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Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE) (SCATE)

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ClinicalTrials.gov Identifier: NCT01531387
Recruitment Status : Terminated (inability to reach a satisfactory endpoint with respect to adequate recruitment)
First Posted : February 13, 2012
Results First Posted : December 9, 2015
Last Update Posted : February 8, 2016
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
St. Jude Children's Research Hospital
Tropical Medicine Research Institute
Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Prevention
Condition Sickle Cell Anemia
Intervention Drug: Hydroxyurea
Enrollment 38
Recruitment Details  
Pre-assignment Details 22 participants were randomized (e.g., a total of 38 participants enrolled, but only 22 participants were randomized to treatment)
Arm/Group Title Hydroxyurea Standard Therapy: Observation
Hide Arm/Group Description

Half of the subjects will be randomized to hydroxyurea, taken as capsules (300 mg, 400 mg, or 500 mg), or as a liquid formulation (100 mg/mL). Hydroxyurea will be administered once daily by mouth. Subjects will be monitored monthly with clinical evaluations, laboratory tests, and TCD endpoint examinations.

Hydroxyurea: Hydroxyurea will be administered once daily, in either capsule form (300mg, 400mg, or 500mg) or as a liquid formulation (100mg/ml). Dosing will commence at 20 mg/kg/day. Dose escalation will occur in 5 mg/kg/day increments, adjusting every 8 weeks unless hematological toxicity occurs.

Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations
Period Title: Overall Study
Started 11 11
Completed 0 0
Not Completed 11 11
Reason Not Completed
Early termination of study             11             11
Arm/Group Title Standard Therapy: Observation Hydroxyurea Total
Hide Arm/Group Description Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations

Half of the subjects will be randomized to hydroxyurea, taken as capsules (300 mg, 400 mg, or 500 mg), or as a liquid formulation (100 mg/mL). Hydroxyurea will be administered once daily by mouth. Subjects will be monitored monthly with clinical evaluations, laboratory tests, and TCD endpoint examinations.

Hydroxyurea: Hydroxyurea will be administered once daily, in either capsule form (300mg, 400mg, or 500mg) or as a liquid formulation (100mg/ml). Dosing will commence at 20 mg/kg/day. Dose escalation will occur in 5 mg/kg/day increments, adjusting every 8 weeks unless hematological toxicity occurs.

Total of all reporting groups
Overall Number of Baseline Participants 11 11 22
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 11 participants 22 participants
<=18 years
11
 100.0%
11
 100.0%
22
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants 11 participants 22 participants
6.3  (1.5) 6  (2.4) 6.15  (1.95)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 11 participants 22 participants
Female
7
  63.6%
7
  63.6%
14
  63.6%
Male
4
  36.4%
4
  36.4%
8
  36.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 11 participants 11 participants 22 participants
Jamaica 7 6 13
Brazil 2 3 5
United States 2 2 4
1.Primary Outcome
Title Conversion to Abnormal Maximum TAMV
Hide Description The primary endpoint of the SCATE trial is the cumulative incidence of conversion to abnormal maximum TAMV (time-averaged mean velocity) measured by transcranial doppler (TCD) ultrasonography. Subjects must have conditional velocities at baseline, defined as 170 - 199 cm/sec, which indicate moderate stroke risk. Abnormal velocities are defined as ≥ 200 cm/sec, which indicate high stroke risk. The number of conversions from conditional velocities to abnormal velocities in each treatment arm will be compared as the primary outcome.
Time Frame 30 months
Hide Outcome Measure Data
Hide Analysis Population Description
No participants reached 30 months of treatment prior to study termination; therefore, the primary outcome measure was not analyzed.
Arm/Group Title Hydroxyurea Standard Therapy: Observation
Hide Arm/Group Description:

Half of the subjects will be randomized to hydroxyurea, taken as capsules (300 mg, 400 mg, or 500 mg), or as a liquid formulation (100 mg/mL). Hydroxyurea will be administered once daily by mouth. Subjects will be monitored monthly with clinical evaluations, laboratory tests, and TCD endpoint examinations.

Hydroxyurea: Hydroxyurea will be administered once daily, in either capsule form (300mg, 400mg, or 500mg) or as a liquid formulation (100mg/ml). Dosing will commence at 20 mg/kg/day. Dose escalation will occur in 5 mg/kg/day increments, adjusting every 8 weeks unless hematological toxicity occurs.

Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Serial TCD Velocities
Hide Description This secondary outcome measure will be the highest TAMV obtained in specific arteries. Serial TCD velocities are measured throughout the SCATE trial and will be compared to the baseline value.
Time Frame 30 months
Hide Outcome Measure Data
Hide Analysis Population Description
No participants reached 30 months of treatment prior to study termination; therefore, the secondary outcome measures were not analyzed.
Arm/Group Title Hydroxyurea Standard Therapy: Observation
Hide Arm/Group Description:

Half of the subjects will be randomized to hydroxyurea, taken as capsules (300 mg, 400 mg, or 500 mg), or as a liquid formulation (100 mg/mL). Hydroxyurea will be administered once daily by mouth. Subjects will be monitored monthly with clinical evaluations, laboratory tests, and TCD endpoint examinations.

