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Decitabine for High-Risk Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT01375608
Recruitment Status : Completed
First Posted : June 17, 2011
Results First Posted : March 1, 2017
Last Update Posted : March 1, 2017
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Neutropenia
Sickle Cell Disease
Intervention Drug: Decitabine
Enrollment 10
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Subcutaneous Decitabine in Sickle Cell Disease
Hide Arm/Group Description 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
Period Title: Overall Study
Started 10
Completed 8
Not Completed 2
Reason Not Completed
Withdrawal by Subject             2
Arm/Group Title Subcutaneous Decitabine in Sickle Cell Disease
Hide Arm/Group Description 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
Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
<=18 years
0
   0.0%
Between 18 and 65 years
10
 100.0%
>=65 years
0
   0.0%
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
5
  50.0%
Male
5
  50.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 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  
Mean (Full Range)
Unit of measure:  g/dL
Number Analyzed 10 participants
8.14
(4.9 to 12)
fetal hemoglobin  
Mean (Full Range)
Unit of measure:  %
Number Analyzed 10 participants
4.8
(1 to 13.5)
1.Primary Outcome
Title The Percentage Change in HbF Level From Baseline to the Average Over the Final 1 Month of Study.
Hide Description [Not Specified]
Time Frame Final 1 month of study
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Subcutaneous Decitabine in Sickle Cell Disease
Hide Arm/Group Description:
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
Overall Number of Participants Analyzed 8
Measure Type: Count of Participants
Unit of Measure: Participants
total Hgb increase by >1g/dL AND HbF by >10%
1
  12.5%
total hgb increase by >1 g/dL OR HbF by 10%
1
  12.5%
total hgb increase by <1 g/dL OR HbF by <10%
6
  75.0%
Time Frame January 2012 to December 2015
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Subcutaneous Decitabine in Sickle Cell Disease
Hide Arm/Group Description 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
All-Cause Mortality
Subcutaneous Decitabine in Sickle Cell Disease
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Subcutaneous Decitabine in Sickle Cell Disease
Affected / at Risk (%) # Events
Total   7/10 (70.00%)    
General disorders   
non sickle related SAE  2/10 (20.00%)  2
Musculoskeletal and connective tissue disorders   
Sickle related  7/10 (70.00%)  30
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Subcutaneous Decitabine in Sickle Cell Disease
Affected / at Risk (%) # Events
Total   10/10 (100.00%)    
Cardiac disorders   
palpitation  2/10 (20.00%)  3
Ear and labyrinth disorders   
ENT [1]  5/10 (50.00%)  10
Gastrointestinal disorders   
GI disturbances [2]  5/10 (50.00%)  10
Musculoskeletal and connective tissue disorders   
temporary musckle weakness with injection  4/10 (40.00%)  6
headache  5/10 (50.00%)  5
Skin and subcutaneous tissue disorders   
related to subcutaneous injection  10/10 (100.00%)  10
rash  5/10 (50.00%)  9
[1]
metallic taste, hearing changes, jaw pain, dry mouth
[2]
Nausea, vomiting, diarrhea, changes in appetite
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
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
First Submitted: June 16, 2011
First Posted: June 17, 2011
Results First Submitted: January 10, 2017
Results First Posted: March 1, 2017
Last Update Posted: March 1, 2017