Clinical Trial News

A monocentric clinical study on Pompe disease (PD) was conducted at University Hospital Erlangen (UHE) and University Medical Center Göttingen (UMG), involving 18+ PD patients and healthy volunteers (HV) matched by age and sex. The study included clinical assessments, ultrasound, MSOT, and MRI imaging. MSOT imaging used a hybrid system capable of acquiring interleaved US images for coregistration, with data analyzed using viewMSOT software. The study aimed to compare PD patients with HV through various imaging techniques and clinical assessments, including lung function tests and muscle strength evaluations.

A Canadian family's relentless quest for a cure for their son's rare disease, SPG50, led to groundbreaking gene therapy treatment in Spain, significantly improving his condition and paving the way for others with the same condition.

A review of 21 studies on SMA therapies (Spinraza, Evrysdi, Zolgensma) found motor function improvement in 1,374 patients over 48 months, with early intervention yielding better outcomes. Safety profiles were favorable, but no significant respiratory or nutritional improvements were noted. The high cost and uncertain long-term benefits pose challenges for coverage decisions.

Asciminib, a BCR::ABL1 inhibitor targeting the ABL myristoyl pocket, shows efficacy against CML-CP, including T315I mutation, with 40 mg b.i.d. and 200 mg b.i.d. dosing regimens. An 80 mg q.d. regimen is proposed for improved patient compliance, supported by model-informed drug development, demonstrating similar efficacy and safety profiles to 40 mg b.i.d.

LEVI-04, a novel neurotrophin-3 inhibitor, demonstrated a 50% reduction in pain for osteoarthritis patients with no adverse effects, in a Phase II study.

I-DXd, a B7-H3–directed antibody–drug conjugate, showed clinically meaningful responses in pretreated ES-SCLC patients, with a confirmed ORR of 54.8% at 12 mg/kg and 26.1% at 8 mg/kg doses. The 12-mg/kg dose was selected as optimal for further study.

French researchers developed a hydrogel delivery system for semaglutide, potentially reducing dosing to once a month, easing adherence for type 2 diabetes and obesity patients. The hydrogel, tested in vitro and on rats, showed sustained release over 1-3 months with no adverse reactions, paving the way for future pig trials and potential clinical trials.

Merck and Daiichi Sankyo announced interim Phase 2 trial results for lung cancer treatment ifinatamab deruxtecan (I-DXd), showing objective response rates of ~55% and ~26% at 12 mg/kg and 8 mg/kg doses, respectively. The 12 mg/kg dose led to 23 partial responses, while the 8 mg/kg dose resulted in one complete and 11 partial responses. Median overall survival was 11.8 months and 9.4 months for the respective doses. Preliminary intracranial ORRs were 50.0% and 66.7% across the two dose groups. Adverse events led to 17% and 6.5% dropouts for the respective doses. The 12 mg/kg dose is selected for the trial's expansion part, with data to be presented at the World Conference on Lung Cancer in San Diego.

Prevent Blindness partners with IAPB for World Sight Day focusing on children's vision, hosting events and launching a tool kit with the National Association of School Nurses. The FDA grants Orphan Drug Designation to SKG1108 for retinitis pigmentosa treatment. InMed Pharmaceuticals secures patents for an ocular drug delivery formulation for AMD. A study validates the accuracy of mobile ophthalmology applications like Eye Handbook for vision assessment.

Lisocabtagene maraleucel (liso-cel) improved response outcomes in relapsed/refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) patients, with consistent results across high-risk features. Lower tumor burden and fewer prior treatments correlated with better responses. The objective response rate (ORR) and complete response (CR) rates varied by disease characteristics, but overall, liso-cel showed efficacy even in high-risk patients. Inflammatory markers, bulky disease, and lower creatine clearance rates increased the risk of neurological toxicity.