Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- Up to 22 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests a blood-forming cell transplant from a partly matched family donor for children and young adults with sickle cell disease. It tests treatment to reduce harm before transplant and medicines afterward to prevent graft-versus-host disease (GVHD), when donor cells attack the body. The goal is for more than 90% of patients to survive without GVHD or rejection of the donor cells.
The purpose of this study it to evaluate a reduced toxicity conditioning regimen for haploidentical donor HCT followed by a GVHD prophylaxis regimen comprising of post-transplant cyclophosphamide, sirolimus and abatacept with the goal to improve the GVHD-free rejection-free survival (GRFS) to greater than 90% after haploidentical donor HCT in children and young adults with SCD. Primary Objective: \- To assess the GVHD-free and rejection free survival (GRFS) after haploidentical donor HCT in children and young adults with SCD. Secondary Objectives: * Assess the overall survival (OS) and disease-free survival (DFS) after haploidentical donor HCT for SCD. * Estimate incidence and severity of acute and chronic GVHD after haploidentical donor HCT for SCD. * Assess the neutrophil and platelet engraftment kinetics after haploidentical donor HCT for SCD.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Patients must be age 22 or younger and have sickle cell disease of any type.
- Patients must lack a suitable fully matched sibling donor and have a willing family donor who matches at least 3 of 6 tissue markers. The donor's full review can happen after enrollment.
- Patients must have at least ONE of the sickle cell problems listed in the next four bullets.
- Abnormal blood flow on at least two brain ultrasound tests, or a brain MRI showing a stroke, including one with no clear symptoms.
- At least two episodes of acute chest syndrome, a serious lung problem, in the past 2 years; or at least two sickle cell pain events needing injected pain medicine in the past year.
- At least two prolonged erections lasting 4 hours or more or needing emergency care; or at least eight red blood cell transfusions in the past year.
- Worsening organ damage that does not respond to medical care and may improve with transplant. Two independent blood specialists must document this before enrollment.
- Donors must be HIV-negative, not pregnant or breastfeeding, and have no major inherited blood disorder. Sickle cell trait is allowed. Donors must pass the required donor review or have a doctor's urgent medical need declaration.
Who can't join
- A score below 60 on a test of ability to do daily activities.
- Pregnant or breastfeeding.
- HIV, or an infection that keeps getting worse despite treatment within 1 month before transplant preparation. Fever or a suspected minor infection must clear before preparation starts.
- Direct bilirubin, a substance measured in blood, above three times the normal limit for age. A rise caused by severe red blood cell breakdown after transfusion may be allowed if it falls back to an acceptable level.
- Heart pumping tests showing a shortening fraction below 25% or an ejection fraction below 40%.
- A kidney filtering test result below 50 mL/min/1.73 m².
- A lung gas-transfer test below 35%, a resting blood oxygen level below 85%, or an artery blood oxygen pressure below 70.
- Immune proteins that attack the donor's tissue markers and do not respond to treatment to lower them.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: Transplant Recipient * Age less than or equal to 22 years. * Patients without a suitable HLA-matched sibling donor but with a suitable single haplotype matched (≥ 3 of 6) family member donor. Potential donors do not need to undergo eligibility determination prior to the recipients enrolling on the study. As long as a potential donor is identified and willing to donate hematopoietic progenitor cells, recipients can enroll on the study. * Patients with SCD (any genotype) who meet any ONE of the following criteria: * History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique (or ≥185 cm/sec by the imaging technique) measured at a minimum of two separate occasions. * History of cerebral infarction on brain MRI (overt stroke, or silent cerebral infarct). * History of two or more episodes of acute chest syndrome (ACS) in the 2-years period preceding enrollment. * History of two or more SCD related pain events requiring treatment with parenteral analgesics in the last 12 months. * History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care). * Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months). * Evidence of progressive end organ damage (eg. cardiomyopathy, nephropathy, pulmonary hypertension etc) that in the opinion of the treating hematologist is not responsive to medical management and may benefit from an HCT. Such a determination must be made in writing by at least two independent hematologists and documented in the patient's electronic medical record prior to enrollment. Donor * An at least single haplotype matched (≥ 3 of 6) family member. * HIV negative * Not pregnant, as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female). * Not breast feeding. * Donor should not have clinically significant hemoglobinopathy. Donors with sickle cell trait are acceptable. * Regarding donation eligibility, is identified as either: * Completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR. * Does not meet 21 CFR 1271 eligibility requirements but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271. Exclusion Criteria: Transplant Recipient * Karnofsky or Lansky performance score \<60. * Pregnant, as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment (if female). * Breast feeding. * Uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms) within 1 month prior to conditioning. Patients with febrile illness or suspected minor infection should await clinical resolution prior to starting conditioning. Patients with confirmed seropositivity or positive NAAT for HIV are excluded. * Serum conjugated (direct) bilirubin \>3x upper limit of normal for age as per local laboratory. Participants with hyperbilirubinemia as the result of hyperhemolysis, or a severe drop in hemoglobin post blood transfusion, are not excluded as long as it downtrends and return to acceptable limits subsequently. * Left ventricular shortening fraction \<25% or ejection fraction \<40% by echocardiogram. * Estimated creatinine clearance less than 50 mL/min/1.73m2. * Diffusion capacity of carbon monoxide (DLCO) \<35% (adjusted for hemoglobin) OR baseline oxygen saturation \<85% or PaO2 \<70. * Presence of anti-donor specific HLA antibodies unresponsive to desensitization.
Study sites (1)
TN
- St Jude Children's Research Hospital · Memphis, TN 38105Akshay Sharma, MD · Study contact · 866-278-5833 · referralinfo@stjude.orgAkshay Sharma, MD · Principal investigatorMore trials in Memphis