Clinical trial 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?
An investigational monoclonal antibody being studied as part of a non‑myeloablative stem cell transplant for severe sickle cell disease. Alemtuzumab is designed to bind CD52 on immune cells and deplete them, helping reduce host immune resistance to donor stem cells.
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 study is testing whether a gentler stem cell transplant works well. It aims to keep donor cells in your body for a long time.
- Who it's for
- You may be able to join if you have severe sickle cell disease or beta-thalassemia. You must need regular red blood cell transfusions.
- What's involved
- You would get a stem cell transplant from a matched donor. The treatment is slightly stronger than older versions to help donor cells stay.
- Time commitment
- The source text does not say how long the study lasts or what visits are needed.
- 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)...
- 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...
- Hepatomegaly of greater than 2 cm below the costochondral margin or by other imaging scans
You likely can't join if
- Karnofsky or Lanksy performance status of \<40
- Diffusing capacity of carbon monoxide \<35% predicted: DLCO corrected for hemoglobin or KCO (corrected for lung volume).
- 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...
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...
- 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...
- 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.
- 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.
- 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
- Cognitively impaired people
The study team makes the final eligibility decision.
Where it is enrolling
- 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, but 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?
This study is enrolling now at Bethesda, Maryland, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.