Modify Clinical study protocol of Iptacopan study in aHUS: CDSCO Panel Tell Novartis

New Delhi: Reviewing the Phase IIIb clinical trial study protocol of Iptacopan, the Subject Expert Committee (SEC) functional under the Central Standard Drug Control Organisation (CDSCO) has opined the drug major Novartis to modify the protocol in line with the inclusion criteria, the duration for which the study drug will be provided to participants, exclusion criteria, and others.

This came after the drug maker Novartis presented Phase IIIb clinical trial study protocol No. CLNP023F12001B. This is a multicenter, single-arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS (atypical hemolytic uremic syndrome).

aHUS (atypical hemolytic uremic syndrome) is a very rare disease that causes tiny blood clots to form in the small blood vessels of the body. These blood clots can block blood flow to important organs, such as kidneys. This can damage the kidneys and lead to kidney failure.

Iptacopan is a novel, oral, selective, and potent inhibitor of factor B, a key component of the complement alternative pathway.

Iptacopan is a factor B inhibitor used to treat paroxysmal nocturnal hemoglobinuria. Iptacopan is a small-molecule factor B inhibitor previously investigated as a potential treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) by inhibiting the complement factor B

At the recent SEC meeting for Renal held on 20th February 2024, the expert panel reviewed the proposal presented by the drug major Novartis to conduct Phase IIIb clinical trial protocol No. CLNP023F12001B.

After detailed deliberation, the committee opined that the following information is to be submitted for further review by the committee.

a. Serial number 3 of inclusion criteria should be revised for more objectivity, defining objective criteria based on which the investigator will assess that the participant has benefitted.

b. In the section describing the duration for which the study drug will be provided to participants, conditions number 2 and 3 should be defined more objectively.

“(Participants no longer derive benefit from Iptacopan” and the benefit-risk profile of the product is no longer positive)”.

c. In exclusion criteria, women of childbearing potential should be redefined.

d. Explanation for spontaneous abortion to be provided.

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