EXpanding Treatment for Existing Neurological Disease (EXTEND)

Study point of contact

Russell Ware, MD, PhD
513-803-4597
[email protected]

Locations

1 Jamaica site

Age

2 to 17 Years

Genotypes

HbSS

Phase

Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

The primary goal of the Phase II EXTEND trial is to investigate the effects of open-label
hydroxyurea treatment, escalated to maximum tolerated dose, for children with Sickle Cell
Anemia and either conditional (170 – 199 cm/sec) or abnormal (≥200 cm/sec) Transcranial
Doppler velocities. The primary endpoint will be measured after 18 months of hydroxyurea but
treatment will continue until a common study termination date.

participation requirements

1. Pediatric participants with a severe form of sickle cell anemia (HbSS, HbSβ0
thalassemia, HbSD, HbSOArab)

2. Age: ≥ 2 and ≤ 17 years of age, at the time of enrollment

3. Time-averaged maximum velocity (TAMV) TCD Velocity in the conditional (170 – 199
cm/sec) or abnormal (≥200 cm/sec) range by Transcranial Doppler ultrasonography
examination within 6 months of enrollment, abnormal or conditional TCD velocity and
currently on commercial hydroxyurea for primary stroke prevention, or previously
enrolled in SCATE, a previous stroke with abnormal or conditional TCD prior to stroke
event.

4. Parent or guardian willing and able to provide informed consent and child gives assent

5. Ability to comply with study related treatments, evaluations, and follow- up visits

participation restrictions

1. Inability to take or tolerate daily oral hydroxyurea, including

– Known allergy to hydroxyurea therapy

– Known positive serology to HIV infection

– Known malignancy

– Current lactation

2. Abnormal historical laboratory values (most recent pre-enrollment values unless
previously enrolled in SCATE):

– Hemoglobin concentration < 6.0 gm/dL - Absolute reticulocyte count < 100 x 109/L with a hemoglobin concentration < 8.0 gm/dL - White Blood Cell (WBC) count < 3.0 x 109/L - Absolute neutrophil count (ANC) < 1.0 x 109/L - Platelet count < 100 x 109/L 3. Use of therapeutic agents for sickle cell disease (e.g., hydroxyurea, arginine, decitabine, magnesium, chronic transfusions) within 3 months of enrollment unless they have an abnormal TCD velocity and receive commercial hydroxyurea for primary stroke prevention or were previously enrolled in the SCATE study or for secondary stroke prevention in a child with a previous stroke. 4. Current participation in other therapeutic clinical trials, except SCATE 5. Known serum creatinine more than twice the upper limit for age AND - 1.0 mg/dL 6. Any condition or chronic illness, which in the opinion of the clinical investigator makes participation ill-advised 7. Pregnancy (for post-menarchal females only) 8. Erythrocyte transfusion within the past 2 months 9. Previous stem cell transplant or other myelosuppressive therapy (unless they have an abnormal TCD velocity and receive commercial hydroxyurea for primary stroke prevention or for secondary stroke prevention in a child with a previous stroke or were previously enrolled in SCATE)

Locations

  • Kingston, Jamaica, Sickle Cell Unit [Recruiting]