Clinical Trials
Oncologists discuss ‘magic wand’ solutions to improve clinical trial diversity
An FDA Oncology Center of Excellence program titled “Real Talk: Our Stories as Black Oncologists at the FDA” included discussion about how to increase diversity in clinical trials.
Nusinersen linked to improved motor function in spinal muscular atrophy
Treatment with nusinersen was linked to increased motor function and reduction of the neurodegenerative biomarker neurofilament light chain in children with spinal muscular atrophy, according to interim results of a study.
Viltolarsen linked to increased pulmonary function in Duchenne muscular dystrophy
Treatment with viltolarsen at 48 weeks improved pulmonary function in a cohort of pediatric patients with Duchenne muscular dystrophy amenable to exon 53 skipping compared with those receiving standard care.
Zolgensma improves motor function at 52 weeks in spinal muscular atrophy
Treatment with Zolgensma improved motor function in a cohort of children with spinal muscular atrophy, according to a poster at the 2024 MDA Clinical & Scientific Conference.
Pamrevlumab nonsuperior to placebo for boys with Duchenne muscular dystrophy
For young ambulatory boys with Duchenne muscular dystrophy, treatment with pamrevlumab was nonsuperior to placebo after 52 weeks, according to a poster presentation at the 2024 MDA Clinical & Scientific Conference.
Atogepant well-tolerated, linked to significant migraine reduction
Treatment with atogepant was safe, effective and well-tolerated over 12 weeks in those with episodic migraine who failed multiple conventional therapies, with significant reductions in mean monthly migraine days compared with placebo.
Quolet completes phase 1 study of oral, lipid-based CBD in healthy volunteers
Quolet Industries has announced the completion of a phase 1 study evaluating a novel, lipid formulation of pure CBD as a potential treatment for various psychiatric and neurologic conditions.
Biopharma firm to commence phase 2 study of oral small molecule drug for Parkinson’s
A New York-based biopharmaceutical company has received a grant from Cure Parkinson’s to initiate a phase 2 clinical trial investigating dapansutrile, an oral small molecule, as a disease-modifying therapy for Parkinson’s disease.
Dosing begun in clinical trial of novel oral chimeric protein degrader
The first subject has been dosed in a phase 1 clinical trial of an oral proteolysis-targeting chimera protein degrader to treat neurodegenerative diseases, according to the manufacturer.
Phase 3 study of agitation drug fails to achieve primary efficacy endpoint
Otsuka has announced that a phase 3 clinical trial of AVP-786, a combination therapy to treat agitation associated with dementia due to Alzheimer’s disease, failed to achieve its primary endpoints.