CDSCO Panel Clears PURE & CURE's Sickle Cell Trial With Conditions On Sample Size, Sites
New Delhi: PURE & CURE Healthcare Pvt. Ltd. has received a recommendation from the Subject Expert Committee (SEC) under the Central Drugs Standard Control Organisation (CDSCO) to conduct a Phase IV clinical trial of Hydroxyurea Oral Suspension 100 mg/mL in patients over 2 years of age suffering from Sickle Cell Disease. However, the approval comes with two key conditions: the sample size must be increased to 200 patients, and the trial sites must be selected from Chhattisgarh and Nagpur, regions with a high prevalence of the disease.
The recommendation came during the 08th meeting of the SEC (Haematology) held on 15th July 2025 at CDSCO headquarters, New Delhi.
The firm had presented its proposal seeking permission to conduct a Phase IV study titled: “A Phase IV Clinical Trial of Hydroxyurea Oral Suspension 100 mg/mL for the Prevention of Vaso-Occlusive Complications of Sickle Cell Disease in Patients Over 2 Years of Age.” The proposed trial will be conducted in accordance with Protocol No. SB-ICMR-CT-001, Version 1.0, dated 22 January 2024.
Hydroxyurea is a widely used therapy in sickle cell disease and works by increasing fetal hemoglobin levels, thereby reducing the frequency of vaso-occlusive crises, which are painful episodes caused by blockage of blood vessels due to sickled red blood cells.
Following a detailed review, the SEC recommended permission to proceed with the trial but asked the firm to incorporate two important changes to the study design. First, the sample size must be increased to 200 patients to enhance the statistical robustness of the findings. Second, the clinical trial sites should be selected from Chhattisgarh and Nagpur, regions with a high prevalence of sickle cell disease, ensuring relevant population representation.
“After detail deliberation, the Committee recommended to grant the permission to conduct clinical trial as per protocol presented with following changes: 1. The sample size should be increased to 200 patients 2. Recruit clinical trial sites from the Chhattisgarh and Nagpur areas where the disease is prevalent,” the SEC noted in its recommendation.
