Recruiting

SCD Curative Therapies

Sponsor:

Vanderbilt University Medical Center

Code:

NCT05153967

Conditions

Sickle Cell Disease

Pulmonary Disease

Renal Disease

Heart Disease

Eligibility Criteria

Sex: All

Age: 4 - 65

Healthy Volunteers: Not accepted

Study Details

Brief summary:

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.

Conditions

Sickle Cell Disease

Pulmonary Disease

Renal Disease

Heart Disease

Study ID

NCT05153967

Start date

Jul 12, 2022

Status verified date

Jul, 2026

Completion date

Feb, 2027

Anticipated

Primary completion date

Nov, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 4 - 65

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

750 participants

Anticipated

Interventions and Outcome Measures

Arms

Pediatric Myeloablative allo-HSCT

Participants ages 4 to 17 years old with SCD who underwent or are scheduled to undergo myeloablative allo-HSCT.

Pediatric Standard Disease-Modifying Therapy

Participants ages 4 to 17 years old with SCD who receive standard therapy.

Adult Non-Myeloablative allo-HSCT

Participants ages 18 to 65 years old with SCD who underwent or are scheduled to undergo non-myeloablative allo-HSCT.

Adult Standard Disease-Modifying Therapy

Participants ages 18 to 65 years old with SCD who receive standard therapy.

Primary outcome measure

  • Measurement of longitudinal change in FEV1 [ Time Frame: Through study completion, an average of four years ]
  • Percent predicted value of longitudinal change in FEV1 [ Time Frame: Through study completion, an average of four years ]
  • Measurement of longitudinal change in FVC [ Time Frame: Through study completion, an average of four years ]
  • Percent predicted value of longitudinal change in FVC [ Time Frame: Through study completion, an average of four years ]
  • FEV1/FVC Ratio Percentage [ Time Frame: Through study completion, an average of four years ]
  • Longitudinal change in eGFR [ Time Frame: Through study completion, an average of four years ]
  • Longitudinal change in albuminuria levels [ Time Frame: Through study completion, an average of four years ]
  • 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 ]
  • 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 ]

Central Contacts and Locations

Central contacts

Leshana Saint Jean, PhD

6158751992leshana.saint.jean@vumc.org

Kristin Wuichet, PhD

6159366098kristin.wuichet@vumc.org

Locations

Vanderbilt University Medical Center

Recruiting

Nashville, Tennessee, United States, 37232-9000

Contacts

Michael R. DeBaun, MD, MPH

615-875-3040m.debaun@vumc.org

More Information

Sponsor

Vanderbilt University Medical Center

Last update posted

Jul 27, 2026

Last verified

Jul, 2026

Keywords

  • Myeloablative Autologous Gene Editing
  • Myeloablative Autologous Gene Therapy
  • Myeloablative allo-HSCT
  • Nonmyeloablative allo-HSCT
  • Disease-Modifying Therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Vanderbilt University Medical Center on 2026-07-27.