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Anti-metabolites

Hydroxyurea for Sickle Cell Disease (HUPrevent Trial)

Phase 2
Waitlist Available
Led By James F. Casella, MD
Research Sponsored by Johns Hopkins University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights

HUPrevent Trial Summary

This trial is testing if hydroxyurea can prevent brain complications in young children with sickle cell disease, compared to a placebo.

Eligible Conditions
  • Sickle Cell Disease
  • Stroke

HUPrevent Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 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
Number of Randomized Participants With Central Nervous System Complications
Secondary outcome measures
Number of Participants Randomized
Severe Adverse Events (SAE) Attributed to Sedated MRIs
Severe Adverse Events (SAE) Attributed to Study Procedures

Side effects data

From 2015 Phase 4 trial • 150 Patients • NCT00202644
25%
Headache
24%
Palpitations
12%
Hypertension
8%
Diarrhoea
8%
Arthralgia
7%
Vertigo
7%
Asthenia
5%
Epistaxis
5%
Anaemia
5%
Chest pain
5%
Upper respiratory tract infection
5%
Urinary tract infection
4%
Ischaemic stroke
3%
Nasopharyngitis
3%
Pharyngitis
1%
Cerebral infarction
1%
Neurological decompensation
1%
Pulmonary embolism
1%
Adenoid cystic carcinoma
1%
Vasculitis cerebral
1%
Ovarian cyst
1%
Type 2 diabetes mellitus
1%
Left ventricular failure
1%
Aphasia
1%
Respiratory distress
1%
Sepsis
1%
Bladder cancer
1%
Crohn's disease
1%
Tachycardia
1%
Laryngitis
1%
Upper gastrointestinal haemorrhage
1%
Sudden death
1%
Ear infection
1%
Leukopenia
1%
Hypertensive crisis
1%
Peripheral artery thrombosis
1%
Iron deficiency anaemia
1%
Angina unstable
1%
Traumatic haematoma
1%
Hyperglycaemia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Anagrelide
Hydroxyurea

HUPrevent Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: HydroxyureaExperimental Treatment1 Intervention
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 Absolute Neutrophil Count (ANC) <4000
Group II: PlaceboPlacebo Group1 Intervention
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

Find a Location

Who is running the clinical trial?

Children's Mercy Hospital Kansas CityOTHER
245 Previous Clinical Trials
935,858 Total Patients Enrolled
Washington University School of MedicineOTHER
1,930 Previous Clinical Trials
2,298,939 Total Patients Enrolled
Vanderbilt University School of MedicineOTHER
15 Previous Clinical Trials
13,913 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
BloodJournal
Inverse Correlation Between Cerebral Blood Flow Measured by Continuous Arterial Spin-labeling (CASL) MRI and Neurocognitive Function in Children with Sickle Cell Anemia (SCA)
Strouse, John J., Christiane S. Cox, Elias R. Melhem, Hanzhang Lu, Michael A. Kraut, Alexander Razumovsky, Kaleb Yohay, Peter C. van Zijl, and James F. Casella. 2006. “Inverse Correlation Between Cerebral Blood Flow Measured by Continuous Arterial Spin-labeling (CASL) MRI and Neurocognitive Function in Children with Sickle Cell Anemia (SCA)”. Blood. American Society of Hematology. doi:10.1182/blood-2005-10-4029.
BloodJournal
Hydroxyurea Therapy Lowers Transcranial Doppler Flow Velocities in Children with Sickle Cell Anemia
Zimmerman, Sherri A., William H. Schultz, Shelly Burgett, Nicole A. Mortier, and Russell E. Ware. 2007. “Hydroxyurea Therapy Lowers Transcranial Doppler Flow Velocities in Children with Sickle Cell Anemia”. Blood. American Society of Hematology. doi:10.1182/blood-2006-11-057893.
Pediatric Hematology and OncologyJournal
Design of the Silent Cerebral Infarct Transfusion (sit) Trial
Casella, James F., Allison A. King, Bruce Barton, Desiree A. White, Michael J. Noetzel, Rebecca N. Ichord, Cindy Terrill, et al.. 2010. “Design of the Silent Cerebral Infarct Transfusion (sit) Trial”. Pediatric Hematology and Oncology. Informa UK Limited. doi:10.3109/08880010903360367.
British Journal of HaematologyJournal
Silent Infarcts in Young Children with Sickle Cell Disease
Kwiatkowski, Janet L., Robert A. Zimmerman, Avrum N. Pollock, Wendy Seto, Kim Smith-Whitley, Justine Shults, Anne Blackwood-Chirchir, and Kwaku Ohene-Frempong. 2009. “Silent Infarcts in Young Children with Sickle Cell Disease”. British Journal of Haematology. Wiley. doi:10.1111/j.1365-2141.2009.07753.x.
~2 spots leftby Apr 2025
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