Sickle Cell Anemia (HbSS)Phase 4ENHANCENCT07177300
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
A Phase 4 study testing PK-optimized oral hydroxyurea at MTD until 15 years of age. in people with Sickle Cell Anemia (HbSS) and Sickle-β0-thalassemia (HbSβ0), run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for people with SCA. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Sickle Cell Anemia (HbSS), Sickle-β0-thalassemia (HbSβ0)
- What is being tested?
- PK-optimized oral hydroxyurea at MTD until 15 years of age.
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Dec 2024to Dec 2028
- Phase
- Phase 4
- 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 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of sickle cell anemia (HbSS) or sickle-β0-thalassemia (HbSβ0)
- Age 6 months at the time of enrollment
- Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea therapy
Who cannot join
- Current treatment with regularly scheduled blood transfusions
- Sickle-hemoglobin C disease (HbSC), sickle-β+-thalassemia (HbSβ+)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Charles T. Quinn, M.D., M.S., 513-803-3086, charles.quinn@cchmc.org
Megan Metcalf, CRC, III, megan.metcalf@cchmc.org
About this study
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for people with SCA.
In this research study, the investigators hope to learn more about how to improve the dosing and monitoring of hydroxyurea and learn more about the long-term effects of hydroxyurea over time. Hydroxyurea is usually dosed based only on your weight. Our study will use a new way to select a starting dose that is based on how each patient absorbs hydroxyurea.
Read the detailed description
The EHANCE study will address key knowledge gaps about hydroxyurea for young children with SCA in nine innovative ways:
- Novel functional assessments of brain, heart, kidneys, spleen, and eyes to assess organ protection in young children who receive hydroxyurea at MTD;
- State-of-the-art assays to assess the benefits of hydroxyurea on growth, development, and reproductive health into puberty including serial measurements of pubertal development and sex hormones;
- A simplified PK-guided strategy to optimize hydroxyurea initiation and dosing, with a long-term goal of validating pharmacogenomic approaches to expand treatment and achieve sustained HbF induction;
- A novel single-cell quantitative HbF/F-cell assay, developed utilizing imaging flow cytometry, will determine the distribution of HbF/F-cell across all F-cells, rather than simply estimating the mean value of HbF/F-cell;
- Collection of genomic DNA samples to allow serial quantitation of clonal hematopoiesis in treated children, to evaluate the possibility for potential emergence of clones with an increased risk of leukemic transformation;
- Studies on primary erythroblasts freshly isolated from patients and control subjects with single cell multiome analysis to evaluate in vivo cis and trans-acting elements that regulate HbF and how they are affected by hydroxyurea;
- Evaluation of cellular mechanisms by which hydroxyurea at MTD can regularly achieve >30% HbF with near-pancellular distribution, similar to levels currently touted with 'curative' gene therapy regimens;
- Exploration of the benefits of early hydroxyurea treatment initiation, in terms of γ-globin de-repression to optimize HbF induction, through unknown cellular mechanisms that may be developmentally regulated.
- SPECIFIC AIMS Aim 1: Document the long-term benefits and risks of long-term hydroxyurea treatment at MTD.
Aim 2: Perform pharmacokinetic (PK) and pharmacodynamic (PD) assessment of hydroxyurea at MTD.
Aim 3: Investigate the cellular mechanisms by which hydroxyurea leads to induction of protective HbF and how timing of treatment initiation and dose optimization affect the efficacy of this process.
- Being tested
- PK-optimized oral hydroxyurea at MTD until 15 years of age.
- Keywords
- sickle cell anemia, SCD, sickle cell, hydroxyurea
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PK-optimized oral hydroxyurea at MTD until 15 years of age.Drug
Also called pharmacokinetic, maximum tolerated dose
Because people are different, we will measure how each participant's body absorbs and eliminates the medicine, hydroxyurea, using blood tests. This information will be used to determine the best dose for each participant (rather than using the same weight-based dose for everyone).
