Novartis Told to Revise Protocol, Justify Sample Size in ITU512 Clinical Trial Proposal
New Delhi: Novartis Healthcare Private Limited has been directed by the Subject Expert Committee (SEC) functional under the Central Drugs Standard Control Organisation (CDSCO) to submit additional data and protocol clarifications for its proposed Phase I/II clinical study on ITU512, an investigational therapy for hemoglobin-related disorders.
The proposal was discussed during the 08th SEC (Haematology) meeting held on 15th July 2025 at CDSCO headquarters, New Delhi. Novartis presented Protocol No.CITU512A12101, Version 01, dated 29 July 2024.
ITU512 is believed to be an advanced therapeutic candidate potentially targeting sickle cell disease or β-thalassemia by modulating fetal hemoglobin levels. However, the SEC raised several scientific and protocol design concerns that require resolution before trial approval.
According to the committee, the primary endpoint must be clearly specified in terms of fetal hemoglobin (HbF) increase >15%, and this threshold must be scientifically justified based on prior studies, regulatory benchmarks, or clinical relevance. The panel also requested a justification for including patients with an absolute fetal hemoglobin percentage <15%.
During the presentation, SEC noted that definitions of the primary and secondary endpoints were not aligned with the submitted protocol. The committee emphasized that the criteria for efficacy evaluation should be explicitly defined. Further, exploratory endpoints such as vaso-occlusive crisis (VOCs) events must be incorporated into the initial protocol design.
Additionally, the firm was asked to submit Phase I data, including safety and hemolysis profiles, from either the current study or any other country where ITU512 has been tested. A detailed justification for the proposed sample size and dosing regimen was also requested.
“Accordingly, the firm shall submit the response for further review by committee,” the SEC recommended.
