Clinical Trial News

Nanorobots guided by magnetic fields could revolutionize stroke prevention by sealing off brain aneurysms more safely and precisely than traditional methods. These bots, which release a clotting agent when heated, have successfully blocked aneurysms in rabbit trials without obstructing overall blood supply. Future research aims to refine this technology for larger animals and deeper brain aneurysms.

FDA advisory panel rejected MDMA-assisted therapy for PTSD due to concerns over clinical trial design, potential abuse, and misconduct allegations. April Pride, CEO of SetSet, views this as a setback for psychedelic medicine, advocating for regulatory reforms and harm-reduction education. Despite the rejection, Pride remains optimistic about the future of psychedelic medicine, expecting innovation in safer compounds and protocols.

BioLab Holdings, a Mesa medical manufacturer, has initiated clinical trials for an FDA-approved amniotic membrane tissue to treat difficult-to-heal venous leg ulcers. The trials aim to assess the product's efficacy and expand treatment options for chronic wounds, which affect 2.5% of the U.S. population and 10.5 million Medicare beneficiaries.

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.