Hydroxyurea in the Emergency Room to Lessen Pain in Sickle Cell Crisis

Phase 2

• Conditions: Anemia, Sickle Cell; Anemia; Sickle-Cell, With Crisis
• Interventions: Drug: Hydroxyurea

• Registration Number: NCT03062501
• Lead Sponsor: Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti

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.

Study Timeline

• Study Start: 2016-11
• Study First Submitted: 2017-01-30
• Status Verified: 2017-02
• Study First Submitted QC: 2017-02-20
• Last Update Submitted: 2017-02-20
• Study First Posted: 2017-02-23
• Last Update Posted: 2017-02-23
• Primary Completion: 2017-04
• Study Completion: 2017-07

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Hydroxyurea	Hydroxyurea	Patients in VOC will receive up to three daily doses of 30-40 mg / kg hydroxyurea.

Primary Outcome Measures

Name	Time	Method
Number of participants with altered laboratory values related to treatment	up to 15 days post last dose
Number of participants with treatment-related adverse events	up to 15 days post last dose	as assessed by CTCAE version 4.03

Secondary Outcome Measures

Name	Time	Method
Time until hospital discharge	Average, up to 7 days post admission
Total opioid use (mg of IV morphine)	From study inclusion until hospital discharge (average, up to 7 days post admission)
Pain score	From admission until hospital discharge (average, up to 7 days post admission)	Numeric pain score rating (0 to 10; 0 = no pain, 10 = worst pain)

Trial Locations

Locations (1)

Hemorio; 🇧🇷 Rio de Janeiro, Brazil