Sickle Cell DiseasePhase 3HOPSNCT03789591
Hydroxyurea Optimization Through Precision Study
A Phase 3 study testing Hydroxyurea in people with Sickle Cell Disease and Sickle Cell Anemia, run by Lifespan at 13 sites in United States.
Trial summary
Hydroxyurea Optimization through Precision Study (HOPS) is a prospective, multi-center, randomized trial that will directly compare a novel, individualized dosing strategy of hydroxyurea to standard weight-based dosing for children with SCA. Read more about this study
- Who is it for?
- Ages 6 months to 21 years
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Anemia
- What is being tested?
- Hydroxyurea
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 104 participants
- Where
- 13 sitesUnited States
- Study dates
- 17 Jan 2019to 19 Mar 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of sickle cell anemia (HbSS, HbSD, HbS/β0-thalassemia, or similarly severe SCA genotype)
- Age 6 months to 21 years at the time of enrollment
- Clinical decision by patient, family, and healthcare providers to initiate hydroxyurea therapy
Who cannot join
- Current treatment with chronic, monthly blood transfusions or erythrocytapheresis
- Treatment with hydroxyurea within the past 3 months
- Hemoglobin SC disease, HbS/β+-thalassemia
- Current treatment with other investigational sickle cell medications
- Current known pregnancy or lactation
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30342
Boston Children's Hospital
Boston, Massachusetts, 02215
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Rainbow Babies / University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Nationwide Children's Hospital.
Columbus, Ohio, 43205
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202
Indiana Hemophilia & Thrombosis Center, Inc. (IHTC)
Indianapolis, Indiana, 46260
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
Cohen Children's Medical Center/Northwell Health
New Hyde Park, New York, 11040
Children's Hospital of Illinois
Peoria, Illinois, 61637
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Carle Foundation Hospital
Urbana, Illinois, 61801
About this study
Official title: Hydroxyurea Optimization Through Precision Study (HOPS): A Prospective, Multi-center, Randomized Trial of Personalized, Pharmacokinetics-guided Dosing of Hydroxyurea Versus Standard Weight-based Dosing for Children With Sickle Cell Anemia.
Hydroxyurea Optimization through Precision Study (HOPS) is a prospective, multi-center, randomized trial that will directly compare a novel, individualized dosing strategy of hydroxyurea to standard weight-based dosing for children with SCA. The primary objective of the study is to evaluate whether a pharmacokinetics-based starting hydroxyurea dose thieves superior fetal hemoglobin response to to standard weight-based initial dosing. Patients will be recruited from the pediatric sickle cell clinic at Cincinnati Children's Hospital Medical Center and from additional pediatric sickle cell centers within the United States.
Read the detailed description
The trial will recruit patients who have decided to initiate hydroxyurea therapy. All participants will have pharmacokinetics studies performed at baseline, following a 20 mg/kg oral dose of hydroxyurea. Pharmacokinetic sampling will use a sparse sampling approach, requiring collection of blood at 3 time points (15 minutes, 60 minutes, 180 minutes) following the hydroxyurea dose. Enrolled participants will be randomized to receive either hydroxyurea using a starting dose of 20 mg/kg/day (Standard Arm) or a personalized PK-guided dose (Alternative Arm) to target an area under the curve (AUC) of 115 mg*h/L based to approximate hydroxyurea exposure seen when patients are escalated to maximum tolerated dose (MTD).
Following randomization and selection of the initial dose, participants in both arms will follow the same procedures of laboratory medication holds for hematological toxicity. The primary endpoint is fetal hemoglobin (HbF) six months following the initiation of hydroxyurea therapy with the hypothesis that participants starting with a PK-guided dose will achieve HbF at least 5% greater than those starting with a 20 mg/kg dose. Based upon the estimated number of new hydroxyurea starts at each site, it is anticipated that it will take 24 months to enroll the 116 participants required to achieve sufficient power to assess the primary endpoint. The study will conclude for each participant 12 months following hydroxyurea initiation.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Being tested
- Hydroxyurea
- Keywords
- Hydroxyurea, HbSS, HbSD, TREAT
- 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.
