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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

TREAT Trial Summary

This trial is testing a new treatment for sickle cell anemia in children, which aims to improve the quality of life for these patients.

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.Check my eligibility

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.See study design

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.

TREAT Trial Eligibility Criteria

Inclusion Criteria

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

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I am between 6 months and 21 years old.

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I have been diagnosed with sickle cell anemia.

TREAT Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ annually, up to ten years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~annually, up to ten years

This trial's timeline: 3 weeks for screening, Varies for treatment, and annually, up to ten years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Time to Reach Maximum Tolerated Dose (months)

Secondary outcome measures

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

Cerebral infarction

Neurological decompensation

Respiratory distress

Aphasia

Type 2 diabetes mellitus

Bladder cancer

Left ventricular failure

Ovarian cyst

Adenoid cystic carcinoma

Pulmonary embolism

Sepsis

Vasculitis cerebral

Crohn's disease

Tachycardia

Laryngitis

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

TREAT 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

~3620

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiLead Sponsor

815 Previous Clinical Trials

6,531,441 Total Patients Enrolled

12 Trials studying Sickle Cell Anemia

1,703 Patients Enrolled for Sickle Cell Anemia

Patrick McGann, MD, MSPrincipal InvestigatorChildren's Hospital Medical Center, Cincinnati

Charles Quinn, MD, MSPrincipal InvestigatorChildren's Hospital Medical Center, Cincinnati

Media Library

Hydroxyurea (Anti-metabolites) Clinical Trial Eligibility Overview. Trial Name: NCT02286154 — N/A

Sickle Cell Anemia Research Study Groups: Hydroxyurea

Sickle Cell Anemia Clinical Trial 2023: Hydroxyurea Highlights & Side Effects. Trial Name: NCT02286154 — N/A

Hydroxyurea (Anti-metabolites) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02286154 — N/A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the eligibility criteria for participation in this clinical trial?

"This medical trial is looking to recruit 150 individuals between 6 months and 21 years old, with a diagnosis of sickle cell anemia or Hbβ0-thalassemia. Further criteria include agreement by the patient, family as well as healthcare providers to begin hydroxyurea therapy; this includes those transitioning from chronic transfusions."

Answered by AI

How many people are eligible to participate in this experiment?

"Confirmative, the data published on clinicaltrials.gov shows that this research is currently recruiting individuals to participate. The trial was first announced in October 2014 and its last update happened in December 2021. A total of 150 patients are sought for enrollment at one site."

Answered by AI

Are those who exceed 25 years of age eligible to participate in this trial?

"The eligibility requirements for this clinical trial necessitate that participants range in age from 6 months to 21 years old. For individuals aged under 18, there are 218 studies currently enrolling patients, and 251 exploratory trials recruiting adults 65 or older."

Answered by AI

Are there any vacancies for volunteers in this experiment?

"According to the clinicaltrials.gov website, this trial is actively seeking patients; it was originally posted on October 1st 2014 and recently updated on December 7th 2021."

Answered by AI

In what cases is Hydroxyurea typically recommended?

"Hydroxyurea is typically deployed to fight malignant melanoma of skin, but it has also proven beneficial for managing hypereosinophilic syndrome (HES), neoplasm metastasis, and myeloid leukemia."

Answered by AI

Recent research and studies

British Journal of HaematologyJournal

Early Initiation of Hydroxyurea (hydroxycarbamide) Using Individualised, Pharmacokinetics‐guided Dosing Can Produce Sustained and Nearly Pancellular Expression of Fetal Haemoglobin in Children with Sickle Cell Anaemia

Quinn, Charles T., Omar Niss, Min Dong, Amanda Pfeiffer, Jennifer Korpik, Mary Reynaud, Holly Bonar, et al.. 2021. “Early Initiation of Hydroxyurea (hydroxycarbamide) Using Individualised, Pharmacokinetics‐guided Dosing Can Produce Sustained and Nearly Pancellular Expression of Fetal Haemoglobin in Children with Sickle Cell Anaemia”. British Journal of Haematology. Wiley. doi:10.1111/bjh.17663.

American Journal of HematologyJournal

Robust Clinical and Laboratory Response to Hydroxyurea Using Pharmacokinetically Guided Dosing for Young Children with Sickle Cell Anemia

McGann, Patrick T., Omar Niss, Min Dong, Anu Marahatta, Thad A. Howard, Tomoyuki Mizuno, Adam Lane, et al.. 2019. “Robust Clinical and Laboratory Response to Hydroxyurea Using Pharmacokinetically Guided Dosing for Young Children with Sickle Cell Anemia”. American Journal of Hematology. Wiley. doi:10.1002/ajh.25510.

British Journal of Clinical PharmacologyJournal

Development of a Pharmacokinetic-guided Dose Individualization Strategy for Hydroxyurea Treatment in Children with Sickle Cell Anaemia

Dong, Min, Patrick T. McGann, Tomoyuki Mizuno, Russell E. Ware, and Alexander A. Vinks. 2016. “Development of a Pharmacokinetic-guided Dose Individualization Strategy for Hydroxyurea Treatment in Children with Sickle Cell Anaemia”. British Journal of Clinical Pharmacology. Wiley. doi:10.1111/bcp.12851.

Blood Cells, Molecules, and DiseasesJournal