Hydroxyurea for Stroke

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Mercy Children's Hospital, Kansas City, MO
Stroke+2 More
Hydroxyurea - Drug
Eligibility
< 18
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether hydroxyurea can reduce the risk of stroke in children with sickle cell disease.

See full description

Eligible Conditions

  • Stroke
  • Sickle Cell Disease (SCD)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether Hydroxyurea will improve 1 primary outcome and 2 secondary outcomes in patients with Stroke. Measurement will happen over the course of 6 months.

3 years
Central Nervous System Complications
Proportion of participants with severe adverse events attributed to study procedures
6 months
Proportion of participants undergoing randomization

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Compared to trials

Side Effects for

Anagrelide
Headache
25%
Palpitations
24%
Hypertension
12%
Arthralgia
8%
Diarrhoea
8%
Vertigo
7%
Asthenia
7%
Upper respiratory tract infection
5%
Chest pain
5%
Epistaxis
5%
Anaemia
5%
Urinary tract infection
5%
Ischaemic stroke
4%
Nasopharyngitis
3%
Pharyngitis
3%
Vasculitis cerebral
1%
Bladder cancer
1%
Adenoid cystic carcinoma
1%
Type 2 diabetes mellitus
1%
Laryngitis
1%
Iron deficiency anaemia
1%
Angina unstable
1%
Left ventricular failure
1%
Crohn's disease
1%
Tachycardia
1%
Sudden death
1%
Sepsis
1%
Upper gastrointestinal haemorrhage
1%
Hyperglycaemia
1%
Ear infection
1%
Traumatic haematoma
1%
Ovarian cyst
1%
Hypertensive crisis
1%
Respiratory distress
1%
Neurological decompensation
1%
Aphasia
1%
Pulmonary embolism
1%
Cerebral infarction
1%
Peripheral artery thrombosis
1%
Leukopenia
1%
Scleroderma
0%
Pancytopenia
0%
Traumatic amputation
0%
Anal fistula
0%
Foot fracture
0%
Inguinal hernia
0%
Gastrointestinal haemorrhage
0%
Malignant melanoma
0%
Tendon calcification
0%
Breast cancer
0%
Deep vein thrombosis
0%
Peripheral nerve sheath tumour malignant
0%
Neutropenia
0%
This histogram enumerates side effects from a completed 2015 Phase 4 trial (NCT00202644) in the Anagrelide ARM group. Side effects include: Headache with 25%, Palpitations with 24%, Hypertension with 12%, Arthralgia with 8%, Diarrhoea with 8%.

Trial Design

2 Treatment Groups

Hydroxyurea
1 of 2
Placebo
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 40 total participants across 2 different treatment groups

This trial involves 2 different treatments. Hydroxyurea is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

Hydroxyurea
Drug
treatment with hydroxyurea 20 mg/kg/day increased by 5 mg/kg every 8 weeks to maximum of 35 mg/kg/day or hematologic toxicity or ANC <4000
Placebo
Drug
Sucrose placebo 0.2 ml/kg/day increased to max of 0.35 ml/kg/day
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Hydroxyurea
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 3 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 3 years for reporting.

Closest Location

Mercy Children's Hospital - Kansas City, MO

Eligibility Criteria

This trial is for patients born any sex aged 18 and younger. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Inclusion Criteria for Screening
Participant must have sickle cell anemia (hemoglobin SS) or sickle Beta-zero (null) thalassemia (hemoglobin S-B0) as confirmed at the local institution by hemoglobin analysis after six months of age.
Participant must be 9 to 48 months of age. All screening procedures except MRI can be completed between 9 and 12 months of age, with the exception of the MRI, for which the child must have reached the age of 12 months.
Informed consent must be signed by the participant's legally authorized guardian acknowledging written consent to join the study.

Patient Q&A Section

How serious can anemia, sickle cell be?

"Significant anemia and SCD are not uncommon in African American and White females in a developing region. In our population, they have similar prevalence and outcome. This suggests that there is unlikely a statistically significant difference between AA and WF with SCD." - Anonymous Online Contributor

Unverified Answer

What causes anemia, sickle cell?

