Clinical trial NCT03062501: Hydroxyurea in the Emergency Room to Lessen Pain in Sickle Cell Crisis
Official title: Protocol for the Administration of Hydroxyurea During Painful Vaso-occlusive Crisis in Sickle Cell Anemia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Anemia, Sickle Cell
Anemia; Sickle-Cell, With Crisis
Lead sponsor: Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti
Sponsor class: OTHER
First posted: 2017-02-23
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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 .

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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Number of participants with treatment-related adverse events
timeFrame: up to 15 days post last dose
measure: Number of participants with altered laboratory values related to treatment
timeFrame: up to 15 days post last dose
Secondary outcomes:
measure: Time until hospital discharge
timeFrame: Average, up to 7 days post admission
measure: Total opioid use (mg of IV morphine)
timeFrame: From study inclusion until hospital discharge (average, up to 7 days post admission)
measure: Pain score
timeFrame: From admission until hospital discharge (average, up to 7 days post admission)
Other outcomes:
Locations:
city: Rio de Janeiro
country: Brazil
facility: Hemorio
Study officials:
References: