Anemia, Sickle CellPhase 2HELPSNCT03062501
Hydroxyurea in the Emergency Room to Lessen Pain in Sickle Cell Crisis
A Phase 2 study testing Hydroxyurea in people with Anemia, Sickle Cell and Anemia; Sickle-Cell, With Crisis, run by Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti at 1 site in Brazil.
Trial summary
This study will investigate the safety, tolerability and potential for the use of up to three daily doses of 30-40 mg/kg HU (daily) upon hospitalization for painful vaso-occlusive crises . Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Anemia, Sickle Cell, Anemia; Sickle-Cell, With Crisis
- What is being tested?
- Hydroxyurea
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteBrazil
- Study dates
- Nov 2016to Jul 2017
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Confirmed diagnosis of homozygous sickle cell anemia (HbSS).
- Hospitalization due to onset of uncomplicated vaso-occlusive crisis (with pain scale≥6 within the last 24 h), confirmed by clinical evaluation.
- Documented and written informed consent
Who cannot join
- Confirmed or suspected pregnancy.
- Initiation of painful crisis> 72h.
- Blood transfusion during the last 8 weeks.
- Admission to Emergency Room due to pain in the last 4 weeks.
- Neutrophil count <2.5 x 109/L or platelet count <95.0 x 109 / L or Hb <4.5 g / dL
- Weight <38 Kg or> 95 Kg.
- Interval longer than 8h since arrival at center.
- Non-consent to participate in the study.
Trial locations and contacts
1 site in 1 country.
Brazil1 site, 1 recruiting
- Recruiting
Hemorio
Rio de Janeiro, Rio de Janeiro
Thais Oliveira, B.Sc., +55 (21) 2505-0750 ext. 2212, cdi@hemorio.rj.gov.br
About this study
Official title: Protocol for the Administration of Hydroxyurea During Painful Vaso-occlusive Crisis in Sickle Cell Anemia
This study will investigate the safety, tolerability and potential for the use of up to three daily doses of 30-40 mg/kg HU (daily) upon hospitalization for painful vaso-occlusive crises .
Read the detailed description
Sickle cell anemia (SCA) is a hereditary hemoglobinopathy; complications of the disease include, spleen enlargement, acute chest syndrome, pulmonary hypertension, stroke and cumulative damage to multiple organs, and painful vaso-occlusive crises (VOC). In Brazil, about 3,500 children are born each year with DF, and the number of individuals with sickle cell disease (DF) in the country is estimated between 25,000 and 30,000 (ANVISA 2012; BRAZIL, 2012).
Hydroxyurea (HU, or hydroxycarbamide) is the only drug approved to date by the American FDA for use in adults with sickle cell disease. The drug modifies the disease process, improving hematological parameters and the hospitalization time of patients, as well as the frequency of vaso-occlusive crises.In addition to its proven effects during chronic use, experimental data indicate that HU has immediate anti-inflammatory effects.
In addition to its proven effects during chronic use, experimental data indicate that HU has immediate anti-inflammatory effects. This study will investigate the safety, tolerability and potential for the use of up to three daily doses of 30-40 mg/kg HU (daily) upon hospitalization for VOC.
- Being tested
- Hydroxyurea
- Medical subject headings
- Anemia, Sickle Cell, Vaso-Occlusive Crises
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxyureaDrug
Patients hospitalized for uncomplicated pain crisis with a pain scale of ≥ 6 during the last 24 hours will receive a dose of 30-40 mg / kg hydroxyurea. This same dose of hydroxyurea will be repeated at 24 h and 48 h after the first dose of hydroxyurea, with dose suspension if the patient is discharged within 48 hours. Patients will also receive the center's usual practice and analgesia protocol.
| Study group | Role | What they receive |
|---|---|---|
| Control (No Hydroxyurea) | No intervention | Patients in VOC will be treated according to the center's usual practice and analgesia protocol. |
| Hydroxyurea | Experimental | Patients in VOC will receive up to three daily doses of 30-40 mg / kg hydroxyurea. Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events
as assessed by CTCAE version 4.03
Measured: up to 15 days post last dose
Number of participants with altered laboratory values related to treatment
Measured: up to 15 days post last dose
Secondary outcomes (3)
Time until hospital discharge
Measured: Average, up to 7 days post admission
Total opioid use (mg of IV morphine)
Measured: From study inclusion until hospital discharge (average, up to 7 days post admission)
Pain score
Numeric pain score rating (0 to 10; 0 = no pain, 10 = worst pain)
Measured: From admission until hospital discharge (average, up to 7 days post admission)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2017
- Study startNov 2016
- Primary completionApr 2017 (estimated)
- Study completionJul 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anemia, Sickle CellStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, CLARISSE LOBO, Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03062501
- Sponsor's protocol ID
- Hemorio 397/16
- Acronym
- HELPS
- Status last verified
- Feb 2017
- First submitted
- 30 Jan 2017
- Last updated
- 23 Feb 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Protocol for the Administration of Hydroxyurea During Painful Vaso-occlusive Crisis in Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT03062501
- Acronym / sponsor ID
- HELPS / Hemorio 397/16
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Instituto Estadual de Hematologia Arthur de Siqueira CavalcantiUniversity, hospital or other
- Study start
- Nov 2016
- Primary completion
- Apr 2017 (estimated)
- Study completion
- Jul 2017 (estimated)
- First posted
- 23 Feb 2017 (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 investigate the safety, tolerability and potential for the use of up to three daily doses of 30-40 mg/kg HU (daily) upon hospitalization for painful vaso-occlusive crises .
Detailed description
Sickle cell anemia (SCA) is a hereditary hemoglobinopathy; complications of the disease include, spleen enlargement, acute chest syndrome, pulmonary hypertension, stroke and cumulative damage to multiple organs, and painful vaso-occlusive crises (VOC). In Brazil, about 3,500 children are born each year with DF, and the number of individuals with sickle cell disease (DF) in the country is estimated between 25,000 and 30,000 (ANVISA 2012; BRAZIL, 2012).
Hydroxyurea (HU, or hydroxycarbamide) is the only drug approved to date by the American FDA for use in adults with sickle cell disease. The drug modifies the disease process, improving hematological parameters and the hospitalization time of patients, as well as the frequency of vaso-occlusive crises.In addition to its proven effects during chronic use, experimental data indicate that HU has immediate anti-inflammatory effects.
In addition to its proven effects during chronic use, experimental data indicate that HU has immediate anti-inflammatory effects. This study will investigate the safety, tolerability and potential for the use of up to three daily doses of 30-40 mg/kg HU (daily) upon hospitalization for VOC.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control (No Hydroxyurea) | No intervention | –Patients in VOC will be treated according to the center's usual practice and analgesia protocol. |
| Hydroxyurea | Experimental | Drug: HydroxyureaPatients in VOC will receive up to three daily doses of 30-40 mg / kg hydroxyurea. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Confirmed diagnosis of homozygous sickle cell anemia (HbSS).
- Hospitalization due to onset of uncomplicated vaso-occlusive crisis (with pain scale≥6 within the last 24 h), confirmed by clinical evaluation.
- Documented and written informed consent
Key exclusion criteria (8 listed)
- Confirmed or suspected pregnancy.
- Initiation of painful crisis> 72h.
- Blood transfusion during the last 8 weeks.
- Admission to Emergency Room due to pain in the last 4 weeks.
- Neutrophil count <2.5 x 109/L or platelet count <95.0 x 109 / L or Hb <4.5 g / dL
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 |
|---|---|---|
| HydroxyureaPatients hospitalized for uncomplicated pain crisis with a pain scale of ≥ 6 during the last 24 hours will receive a dose of 30-40 mg / kg hydroxyurea. This same dose of hydroxyurea will be repeated at 24 h and 48 h after the first dose of hydroxyurea, with dose suspension if the patient is discharged within 48 hours. Patients will also receive the center's usual practice and analgesia protocol. | Drug | Hydroxyurea |
Primary outcome
Number of participants with treatment-related adverse events
Time frame: up to 15 days post last dose
as assessed by CTCAE version 4.03
Number of participants with altered laboratory values related to treatment
Time frame: up to 15 days post last dose
3 secondary outcomes
Time until hospital discharge
Time frame: Average, up to 7 days post admission
Total opioid use (mg of IV morphine)
Time frame: From study inclusion until hospital discharge (average, up to 7 days post admission)
Pain score
Time frame: From admission until hospital discharge (average, up to 7 days post admission)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2017.
- 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.
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 Anemia, Sickle Cell and Anemia; Sickle-Cell, With Crisis, run by Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti at 1 site in Brazil.
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 8 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: status not confirmed, as of the sponsor's last update on 23 Feb 2017. 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 Brazil. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Clarisse Lobo, MD (+ 55 (21) 98133-3606, lobo.clarisse@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-related adverse events (time frame: up to 15 days post last dose). The study lists 2 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 23 Feb 2017.