Hydroxyurea for Children and Young Adults With Sickle Cell Disease and Pulmonary Hypertension

NCT00350844 | Terminated Phase 1 Results

• 1 other identifier: 12735
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Sickle Cell Disease; Pulmonary Hypertension

Keywords

Sickle Cell Disease; Pulmonary Hypertension

Outcome Measures

Primary Outcomes (1)

• Tricuspid Regurgitant Jet Velocity

  Primary outcome measure was tricuspid regurgitant jet velocity (TRJV) by echocardiogram after 6 and 12 months of hydroxyurea therapy.

  6 and 12 months after HU therapy begins

Secondary Outcomes (1)

• Compliance

  Throughout study

Study Arms (1)

Hydroxyurea

EXPERIMENTAL

Drug: Hydroxyurea

Interventions

Hydroxyurea DRUG

20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.

Hydroxyurea

Eligibility Criteria

• Age: 10 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (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

Sponsors & Collaborators

• Ann & Robert H Lurie Children's Hospital of Chicago: lead

Study Sites (1)

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Hypertension, Pulmonary

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals

Limitations and Caveats

Early termination leading to no data analysis completed.

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: August 6, 2019
• Results First Posted: May 8, 2013

Record last verified: 2019-07

Locations

US (1)