Clinical Trials
Positive topline data reported in phase 2b trial of oral, fixed-dose ALS drug
A company specializing in treatment for neurodegenerative diseases announced positive topline data from a phase 2b clinical trial of a novel, extended release oral fixed-dose therapeutic to treat amyotrophic lateral sclerosis.
Most multiple myeloma studies do not address 'elephant in the room'
Improving representation of African American patients in multiple myeloma research is essential to preventing outcome disparities in this population, according to a spotlight session at ASH Annual Meeting and Exposition.
FDA, BrainStorm confer regarding investigational ALS treatment
The FDA has conferred with BrainStorm Cell Therapeutics Inc. regarding NurOwn, an investigational technology platform developed to treat amyotrophic lateral sclerosis.
‘Multi-pronged approach’ needed to increase diversity in clinical trials
In a special scientific session on race and science at ASH Annual Meeting an Exposition, Saad Z. Usmani, MD, MBA, FACP, FRCP, examined clinical trial inclusion from an investigator’s perspective.
Clinical trials for rare diseases need new, specific methodological approaches
ROME — The total estimated costs from inherited retinal diseases in the United States amounts to $31.8 billion. And yet, research barriers slow down the progress toward innovative treatment pathways, such as gene and cell therapies.
Rett syndrome gene therapy trial expanded to include patients aged 12 years and older
Health Canada has authorized an amendment to the ongoing REVEAL phase 1/2 trial evaluating an AAV9 investigational gene transfer therapy that expands eligibility to patients with Rett syndrome aged 12 years and older.
Joint statement calls for FDA to require drug trials to include people with obesity
Five obesity advocacy organization have issued a joint statement asking the FDA to mandate that all drug approval trials include people with obesity.
First patient enrolled in phase 2b trial of novel therapy for mild Alzheimer’s
The first patient has been enrolled in a European phase 2b clinical trial for AD04, a novel immunotherapy for the treatment of mild Alzheimer’s disease, according to the manufacturer.
Positive interim results announced in phase 2 trial of leukoencephalopathy drug
Vigil Neuroscience Inc. has announced positive interim data from a phase 2 proof-of-concept clinical trial of a monoclonal antibody to treat those suffering from adult-onset leukoencephalopathy with axonal spheroids and pigmented glia.
First patient dosed in phase 2 trial of novel therapy for early Parkinson’s disease
A precision therapeutics company announced that the first participant has been dosed in a phase 2 clinical trial of a non-dopamine selective modulator to treat those with early-stage Parkinson’s disease.