Citius Pharmaceuticals announced the results from the clinical study of Halo-Lido (CITI-002) yesterday (June 20th).
The trial was designed to assess the efficacy of the combination product, select the appropriate dose and endpoints for a phase 3 trial and to validate the patient recorded outcome (PRO).
The high dose formulation of CITI-002, a lidocaine and halobetasol propionate combination formulation, reduced symptom severity, as reported by patients, when compared to individual components alone.
There were no reported significant adverse events and CITI-002 was well-tolerated by patients in the study. The company intends to schedule an end of phase 2 meeting with the US Food and Drug Administration (FDA) to begin planning the next steps in the regulatory and clinical development program for CITI-002.
FDA directions
Approximately 300 adults with a clinical diagnosis of symptomatic hemorrhoids were enrolled in the Halo-Lido phase 2b study, a multi-center, randomized, dose-ranging, double-blind, parallel group comparison clinical trial.
The study assessed a high dose, CITI-002H, and low dose, CITI-002L, formulation of the combination drug products in comparison to the single active drug monads. The combinations were high dose halobetasol, low dose halobetasol and lidocaine.
The FDA directed Citius to develop a ‘fit for purpose’ PRO instrument to assess the efficacy of treatments in this disease. Symptom intensity and impact data (Hemorrhoid Quality of Life Index or HQLI) were recorded by patients utilizing a proprietary mobile-enabled PRO instrument developed by the company for this study.
Data collected using the HQLI was analyzed to derive a meaningful change threshold (MCT) to test for the change in hemorrhoidal symptoms considered relevant to the patient during and following treatment.
Trial Results
Treatment effect on hemorrhoidal symptoms was analyzed using the MCT. At the end of the 7-day treatment period, 42% of the patients in the high dose CITI-002 group reached MCT compared to patients treated with high dose halobetasol alone (29%) or patients treated with lidocaine alone (21%).
The study also found that proportionally more patients in the CITI-002H cohort reported meaningful and statistically significant improvement as compared to patients treated with lidocaine alone.
The researchers said: “We additionally assessed clinical treatment efficacy outcomes during 7-day treatment and seven-day follow-up periods using an analysis of covariance, which analyzed changes from baseline.
“Substantial improvements were seen across all active treatment groups. Although no statistical significance was determined in the changes between the comparison groups, directionally the data signaled that the combination products provided faster relief compared to individual monads, and the relief persisted after completing treatment.”
Data from the end of phase 2 will be presented to the FDA.
Myron Czuczman, chief medical officer of Citius said: “The encouraging phase 2b results indicate that CITI-002 may have the potential to provide patients with relief from the pain and discomfort associated with hemorrhoids. As far as we know, this is the first study of its kind, utilizing a novel PRO, conducted in hemorrhoid patients. We now better understand how to design the pivotal phase 3 study with relevant end points and look forward to further use of our PRO tool to evaluate efficacy.”
