Clinical Trials

A nonprofit focused on treatments and cures for Charcot-Marie-Tooth disease will partner with a biotechnology firm to advance a novel intrathecal miRNA therapy to address CMT type 1a in adults with the condition.

In adults with epilepsy, treatment with a novel, oral adjunctive therapeutic significantly reduced monthly occurrence of focal-onset seizures across three doses compared with placebo, according to research in JAMA Neurology.

The FDA has cleared an investigational new drug application for AVB-101 to treat those with frontotemporal dementia with mutations in the progranulin gene, according to a release from the manufacturer.

Nido Biosciences has announced positive results from a phase 1 study of its novel, small molecule therapeutic to treat spinal and bulbar muscular atrophy in healthy male participants.

Epion Therapeutics has initiated its phase 3 Apricity trials for EpiSmart, a minimally invasive drug-device combination indicated for epithelium-on cross-linking in the treatment of keratoconus.

Annovis Bio has been permitted by the Data and Safety Monitoring Board to continue its phase 2/3 trial of buntanetap, an oral protein inhibitor for moderate to mild Alzheimer’s disease, according to a company release.

Participation in memory-based digital cognitive testing and blood-based biomarker assays for Alzheimer’s-related pathology was linked to successful enrollment into clinical trials for disease-modifying treatment, according to research.

Compared with placebo, donanemab slowed disease progression and reduced amyloid presence at 76 weeks for carriers and noncarriers of the APOE ε4 allele in early symptomatic Alzheimer’s disease, according to data presented at CTAD.

Greater travel time appeared associated with reduced participation in genotype-matched clinical trials among patients with cancer who underwent comprehensive genomic profiling, according to results of a study conducted in Japan.

For those with relapsing-remitting MS, treatment with diroximel fumarate was linked to better outcomes at 96 weeks compared with those given interferon beta-1a, according to a presentation at ECTRIMS 2023.