What is the mechanism of action of this treatment?

Hydroxyurea, a common treatment for Sickle Cell Anemia, increases the production of fetal hemoglobin (Hb F). This is important because Hb F reduces the sickling of red blood cells, which can block blood flow and cause pain and organ damage. By increasing Hb F levels, hydroxyurea helps to prevent these complications, thereby improving the quality of life and potentially increasing the life expectancy of patients with Sickle Cell Anemia.

What side effects are associated with this type of intervention?

Hydroxyurea, a common treatment for Sickle Cell Anemia, is generally well-tolerated but can cause side effects such as bone marrow suppression, leading to neutropenia, thrombocytopenia, and anemia. Long-term use has not shown significant adverse effects on growth or development in children. L-glutamine, another treatment, has minimal side effects, primarily mild gastrointestinal symptoms. Both treatments aim to reduce the frequency of painful episodes and other complications associated with Sickle Cell Anemia.

What prior FDA approvals exist?

Hydroxyurea is an FDA-approved drug for the treatment of Sickle Cell Anemia, primarily because it increases fetal hemoglobin (Hb F) production, which helps reduce red blood cell sickling and related complications. Other FDA-approved treatments for Sickle Cell Anemia include L-glutamine (Endari), which reduces oxidative stress in red blood cells, and voxelotor (Oxbryta), which increases hemoglobin's affinity for oxygen, thereby reducing sickling. Crizanlizumab (Adakveo) is another approved drug that works by inhibiting P-selectin, reducing the frequency of vaso-occlusive crises. These treatments aim to manage and reduce the complications associated with Sickle Cell Anemia.

What other types of research exist?

Crizanlizumab is a promising novel alternative to Hydroxyurea for Sickle Cell Anemia patients. It has been shown to reduce the frequency of pain crises in patients with SCA. Additionally, chronic red blood cell transfusions are effective for stroke prevention and managing severe complications not responsive to Hydroxyurea.

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Anti-metabolites

Hydroxyurea for Sickle Cell Anemia (TREAT Trial)

N/A

Recruiting

Led By Patrick McGann, MD, MS

Research Sponsored by Children's Hospital Medical Center, Cincinnati

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 6 months to 21 years at the time of enrollment

Diagnosis of sickle cell anemia (HbSS or Hbβ0-thalassemia)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up annually up to ten years

Awards & highlights

No Placebo-Only Group

Summary

This trial studies how to best use hydroxyurea, a medication that helps blood cells stay round and flexible, in children with sickle cell anemia. It aims to find the right dose for each child and monitor their health. The study also looks at ways to check if children are taking their medication regularly. Hydroxyurea has been used for over 25 years to treat sickle cell anemia, showing efficacy in both adults and children.

Who is the study for?

The TREAT study is for children and young adults aged 6 months to 21 years with sickle cell anemia who, along with their families and healthcare providers, decide to start hydroxyurea therapy. This includes those switching from transfusions to hydroxyurea. Families must be willing to consent and comply with the study.

What is being tested?

This trial tests hydroxyurea in treating sickle cell anemia. It aims to predict the maximum tolerated dose using a model that considers how the body processes the drug (pharmacokinetics/pharmacodynamics), find urine markers of medication adherence, explore genetic factors affecting dosage tolerance, and monitor long-term effects on organ function and life quality.

What are the potential side effects?

Hydroxyurea can cause side effects such as bone marrow suppression leading to low blood counts, digestive issues like nausea or vomiting, skin changes including rashes or ulcers, hair loss, headache, fever, drowsiness or fatigue.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am between 6 months and 21 years old.

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I have been diagnosed with sickle cell anemia.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ yearly

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~yearly

This trial's timeline: 3 weeks for screening, Varies for treatment, and yearly for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Time to Reach Maximum Tolerated Dose (months)

Secondary study objectives

Assessment of Growth

Cardiac function (assessment and growth)

Hydroxyurea adherence

+4 more

Side effects data

From 2015 Phase 4 trial • 150 Patients • NCT00202644

25%

Headache

24%

Palpitations

12%

Hypertension

Diarrhoea

Arthralgia

Vertigo

Asthenia

Epistaxis

Anaemia

Chest pain

Upper respiratory tract infection

Urinary tract infection

Ischaemic stroke

Nasopharyngitis

Pharyngitis

Crohn's disease

Respiratory distress

Bladder cancer

Laryngitis

Sepsis

Neurological decompensation

Pulmonary embolism

Vasculitis cerebral

Tachycardia

Aphasia

Cerebral infarction

Type 2 diabetes mellitus

Adenoid cystic carcinoma

Left ventricular failure

Ovarian cyst

Upper gastrointestinal haemorrhage

Sudden death

Ear infection

Leukopenia

Hypertensive crisis

Peripheral artery thrombosis

Iron deficiency anaemia

Angina unstable

Traumatic haematoma

Hyperglycaemia

100%

80%

60%

40%

20%

Study treatment Arm

Anagrelide

Hydroxyurea

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: HydroxyureaExperimental Treatment1 Intervention

All enrolled participants will receive hydroxyurea, but upon enrollment, participants will be identified as part of the "New Cohort" or "Old Cohort" "New Cohort" participants include those who are not receiving hydroxyurea therapy upon study entry. "Old Cohort" participants include those who are already receiving hydroxyurea therapy upon study entry. New Cohort participants will have starting dose predicted using PK/PD data and Old Cohort participants will continue dosing per clinical guidelines.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Hydroxyurea

2006

Completed Phase 4

~3490

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Hydroxyurea, a common treatment for Sickle Cell Anemia, increases the production of fetal hemoglobin (Hb F). This is important because Hb F reduces the sickling of red blood cells, which can block blood flow and cause pain and organ damage. By increasing Hb F levels, hydroxyurea helps to prevent these complications, thereby improving the quality of life and potentially increasing the life expectancy of patients with Sickle Cell Anemia.