSF, alemtuzumab) used in the study
- Active psychiatric disorder which is deemed by the PI to have significant risk of compromising compliance with the transplant protocol or which does not allow for appropriate informed consent
- Pregnant women are excluded from this study because the study agents have the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants...
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance...
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
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 5 years 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 69 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, 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.