Sickle cell breakthrough: can curative therapies heal damaged organs?
NCT ID NCT05213572
First seen Jun 27, 2026 · Last updated Aug 28, 2026 · Updated 10 times
Summary
This study looks at adults with sickle cell disease to see how their heart, brain, kidneys, liver, and lungs change after treatments meant to cure the disease. Researchers will compare people getting curative therapy (like a bone marrow transplant or gene therapy) with those who are not. Participants undergo tests like MRIs, blood work, and lung function tests at the start and again after two years.
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Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 200 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2022
- Expected to finish
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Jun 2035
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.
Who is studied
Patients aged \>or= 18 years who will undergo curative therapies at the NIH Clinical Center and who have a diagnosis of any type of SCD (including HbSS, HbSC, HbSbeta0-thal, HbSbeta+-thal).
- Ages
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18 to 100 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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: In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Stated willingness to comply with all study procedures and availability for the duration of the study 2. Male or female, aged \>=18 years 3. Patients with current or previous diagnosis of any type of SCD (including HbSS, HbSC, HbSbeta\^0-thal, HbSbeta\^+-thal) who: 1. plan to receive an allogeneic HCT or gene therapy, Or 2. are receiving non-curative treatment (standard of care or investigational) and who do not plan to receive an allogeneic HCT or gene therapy 4. Ability to travel to the NIH Clinical Center 5. Ability of subject to understand a written informed consent document. 6. At least one of the following eligibility criteria: * History of stroke (including silent stroke) or abnormal transcranial doppler examination (\>= 200 m/s) * Progression of central nervous system vasculopathy on MRA * History of SCD-related renal insufficiency defined as nephrotic syndrome OR creatinine clearance \< 60mL/min OR a creatinine level \>=1.3 mg/dL AND kidney biopsy consistent with sickle cell nephropathy * Tricuspid regurgitant velocity \>= 2.5 m/s * Recurrent tricorporal priapism defined as at least 2 episodes of an erection lasting \> 4 hours involving the corpora cavernosa and corpus spongiosa * SCD-associated liver disease defined as EITHER ferritin \> 1000 mcg/L OR direct bilirubin \> 0.4 mg/dL * \> 1 hospitalization per year for vaso-occlusive crises while receiving hydroxyurea treatment * A history of 2 or more acute chest syndrome episodes in the participant's lifetime. * Avascular necrosis of 2 or more joints * Red cell alloimmunization (delayed hemolytic transfusion reaction or significant red blood cell alloimmunization that makes transfusion difficult). * Administration of regular red blood cell (RBC) transfusion therapy (\>=8 red cell transfusions in the previous 12 months) to prevent SCD complications (i.e. pain, stroke, or acute chest syndrome). * Eligible for an allogeneic HCT or gene therapy at an outside transplant center. EXCLUSION CRITERIA: All individuals meeting any of the exclusion criteria at baseline will be excluded from study participation. 1. Prior transplantation (including but not limited to HSCT and kidney transplant) 2. Pregnant or breastfeeding 3. Patients with allergy to iodine or iodinated contrast solutions will not undergo Iothalamate or Iohexol GFR clearance testing but can undergo the other deep phenotype testing 4. Implanted metal object that is not compatible with MRI (e.g.: cerebral aneurysm clip, cochlear implant, or pacemaker) 5. Patients with a pacemaker or automated implantable cardioverter defibrillator will not undergo VCTE but can enroll and undergo the other deep phenotype testing as long as the device is compatible with MRI and MRI testing can be performed 6. Patients requiring peritoneal or hemodialysis 7. Uncontrolled infection or acute illness
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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?