A study to assess the efficacy of 10 mg/kg/day hydroxyurea versus 20 mg/kg/day hydroxyurea in treatment of Indian sickle cell disease patients.

A non-inferiority randomized trial to assess the efficacy of 10mg hydroxyurea/kg/day versus 20mg hydroxyurea/kg/day in treatment of Sickle cell disease patients of tribal and non tribal areas in India.

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2022/04/042006

Enrollment

560

Registered

2022-04-21

Start date

Unknown

Completion date

Unknown

Last updated

2022-05-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Health Condition 1: D578- Other sickle-cell disorders

Interventions

Intervention1: Hydroxyurea oral drug: 10mg/kg/day intervention in arm 1 for 24 months Control Intervention1: Hydroxyurea: 20mg/kg/day intervention in arm 2 for 24 months.

Eligibility

Inclusion criteria

Inclusion criteria: 1. Confirmed diagnosis of Sickle cell disease and not on hydroxyurea treatment for last 6 months. 2. Age 2-14 years of age. 3. Creatinine less than or equal to 1.4 mg/dl and/or estimated glomerular filtration rate greater than 45ml/min/1.73 m2 4. Liver function tests (especially ALT and conjugated bilirubin) less than or equal to 4 times upper limit of normal.

Exclusion criteria

Exclusion criteria: 1) Red blood cell transfusion in the last 3 months. 2) Patient not willing to participate in the study.

Design outcomes

Primary

Measure	Time frame
Proportion of children with average hemoglobin rise of more than 2g/dl over 24 month period or average HbF rise by 4 points over 24 month period.Timepoint: 24 months	—

Secondary

Measure	Time frame
SCA-related morbidity (over 24 month period on study drug) - Pain Event - Dactylitis - Acute Chest Syndrome - Hepatic Sequestration - Splenic Sequestration - Cerebrovascular events - Blood Transfusion requirements b) Incidence of severe adverse events (SAE) over 24 month period Death, Hospitalization more than 7 days, Life threatening events c) Incidence of hematologic toxicities over 24 month period on study drug. Hemoglobin (Hb) 4.0gm/dl Hb6.0 g/dl and absolute reticulocyte count (ARC) 100X109 /L. Hb 7.0g/dl and ARC 80 X109/L Platelets 80X 109/L Absolute neutrophil count (ANC) 1.5X109/L d) Change in creatinine levels over 24 month period as a measure of renal function. Timepoint: 24 months	—

Measure

Time frame

SCA-related morbidity (over 24 month period on study drug) - Pain Event - Dactylitis - Acute Chest Syndrome - Hepatic Sequestration - Splenic Sequestration - Cerebrovascular events - Blood Transfusion requirements b) Incidence of severe adverse events (SAE) over 24 month period Death, Hospitalization more than 7 days, Life threatening events c) Incidence of hematologic toxicities over 24 month period on study drug. Hemoglobin (Hb) 4.0gm/dl Hb6.0 g/dl and absolute reticulocyte count (ARC) 100X109 /L. Hb 7.0g/dl and ARC 80 X109/L Platelets 80X 109/L Absolute neutrophil count (ANC) 1.5X109/L d) Change in creatinine levels over 24 month period as a measure of renal function. Timepoint: 24 months

—

Countries

India

Contacts

Public ContactNaveen Khargekar

ICMR-National Institute of Immunohaematology

madkaikarmanisha@gmail.com9892472552

Outcome results

None listed

Source: CTRI (via WHO ICTRP) · Data processed: Feb 4, 2026