Prospective Identification of Variables as Outcomes for Treatment (PIVOT):

Conditions

Genetic Diseases Haematological Disorders Paediatrics

Catherine I. Segbefia, Luke R. Smart, Susan E. Stuber, Kwesi Nyan Amissah-Arthur, Klenam Dzefi‐Tettey et al. (15 authors)

British Journal of Haematology2024-10-153 citations

10.1111/bjh.19832

Prospective Identification of Variables As Outcomes for Treatment (PIVOT): A Phase II Clinical Trial of Hydroxyurea for Children and Adults with HbSC Disease

Catherine Segbefia, Luke R. Smart, Susan E. Stuber, Priscilla Ekpale, Kwesi Nyan Amissah-Arthur et al. (12 authors)

Blood2023-11-02

10.1182/blood-2023-190351

Prospective identification of variables as outcomes for treatment (PIVOT): study protocol for a randomised, placebo-controlled trial of hydroxyurea for Ghanaian children and adults with haemoglobin SC disease.

Smart LR, Segbefia CI, Latham TS, Stuber SE, Amissah-Arthur KN, Dzefi-Tettey K, Lane AC, Dei-Adomakoh YA, Ware RE.

2023-09-223 citations

10.1186/s13063-023-07649-7

Interventions

Placebo

Hydroxyurea

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Study participants who meet all of the following criteria are eligible for enrolment into the study: 1. Patients with documented HbSC disease 2. Age: = 5.0 and = 50.0 years of age, at the time of enrolment 3. Steady-state laboratory values in the following ranges: a. Hemoglobin concentration between 6.0 g/dL and 12.0 g/dL b. Absolute reticulocyte count (ARC) > 50 x 109/L c. White blood cell (WBC) count > 2.0 x 109/L d. Absolute neutrophil count (ANC) > 1.0 x 109/L e. Platelet count > 75 x 109/L 4. Patient, parent, or guardian willing and able to provide informed consent 5. Ability to comply with all study related treatments, evaluations, and follow-up

Exclusion criteria

Exclusion criteria: Study participants who meet any of the following criteria are disqualified from enrolment in the study: 1. Current hydroxyurea treatment (or within the past 6 months) 2. Regular blood transfusions (6 or more within the past 12 months) 3. More than 10 hospitalizations in the past 12 months 4. Known serum creatinine more than twice the upper limit for age AND = 176.8 µmol/L 5. Any underlying condition or illness, including chronic pain, co-morbid chronic disease (e.g. TB, retroviral infection etc.) which makes study participation ill-advised 6. Use of other therapeutic agents for sickle cell disease such as glutamine, arginine, crizanlizumab, rivipansel, voxelotor, decitabine, or magnesium within the past six months (temporary exclusion). 7. Previous stem cell transplant or other myelosuppressive therapy 8. Inability to take or tolerate daily oral hydroxyurea, including a. Known allergy to hydroxyurea therapy b. Known malignancy 9. Pregnancy (for post-menarcheal females only) 10. Current participation in other therapeutic clinical trials, or within 6 months of prior disease-modifying treatments 11. Blood transfusion within the past 2 months (temporary exclusion)

Design outcomes

Primary

Measure	Time frame
For children and adults with HbSC disease the trial will determine the safety of hydroxyurea in this patient population, and identify measurable changes in a variety of laboratory and clinical parameters, suitable for prospective testing in a definitive Phase 3 clinical trial.	—

Secondary

Measure	Time frame
To measure the toxicities of hydroxyurea treatment on laboratory parameters.;To assess the effects of hydroxyurea treatment on a variety of sickle-related clinical and laboratory parameters in a large cohort of children and adults with HbSC disease.	—

Countries

Ghana

Contacts

Public ContactSusan Stuber

Project Manager

susan.stuber@cchmc.org5136364200

Outcome results

None listed