"The causes of anemia are complex and need to be viewed in the context of the patient's medical history and physical examination data. In Africa, where most of the world's sickle cell-disease victims are found, the most common cause of anemia is malaria. In this region of the world, malaria is also the leading cause of anemia-associated mortality. In many instances, malaria was also a major reason for moving to urban areas. Many factors interact to cause changes in hemoglobin concentration. In industrialized nations, such as the United States, malaria and sickling defects are rare causes of acute or nonacute anemia." - Anonymous Online Contributor

Unverified Answer

What are the signs of anemia, sickle cell?

"Signs and symptoms of anemia in children are not unique to sickle cell disease. Children who are sickle cell disease do not usually present with symptoms of anemia, so it is important for GPs to be alert to the existence of anemia but to seek further investigation if it is identified." - Anonymous Online Contributor

Unverified Answer

Can anemia, sickle cell be cured?

"It has not been definitively proved that anemia and sickle cell trait can be cured. This has not only significant economic and social consequences for a population with a high morbidity of anemia and sickle cell, but also for family members living with sickle-cell-thalassemia." - Anonymous Online Contributor

Unverified Answer

What is anemia, sickle cell?

"Sickle cell anemia arises from heterozygosity of HbS with the normal HbA. It is characterized by shortened life of red blood cells. In the absence of other hemoglobinopathies, it is invariably associated with sickle-cell disease." - Anonymous Online Contributor

Unverified Answer

How many people get anemia, sickle cell a year in the United States?

"In the United States, anemia is relatively common. In 2012, about 6 million people are believed to have [sickle cell anemia](https://www.withpower.com/clinical-trials/sickle-cell-anemia). In 2012, approximately 16,700 people died of sickle cell disease." - Anonymous Online Contributor

Unverified Answer

What are common treatments for anemia, sickle cell?

"There are many common treatments for anemia, particularly those with sickle cell disease. Patients with these diseases may benefit from additional treatment based on clinical, diagnostic, and hemoglobin profile evaluation." - Anonymous Online Contributor

Unverified Answer

How does hydroxyurea work?

"HU has been found to be particularly effective in lowering hemoglobin, particularly in patients with hereditary hemoglobinopathies, such as sickle cell anemia (SCA). We present an analysis of the effect of HU on adult SCA patients and summarize the clinical relevance of these findings through an analysis of the relationship between Hb levels, the number of HbF molecules, and clinical outcomes." - Anonymous Online Contributor

Unverified Answer

Does hydroxyurea improve quality of life for those with anemia, sickle cell?

"Increasing Hb levels with hydroxyurea was associated with improved quality of life and life satisfaction but not increased exercise capacity or Hb levels. There was no increased risk of adverse effects with hydroxyurea and no dose response. Prospective trials to study the long-term impact and safety of hydroxyurea and examine the optimal target Hb on QoL for these patients are required." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets anemia, sickle cell?

"Although there is an understanding of the disease pathogenesis, an understanding of the disease process, and guidelines for patient management, more information is needed about how the disease process presents, progresses, and how it differs depending on age. Future prospective studies on the disease process and for patients and their families are warranted, in order to help clinicians provide early diagnosis to avoid complications and to prevent disease progression." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of hydroxyurea?

"Hydroxyurea side effects can be as common and as unpleasant as the chronic illness. The side effects of hydroxyurea can be attributed to its ability to block the production of the enzyme ribonuclease (RNase). This inability, in turn, results in a deficiency in a molecule of the enzyme that is essential to the process of RNA and DNA synthesis. In addition, hydroxyurea interferes with heme synthesis. Heme functions depend on the process of metabolism which requires oxygen, and hydroxyurea affects its production. Moreover, hydroxyurea can interfere with the transport of a variety of chemicals, including the antihistamine diphenhydramine, which helps control allergic reactions." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for anemia, sickle cell?

"Anemia and sickle cell (SCD, HbC, or HbS) should be considered for clinical trials. summary: Recent findings assessed the efficacy and safety of different treatment regimens for patients with SCD-related anemia (high red cell distribution width (RDW), normal hematocrit (Hct), or normal Hb)." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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