Trial Outcomes & Findings for Decitabine for High-Risk Sickle Cell Disease (NCT NCT01375608)

Last Updated: 2017-03-01

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

Final 1 month of study

Results posted on

2017-03-01

Participant milestones
Measure	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
Overall Study STARTED	10
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	2

Reasons for withdrawal
Measure	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
Overall Study Withdrawal by Subject	2

Decitabine for High-Risk Sickle Cell Disease

Baseline characteristics by cohort
Measure	Subcutaneous Decitabine in Sickle Cell Disease n=10 Participants 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
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Gender Female	5 Participants n=5 Participants
Gender Male	5 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	10 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
total hemoglobin	8.14 g/dL n=5 Participants
fetal hemoglobin	4.8 % n=5 Participants

Timeframe: Final 1 month of study

Outcome measures
Measure	Subcutaneous Decitabine in Sickle Cell Disease n=8 Participants 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
The Percentage Change in HbF Level From Baseline to the Average Over the Final 1 Month of Study. total Hgb increase by >1g/dL AND HbF by >10%	1 Participants
The Percentage Change in HbF Level From Baseline to the Average Over the Final 1 Month of Study. total hgb increase by >1 g/dL OR HbF by 10%	1 Participants
The Percentage Change in HbF Level From Baseline to the Average Over the Final 1 Month of Study. total hgb increase by <1 g/dL OR HbF by <10%	6 Participants

Serious events: 7 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events
Measure	Subcutaneous Decitabine in Sickle Cell Disease n=10 participants at risk 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
Musculoskeletal and connective tissue disorders Sickle related	70.0% 7/10 • Number of events 30 • January 2012 to December 2015
General disorders non sickle related SAE	20.0% 2/10 • Number of events 2 • January 2012 to December 2015

Other adverse events
Measure	Subcutaneous Decitabine in Sickle Cell Disease n=10 participants at risk 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
Skin and subcutaneous tissue disorders related to subcutaneous injection	100.0% 10/10 • Number of events 10 • January 2012 to December 2015
Musculoskeletal and connective tissue disorders temporary musckle weakness with injection	40.0% 4/10 • Number of events 6 • January 2012 to December 2015
Skin and subcutaneous tissue disorders rash	50.0% 5/10 • Number of events 9 • January 2012 to December 2015
Gastrointestinal disorders GI disturbances	50.0% 5/10 • Number of events 10 • January 2012 to December 2015
Musculoskeletal and connective tissue disorders headache	50.0% 5/10 • Number of events 5 • January 2012 to December 2015
Cardiac disorders palpitation	20.0% 2/10 • Number of events 3 • January 2012 to December 2015
Ear and labyrinth disorders ENT	50.0% 5/10 • Number of events 10 • January 2012 to December 2015

NIH, NHLBI, MCHB

Phone: 301-402-7687