NCT00350844

Brief Summary

The goal of this study is to test the hypothesis that hydroxyurea is effective for the specific treatment of secondary pulmonary hypertension found on screening in children and young adults with sickle cell disease.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jul 2006

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2006

Completed
9 days until next milestone

First Submitted

Initial submission to the registry

July 10, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 11, 2006

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2008

Completed
4.9 years until next milestone

Results Posted

Study results publicly available

May 8, 2013

Completed
Last Updated

September 24, 2026

Status Verified

August 1, 2026

Enrollment Period

1.9 years

First QC Date

July 10, 2006

Results QC Date

February 18, 2013

Last Update Submit

August 31, 2026

Conditions

Sickle Cell DiseasePulmonary Hypertension

Keywords

Sickle Cell DiseasePulmonary Hypertension

Outcome Measures

Primary Outcomes (2)

  • Mean Reduction in TRJV at M6

    6 months after initiation of hydroxyurea

  • Mean Reduction in TRJV at M12

    12 months after initiation of hydroxyurea

Secondary Outcomes (6)

  • Mean Increase Hb F% at M6

    6 months after initiation of hydroxyurea

  • Mean Increase in HbF% at M12

    12 months after initiation of hydroxyurea

  • Mean Reduction in LDH at M6

    6 months after initiation of hydroxyurea

  • Mean Reduction in LDH at M12

    12 months after initiation of hydroxyurea

  • Mean Reduction in Retic% at M6

    6 months after initiation of hydroxyurea

  • +1 more secondary outcomes

Study Arms (1)

Hydroxyurea

EXPERIMENTAL

Single arm pilot of hydroxyurea in the experimental arm with starting dose of 20 mg/kg/day po and dose escalation every 2 months until maximum tolerated dose.

Drug: Hydroxyurea

Interventions

HydroxyureaDRUG

Drug to be initiated at standard starting dose with dose escalation until maximum tolerated dose reached.

Also known as: Experimental arm
Hydroxyurea

Eligibility Criteria

Age10 Years - 25 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age between 10 and 25 years old
  • Sickle cell disease with hemoglobin SS, SC or S-B\^0 thalassemia confirmed on hemoglobin electrophoresis
  • Tricuspid regurgitant jet velocity (TRJV) equal to or greater than 2.5 m/sec on 2 baseline Doppler echocardiograms at least 3 months apart

You may not qualify if:

  • Patients already being treated with hydroxyurea
  • Patients on a chronic transfusion protocol
  • Patients with evidence of hepatic (alanine aminotransferase \[ALT\] equal to or greater than 2 SD above normal) or renal dysfunction (creatinine \[Cr\] equal to or greater than 2 SD above normal)
  • Patients who are pregnant
  • Patients with documented causes of severe pulmonary hypertension other than from SCD

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

MeSH Terms

Conditions

Anemia, Sickle CellHypertension, Pulmonary

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLung DiseasesRespiratory Tract DiseasesHypertensionVascular DiseasesCardiovascular Diseases

Intervention Hierarchy (Ancestors)

UreaAmidesOrganic Chemicals

Limitations and Caveats

Given our small sample size, no other secondary data were evaluated and no further statistical analysis was performed on reported primary or secondary data.

Results Point of Contact

Title
Dr. Robert I. Liem
Organization
Ann and Robert H. Lurie Children's Hospital of Chicago
Rliem@luriechildrens.org
312-227-4842

Study Officials

  • Robert I Liem, MD

    Ann & Robert H Lurie Children's Hospital of Chicago

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, Assistant Professor of Pediatics, NU Feinberg School of Medicine

Study Record Dates

First Submitted

July 10, 2006

First Posted

July 11, 2006

Study Start

July 1, 2006

Primary Completion

June 1, 2008

Study Completion

June 1, 2008

Last Updated

September 24, 2026

Results First Posted

May 8, 2013

Record last verified: 2026-08

Locations

US(1)