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

This study has been terminated.
(inability to reach a satisfactory endpoint with respect to adequate recruitment)
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
St. Jude Children's Research Hospital
Tropical Medicine Research Institute
HEMORIO – State institute of Hematology Arthur de Siqueira Cavalcanti (English)
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT01531387
First received: February 6, 2012
Last updated: January 13, 2016
Last verified: January 2016
Results First Received: June 19, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Prevention
Condition: Sickle Cell Anemia
Intervention: Drug: Hydroxyurea

  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
22 participants were randomized (e.g., a total of 38 participants enrolled, but only 22 participants were randomized to treatment)

Reporting Groups
  Description
Hydroxyurea

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.

Standard Therapy: Observation Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations

Participant Flow:   Overall Study
    Hydroxyurea     Standard Therapy: Observation  
STARTED     11     11  
COMPLETED     0     0  
NOT COMPLETED     11     11  
Early termination of study                 11                 11  



  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
Standard Therapy: Observation Half of the subjects will be randomized to clinical observation only, which includes monthly visits with clinical evaluations, laboratory tests, and TCD endpoint examinations
Hydroxyurea

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 Total of all reporting groups

Baseline Measures
    Standard Therapy: Observation     Hydroxyurea     Total  
Number of Participants  
[units: participants]
  11     11     22  
Age  
[units: participants]
     
<=18 years     11     11     22  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Age  
[units: Years]
Mean (Standard Deviation)
  6.3  (1.5)     6  (2.4)     6.15  (1.95)  
Gender  
[units: participants]
     
Female     7     7     14  
Male     4     4     8  
Region of Enrollment  
[units: participants]
     
Jamaica     7     6     13  
Brazil     2     3     5  
United States     2     2     4  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Conversion to Abnormal Maximum TAMV   [ Time Frame: 30 months ]

2.  Secondary:   Serial TCD Velocities   [ Time Frame: 30 months ]

3.  Secondary:   Cumulative Incidence of Neurological Events   [ Time Frame: 30 months ]

4.  Secondary:   Cumulative Incidence of Non-Neurological Events   [ Time Frame: 30 months ]

5.  Secondary:   Quality of Life   [ Time Frame: 30 months ]


  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:  
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:  
Name/Title: James M. Boyett, PhD
Organization: St. Jude Children's Research Hospital
phone: 901-595-4986
e-mail: james.boyett@stjude.org


Publications of Results:

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01531387     History of Changes
Other Study ID Numbers: H-29205 SCATE
R01HL098239 ( US NIH Grant/Contract Award Number )
Study First Received: February 6, 2012
Results First Received: June 19, 2015
Last Updated: January 13, 2016
Health Authority: United States: Food and Drug Administration
United States: Federal Government