Clinical Trial News

Mark Bleackley discusses challenges in developing IHL-42X for obstructive sleep apnea (OSA), including lack of a standard for clinical trial endpoints. Adverse events observed were consistent with known effects of THC and acetazolamide, mitigated by dose reduction. Surprising findings included a nonlinear dose response in efficacy.

The Foundation Fighting Blindness, funded by the Usher III Initiative, is conducting a natural history study on Usher syndrome type 3 (USH3) caused by the N48K mutation of the CLRN1 gene. This study aims to identify potential patients for future clinical trials, understand disease progression, and optimize clinical trial designs for therapies. The 4-year study will involve up to 20 patients as part of the broader Uni-Rare natural history study, which aims to enroll up to 1,500 patients with inherited retinal diseases (IRD) across multiple sites globally.

A novel combination of low doses of three anti-hypertensive drugs in a single pill (GMRx2) was found superior to standard care in lowering blood pressure in uncontrolled hypertension patients, according to the VERONICA-Nigeria trial results presented at the ESC Congress 2024 and published in JAMA. GMRx2 showed significant reductions in systolic blood pressure and improved blood pressure control rates, with good tolerability.

Low-dose triple-pill protocol significantly reduced home systolic blood pressure by 5.8 mm Hg in a randomized trial in Nigeria among 300 Black African patients with uncontrolled hypertension, compared to standard-care protocol, with no increase in treatment discontinuation due to adverse events. This suggests that the triple-pill protocol can improve blood pressure lowering compared to standard care, potentially addressing the high burden of hypertension in sub-Saharan Africa.

A phase 1b study of abemaciclib monotherapy in heavily pretreated patients with metastatic ccRCC showed no clinically meaningful activity, with no objective responses and rapid disease progression in most patients. Despite the negative results, these findings are crucial for understanding the potential of CDK4/6 inhibitors in combination therapies for RCC, guiding future trials.

At ESC 2024, Dr. Erin Michos discussed the success of obicetrapib trials, showing its ability to reduce LDL. Completion of Phase III TANDEM trial enrollment brings NewAmsterdam closer to launching obicetrapib for HeFH/ASCVD. If successful, it could offer a new treatment for high cholesterol, addressing the unmet need for Lp(a) reduction.

CGTLive®’s Weekly Rewind covers FDA actions, research, and interviews in gene and cell therapies. Highlights include no FVIII-specific responses in hemophilia A patients, mouse research findings at ASGCT’s 2024 conference, Galpagos expanding its ATALANTA-1 trial to the US, NMPA’s decision based on the CARTIFAN-1 trial, and Dr. Mark Walters discussing gene therapy’s impact on treatment.

NXP800, a novel GCN2 kinase activator, received orphan drug designation from the FDA for ARID1a-deficient ovarian, fallopian tube, and primary peritoneal cancers. It is an oral small molecule inhibitor targeting the HSF1 pathway, showing strong antitumor effects in ARID1a-mutated ovarian carcinoma. A phase 1b clinical trial (NCT05226507) is ongoing, evaluating NXP800 in platinum-resistant, ARID1a-mutated ovarian cancers.

A study at St Vincent's University Hospital challenges the belief that weight loss medications like Ozempic, Wegovy, or Monjaro work solely by promoting satiety. The randomized controlled trial, led by Professor Donal O'Shea, found a strong relationship between the increase in metabolic activity caused by GLP-1 treatment and weight loss, with low metabolic activity prior to treatment showing the most benefit.

The SELECT trial at ESC Congress 2024 evaluated semaglutide for obesity-related HFpEF, showing reduced risk of MACEs in females with overweight/obesity and ASCVD, without diabetes, and significant reductions in body weight, waist circumference, and hsCRP with semaglutide compared to placebo, though ARR was higher in males. Semaglutide's efficacy was consistent by sex across cardiovascular, metabolic, and kidney endpoints, with comparable safety profiles between sexes.