Hydroxyurea: Hydroxyurea will be administered once daily, in either capsule form (300mg, 400mg, or 500mg) or as a liquid formulation (100mg/ml). Dosing will commence at 20 mg/kg/day. Dose escalation will occur in 5 mg/kg/day increments, adjusting every 8 weeks unless hematological toxicity occurs.

Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Cumulative Incidence of Neurological Events
Hide Description The cumulative incidence of neurological events as a secondary endpoint, which include both stroke and non-stroke neurological events, will be determined over the treatment period for both standard and alternative arms.
Time Frame 30 months
Hide Outcome Measure Data
Hide Analysis Population Description
No participants reached 30 months of treatment prior to study termination; therefore, the secondary outcome measures were not analyzed.
Arm/Group Title Hydroxyurea Standard Therapy: Observation
Hide Arm/Group Description:

Half of the subjects will be randomized to hydroxyurea, taken as capsules (300 mg, 400 mg, or 500 mg), or as a liquid formulation (100 mg/mL). Hydroxyurea will be administered once daily by mouth. Subjects will be monitored monthly with clinical evaluations, laboratory tests, and TCD endpoint examinations.

Hydroxyurea: Hydroxyurea will be administered once daily, in either capsule form (300mg, 400mg, or 500mg) or as a liquid formulation (100mg/ml). Dosing will commence at 20 mg/kg/day. Dose escalation will occur in 5 mg/kg/day increments, adjusting every 8 weeks unless hematological toxicity occurs.

Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Cumulative Incidence of Non-Neurological Events
Hide Description The cumulative incidence of non-neurological sickle cell-related events, including vaso-occlusion and splenic sequestration, will be estimated over the treatment period for both standard and alternative arms.
Time Frame 30 months
Hide Outcome Measure Data
Hide Analysis Population Description
No participants reached 30 months of treatment prior to study termination; therefore, the secondary outcome measures were not analyzed.
Arm/Group Title Hydroxyurea Standard Therapy: Observation
Hide Arm/Group Description:

Half of the subjects will be randomized to hydroxyurea, taken as capsules (300 mg, 400 mg, or 500 mg), or as a liquid formulation (100 mg/mL). Hydroxyurea will be administered once daily by mouth. Subjects will be monitored monthly with clinical evaluations, laboratory tests, and TCD endpoint examinations.

Hydroxyurea: Hydroxyurea will be administered once daily, in either capsule form (300mg, 400mg, or 500mg) or as a liquid formulation (100mg/ml). Dosing will commence at 20 mg/kg/day. Dose escalation will occur in 5 mg/kg/day increments, adjusting every 8 weeks unless hematological toxicity occurs.

Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Quality of Life
Hide Description Standard Quality of Life measure will be taken during specific time points, as well as one newly-developed Sickle Cell Disease Quality of Life measure.
Time Frame 30 months
Hide Outcome Measure Data
Hide Analysis Population Description
No participants reached 30 months of treatment prior to study termination; therefore, the secondary outcome measures were not analyzed.
Arm/Group Title Hydroxyurea Standard Therapy: Observation
Hide Arm/Group Description:

Half of the subjects will be randomized to hydroxyurea, taken as capsules (300 mg, 400 mg, or 500 mg), or as a liquid formulation (100 mg/mL). Hydroxyurea will be administered once daily by mouth. Subjects will be monitored monthly with clinical evaluations, laboratory tests, and TCD endpoint examinations.

Hydroxyurea: Hydroxyurea will be administered once daily, in either capsule form (300mg, 400mg, or 500mg) or as a liquid formulation (100mg/ml). Dosing will commence at 20 mg/kg/day. Dose escalation will occur in 5 mg/kg/day increments, adjusting every 8 weeks unless hematological toxicity occurs.

Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame May 2012 - Jan 2014 = 20 months
Adverse Event Reporting Description AEs below grade 2 were not recorded for the SCATE trial
 
Arm/Group Title Hydroxyurea Standard Therapy: Observation
Hide Arm/Group Description

Half of the subjects will be randomized to hydroxyurea, taken as capsules (300 mg, 400 mg, or 500 mg), or as a liquid formulation (100 mg/mL). Hydroxyurea will be administered once daily by mouth. Subjects will be monitored monthly with clinical evaluations, laboratory tests, and TCD endpoint examinations.

Hydroxyurea: Hydroxyurea will be administered once daily, in either capsule form (300mg, 400mg, or 500mg) or as a liquid formulation (100mg/ml). Dosing will commence at 20 mg/kg/day. Dose escalation will occur in 5 mg/kg/day increments, adjusting every 8 weeks unless hematological toxicity occurs.

Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations
All-Cause Mortality
Hydroxyurea Standard Therapy: Observation
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Hide Serious Adverse Events
Hydroxyurea Standard Therapy: Observation
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   6/11 (54.55%)      11/11 (100.00%)    
Blood and lymphatic system disorders     
Hemoglobin  1  1/11 (9.09%)  11 3/11 (27.27%)  11
General disorders     
Fever  1  1/11 (9.09%)  11 0/11 (0.00%)  0
Acute Chest Syndrome  1  0/11 (0.00%)  0 1/11 (9.09%)  11
Acute Splenic Sequestration  1  1/11 (9.09%)  11 1/11 (9.09%)  11
Vaso-occlusive Event  1  0/11 (0.00%)  0 1/11 (9.09%)  11
AST  1  1/11 (9.09%)  11 0/11 (0.00%)  0
Hepatobiliary disorders     
Hepatobiliary Disorder  1  2/11 (18.18%)  11 0/11 (0.00%)  0
Infections and infestations     
Infections and Infestations  1  1/11 (9.09%)  11 0/11 (0.00%)  0
Lung Infection  1  0/11 (0.00%)  0 1/11 (9.09%)  11
Lymph gland infection  1  0/11 (0.00%)  0 1/11 (9.09%)  11
Renal and urinary disorders     
Hematuria  1  0/11 (0.00%)  0 1/11 (9.09%)  11
Respiratory, thoracic and mediastinal disorders     
Bronchospasm  1  0/11 (0.00%)  0 1/11 (9.09%)  11
Vascular disorders     
Hypertension  1  0/11 (0.00%)  0 1/11 (9.09%)  11
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
Hydroxyurea Standard Therapy: Observation
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/11 (63.64%)      7/11 (63.64%)    
Blood and lymphatic system disorders     
Acute Chest Syndrome  1  0/11 (0.00%)  0 1/11 (9.09%)  1
Acute Stlenic sequestration  1  1/11 (9.09%)  1 1/11 (9.09%)  1
Vaso-occlusive Event  1  2/11 (18.18%)  2 3/11 (27.27%)  4
ANC  1  1/11 (9.09%)  1 0/11 (0.00%)  0
ARC  1  1/11 (9.09%)  1 1/11 (9.09%)  1
AST  1  1/11 (9.09%)  1 0/11 (0.00%)  0
Hemoglobin  1  1/11 (9.09%)  1 2/11 (18.18%)  3
Bilirubin  1  2/11 (18.18%)  2 0/11 (0.00%)  0
Gastrointestinal disorders     
Abdominal pain  1  1/11 (9.09%)  1 0/11 (0.00%)  0
Gastritis  1  0/11 (0.00%)  0 1/11 (9.09%)  1
Vomiting  1  1/11 (9.09%)  1 0/11 (0.00%)  0
General disorders     
Fever  1  2/11 (18.18%)  2 2/11 (18.18%)  2
Flu Like Symptoms  1  1/11 (9.09%)  1 1/11 (9.09%)  1
Non Cardiac Chest Pain  1  1/11 (9.09%)  1 0/11 (0.00%)  0
Pain  1  0/11 (0.00%)  0 1/11 (9.09%)  1
Hepatobiliary disorders     
Hepatobiliary Disorders  1  1/11 (9.09%)  1 0/11 (0.00%)  0
Infections and infestations     
Infections and Infestations  1  1/11 (9.09%)  1 1/11 (9.09%)  1
Lung infections  1  0/11 (0.00%)  0 1/11 (9.09%)  1
Lymph Glad Infection  1  0/11 (0.00%)  0 1/11 (9.09%)  1
Tooth Infection  1  1/11 (9.09%)  1 0/11 (0.00%)  0
Upper Respiratory Infection  1  2/11 (18.18%)  2 1/11 (9.09%)  1
Metabolism and nutrition disorders     
Metabolism and Nutrition Disorders  1  1/11 (9.09%)  1 0/11 (0.00%)  0
Nervous system disorders     
Dizziness  1  1/11 (9.09%)  1 0/11 (0.00%)  0
Headache  1  1/11 (9.09%)  1 1/11 (9.09%)  1
Renal and urinary disorders     
Hematuria  1  0/11 (0.00%)  0 1/11 (9.09%)  1
Proteinuria  1  0/11 (0.00%)  0 1/11 (9.09%)  1
Respiratory, thoracic and mediastinal disorders     
Allergic Rhinitis  1  1/11 (9.09%)  3 1/11 (9.09%)  1
Bronchospasm  1  1/11 (9.09%)  1 1/11 (9.09%)  1
Cough  1  1/11 (9.09%)  3 2/11 (18.18%)  2
Dysnea  1  1/11 (9.09%)  1 0/11 (0.00%)  0
Wheezing  1  0/11 (0.00%)  0 1/11 (9.09%)  1
Skin and subcutaneous tissue disorders     
Skin and subcutaneous tissue dissorder other  1  1/11 (9.09%)  1 1/11 (9.09%)  1
Vascular disorders     
hypertension  1  0/11 (0.00%)  0 1/11 (9.09%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: James M. Boyett, PhD
Organization: St. Jude Children's Research Hospital
Phone: 901-595-4986
EMail: james.boyett@stjude.org
Layout table for additonal information
Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01531387    
Other Study ID Numbers: H-29205 SCATE
R01HL098239 ( U.S. NIH Grant/Contract )
First Submitted: February 6, 2012
First Posted: February 13, 2012
Results First Submitted: June 19, 2015
Results First Posted: December 9, 2015
Last Update Posted: February 8, 2016