| Study group | Role | What they receive |
|---|---|---|
| Observational Treatment Group (Single Arm) | Experimental | All children with sickle cell anemia who are started on hydroxyurea for clinical indicators between 6 months and 5 years of age can do so on this observational study with PK-optimized hydroxyurea dosing. Receives: Drug: PK-optimized oral hydroxyurea at MTD until 15 years of age. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite Organ Injury
Evidence of injury in any of four critical organ systems: brain, kidney, heart, or spleen. Participants will be classified as having met the composite endpoint if they fulfill at least one of the organ-specific criteria listed: cerebral infarction (silent or overt) or steno-occlusive vasculopathy by MRI of brain; urine albumin-to-creatinine ratio (UACR) > 300 mg/g; extracellular volume fraction (ECV) > 0.35 on cardiac MRI; or erythrocyte pit count < 5%
Measured: Through study completion, an average of 10 years
Secondary outcomes (7)
longitudinal change in fetal hemoglobin percentage (HbF%)
HbF is a well-established mechanistic surrogate marker in SCA that is strongly associated with reduced sickling and organ injury across multiple organ systems. This endpoint will quantify the sustained biological effectiveness of early PK-guided hydroxyurea dosing.
Measured: Through study completion, an average of 10 years
Longitudinal change in hemoglobin concentration (g/dL)
A beneficial laboratory response to hydroxyurea would include less severe anemia (measured by hemoglobin concentration). This is reported in a complete blood count (CBC).
Measured: Through study completion, an average of 10 years.
Longitudinal change in reticulocyte count (10^9/L)
A beneficial laboratory response to hydroxyurea would include a decreased reticulocyte count, indicating less severe anemia. This is reported with a complete blood count (CBC).
Measured: Through study completion, an average of 10 years.
Longitudinal change in absolute neutrophil count (10^9/L)
A surrogate marker of hydroxyurea effectiveness and adherence is the absolute neutrophil count (ANC). Hydroxyurea is titrated according to the ANC. This is reported in a complete blood count (CBC) with differential leukocyte count.
Measured: Through study completion, an average of 10 years.
Longitudinal change in mean cell volume (fL)
A surrogate marker of hydroxyurea effectiveness and adherence is the mean cell volume (MCV). This is reported in a complete blood count (CBC).
Measured: Through study completion, an average of 10 years.
F-cell fraction (%)
Flow cytometric determination of the fraction of HbF-containing red blood cells (F-cells).
Measured: Through study completion, an average of 10 years.
Adverse events
The occurrence of adverse events while on study will be continually monitored and recorded.
Measured: Through study completion, an average of 10 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2025
- Study start19 Dec 2024
- Primary completionDec 2026 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (13)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell Anemia (HbSS)Studies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Prospective, open-label, single-arm therapeutic trial of pharmacokinetics (PK)-optimized oral hydroxyurea at maximum tolerated dose (MTD) for the treatment of sickle cell anemia (SCA) with long-term follow-up.
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Greater Cincinnati Foundation
- Responsible party
- Sponsor
- Investigators
- Charles T. Quinn, Professor of Pediatrics, M.D., M.S., Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07177300
- Sponsor's protocol ID
- ENHANCE
- Other IDs
- GCF_Quinn
- Acronym
- ENHANCE
- Status last verified
- Aug 2025
- First submitted
- 18 Aug 2025
- Last updated
- 16 Sep 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Hay SB, Ferchen K, Chetal K, Grimes HL, Salomonis N. The Human Cell Atlas bone marrow single-cell interactive web portal. Exp Hematol. 2018 Dec;68:51-61. doi: 10.1016/j.exphem.2018.09.004. Epub 2018 Sep 21.
PubMed 30243574Marahatta A, Megaraj V, McGann PT, Ware RE, Setchell KD. Stable-Isotope Dilution HPLC-Electrospray Ionization Tandem Mass Spectrometry Method for Quantifying Hydroxyurea in Dried Blood Samples. Clin Chem. 2016 Dec;62(12):1593-1601. doi: 10.1373/clinchem.2016.263715. Epub 2016 Sep 30.
PubMed 27694393Vassiliou G. Telomere Length and Clonal Hematopoiesis. N Engl J Med. 2023 Jun 29;388(26):2481-2484. doi: 10.1056/NEJMe2303022. Epub 2023 May 4. No abstract available.
PubMed 37140164Bowman RL, Busque L, Levine RL. Clonal Hematopoiesis and Evolution to Hematopoietic Malignancies. Cell Stem Cell. 2018 Feb 1;22(2):157-170. doi: 10.1016/j.stem.2018.01.011.
PubMed 29395053Sankaran VG, Xu J, Orkin SH. Advances in the understanding of haemoglobin switching. Br J Haematol. 2010 Apr;149(2):181-94. doi: 10.1111/j.1365-2141.2010.08105.x. Epub 2010 Mar 1.