HydroxyureaDrug
The alternative arm will use PK data to choose a starting hydroxyurea dose to achieve an AUC of 115 mg*h/L to approximate maximum tolerated dose. On the standard arm, participants will start at the traditional, weight-based dose of 20 mg/kg/day. Following selection of the starting dose, all participants will follow the same dose escalation and laboratory monitoring procedures.
| Study group | Role | What they receive |
|---|---|---|
| Standard Arm | Active comparator | Participants randomized to the standard arm will receive a starting dose of hydroxyurea of 20 mg/kg/day. Receives: Drug: Hydroxyurea |
| Alternative Arm | Experimental | Participants randomized to the alternative arm will receive a pharmacokinetic guided starting dose of hydroxyurea based on PK labs drawn at a baseline visit to target an area under the curve (AUC) of 115 mg*h/L in an attempt to approximate maximum tolerated dose (MTD). This dose will not exceed the maximum tolerated dose of 35 mg/kg/day. Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Fetal Hemoglobin (HbF) Response Following Six Months of Hydroxyurea Therapy
The primary outcome will be HbF response six months after starting hydroxyurea therapy with the hypothesis that participants in the Alternative Arm (PK-guided starting dose) will have at least 5% higher HbF than the Standard Arm (20 mg/kg starting dose)
Measured: 6 months after starting daily hydroxyurea therapy
Secondary outcomes (2)
F Cells
In addition to traditional %HbF measurement, F cells will be measured at baseline, 6 months, and 12 months
Measured: Baseline, 6 and 12 months after initiating daily hydroxyurea therapy
Gene Expression Patterns of Study Participants
The epigenomic signature and gene expression patterns of study participants receiving hydroxyurea therapy at MTD. MTD is defined as a stable dose without any dose increases (except to account for weight gain), holds, or decreases within 8 weeks with laboratory criteria within the target range. This outcome will explain the mechanisms that yield high HbF responses.
Measured: 6 Months after initial Hydroxyurea therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2018
- Study start17 Jan 2019
- Primary completion19 Mar 2025
- Study completion19 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 104 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lifespan
- Sponsor type
- University, hospital or other
- Collaborators
- Doris Duke Charitable Foundation
- Responsible party
- Principal investigator, Patrick McGann, Lifespan
- Investigators
- Patrick Niss, MD, Principal investigator, Lifespan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03789591
- Sponsor's protocol ID
- CCHMC_HOPS
- Acronym
- HOPS
- Status last verified
- Apr 2025
- First submitted
- 27 Nov 2018
- Last updated
- 18 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Meier ER, Creary SE, Heeney MM, Dong M, Appiah-Kubi AO, Nelson SC, Niss O, Piccone C, Quarmyne MO, Quinn CT, Saving KL, Scott JP, Talati R, Latham TS, Pfeiffer A, Shook LM, Vinks AA, Lane A, McGann PT. Hydroxyurea Optimization through Precision Study (HOPS): study protocol for a randomized, multicenter trial in children with sickle cell anemia. Trials. 2020 Nov 27;21(1):983. doi: 10.1186/s13063-020-04912-z.
PubMed 33246482
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hydroxyurea Optimization Through Precision Study (HOPS): A Prospective, Multi-center, Randomized Trial of Personalized, Pharmacokinetics-guided Dosing of Hydroxyurea Versus Standard Weight-based Dosing for Children With Sickle Cell Anemia.
- ClinicalTrials.gov ID
- NCT03789591
- Acronym / sponsor ID
- HOPS / CCHMC_HOPS
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Keywords
- Hydroxyurea, HbSS, HbSD, TREAT
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 104 participants (actual)
- Lead sponsor
- LifespanUniversity, hospital or other
- Collaborators
- Doris Duke Charitable Foundation
- Study start
- 17 Jan 2019 (actual)
- Primary completion
- 19 Mar 2025 (actual)
- Study completion
- 19 Mar 2025 (actual)
- First posted
- 28 Dec 2018 (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.
Hydroxyurea Optimization through Precision Study (HOPS) is a prospective, multi-center, randomized trial that will directly compare a novel, individualized dosing strategy of hydroxyurea to standard weight-based dosing for children with SCA. The primary objective of the study is to evaluate whether a pharmacokinetics-based starting hydroxyurea dose thieves superior fetal hemoglobin response to to standard weight-based initial dosing. Patients will be recruited from the pediatric sickle cell clinic at Cincinnati Children's Hospital Medical Center and from additional pediatric sickle cell centers within the United States.
