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

This study has been terminated.
(Low subject accrual)
Sponsor:
Information provided by (Responsible Party):
Robert I. Liem, Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier:
NCT00350844
First received: July 10, 2006
Last updated: March 28, 2013
Last verified: March 2013
Results First Received: February 18, 2013  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Sickle Cell Disease
Pulmonary Hypertension
Intervention: Drug: Hydroxyurea

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
This study is currently closed to patient accrual at all sites due to poor enrollment, including Children’s Memorial Hospital, Washington University, Johns Hopkins and the Medical College of Wisconsin. The FDA was notified in 2010, and the IND was removed for this study.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Hydroxyurea Hydroxyurea : 20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.

Participant Flow:   Overall Study
    Hydroxyurea  
STARTED     6  
COMPLETED     5  
NOT COMPLETED     1  
Lost to Follow-up                 1  



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Hydroxyurea Hydroxyurea : 20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.

Baseline Measures
    Hydroxyurea  
Number of Participants  
[units: participants]
  6  
Age  
[units: participants]
 
<=18 years     6  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  NA  ± NA [1]
Gender  
[units: participants]
 
Female     2  
Male     4  
Region of Enrollment  
[units: participants]
 
United States     6  
[1] Study was terminated due to low enrollment.



  Outcome Measures

1.  Primary:   Tricuspid Regurgitant Jet Velocity   [ Time Frame: 6 and 12 months after HU therapy begins ]

2.  Secondary:   Compliance   [ Time Frame: Throughout study ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  More Information
  Hide More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Early termination leading to no data analysis completed.  


Results Point of Contact:  
Name/Title: Dr. Robert I. Liem
Organization: Ann and Robert H. Lurie Children's Hospital of Chicago
phone: 312-227-4842
e-mail: Rliem@luriechildrens.org


No publications provided


Responsible Party: Robert I. Liem, Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier: NCT00350844     History of Changes
Other Study ID Numbers: 12735
Study First Received: July 10, 2006
Results First Received: February 18, 2013
Last Updated: March 28, 2013
Health Authority: United States: Food and Drug Administration