PubMed 20201948Ware RE, Davis BR, Schultz WH, Brown RC, Aygun B, Sarnaik S, Odame I, Fuh B, George A, Owen W, Luchtman-Jones L, Rogers ZR, Hilliard L, Gauger C, Piccone C, Lee MT, Kwiatkowski JL, Jackson S, Miller ST, Roberts C, Heeney MM, Kalfa TA, Nelson S, Imran H, Nottage K, Alvarez O, Rhodes M, Thompson AA, Rothman JA, Helton KJ, Roberts D, Coleman J, Bonner MJ, Kutlar A, Patel N, Wood J, Piller L, Wei P, Luden J, Mortier NA, Stuber SE, Luban NLC, Cohen AR, Pressel S, Adams RJ. Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial. Lancet. 2016 Feb 13;387(10019):661-670. doi: 10.1016/S0140-6736(15)01041-7. Epub 2015 Dec 6.
PubMed 26670617Hankins JS, Helton KJ, McCarville MB, Li CS, Wang WC, Ware RE. Preservation of spleen and brain function in children with sickle cell anemia treated with hydroxyurea. Pediatr Blood Cancer. 2008 Feb;50(2):293-7. doi: 10.1002/pbc.21271.
PubMed 17554794Letvin NL, Linch DC, Beardsley GP, McIntyre KW, Nathan DG. Augmentation of fetal-hemoglobin production in anemic monkeys by hydroxyurea. N Engl J Med. 1984 Apr 5;310(14):869-73. doi: 10.1056/NEJM198404053101401.
PubMed 6199670Platt OS. Hydroxyurea for the treatment of sickle cell anemia. N Engl J Med. 2008 Mar 27;358(13):1362-9. doi: 10.1056/NEJMct0708272. No abstract available.
PubMed 18367739Ware RE. Optimizing hydroxyurea therapy for sickle cell anemia. Hematology Am Soc Hematol Educ Program. 2015;2015:436-43. doi: 10.1182/asheducation-2015.1.436.
PubMed 26637755Lanzkron S, Carroll CP, Haywood C Jr. Mortality rates and age at death from sickle cell disease: U.S., 1979-2005. Public Health Rep. 2013 Mar-Apr;128(2):110-6. doi: 10.1177/003335491312800206.
PubMed 23450875Platt OS, Brambilla DJ, Rosse WF, Milner PF, Castro O, Steinberg MH, Klug PP. Mortality in sickle cell disease. Life expectancy and risk factors for early death. N Engl J Med. 1994 Jun 9;330(23):1639-44. doi: 10.1056/NEJM199406093302303.
PubMed 7993409Vichinsky E, Hurst D, Earles A, Kleman K, Lubin B. Newborn screening for sickle cell disease: effect on mortality. Pediatrics. 1988 Jun;81(6):749-55.
PubMed 3368274
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
- ClinicalTrials.gov ID
- NCT07177300
- Acronym / sponsor ID
- ENHANCE / ENHANCE
- Keywords
- sickle cell anemia, SCD, sickle cell, hydroxyurea
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Greater Cincinnati Foundation
- Study start
- 19 Dec 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 16 Sep 2025 (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.
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for people with SCA.
In this research study, the investigators hope to learn more about how to improve the dosing and monitoring of hydroxyurea and learn more about the long-term effects of hydroxyurea over time. Hydroxyurea is usually dosed based only on your weight. Our study will use a new way to select a starting dose that is based on how each patient absorbs hydroxyurea.
Detailed description
The EHANCE study will address key knowledge gaps about hydroxyurea for young children with SCA in nine innovative ways:
- Novel functional assessments of brain, heart, kidneys, spleen, and eyes to assess organ protection in young children who receive hydroxyurea at MTD;
- State-of-the-art assays to assess the benefits of hydroxyurea on growth, development, and reproductive health into puberty including serial measurements of pubertal development and sex hormones;
- A simplified PK-guided strategy to optimize hydroxyurea initiation and dosing, with a long-term goal of validating pharmacogenomic approaches to expand treatment and achieve sustained HbF induction;
- A novel single-cell quantitative HbF/F-cell assay, developed utilizing imaging flow cytometry, will determine the distribution of HbF/F-cell across all F-cells, rather than simply estimating the mean value of HbF/F-cell;
- Collection of genomic DNA samples to allow serial quantitation of clonal hematopoiesis in treated children, to evaluate the possibility for potential emergence of clones with an increased risk of leukemic transformation;
- Studies on primary erythroblasts freshly isolated from patients and control subjects with single cell multiome analysis to evaluate in vivo cis and trans-acting elements that regulate HbF and how they are affected by hydroxyurea;
- Evaluation of cellular mechanisms by which hydroxyurea at MTD can regularly achieve >30% HbF with near-pancellular distribution, similar to levels currently touted with 'curative' gene therapy regimens;
- Exploration of the benefits of early hydroxyurea treatment initiation, in terms of γ-globin de-repression to optimize HbF induction, through unknown cellular mechanisms that may be developmentally regulated.
