RECRUITING

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 Organ Damage Sickle Cell Life Expectancy and Risk Factors for Early Death Sickle Cell Lung Disease and Sudden Death Hydroxyurea Hydroxyurea-Increased Fetal Hemoglobin sickle cell anemi Patterns of mortality in sickle cell disease Mortality rates and age at death from sickle cell Natural History

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2022-03-24

Study Completion:2035-06-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05213572

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 100 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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. Current or previous diagnosis of any type of SCD (including HbSS, HbSC, HbSbeta0-thal, HbSbeta+-thal) 4. Ability to travel to the NIH Clinical Center 5. Ability of subject to understand and the willingness to sign a written informed consent document. 6. Ability to undergo required studies except as specified in the Exclusion Criteria. 7. Curative Therapies Group 8. Non-Curative Therapy Group 9. 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	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 Iohexal 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 8. Criteria specific to the non-curative therapy group: 1. Hydroxyurea initiation or dose adjustment \<2 months prior 2. Initiation of chronic transfusion therapy \<2 months prior 3. Antihypertensive medication initiation or dose adjustment \<1 month prior

Inclusion Criteria

Exclusion 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. Current or previous diagnosis of any type of SCD (including HbSS, HbSC, HbSbeta0-thal, HbSbeta+-thal)
4. Ability to travel to the NIH Clinical Center
5. Ability of subject to understand and the willingness to sign a written informed consent document.
6. Ability to undergo required studies except as specified in the Exclusion Criteria.
7. Curative Therapies Group
8. Non-Curative Therapy Group
9. 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

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 Iohexal 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
8. Criteria specific to the non-curative therapy group:
1. Hydroxyurea initiation or dose adjustment \<2 months prior
2. Initiation of chronic transfusion therapy \<2 months prior
3. Antihypertensive medication initiation or dose adjustment \<1 month prior

Contacts and Locations

Study Contact

Jennifer L Brooks, R.N.

CONTACT

(301) 480-6149

jennifer.brooks2@nih.gov

Courtney F Joseph, M.D.

CONTACT

(301) 402-6496

courtney.fitzhugh@nih.gov

Principal Investigator

Courtney F Joseph, M.D.

PRINCIPAL_INVESTIGATOR

National Heart, Lung, and Blood Institute (NHLBI)

Study Locations (Sites)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892

United States

Collaborators and Investigators

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Courtney F Joseph, M.D., PRINCIPAL_INVESTIGATOR, National Heart, Lung, and Blood Institute (NHLBI)

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-03-24

Study Completion Date2035-06-01

Study Record Updates

Study Start Date2022-03-24

Study Completion Date2035-06-01

Terms related to this study

Keywords Provided by Researchers

Hydroxyurea
Hydroxyurea-Increased Fetal Hemoglobin
sickle cell anemi
Patterns of mortality in sickle cell disease
Mortality rates and age at death from sickle cell
Natural History

Additional Relevant MeSH Terms

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