Articles related to CLINICAL TRIALS

A phase 3, multicenter, double-blind, randomized, placebo-controlled trial was conducted to evaluate the efficacy and safety of epicutaneous immunotherapy with a peanut patch in children aged 1 to 3 years with peanut allergy. No approved treatment for peanut allergy exists for children under 4 years old. Participants with confirmed peanut allergy were assigned to either receive the peanut patch or a placebo daily for 12 months. The primary endpoint was the treatment response measured by the eliciting dose of peanut protein at 12 months. The trial showed that 67.0% of children in the intervention group demonstrated a treatment response compared to 33.5% in the placebo group. Adverse events occurred in both groups, with serious adverse events and anaphylaxis observed more frequently in the intervention group. However, serious treatment-related adverse events were rare. In conclusion, this trial demonstrated that epicutaneous immunotherapy with a peanut patch for 12 months was more effective than placebo in desensitizing children aged 1 to 3 years with peanut allergy and increasing their tolerance to peanut protein. Although adverse events were observed in both groups, serious treatment-related adverse events were uncommon. These findings suggest that epicutaneous immunotherapy may be a potential treatment option for young children with […]

Researchers presented findings from the AB10015 Phase 2/3 clinical trial, demonstrating that masitinib, an oral add-on therapy to Rilutek, offers significant benefits for patients with mild to moderate amyotrophic lateral sclerosis (ALS). Although the entire study population did not experience improved survival with masitinib, patients with moderate ALS who received masitinib in addition to Rilutek had significantly longer survival compared to those who received a placebo. The combined treatment also slowed disease progression, improved lung function, and enhanced quality of life in these patients. Masitinib works by blocking specific tyrosine kinase enzymes that regulate immune cell activity and inflammation, aiming to slow disease progression and alleviate symptoms. The analysis focused on normally progressing participants with moderate ALS at the study’s start, and in this subgroup, masitinib combined with Rilutek significantly slowed disease progression by 42% compared to Rilutek alone. Additionally, patients with moderate ALS experienced a median survival of 69 months with masitinib, representing a 44% reduction in mortality risk compared to placebo-treated patients. The combination therapy also showed positive effects on quality of life and lung function in patients progressing normally. These findings suggest that initiating masitinib at an earlier stage of the disease could lead to substantial reductions […]

Cabozantinib plus nivolumab and ipilimumab (C-N-I) is a new combination therapy for patients with advanced renal cell carcinoma (RCC). In a phase 3 clinical trial, C-N-I was shown to be superior to sunitinib in terms of progression-free survival (PFS) and overall survival (OS). The trial enrolled 823 patients with advanced RCC who had not received prior systemic therapy. Patients were randomly assigned to receive either C-N-I (n=412) or sunitinib (n=411). The primary endpoint of the trial was PFS, and the secondary endpoint was OS. At a median follow-up of 24 months, patients who received C-N-I had a significantly longer PFS than patients who received sunitinib. The median OS for patients who received C-N-I was not reached, while the median OS for patients who received sunitinib was 22.8 months.

This study compared the efficacy and safety of four different treatment regimens for patients with chronic lymphocytic leukemia (CLL): chemoimmunotherapy, venetoclax-rituximab, venetoclax-obinutuzumab, and venetoclax-obinutuzumab-ibrutinib. The results showed that the venetoclax-obinutuzumab-ibrutinib regimen was the most effective, with significantly higher rates of undetectable minimal residual disease (MRD) and progression-free survival (PFS) at 15 months and 3 years, respectively, compared to the other regimens. The venetoclax-obinutuzumab regimen was also effective, with significantly higher rates of undetectable MRD at 15 months compared to chemoimmunotherapy. However, there was no significant difference in PFS at 3 years between the venetoclax-obinutuzumab and chemoimmunotherapy regimens. The venetoclax-rituximab regimen was the least effective, with no significant difference in undetectable MRD or PFS at 15 months or 3 years compared to chemoimmunotherapy. The most common adverse events were infections, which were more common with chemoimmunotherapy and venetoclax-obinutuzumab-ibrutinib than with the other regimens.

More trials in larger groups of patients will be required to determine whether this drug, BIIB080 (/IONIS-MAPTRx), an antisense oligonucleotide (used to stop RNA from producing a protein), leads to clinical benefit, but the phase 1 results published in Nature Medicine – with results from 46 patients – are the first indication that this method has a biological effect. BIIB080 ‘silences’ the tau protein-coding gene, known as the microtubule-associated protein tau (MAPT) gene. This inhibits the gene from being dosed and reversibly translated into protein. It will also reduce the creation of that protein, changing the course of the condition.

The investigational combination therapy of extended-release pramipexole and extended-release rasagiline controlled symptoms similar to pramipexole alone in this phase 3 trial, but with less sleepiness and orthostatic hypotension. Pramipexole mimics dopamine activity in the brain, and rasagiline increases dopamine levels by blocking dopamine reuptake.