- SPECIFIC AIMS Aim 1: Document the long-term benefits and risks of long-term hydroxyurea treatment at MTD.
Aim 2: Perform pharmacokinetic (PK) and pharmacodynamic (PD) assessment of hydroxyurea at MTD.
Aim 3: Investigate the cellular mechanisms by which hydroxyurea leads to induction of protective HbF and how timing of treatment initiation and dose optimization affect the efficacy of this process.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Prospective, open-label, single-arm therapeutic trial of pharmacokinetics (PK)-optimized oral hydroxyurea at maximum tolerated dose (MTD) for the treatment of sickle cell anemia (SCA) with long-term follow-up.
| Arm | Type | Receives |
|---|---|---|
| Observational Treatment Group (Single Arm) | Experimental | Drug: PK-optimized oral hydroxyurea at MTD until 15 years of age.All children with sickle cell anemia who are started on hydroxyurea for clinical indicators between 6 months and 5 years of age can do so on this observational study with PK-optimized hydroxyurea dosing. |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of sickle cell anemia (HbSS) or sickle-β0-thalassemia (HbSβ0)
- Age 6 months at the time of enrollment
- Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea therapy
Key exclusion criteria (2 listed)
- Current treatment with regularly scheduled blood transfusions
- Sickle-hemoglobin C disease (HbSC), sickle-β+-thalassemia (HbSβ+)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PK-optimized oral hydroxyurea at MTD until 15 years of age.Also: pharmacokinetic, maximum tolerated doseBecause people are different, we will measure how each participant's body absorbs and eliminates the medicine, hydroxyurea, using blood tests. This information will be used to determine the best dose for each participant (rather than using the same weight-based dose for everyone). | Drug | Observational Treatment Group (Single Arm) |
Primary outcome
Composite Organ Injury
Time frame: Through study completion, an average of 10 years
Evidence of injury in any of four critical organ systems: brain, kidney, heart, or spleen. Participants will be classified as having met the composite endpoint if they fulfill at least one of the organ-specific criteria listed: cerebral infarction (silent or overt) or steno-occlusive vasculopathy by MRI of brain; urine albumin-to-creatinine ratio (UACR) > 300 mg/g; extracellular volume fraction (ECV) > 0.35 on cardiac MRI; or erythrocyte pit count < 5%
7 secondary outcomes
longitudinal change in fetal hemoglobin percentage (HbF%)
Time frame: Through study completion, an average of 10 years
Longitudinal change in hemoglobin concentration (g/dL)
Time frame: Through study completion, an average of 10 years.
Longitudinal change in reticulocyte count (10^9/L)
Time frame: Through study completion, an average of 10 years.
Longitudinal change in absolute neutrophil count (10^9/L)
Time frame: Through study completion, an average of 10 years.
Longitudinal change in mean cell volume (fL)
Time frame: Through study completion, an average of 10 years.
F-cell fraction (%)
Time frame: Through study completion, an average of 10 years.
Adverse events
Time frame: Through study completion, an average of 10 years.
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 16 Sep 2025.
- Background: PubMed 30243574Hay SB, Ferchen K, Chetal K, Grimes HL, Salomonis N. The Human Cell Atlas bone marrow single-cell interactive web portal. Exp Hematol. 2018 Dec;68:51-61. doi: 10.1016/j.exphem.2018.09.004. Epub 2018 Sep 21.
- Background: PubMed 27694393Marahatta A, Megaraj V, McGann PT, Ware RE, Setchell KD. Stable-Isotope Dilution HPLC-Electrospray Ionization Tandem Mass Spectrometry Method for Quantifying Hydroxyurea in Dried Blood Samples. Clin Chem. 2016 Dec;62(12):1593-1601. doi: 10.1373/clinchem.2016.263715. Epub 2016 Sep 30.
