Adakveo (crizanlizumab-tmca)

P1/2, N=45, Terminated, Novartis Pharmaceuticals | Completed --> Terminated; Sponsor Decision

P2, N=117, Completed, Novartis Pharmaceuticals | Active, not recruiting --> Completed

P2, N=20, Active, not recruiting, Washington University School of Medicine | Trial completion date: Aug 2027 --> Dec 2025 | Trial primary completion date: Jan 2024 --> Apr 2025

P4, N=130, Recruiting, Novartis Pharmaceuticals | Trial completion date: Oct 2031 --> Jun 2031 | Trial primary completion date: Oct 2031 --> Jun 2031

Forty-four patients were enrolled in Part 1 of the study of ruxolitinib in combination with siremadlin, rineterkib, sabatolimab, crizanlizumab, or NIS793. Overall, available data from ADORE suggest the feasibility and benefits of combining novel agents with ruxolitinib in patients with suboptimal response to ruxolitinib alone. This trial was registered at www.clinicaltrials.gov as #NCT04097821.

Notably, pharmaceutical approaches like hydroxyurea, l-glutamine, voxelotor, and crizanlizumab, in addition to therapeutic techniques like gene therapies like Casgevy and Lyfgenia, signify noteworthy advancements in the management of issues connected to SCD. It has been demonstrated that inhibiting these targets can prevent the silencing of the gene encoding for the formation of γ-chains and, in turn, increase the synthesis of HbF, providing a possible treatment option for SCD symptoms. These approaches could pave the way for innovative, mechanism-driven therapies that address the unmet medical needs of SCD patients.

P3, N=315, Recruiting, Novartis Pharmaceuticals | Not yet recruiting --> Recruiting

P=N/A, N=376, Completed, Novartis

P2, N=117, Active, not recruiting, Novartis Pharmaceuticals | Recruiting --> Active, not recruiting

P1/2, N=45, Completed, Novartis Pharmaceuticals | Active, not recruiting --> Completed

P3, N=315, Not yet recruiting, Novartis Pharmaceuticals

P2, N=119, Recruiting, Novartis Pharmaceuticals | Trial completion date: Jun 2026 --> Jan 2025