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Decitabine for High-Risk Sickle Cell Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier:
NCT01375608
First received: June 16, 2011
Last updated: January 10, 2017
Last verified: January 2017
Results First Received: January 10, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Neutropenia
Sickle Cell Disease
Intervention: Drug: Decitabine

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

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
Subcutaneous Decitabine in Sickle Cell Disease decitabine (starting dose of 0.20 mg/kg, 2 days per week) to induce fetal hemoglobin (HbF) in patients with sickle cell anemia who are refractory to or intolerant of hydroxyurea

Participant Flow:   Overall Study
    Subcutaneous Decitabine in Sickle Cell Disease
STARTED   10 
COMPLETED   8 
NOT COMPLETED   2 
Withdrawal by Subject                2 



  Baseline Characteristics
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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
Subcutaneous Decitabine in Sickle Cell Disease decitabine (starting dose of 0.20 mg/kg, 2 days per week) to induce fetal hemoglobin (HbF) in patients with sickle cell anemia who are refractory to or intolerant of hydroxyurea

Baseline Measures
   Subcutaneous Decitabine in Sickle Cell Disease 
Overall Participants Analyzed 
[Units: Participants]
 10 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      0   0.0% 
Between 18 and 65 years      10 100.0% 
>=65 years      0   0.0% 
Gender 
[Units: Participants]
Count of Participants
 
Female      5  50.0% 
Male      5  50.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      10 100.0% 
White      0   0.0% 
More than one race      0   0.0% 
Unknown or Not Reported      0   0.0% 
total hemoglobin 
[Units: g/dL]
Mean (Full Range)
 8.14 
 (4.9 to 12) 
fetal hemoglobin 
[Units: %]
Mean (Full Range)
 4.8 
 (1 to 13.5) 


  Outcome Measures

1.  Primary:   The Percentage Change in HbF Level From Baseline to the Average Over the Final 1 Month of Study.   [ Time Frame: Final 1 month of study ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr Matthew Hsieh
Organization: NIH, NHLBI, MCHB
phone: 301-402-7687
e-mail: matthewhs@nhlbi.nih.gov


Publications:

Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT01375608     History of Changes
Other Study ID Numbers: 110184
11-H-0184
Study First Received: June 16, 2011
Results First Received: January 10, 2017
Last Updated: January 10, 2017