Sickle Cell DiseasePhase 2HATNCT03644953
Hydroxyurea and Transfusion
A Phase 2 study testing Hydroxyurea in people with Sickle Cell Disease, run by Children's National Research Institute at 1 site in United States.
Trial summary
This study will prospectively investigate the feasibility, safety, and transfusion requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle cell anemia already on chronic transfusions. Read more about this study
- Who is it for?
- Ages 2 to 24 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Hydroxyurea
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Oct 2018to 1 Jul 2021
- Phase
- Phase 2
- 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 2 to 24 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of SCA (Hb SS or Sβ0 thalassemia).
- On simple chronic transfusion therapy for stroke prevention (primary or secondary prevention) for ≥1 year with no plans to stop simple chronic transfusion in the next year.
Who cannot join
- Poor adherence to simple transfusion regimen as defined by having an HbS >45% at any time in the last year AND a transfusion interval >5 weeks.
- Treatment with hydroxyurea in the 12 months prior to study enrollment.
- Abnormal initial laboratory values (temporary exclusions):
- Absolute neutrophil count <1.5 x 10^9/L
- Platelet count <100 x 10^9/L
- Serum creatinine more than twice upper limit for age
- Pregnancy or unwillingness to use a medically acceptable form of contraception if sexually active.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's National Health System
Washington D.C., District of Columbia, 20010
About this study
Official title: Hydroxyurea and Transfusion: Pilot Study of Combination Therapy for Patients With Sickle Cell Anemia
This study will prospectively investigate the feasibility, safety, and transfusion requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle cell anemia already on chronic transfusions.
Read the detailed description
This is a single-arm, prospective study of hydroxyurea added to simple chronic transfusions, combination treatment termed hydroxyurea and transfusion (HAT). The primary objective of the study is to determine the feasibility of HAT for patients with sickle cell anemia (SCA) currently being treated only with simple transfusions for stroke prevention. Secondary objectives include: to evaluate the safety of HAT and to determine if HAT decreases transfusion requirements in this patient population. Exploratory objectives include: to evaluate with HAT changes in pre-transfusion laboratories and biomarkers of cerebrovascular disease progression, and to describe changes on brain imaging.
- Conditions
- Sickle Cell Disease
- Being tested
- Hydroxyurea
- Keywords
- Hydroxyurea, Transfusion
- 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
Participants will be started on hydroxyurea 20 ± 2.5 mg/kg/day in addition to continuing simple chronic transfusion every 4 weeks ± 1 week. Hydroxyurea will be dose escalated to achieve a HAT target dose (HAT-TD). Hydroxyurea will be increased by 5 mg/kg/day after 8 weeks of a dose if HAT-TD is not achieved (maximum daily dose 2,000 mg). After HAT-TD has been achieved, minor dose increases may occur if subject grows a significant amount to maintain the same mg/kg dose.
| Study group | Role | What they receive |
|---|---|---|
| Hydroxyurea and Transfusion (HAT) | Experimental | Combination hydroxyurea and simple chronic transfusion therapy Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recruitment ratio
number of participants who enroll on the study / total number of eligible subjects
Measured: 1 year
Retention ratio
number participants who remain on study 1 year after HAT target dose / total number enrolled participants
Measured: 1 year
Hydroxyurea adherence ratio
(hydroxyurea amount dispensed - amount returned) / prescribed amount between visits
Measured: 1 year
Secondary outcomes (2)
Proportion of subjects who develop an hemoglobin (Hb) S >45% AND an Hb >11.0 g/dL
incidence of above safety event will be monitored closely throughout the trial
Measured: 1 year
Volume of red blood cells transfused per patient weight
measure to evaluate the transfusion requirement of HAT
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2018
- Study start30 Oct 2018
- Primary completion1 Jul 2021
- Study completion1 Jul 2021
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 Children's National Research InstituteThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Prospective, single arm, open label study of combination hydroxyurea and simple chronic transfusion.