Detailed description
The trial will recruit patients who have decided to initiate hydroxyurea therapy. All participants will have pharmacokinetics studies performed at baseline, following a 20 mg/kg oral dose of hydroxyurea. Pharmacokinetic sampling will use a sparse sampling approach, requiring collection of blood at 3 time points (15 minutes, 60 minutes, 180 minutes) following the hydroxyurea dose. Enrolled participants will be randomized to receive either hydroxyurea using a starting dose of 20 mg/kg/day (Standard Arm) or a personalized PK-guided dose (Alternative Arm) to target an area under the curve (AUC) of 115 mg*h/L based to approximate hydroxyurea exposure seen when patients are escalated to maximum tolerated dose (MTD).
Following randomization and selection of the initial dose, participants in both arms will follow the same procedures of laboratory medication holds for hematological toxicity. The primary endpoint is fetal hemoglobin (HbF) six months following the initiation of hydroxyurea therapy with the hypothesis that participants starting with a PK-guided dose will achieve HbF at least 5% greater than those starting with a 20 mg/kg dose. Based upon the estimated number of new hydroxyurea starts at each site, it is anticipated that it will take 24 months to enroll the 116 participants required to achieve sufficient power to assess the primary endpoint. The study will conclude for each participant 12 months following hydroxyurea initiation.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard Arm | Active comparator | Drug: HydroxyureaParticipants randomized to the standard arm will receive a starting dose of hydroxyurea of 20 mg/kg/day. |
| Alternative Arm | Experimental | Drug: HydroxyureaParticipants randomized to the alternative arm will receive a pharmacokinetic guided starting dose of hydroxyurea based on PK labs drawn at a baseline visit to target an area under the curve (AUC) of 115 mg*h/L in an attempt to approximate maximum tolerated dose (MTD). This dose will not exceed the maximum tolerated dose of 35 mg/kg/day. |
Who may qualify
- Ages
- 6 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Diagnosis of sickle cell anemia (HbSS, HbSD, HbS/β0-thalassemia, or similarly severe SCA genotype)
- Age 6 months to 21 years at the time of enrollment
- Clinical decision by patient, family, and healthcare providers to initiate hydroxyurea therapy
Key exclusion criteria (5 listed)
- Current treatment with chronic, monthly blood transfusions or erythrocytapheresis
- Treatment with hydroxyurea within the past 3 months
- Hemoglobin SC disease, HbS/β+-thalassemia
- Current treatment with other investigational sickle cell medications
- Current known pregnancy or lactation
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 |
|---|---|---|
| HydroxyureaThe alternative arm will use PK data to choose a starting hydroxyurea dose to achieve an AUC of 115 mg*h/L to approximate maximum tolerated dose. On the standard arm, participants will start at the traditional, weight-based dose of 20 mg/kg/day. Following selection of the starting dose, all participants will follow the same dose escalation and laboratory monitoring procedures. | Drug | Alternative Arm, Standard Arm |
Primary outcome
Fetal Hemoglobin (HbF) Response Following Six Months of Hydroxyurea Therapy
Time frame: 6 months after starting daily hydroxyurea therapy
The primary outcome will be HbF response six months after starting hydroxyurea therapy with the hypothesis that participants in the Alternative Arm (PK-guided starting dose) will have at least 5% higher HbF than the Standard Arm (20 mg/kg starting dose)
2 secondary outcomes
F Cells
Time frame: Baseline, 6 and 12 months after initiating daily hydroxyurea therapy
Gene Expression Patterns of Study Participants
Time frame: 6 Months after initial Hydroxyurea therapy
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2025.
- Derived from this study: PubMed 33246482Meier ER, Creary SE, Heeney MM, Dong M, Appiah-Kubi AO, Nelson SC, Niss O, Piccone C, Quarmyne MO, Quinn CT, Saving KL, Scott JP, Talati R, Latham TS, Pfeiffer A, Shook LM, Vinks AA, Lane A, McGann PT. Hydroxyurea Optimization through Precision Study (HOPS): study protocol for a randomized, multicenter trial in children with sickle cell anemia. Trials. 2020 Nov 27;21(1):983. doi: 10.1186/s13063-020-04912-z.
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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.
Patrick Niss, MD principal investigator
Lifespan
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Hydroxyurea in people with Sickle Cell Disease and Sickle Cell Anemia, run by Lifespan at 13 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 3 inclusion and 5 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: finished, as of the sponsor's last update on 18 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Fetal Hemoglobin (HbF) Response Following Six Months of Hydroxyurea Therapy (time frame: 6 months after starting daily hydroxyurea therapy). 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 NCT03789591, which also lists 1 linked publication.
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 18 Jun 2025.