Hemoglobin SC DiseasePhase 2SCYTHENCT02336373
Treatment of Hemoglobin SC Disease With Hydroxyurea
A Phase 2 study testing hydroxyurea in people with Hemoglobin SC Disease, run by Baylor College of Medicine at 1 site in United States.
Trial summary
Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications. Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. Read more about this study
- Who is it for?
- Ages 5 to 21 years
- Conditions the study is for
- Hemoglobin SC Disease
- What is being tested?
- hydroxyurea
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2014to 31 Mar 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 5 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of HbSC disease
- Score equal or lower than 80 on the PedsQL™ Sickle Cell Disease Module version 3.0
- Have experienced a sickle cell disease related complication
Who cannot join
- Failure to meet inclusion criteria.
- Hydroxyurea usage in the last 3 months.
- Chronic RBC transfusion therapy.
- Packed red blood cell transfusion in the last 3 months (temporary exclusion).
- Pregnancy, or refusal to use medically effective birth control if female and sexually active.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: SC Youth Treatment With Hydroxyurea Effects
Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications. Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, and damage to body organs. One medication used to treat sickle cell disease is hydroxyurea.
Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs). Hydroxyurea has been given to many HbSC patients but HbSC patients were not included in the large clinical trials used to test hydroxyurea in SCD, so less is known about how HbSC patients respond to hydroxyurea.
The purpose of this research study is to see if hydroxyurea, a medication given to many children with the most common type of sickle cell, those who are homozygous for the sickle mutation (HbSS), helps children who have HbSC. The investigators will see if it helps by giving a questionaire when the medication is started, and then every two months at a clinic visit. The questionaire, called the Pediatric Quality of Life Inventory (PedsQL™) Sickle Cell Disease Module version 3.0, measures quality of life. The investigators will also see how hydroxyurea changes laboratory test numbers, and blood thickness.
Read the detailed description
To be eligible to participate in this study, patients must have HbSC disease, have experienced a sickle cell disease related complication, or have a score of 80 or lower on the PedsQL™ Sickle Cell Disease Module version 3.0. This questionnaire will be offered to all patients with HbSC seen in our clinic that consent to this study. If the patient is sexually active, they will be offered birth control. If the patient chooses not to initiate effective birth control, they will be tested at their scheduled vist with a urine pregnancy test. If the patient becomes pregnant they will be removed from the study.
The maximum time patients will be on the study is 12 months after starting hydroxyurea therapy, with an option to participate in a 2 year observation study following the end of the study.
Patients will be assessed in the clinic every two months after starting treatment. Hydroxyurea will be started at 10 mg/kg/day, and increased by 5 mg/kg/day at eight week intervals if needed to reach a maximum tolerated dose (MTD). The most common side effect of the drug is a drop in infection fighting cells, or white blood cells, so the medication will be started at a low dose and the dose will be increased only if it is safe to do so.
Patients will be asked to allow the investigators to review information from their medical records at the start of the study, and throughout the study. If the patient would like to participate in the two year follow-up, their records will be reviewed during that period as well.
- Conditions
- Hemoglobin SC Disease
- Being tested
- hydroxyurea
- Keywords
- Hemoglobin SC disease, hydroxyurea, quality of life, viscosity, red cell density
- Medical subject headings
- Hemoglobin SC Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hydroxyureaDrug
Also called Hydrea, Droxia
Treat symptomatic HbSC patients to MTD on hydroxyurea, and assess for clinical improvement using the PedsQL™ Sickle Cell Disease Module version 3.0 after 6 months at MTD, compared to entrance scores
| Study group | Role | What they receive |
|---|---|---|
| Hydroxurea | Experimental | Initiate hydroxyurea at 10 mg/kg daily and escalate hydroxyurea dose by 5 mg/kg/day every 8 weeks up to a maximum dose of 35 mg/kg/day if blood counts meet escalation criteria on at least 2 blood tests over eight weeks prior to dose increase. Receives: Drug: hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in PedsQL SCDM
Mean change in PedsQL™ Sickle Cell Disease Module version 3.0 score after achieving MTD compared with baseline.
PedsQL™ Sickle Cell Disease Module version 3.0 scores are on a 100 point scale ranging from 0 to 100 with higher values indicating better quality of life.
PedsQL: pediatric qulaity of life
Measured: 6 months
Secondary outcomes (11)
Change in HVR at 45s-1
Change in hematocrit to viscosity ratio (HVR) at 45s-1 at follow-up versus baseline. This is a measure of oxygen carrying capacity, as a higher hematocrit with lower viscosity indicates ability to deliver oxygen without slowed blood flow due to high viscosity or blood thickness. Higher values are associated with improvement.
