Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies (NCT05213572) | Clinical Trial Compass
RecruitingNot Applicable
Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies
United States200 participantsStarted 2022-03-24
Plain-language 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 how and why this happens.
Objective:
To study the benefits of treatments that are intended to cure SCD.
Eligibility:
People aged 18 and older with SCD who are either receiving curative therapy in the next 3 months or don t have any plans to receive a curative therapy in the next 2 years.
Design:
At their first visit, participants will be screened with their medical history and a physical exam.
Participants will then have a baseline visit. This will take about a week to complete and will include:
Blood and heart tests
MRI of the brain, heart, and lungs. Participants will lie on a bed that will move into the MRI scanner. Special padding may be placed around their head to keep it still.
Interactive games. Participants will complete computer games that test memory, attention, problem solving, language, spatial orientation, processing speed, and emotion.
Questionnaire rating quality of life
Iothalamate test. An IV catheter will be placed into a vein. A contrast agent will be injected through the IV. Blood will then be collected at different time points.
Lung function tests and a 6-minute walk test
Vibration controlled transient elastography. A probe placed on the abdomen will measure liver scarring.
DOS test. A light attached to the finger or toe will measure blood oxygen.
Participants will have an end-of-study visit about 2 years after their baseline visit. This will include repeats of the baseline visit tests.
Who can participate
Age range18 Years – 100 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Stated willingness to comply with all study procedures and availability for the duration of the study
✓. Male or female, aged \>=18 years
✓. Patients with current or previous diagnosis of any type of SCD (including HbSS, HbSC, HbSbeta0-thal, HbSbeta+-thal) who:
✓. plan to receive an allogeneic HCT or gene therapy,
✓. are receiving non-curative treatment (standard of care or investigational) and who do not plan to receive an allogeneic HCT or gene therapy
✓. Ability to travel to the NIH Clinical Center
✓. Ability of subject to understand a written informed consent document.
✓. At least one of the following eligibility criteria:
Exclusion criteria
✕. Prior transplantation (including but not limited to HSCT and kidney transplant)
✕
What they're measuring
1
Change in LVEDV/BSA in patients with SCD who undergo curative therapies as compared to patients who receive non-curative treatment
Timeframe: 2 years after initial testing
Trial details
NCT IDNCT05213572
SponsorNational Heart, Lung, and Blood Institute (NHLBI)
✕. 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