Status:
RECRUITING
Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Mortality in Sickle Cell
Sickle Cell Cardiopulmonary Complications
Eligibility:
All Genders
18-100 years
Brief Summary
Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn ...
Detailed Description
Study Description: This study seeks to evaluate heart, lung, liver, kidney, brain, and neurocognitive function post-hematopoietic stem cell transplant (HSCT) in patients with sickle cell disease (SCD...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, aged \>=18 years
- Current or previous diagnosis of any type of SCD (including HbSS, HbSC, HbSbeta0-thal, HbSbeta+-thal)
- Ability to travel to the NIH Clinical Center
- Ability of subject to understand and the willingness to sign a written informed consent document.
- Ability to undergo required studies except as specified in the Exclusion Criteria.
- Curative Therapies Group
- -Plan to receive conditioning for curative therapy at the NIH Clinical Center, Vanderbilt University Medical Center, or Johns Hopkins Hospital
- Non-Curative Therapy Group
- \-- No plan to undergo curative therapy within 2 years
- At least one of the following eligibility criteria:
- History of stroke or abnormal transcranial doppler examination (\>= 200 m/s)
- History of SCD-related renal insufficiency defined as a creatinine level \>=1.3 mg/dL and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance \< 50mL/min
- 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 on a therapeutic dose of hydroxyurea
- Any acute chest syndrome while on a therapeutic dose of hydroxyurea
- Osteonecrosis of 2 or more joints
- Red cell alloimmunization
- EXCLUSION CRITERIA:
- All individuals meeting any of the exclusion criteria at baseline will be excluded from study participation.
- Prior transplantation (including but not limited to HSCT and kidney transplant)
- Pregnant or breastfeeding
- Patients with allergy to iodine or iodinated contrast solutions will not undergo Iothalamate or Iohexal GFR clearance testing but can undergo the other deep phenotype testing
- Implanted metal object that is not compatible with MRI (e.g.: cerebral aneurysm clip, cochlear implant, or pacemaker)
- 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
- Patients requiring peritoneal or hemodialysis
- Uncontrolled infection or acute illness
- Criteria specific to the non-curative therapy group:
- Hydroxyurea initiation or dose adjustment \<2 months prior
- Initiation of chronic transfusion therapy \<2 months prior
- Antihypertensive medication initiation or dose adjustment \<1 month prior
Exclusion
Key Trial Info
Start Date :
March 24 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
June 1 2035
Estimated Enrollment :
200 Patients enrolled
Trial Details
Trial ID
NCT05213572
Start Date
March 24 2022
End Date
June 1 2035
Last Update
November 10 2025
Active Locations (1)
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1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892