Second chance: stem cell transplant trial aims to rescue failing sickle cell treatments
NCT ID NCT04008368
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study offers a second stem cell transplant to people with sickle cell disease or beta-thalassemia whose first transplant is losing its effect. Participants receive donor stem cells along with drugs to help their body accept them. The goal is to restore donor cell levels and control the disease long-term.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Donor stem cells (CD34-selected cells)
- What this could lead to
- If successful, this could offer a way to regain disease control for people whose first stem cell transplant is wearing off, potentially reducing symptoms and complications.
- What could go wrong
- This is an early-phase trial with only 32 participants, so results may not apply to everyone. There are risks of graft rejection, graft-versus-host disease, and side effects from the conditioning drugs.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 32 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Oct 2019
- Expected to finish
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Dec 2037
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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2 to 80 years
- Sex
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Anyone
- Healthy volunteers
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Accepted
You do not need to have the condition being studied to take part.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: Inclusion criteria- recipient 1. Patient with history of SCD or beta thalassemia who previously underwent allogeneic hematopoietic stem cell transplantation (HSCT) 2. Patient with recurrent SCD defined as HbS greater than or equal to 50% for donors with sickle cell trait and greater than or equal to 10% for donors with HbAA with recurrent clinical manifestations (for example but not limited to recurrent painful crises, acute chest syndrome, priapism, or severe anemia) or patients with recurrent beta thalassemia defined as clinical manifestations such as transfusion-dependence or evidence of extramedullary hematopoiesis. The HbS requirement may be waived by the PI or designee for reasons such as the patient is requiring chronic transfusion therapy or otherwise meets clinical criteria for return of SCD. 3. Persistent donor chimerism levels 4. Age greater than or equal to 4 years 5. Negative beta-HCG 6. Ejection fraction greater than or equal to 35% 7. DLCO greater than or equal to 35% (Note: This criterion may be omitted in young children (e.g. near the age of 5 years old) or other individuals who may have difficulty understanding or complying with the instructions for accurate testing.) Inclusion- donor Related donor from original transplant, deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Related donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Age \>= 2 years \<80 years old EXCLUSION CRITERIA: Exclusion criteria- recipient 1. ECOG performance status of 3 or more or if \<16 years of age Lansky score of 50 or lower. 2. Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to signing the consent 3. Patients with fever or suspected minor infection should await resolution of symptoms before signing the consent 4. Major anticipated illness or organ failure incompatible with survival from PBSC transplant 5. Pregnant or breastfeeding 6. History of secondary malignancies (other than localized skin cancer) 7. Donor-specific HLA antibodies with mean fluorescent intensity \>= 2,000 Exclusion- donor None
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can adding common pain drugs reduce morphine needs in sickle cell crises?
- Gene editing offers hope for a One-Time sickle cell cure
- Tiny biochip could reveal sickle cell severity
- Can a milder transplant cure sickle cell and thalassemia in adults?
- Can an antioxidant supplement calm sickle cell blood cells?
- Can a softer transplant cure sickle cell disease?