Sickle Cell DiseaseCOALESCENCT05153967
Cooperative Assessment of Late Effects for SCD Curative Therapies
An observational study of Sickle Cell Disease and Pulmonary Disease, run by Vanderbilt University Medical Center at 5 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 65 years
- Conditions the study is for
- Sickle Cell Disease, Pulmonary Disease, Renal Disease
- How many people
- 377 participants
- Where
- 5 sitesUnited States
- Study dates
- 12 Jul 2022to Feb 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants will be patient volunteers from the sickle cell disease clinics and transplant centers of participating study sites.
Who may be able to join
- 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
Who cannot join
- •History of non-compliance
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
Johns Hopkins Hospital
Baltimore, Maryland, 21287
National Institutes of Health Clinical Center
Bethesda, Maryland, 20814
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-9000
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: U01 Cooperative Assessment of Late Effects for Sickle Cell Disease Curative Therapies
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.
Read the detailed description
Our primary objective is initiating a personalized approach to curative therapies in children and adults with sickle cell disease (SCD) to maximize benefits and limit adverse outcomes. Limited clinical studies exist to determine the long-term health outcomes following curative therapies for SCD. With emerging curative therapies for SCD (allogeneic [allo] hematopoietic stem cell transplant [HSCT], gene therapy/editing), long-term health outcomes studies are critical to inform personalized choices. Unfortunately, adverse outcomes have started to emerge after SCD curative therapy. Thus, risks of a cure in SCD must be measured against the benefits of a cure, including stabilization of lung function (FEV1) and improved tricuspid regurgitant jet velocity [TRJV]. Ultimately, the shortened lifespan of individuals with SCD, attributable to declining heart (elevated TRJV), lung (decreased FEV1), and kidney (decreased eGFR) function, for which curative therapies were designed to ameliorate, must be measured against favorable and unfavorable late outcomes. In our multicenter retrospective-prospective cohort, the investigators will test the following hypotheses: 1a): myeloablative curative therapies for children with SCD will result in progressive pulmonary and renal dysfunction when compared to children with SCD receiving standard therapy; 1b): nonmyeloablative HSCT for adults with SCD will result in no significant change in FEV1% predicted, but will lead to accelerated decline in eGFR when compared to adults receiving standard therapy; 2) nonmyeloablative HSCT for adults with SCD will be associated with a clinically significant improvement in TRJV following HSCT; and 3) in adults with SCD, proliferative and genotoxic stress uniformly related to nonmyeloablative allo-HSCT and myeloablative gene editing will lead to post-HSCT therapy-related myeloid neoplasm of recipient origin. The investigators will address these hypotheses with the following aims: 1) evaluate the incidence of pulmonary and renal function in 1a: children with SCD receiving myeloablative curative therapies; and 1b: adults with SCD receiving nonmyeloablative allo-HSCT, compared to a pre-existing cohort of children and adults with SCD; 2) determine whether there is a clinically significant improvement in TRJV in adults with SCD, at least half having TRJV > 2.5 m/s, following nonmyeloablative allo-HSCT, 3) evaluate the prevalence, incidence and evolution of Clonal hematopoiesis of indeterminate potential (CHIP) concerning therapy-related myeloid neoplasm development following non-myeloablative HSCT or myeloablative gene editing in adults and children with SCD, and 4) evaluate accuracy and gaps involved in collecting clinical health record data directly from patients and family members in comparison to clinical health record data collected by research coordinators.
- Conditions
- Sickle Cell Disease, Pulmonary Disease, Renal Disease, Heart Disease
- Keywords
- Myeloablative Autologous Gene Editing, Myeloablative Autologous Gene Therapy, Myeloablative allo-HSCT, Nonmyeloablative allo-HSCT, Disease-Modifying Therapy
- Medical subject headings
- Anemia, Sickle Cell, Lung Diseases, Kidney Diseases, Heart Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measurement of longitudinal change in FEV1
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).
Measured: Through study completion, an average of four years
Percent predicted value of longitudinal change in FEV1
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).
Measured: Through study completion, an average of four years
Measurement of longitudinal change in FVC
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).
Measured: Through study completion, an average of four years
Percent predicted value of longitudinal change in FVC
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).
Measured: Through study completion, an average of four years
FEV1/FVC Ratio Percentage
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.
Measured: Through study completion, an average of four years
Longitudinal change in eGFR
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.
Measured: Through study completion, an average of four years
Longitudinal change in albuminuria levels
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.
Measured: Through study completion, an average of four years
Longitudinal change in TRJV in adults with SCD treated with nonmyeloablative allo HSCT in adults
Improvement in TRJV in adults with SCD following HSCT will be acquired. Results will be reported in m/sec.
Measured: Through study completion, an average of four years
Longitudinal change in SBP/DBP in adults with SCD treated with nonmyeloablative allo HSCT in adults
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)).
