NCT05153967 · Vanderbilt University Medical Center
Cooperative Assessment of Late Effects for SCD Curative Therapies
(COALESCE)
What this study is about
Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States.
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Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States. Mortality for children with SCD has decreased substantially over the past 4 decades, with \>99% of those born in high resource settings, including the United States, France, and England, now surviving to 18 years of age. However, the life expectancy of adults with SCD is severely shortened. Dysfunction of the heart, lung, and kidney is directly associated with decreased life expectancy. With the variety of curative therapies that are now available for SCD, long-term health outcomes studies are time-sensitive. As of now, efforts to determine long-term health outcomes following curative therapies for SCD have been limited. Though curative therapies initially should provide a cure for symptoms of SCD, there is the risk of late health outcomes to consider. Defining health outcomes following curative therapy is essential to improve personalized decision-making when considering curative versus disease-modifying therapeutic options. The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.
Primary outcome measures
Measurement of longitudinal change in FEV1
Time frame: Through study completion, an average of four years
Measurements of forced expiratory volume in 1 second (FEV1) based on the global lung index will be acquired from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. FEV1 will be reported in liters. (References: Eur Respir J. Volume 40 Issue 6: pages 1324-1343, 2012 June 27; Am J Hematol. Volume 93 Issue 3: pages 408-415, 2018 March).
Percent predicted value of longitudinal change in FEV1
Time frame: Through study completion, an average of four years
Percent predicted of forced expiratory volume in 1 second (FEV1) based on the global lung index will be acquired from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. FEV1 will be reported in percentage. (References: Eur Respir J. Volume 40 Issue 6: pages 1324-1343, 2012 June 27; Am J Hematol. Volume 93 Issue 3: pages 408-415, 2018 March).
Measurement of longitudinal change in FVC
Time frame: Through study completion, an average of four years
Measurements of forced volume capacity (FVC) based on the global lung index will be acquired from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. FVC will be reported in liters. (References: Eur Respir J. Volume 40 Issue 6: pages 1324-1343, 2012 June 27; Am J Hematol. Volume 93 Issue 3: pages 408-415, 2018 March).
Percent predicted value of longitudinal change in FVC
Time frame: Through study completion, an average of four years
Percent predicted of forced volume capacity (FVC) on the global lung index will be acquired from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. FVC will be reported in percentage. (References: Eur Respir J. Volume 40 Issue 6: pages 1324-1343, 2012 June 27; Am J Hematol. Volume 93 Issue 3: pages 408-415, 2018 March).
FEV1/FVC Ratio Percentage
Time frame: Through study completion, an average of four years
Percentage of FEV1/FVC ratio based on the global lung index will be acquired from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. (References: Eur Respir J. Volume 40 Issue 6: pages 1324-1343, 2012 June 27; Am J Hematol. Volume 93 Issue 3: pages 408-415, 2018 March). FEV1/FVC will be reported in percentage.
Longitudinal change in eGFR
Time frame: Through study completion, an average of four years
Estimated GFR (eGFR) as a determinant of kidney disease will be collected from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. The eGFR will be tested as a linear variable and using eGFR Categories according to the KDIGO 2012 Guidelines (Table 5 - Reference: Kidney Int Suppl. Volume 3 Issue 1: pages 19-62, 2013 Jan) as follows: i.G1 \>/ 90 mL/min/1.73m2 ii.G2 60 - 89 mL/min/1.73m2 iii.G3a 45 - 59 mL/min/1.73m2 iv.G3b 30 - 44 mL/min/1.73m2 v.G4 15 - 20 mL/min/1.73m2 vi.G5 \< 15 mL/min/1.73m2 The standard technique of measured GFR will be used and reported in mL/min/1.73m2.
Longitudinal change in albuminuria levels
Time frame: Through study completion, an average of four years
Data pertaining to persistent albuminuria (defined as \>/ 30 mg/g creatinine on 2 evaluations) associated with a more rapid decline in eGFR on longitudinal follow-up (Reference: Blood Adv. Volume 4 Issue 7: pages 1501-1511, 2020 Apr 14) will be tested. Results will be reported in mg/g.
Longitudinal change in TRJV in adults with SCD treated with nonmyeloablative allo HSCT in adults
Time frame: Through study completion, an average of four years
Improvement in TRJV in adults with SCD following HSCT will be acquired. Results will be reported in m/sec.
Longitudinal change in SBP/DBP in adults with SCD treated with nonmyeloablative allo HSCT in adults
Time frame: Through study completion, an average of four years
Measurements of Systolic blood pressure (SBP)/Diastolic blood pressure (DBP) in adults with SCD following HSCT will be acquired. Results will be reported as a ratio (SBP (mmHg)/DBP (mmHg)).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Confirmed laboratory diagnosis of SCD
- Ability to give informed consent
- Ability to provide pre- and post-curative therapy data
- Treated with either one HSCT or with standard disease-modifying therapy
Exclusion criteria
- •History of non-compliance
Where
- Washington D.C., District of Columbia
- Atlanta, Georgia
- Baltimore, Maryland
- Bethesda, Maryland
- Nashville, Tennessee
Collaborators
Johns Hopkins University, Children's National Research Institute, Emory University, University of Illinois at Chicago, Children's Healthcare of Atlanta
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 27, 2026 · Source of record for eligibility and locations