Measured: up to 7 months
Change in HVR at 225s-1
Change in hematocrit viscosity ratio at 225s
Measured: up to 7 months
DRBC
Change in percent dense red blood cells
Measured: up to 7 months
Change in HbF
Change in fetal hemaglobin
Measured: up to 7 months
Change in MCV
Change in mean corpuscular volume
Measured: up to 7 months
Change in MCHC
Change in mean corpuscular hemoglobin concentration
Measured: up to 7 months
Change in Hb
Change in hemoglobin
Measured: up to 7 months
Change in ARC
Change in absolute reticulocyte count
Measured: up to 7 months
Change in ANC
Change in absolute neutrophil count
Measured: up to 7 months
Change in LDH
Change in lactate dehydrogenase
Measured: up to 7 months
Change in UB Levels
Change in unconjugated bilirubin levels
Measured: up to 7 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2015 (estimated)
- Study startDec 2014
- Primary completionFeb 2017
- Study completion31 Mar 2017
Why it stopped: low enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemoglobin SC DiseaseStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe 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
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Vivien Sheehan, Baylor College of Medicine
- Investigators
- Vivien Sheehan, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02336373
- Sponsor's protocol ID
- H-35010 SCYTHE
- Acronym
- SCYTHE
- Status last verified
- Sep 2020
- First submitted
- 8 Jan 2015
- Last updated
- 9 Sep 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- SC Youth Treatment With Hydroxyurea Effects
- ClinicalTrials.gov ID
- NCT02336373
- Acronym / sponsor ID
- SCYTHE / H-35010 SCYTHE
- Conditions
- Hemoglobin SC Disease
- Keywords
- Hemoglobin SC disease, hydroxyurea, quality of life, viscosity, red cell density
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: low enrollment
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Study start
- Dec 2014
- Primary completion
- Feb 2017 (actual)
- Study completion
- 31 Mar 2017 (actual)
- First posted
- 13 Jan 2015 (estimated)
- Results
- Posted 9 Sep 2020
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 (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications. Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, and damage to body organs. One medication used to treat sickle cell disease is hydroxyurea.
Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs). Hydroxyurea has been given to many HbSC patients but HbSC patients were not included in the large clinical trials used to test hydroxyurea in SCD, so less is known about how HbSC patients respond to hydroxyurea.
The purpose of this research study is to see if hydroxyurea, a medication given to many children with the most common type of sickle cell, those who are homozygous for the sickle mutation (HbSS), helps children who have HbSC. The investigators will see if it helps by giving a questionaire when the medication is started, and then every two months at a clinic visit. The questionaire, called the Pediatric Quality of Life Inventory (PedsQL™) Sickle Cell Disease Module version 3.0, measures quality of life. The investigators will also see how hydroxyurea changes laboratory test numbers, and blood thickness.
Detailed description
To be eligible to participate in this study, patients must have HbSC disease, have experienced a sickle cell disease related complication, or have a score of 80 or lower on the PedsQL™ Sickle Cell Disease Module version 3.0. This questionnaire will be offered to all patients with HbSC seen in our clinic that consent to this study. If the patient is sexually active, they will be offered birth control. If the patient chooses not to initiate effective birth control, they will be tested at their scheduled vist with a urine pregnancy test. If the patient becomes pregnant they will be removed from the study.
The maximum time patients will be on the study is 12 months after starting hydroxyurea therapy, with an option to participate in a 2 year observation study following the end of the study.
Patients will be assessed in the clinic every two months after starting treatment. Hydroxyurea will be started at 10 mg/kg/day, and increased by 5 mg/kg/day at eight week intervals if needed to reach a maximum tolerated dose (MTD). The most common side effect of the drug is a drop in infection fighting cells, or white blood cells, so the medication will be started at a low dose and the dose will be increased only if it is safe to do so.
Patients will be asked to allow the investigators to review information from their medical records at the start of the study, and throughout the study. If the patient would like to participate in the two year follow-up, their records will be reviewed during that period as well.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hydroxurea | Experimental | Drug: hydroxyureaInitiate hydroxyurea at 10 mg/kg daily and escalate hydroxyurea dose by 5 mg/kg/day every 8 weeks up to a maximum dose of 35 mg/kg/day if blood counts meet escalation criteria on at least 2 blood tests over eight weeks prior to dose increase. |
Who may qualify
- Ages
- 5 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Diagnosis of HbSC disease
- Score equal or lower than 80 on the PedsQL™ Sickle Cell Disease Module version 3.0
- Have experienced a sickle cell disease related complication
Key exclusion criteria (5 listed)
- Failure to meet inclusion criteria.
- Hydroxyurea usage in the last 3 months.
- Chronic RBC transfusion therapy.
- Packed red blood cell transfusion in the last 3 months (temporary exclusion).
- Pregnancy, or refusal to use medically effective birth control if female and 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 |
|---|---|---|
| hydroxyureaAlso: Hydrea, DroxiaTreat symptomatic HbSC patients to MTD on hydroxyurea, and assess for clinical improvement using the PedsQL™ Sickle Cell Disease Module version 3.0 after 6 months at MTD, compared to entrance scores | Drug | Hydroxurea |
Primary outcome
Change in PedsQL SCDM
Time frame: 6 months
Mean change in PedsQL™ Sickle Cell Disease Module version 3.0 score after achieving MTD compared with baseline. PedsQL™ Sickle Cell Disease Module version 3.0 scores are on a 100 point scale ranging from 0 to 100 with higher values indicating better quality of life. PedsQL: pediatric qulaity of life
11 secondary outcomes
Change in HVR at 45s-1
Time frame: up to 7 months
Change in HVR at 225s-1
Time frame: up to 7 months
DRBC
Time frame: up to 7 months
Change in HbF
Time frame: up to 7 months
Change in MCV
Time frame: up to 7 months
Change in MCHC
Time frame: up to 7 months
Change in Hb
Time frame: up to 7 months
Change in ARC
Time frame: up to 7 months
Change in ANC
Time frame: up to 7 months
Change in LDH
Time frame: up to 7 months
Change in UB Levels
Time frame: up to 7 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Hemoglobin SC Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Active, not recruitingObservational study
Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
Sickle Cell Anemia, Sickle Cell Thalassemia
RecruitingObservational study
Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders
Sickle Cell Disease
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.
Vivien Sheehan, MD principal investigator
Baylor College of Medicine
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 Hemoglobin SC Disease, run by Baylor College of Medicine 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 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: stopped early, as of the sponsor's last update on 9 Sep 2020. 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?
Yes. Results are posted in the registry (first posted 9 Sep 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change in PedsQL SCDM (time frame: 6 months). 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 9 Sep 2020.