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

Phase 2

Recruiting

• Conditions: Genetic Diseases; Haematological Disorders; Paediatrics

• Registration Number: PACTR202108893981080
• Lead Sponsor: Cincinnati Childrens Hospital Medical Center

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-08-11
• Last Update Posted: 29 May 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 240

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

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)

Study & Design

• Study Type: Interventional
• Study Design: Not specified