Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea (NCT01425307) | Clinical Trial Compass
TerminatedPhase 3
Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea
Stopped: The study was stopped early due to successfully meeting the primary endpoint
159 participantsStarted 2011-08
Plain-language summary
The primary goal of the Phase III TWiTCH trial is to compare 24 months of alternative therapy (hydroxyurea) to standard therapy (transfusions) for pediatric subjects with sickle cell anemia and abnormally high (≥200 cm/sec) Transcranial Doppler (TCD) velocities, who currently receive chronic transfusions to reduce the risk of primary stroke. For the alternative treatment regimen (hydroxyurea) to be declared non-inferior to the standard treatment regimen (transfusions), after adjusting for baseline differences, the hydroxyurea-treated group must have a mean TCD velocity similar to that observed with transfusion prophylaxis.
Who can participate
Age range4 Years – 15 Years
SexALL
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Inclusion criteria
✓. Pediatric subjects with severe forms of sickle cell anemia (HbSS, HbSβ0 thalassemia,HbSOArab)
✓. Age range of 4.0-15.99 years, inclusive, at the time of enrollment
✓. Documented index (pre-treatment) abnormally high TCD Velocity by Transcranial Doppler ultrasonography. An abnormally high index TCD is defined as TCD V greater than or equal to 200 cm/sec, or abnormally high TCDi V greater than or equal to185cm/sec, or TCD maximum V greater than or equal to 250 cm/sec.
✓. At least 12 months of chronic monthly erythrocyte transfusions since the index abnormal TCD examination
✓. Adequate monthly erythrocyte transfusions with average HbS less than or equal to 45% (the upper limit of the established academic community standard) for the past 6 months before enrollment
✓. Parent or guardian willing and able to provide informed consent with verbal or written assent from the child
✓. Ability to comply with study related treatments, evaluations, and follow-up
Exclusion criteria
✕
What they're measuring
1
Difference in TCD Time-averaged Mean Velocity (TAMV) on the Index Side
Timeframe: Since the study was terminated early, time frame is from beginning of treatment until end of treatment (up to 24 Months).
Trial details
NCT IDNCT01425307
SponsorChildren's Hospital Medical Center, Cincinnati
✕. Inability to obtain TCD velocities due to anatomical abnormalities such as a) Inadequate bone windows b) Previous revascularization procedures (e.g., EDAS)
✕. Known severe vasculopathy or moya-moya disease on brain MRA
✕. Inability to receive or tolerate chronic red blood cell (RBC) transfusion therapy, due to any of the following: a) Multiple RBC alloantibodies making cross-matching difficult or impossible b) RBC autoantibodies making cross-matching difficult or impossible c) Religious objection to transfusions that preclude their chronic use d) Non-compliance with transfusions over the past 6 months before enrollment (temporary exclusion)
✕. Inability to take or tolerate daily oral hydroxyurea, including a) Known allergy to hydroxyurea therapy b) Positive serology to HIV infection c) Malignancy d) Current lactation e) Previous stem cell transplant or other myelosuppressive therapy
✕. Clinical and laboratory evidence of hypersplenism (temporary exclusions): a) Palpable splenomegaly greater than 5cm below the left costal margin AND b) Transfusion requirement greater than 250 mL/kg over the previous 12 months
✕. Abnormal laboratory values at initial evaluation (temporary exclusions): a) Pre-transfusion hemoglobin concentration less than 8.0 gm/dL b) WBC count less than 3.0 x 10\^9/L c) Absolute neutrophil count (ANC) less than 1.5 x 10\^9/L d) Platelet count less than 100 x 10\^9/L e) Serum creatinine more than twice the upper limit for age OR greater than or equal to 1.0 mg/dL
✕. Current participation in other therapeutic clinical trials