Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies

Conditions

• Mortality in Sickle Cell
• Sickle Cell Cardiopulmonary Complications
• Sickle Cell Life Expectancy and Risk Factors for Early Death
• Sickle Cell Lung Disease and Sudden Death
• Sickle Cell Organ Damage

Eligibility Criteria

Sex

ALL

Age

18 Years - 100 Years

Healthy Volunteers

Not accepted

Study Details

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.

Key Dates

Start date

Mar 24, 2022

Status verified

May 2026

Primary completion

Jun 1, 2035

Completion

Jun 1, 2035

Study Design

Enrollment

200 participants (estimated)

Arms

• Arm: subjects w sickle cell disease
  to evaluate heart, lung, liver, kidney, brain, and neurocognitive function post-hematopoietic stem cell transplant (HSCT) in subjects with sickle cell disease (SCD) who undergo curative therapies

Primary Outcome Measure

Change in LVEDV/BSA in patients with SCD who undergo curative therapies as compared to patients who receive non-curative treatment [ Time Frame: 2 years after initial testing ]

Central Contacts

• Julia M Varga, R.N.
  (301) 402-3595
  [email protected]
• Courtney F Joseph, M.D.
  (301) 402-6496
  [email protected]

Locations (1)

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