EXTEND EXpanding Treatment for Existing Neurological Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 29 January 2024
• Main ID: NCT02556099
• Date of registration: 18/05/2015
• Prospective Registration: No
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: EXTEND EXpanding Treatment for Existing Neurological Disease
• Scientific title: EXpanding Treatment for Existing Neurological Disease (EXTEND)
• Date of first enrolment: August 2014
• Target sample size: 100
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02556099
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Jamaica

Contacts

• Name: Russell Ware, MD, PhD
• Affiliation: Cincinnati Children's

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Pediatric participants with a severe form of sickle cell anemia (HbSS, HbSß0
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

Exclusion Criteria:

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)



Age minimum: 2 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Sickle Cell Anemia

Intervention(s)

Drug: Hydroxyurea

Primary Outcome(s)

Maximum Time-Averaged Mean velocity (TAMV) on TCD exam [Time Frame: 18 months]

Secondary Outcome(s)

Cumulative Incidence of Non-Neurological Events [Time Frame: Screening/Baseline and approximately 3 years after the first enrollment]

Quality of Life Assessment [Time Frame: Baseline, 18 months, and approximately 3 years after the first enrollment]

The cumulative incidence of neurological events [Time Frame: Screening/Baseline and approximately 3 years after the first enrollment]

Neuropsychological Assessment [Time Frame: Baseline, after 18 months]

Serial TCD velocities [Time Frame: Screening, Baseline, month 6, month 12, month 18]

Secondary ID(s)

2014-2875 EXTEND

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Caribbean Institute for Health Research (CAIHR)