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Robert Nickel, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03644953
- Sponsor's protocol ID
- Pro00010541
- Acronym
- HAT
- Status last verified
- Oct 2021
- First submitted
- 21 Aug 2018
- Last updated
- 21 Oct 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hydroxyurea and Transfusion: Pilot Study of Combination Therapy for Patients With Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT03644953
- Acronym / sponsor ID
- HAT / Pro00010541
- Conditions
- Sickle Cell Disease
- Keywords
- Hydroxyurea, Transfusion
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Study start
- 30 Oct 2018 (actual)
- Primary completion
- 1 Jul 2021 (actual)
- Study completion
- 1 Jul 2021 (actual)
- First posted
- 23 Aug 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.
This study will prospectively investigate the feasibility, safety, and transfusion requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle cell anemia already on chronic transfusions.
Detailed description
This is a single-arm, prospective study of hydroxyurea added to simple chronic transfusions, combination treatment termed hydroxyurea and transfusion (HAT). The primary objective of the study is to determine the feasibility of HAT for patients with sickle cell anemia (SCA) currently being treated only with simple transfusions for stroke prevention. Secondary objectives include: to evaluate the safety of HAT and to determine if HAT decreases transfusion requirements in this patient population. Exploratory objectives include: to evaluate with HAT changes in pre-transfusion laboratories and biomarkers of cerebrovascular disease progression, and to describe changes on brain imaging.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Prospective, single arm, open label study of combination hydroxyurea and simple chronic transfusion.
| Arm | Type | Receives |
|---|---|---|
| Hydroxyurea and Transfusion (HAT) | Experimental | Drug: HydroxyureaCombination hydroxyurea and simple chronic transfusion therapy |
Who may qualify
- Ages
- 2 to 24 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Diagnosis of SCA (Hb SS or Sβ0 thalassemia).
- On simple chronic transfusion therapy for stroke prevention (primary or secondary prevention) for ≥1 year with no plans to stop simple chronic transfusion in the next year.
Key exclusion criteria (7 listed)
- Poor adherence to simple transfusion regimen as defined by having an HbS >45% at any time in the last year AND a transfusion interval >5 weeks.
- Treatment with hydroxyurea in the 12 months prior to study enrollment.
- Abnormal initial laboratory values (temporary exclusions):
- Pregnancy or unwillingness to use a medically acceptable form of contraception if sexually active.
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 |
|---|---|---|
| HydroxyureaParticipants will be started on hydroxyurea 20 ± 2.5 mg/kg/day in addition to continuing simple chronic transfusion every 4 weeks ± 1 week. Hydroxyurea will be dose escalated to achieve a HAT target dose (HAT-TD). Hydroxyurea will be increased by 5 mg/kg/day after 8 weeks of a dose if HAT-TD is not achieved (maximum daily dose 2,000 mg). After HAT-TD has been achieved, minor dose increases may occur if subject grows a significant amount to maintain the same mg/kg dose. | Drug | Hydroxyurea and Transfusion (HAT) |
Primary outcome
Recruitment ratio
Time frame: 1 year
number of participants who enroll on the study / total number of eligible subjects
Retention ratio
Time frame: 1 year
number participants who remain on study 1 year after HAT target dose / total number enrolled participants
Hydroxyurea adherence ratio
Time frame: 1 year
(hydroxyurea amount dispensed - amount returned) / prescribed amount between visits
2 secondary outcomes
Proportion of subjects who develop an hemoglobin (Hb) S >45% AND an Hb >11.0 g/dL
Time frame: 1 year
Volume of red blood cells transfused per patient weight
Time frame: 1 year
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 21 Oct 2021.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxyurea in people with Sickle Cell Disease, run by Children's National Research Institute 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 2 inclusion and 7 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 21 Oct 2021. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Recruitment ratio (time frame: 1 year). The study lists 3 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 21 Oct 2021.