Measured: Through study completion, an average of four years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2021
- Study start12 Jul 2022
- Primary completionNov 2026 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 377 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Johns Hopkins University, Children's National Research Institute, Emory University, University of Illinois Chicago, Children's Healthcare of Atlanta
- Responsible party
- Principal investigator, Michael DeBaun, Vanderbilt University Medical Center
- Investigators
- Michael R DeBaun, MD, MPH, Principal investigator, Vanderbilt University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05153967
- Sponsor's protocol ID
- 210806
- Acronym
- COALESCE
- Status last verified
- Oct 2026
- First submitted
- 25 Oct 2021
- Last updated
- 6 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: This research study's sharing plan involves study data, publications, and outcomes. We will adhere to the NIH Public Access Policy (NOT-OD-08-033) and provide publications generated through funding by this project at the time the final, peer- reviewed papers are accepted for journal publication to the National Library of Medicine's PubMed Central, either through NIHMS (NIH Manuscript Submission) or through the Publisher. All applicable proposals, applications, and reports will include the PubMed Central number (PMCID) for all NIH-funded papers. All sequence data will be uploaded to database of Genotypes and Phenotype (dbGaP).
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- U01 Cooperative Assessment of Late Effects for Sickle Cell Disease Curative Therapies
- ClinicalTrials.gov ID
- NCT05153967
- Acronym / sponsor ID
- COALESCE / 210806
- Conditions
- Sickle Cell Disease, Pulmonary Disease, Renal Disease, Heart Disease
- Keywords
- Myeloablative Autologous Gene Editing, Myeloablative Autologous Gene Therapy, Myeloablative allo-HSCT, Nonmyeloablative allo-HSCT, Disease-Modifying Therapy
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 377 participants (actual)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Collaborators
- Johns Hopkins University, Children's National Research Institute, Emory University, University of Illinois Chicago, Children's Healthcare of Atlanta
- Study start
- 12 Jul 2022 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 10 Dec 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Our primary objective is initiating a personalized approach to curative therapies in children and adults with sickle cell disease (SCD) to maximize benefits and limit adverse outcomes. Limited clinical studies exist to determine the long-term health outcomes following curative therapies for SCD. With emerging curative therapies for SCD (allogeneic [allo] hematopoietic stem cell transplant [HSCT], gene therapy/editing), long-term health outcomes studies are critical to inform personalized choices. Unfortunately, adverse outcomes have started to emerge after SCD curative therapy. Thus, risks of a cure in SCD must be measured against the benefits of a cure, including stabilization of lung function (FEV1) and improved tricuspid regurgitant jet velocity [TRJV]. Ultimately, the shortened lifespan of individuals with SCD, attributable to declining heart (elevated TRJV), lung (decreased FEV1), and kidney (decreased eGFR) function, for which curative therapies were designed to ameliorate, must be measured against favorable and unfavorable late outcomes. In our multicenter retrospective-prospective cohort, the investigators will test the following hypotheses: 1a): myeloablative curative therapies for children with SCD will result in progressive pulmonary and renal dysfunction when compared to children with SCD receiving standard therapy; 1b): nonmyeloablative HSCT for adults with SCD will result in no significant change in FEV1% predicted, but will lead to accelerated decline in eGFR when compared to adults receiving standard therapy; 2) nonmyeloablative HSCT for adults with SCD will be associated with a clinically significant improvement in TRJV following HSCT; and 3) in adults with SCD, proliferative and genotoxic stress uniformly related to nonmyeloablative allo-HSCT and myeloablative gene editing will lead to post-HSCT therapy-related myeloid neoplasm of recipient origin. The investigators will address these hypotheses with the following aims: 1) evaluate the incidence of pulmonary and renal function in 1a: children with SCD receiving myeloablative curative therapies; and 1b: adults with SCD receiving nonmyeloablative allo-HSCT, compared to a pre-existing cohort of children and adults with SCD; 2) determine whether there is a clinically significant improvement in TRJV in adults with SCD, at least half having TRJV > 2.5 m/s, following nonmyeloablative allo-HSCT, 3) evaluate the prevalence, incidence and evolution of Clonal hematopoiesis of indeterminate potential (CHIP) concerning therapy-related myeloid neoplasm development following non-myeloablative HSCT or myeloablative gene editing in adults and children with SCD, and 4) evaluate accuracy and gaps involved in collecting clinical health record data directly from patients and family members in comparison to clinical health record data collected by research coordinators.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 4 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants will be patient volunteers from the sickle cell disease clinics and transplant centers of participating study sites.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- •History of non-compliance
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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)).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2026.
- Individual participant dataSharing planned: Yes. This research study's sharing plan involves study data, publications, and outcomes. We will adhere to the NIH Public Access Policy (NOT-OD-08-033) and provide publications generated through funding by this project at the time the final, peer- reviewed papers are accepted for journal publication to the National Library of Medicine's PubMed Central, either through NIHMS (NIH Manuscript Submission) or through the Publisher. All applicable proposals, applications, and reports will include the PubMed Central number (PMCID) for all NIH-funded papers. All sequence data will be uploaded to database of Genotypes and Phenotype (dbGaP).
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael R DeBaun, MD, MPH principal investigator
Vanderbilt University Medical Center
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease and Pulmonary Disease, run by Vanderbilt University Medical Center at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 6 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measurement of longitudinal change in FEV1 (time frame: Through study completion, an average of four years). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2026.