Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
Matched Related Donor Non-myeloablative Hematopoietic Cell Transplantation With Alemtuzumab, 400 cGy TBI, and Abatacept for Sickle Cell Disease and Beta-Thalassemia
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 4 Years to 65 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 partial stem cell transplant from a fully matched donor. It is for people with severe sickle cell disease or beta-thalassemia, a blood disorder that requires red blood cell transfusions. The transplant is slightly stronger than earlier treatments, with the goal of keeping more donor cells in the body long term.
This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Recipients must be age 4 to 64 and meet the sickle cell disease or beta-thalassemia rules below.
- People with sickle cell disease must have a qualifying problem, such as brain injury, abnormal brain blood flow, kidney or liver damage, an abnormal heart scan, repeated painful erections, or pain crises or acute chest syndrome despite treatment.
- People with beta-thalassemia must have grade 2 or 3 iron buildup, with at least two of these: liver scarring, a history of not getting enough iron-removing treatment, or an enlarged liver.
- Recipients must have a fully matched HLA donor. HLA markers help match the donor's tissue type to the recipient's.
- Recipients must understand and agree to take part. Children must also agree when appropriate. A pregnancy test must be negative when needed.
- Recipients must follow birth control rules during the study, until medicines that lower immune defenses are stopped, and for 3 months after abatacept or sirolimus.
- Donors must be at least age 4 and weigh at least 15 kilograms, unless the medical team approves another weight.
- Donors must pass the NIH checks for safe stem cell donation.
Who can't join
- Recipients with a score below 40 on a test of how well they can do daily activities.
- Recipients with lung test results below 35% of expected. This test may be skipped for young children or others who cannot follow the test instructions.
- Recipients with blood oxygen levels below the study's limits.
- Recipients whose heart pumps out less than 35% of its blood with each beat.
- Recipients with liver enzyme levels more than 5 times the normal upper limit for their age.
- Recipients with an infection that keeps getting worse despite medicine within the month before transplant preparation.
- Recipients with a serious illness or organ failure that would make surviving the transplant unlikely.
- Recipients or donors who are pregnant or breastfeeding; donors with problems with thinking or understanding.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
* INCLUSION CRITERIA RECIPIENT: Participants must fulfill one disease category (1 or 2) and 3 1. Patients with sickle cell disease at high risk for disease related morbidity or mortality, defined by having an end-organ damage (A, B, C, D, OR E) or complication(s) not ameliorated by sickle cell-specific therapies (F): A. Stroke defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI ORb B. Abnormal trans-cranial Doppler examination (\>=200 cm/s); OR C. Silent cerebral infarct defined as an infarct-like lesion based on an MRI signal abnormality at least 3 mm in one dimension and visible in two planes on FLAIR or T2- weighted images (or similar image with 3D imaging) and documented neurological examination performed by a neurologist demonstrating the participant has a normal neurologic examination, or an abnormality on examination that could not be explained by the location of the brain lesion(s); OR D. Sickle cell related renal insufficiency defined by a creatinine level \>=1.5 times the upper limit of normal and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance \<60mL/min/1.73m2 for patients \<16 years of age or \<50mL/min for patients \>16 years of age OR requiring peritoneal or hemodialysis; OR Age (Years): \<= 5 / Upper limit of normal serum creatinine (mg/dl): 0.8 Age (Years): 5 \< age \<= 10 / Upper limit of normal serum creatinine (mg/dl): 1.0 Age (Years): 10 \< age \<= 15 / Upper limit of normal serum creatinine (mg/dl): 1.2 Age (Years): \> 15 / Upper limit of normal serum creatinine (mg/dl): 1.3 E. Tricuspid regurgitant jet velocity (TRV) of \>=2.5 m/s in patients at least 3 weeks after a vaso-occlusive crisis; OR F. Recurrent severe priapism defined as at least two episodes of an erection lasting \>=4 hours requiring medical intervention (e.g. aspiration, injection of vasoconstrictor, prior penile surgery.); OR G. Sickle hepatopathy defined as EITHER ferritin \>1000mcg/L OR direct bilirubin \>0.4 mg/dL at baseline; OR H. Vaso-occlusive crises: more than 1 hospital admission per year while on a therapeutic dose of sickle cell treatment /medication; OR I. Acute chest syndrome (ACS): any ACS while on sickle cell treatment /medication 2. Patients with beta-thalassemia who have grade 2 or 3 iron overload, determined by the presence of 2 or more of the following: * Portal fibrosis by liver biopsy * Inadequate chelation history (defined as failure to maintain adequate compliance with chelation with deferoxamine initiated within 18 months of the first transfusion and administered at least 5 days each week) * Hepatomegaly of greater than 2 cm below the costochondral margin or by other imaging scans 3. Non disease specific * Ages \>=4 years and less than 65 years old * Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10) * Ability to comprehend and willing to sign an informed consent, assent obtained from minors when applicable. Negative serum or urine beta-HCG, when applicable * Agree to use birth control throughout the study and 3 months after abatacept or sirolimus administration. * Female subjects must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or implant/injection from start of screening until immunosuppression is stopped. * Male subjects must agree to use effective contraception (including condoms) from start of screening until immunosuppression is stopped. DONOR: * Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10) are intended for this study. * Donors age 4 or older and \>=15 kg (or weight deemed acceptable by IR for line placement, DTM for apheresis, and pediatric consult service) eligible to donate hematopoietic stem cells, are eligible for this study. * Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Donors will sign on a separate protocol, 20-H-0099 NHLBI standard of care protocol for the mobilization and collection of HSCs. Note that participation in this study is offered to all eligible donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study. EXCLUSION CRITERIA RECIPIENT: * Karnofsky or Lanksy performance status of \<40 * Diffusing capacity of carbon monoxide \<35% predicted: DLCO corrected for hemoglobin or KCO (corrected for lung volume). This criterion may be omitted in young children (e.g. near age 5) or other individuals who may have difficulty understanding or complying with instructions of testing. * Baseline oxygen saturation of \<85% or PaO2 \<70 * Left ventricular ejection fraction: \<35% estimated by ECHO * Transaminases \>5x upper limit of normal for age * Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to starting the conditioning regimen * Major anticipated illness or organ failure incompatible with survival from HCT * Pregnant or breastfeeding DONOR: * Pregnant or breastfeeding * Cognitively impaired subjects
Study sites (1)
MD
- National Institutes of Health Clinical Center · Bethesda, MD 20892NIH Clinical Center Office of Patient Recruitment (OPR) · Study contact · 800-411-1222 ext. TTY dial 711 · ccopr@nih.govMore trials in Bethesda