- Background: PubMed 37140164Vassiliou G. Telomere Length and Clonal Hematopoiesis. N Engl J Med. 2023 Jun 29;388(26):2481-2484. doi: 10.1056/NEJMe2303022. Epub 2023 May 4. No abstract available.
- Background: PubMed 29395053Bowman RL, Busque L, Levine RL. Clonal Hematopoiesis and Evolution to Hematopoietic Malignancies. Cell Stem Cell. 2018 Feb 1;22(2):157-170. doi: 10.1016/j.stem.2018.01.011.
- Background: PubMed 20201948Sankaran VG, Xu J, Orkin SH. Advances in the understanding of haemoglobin switching. Br J Haematol. 2010 Apr;149(2):181-94. doi: 10.1111/j.1365-2141.2010.08105.x. Epub 2010 Mar 1.
- Background: PubMed 26670617Ware RE, Davis BR, Schultz WH, Brown RC, Aygun B, Sarnaik S, Odame I, Fuh B, George A, Owen W, Luchtman-Jones L, Rogers ZR, Hilliard L, Gauger C, Piccone C, Lee MT, Kwiatkowski JL, Jackson S, Miller ST, Roberts C, Heeney MM, Kalfa TA, Nelson S, Imran H, Nottage K, Alvarez O, Rhodes M, Thompson AA, Rothman JA, Helton KJ, Roberts D, Coleman J, Bonner MJ, Kutlar A, Patel N, Wood J, Piller L, Wei P, Luden J, Mortier NA, Stuber SE, Luban NLC, Cohen AR, Pressel S, Adams RJ. Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial. Lancet. 2016 Feb 13;387(10019):661-670. doi: 10.1016/S0140-6736(15)01041-7. Epub 2015 Dec 6.
- Background: PubMed 17554794Hankins JS, Helton KJ, McCarville MB, Li CS, Wang WC, Ware RE. Preservation of spleen and brain function in children with sickle cell anemia treated with hydroxyurea. Pediatr Blood Cancer. 2008 Feb;50(2):293-7. doi: 10.1002/pbc.21271.
- Background: PubMed 6199670Letvin NL, Linch DC, Beardsley GP, McIntyre KW, Nathan DG. Augmentation of fetal-hemoglobin production in anemic monkeys by hydroxyurea. N Engl J Med. 1984 Apr 5;310(14):869-73. doi: 10.1056/NEJM198404053101401.
- Background: PubMed 18367739Platt OS. Hydroxyurea for the treatment of sickle cell anemia. N Engl J Med. 2008 Mar 27;358(13):1362-9. doi: 10.1056/NEJMct0708272. No abstract available.
- Background: PubMed 26637755Ware RE. Optimizing hydroxyurea therapy for sickle cell anemia. Hematology Am Soc Hematol Educ Program. 2015;2015:436-43. doi: 10.1182/asheducation-2015.1.436.
- Background: PubMed 23450875Lanzkron S, Carroll CP, Haywood C Jr. Mortality rates and age at death from sickle cell disease: U.S., 1979-2005. Public Health Rep. 2013 Mar-Apr;128(2):110-6. doi: 10.1177/003335491312800206.
- Background: PubMed 7993409Platt OS, Brambilla DJ, Rosse WF, Milner PF, Castro O, Steinberg MH, Klug PP. Mortality in sickle cell disease. Life expectancy and risk factors for early death. N Engl J Med. 1994 Jun 9;330(23):1639-44. doi: 10.1056/NEJM199406093302303.
- Individual participant dataSharing planned: No
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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.
Charles T. Quinn, Professor of Pediatrics, M.D., M.S. principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing PK-optimized oral hydroxyurea at MTD until 15 years of age. in people with Sickle Cell Anemia (HbSS) and Sickle-β0-thalassemia (HbSβ0), run by Children's Hospital Medical Center, Cincinnati at 1 site 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 3 inclusion and 2 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: looking for participants now, as of the sponsor's last update on 16 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact Wendi L. Long, Sr. Regulatory Specialist, BS, CCRC (513-803-3064, wendi.long@cchmc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Composite Organ Injury (time frame: Through study completion, an average of 10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07177300, which also lists 13 linked publications.
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 16 Sep 2025.