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New treatment option for Sickle Cell Disease
Official title Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
ClinicalTrials.gov ID: NCT07599176
What this study is testing
What is Alemtuzumab?
Alemtuzumab is an investigational medicine, being studied as a potential treatment for sickle cell disease.
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
- 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.
- Phase 2: a mid-size study of how well it works
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 65
You may be able to join if
- RECIPIENT: Participants must fulfill one disease category (1 or 2) and 3
- 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)...
- 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...
You likely can't join if
- 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...
- Baseline oxygen saturation of \<85% or PaO2 \<70
- Left ventricular ejection fraction: \<35% estimated by ECHO
- Transaminases \>5x upper limit of normal for age
See the full eligibility criteria
- RECIPIENT: Participants must fulfill one disease category (1 or 2) and 3
- 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...
- 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
- 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 people 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...
- Male people 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...
- 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...
- 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 people
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, 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 listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years to 65 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.