Hopkins

Researchers are evaluating the efficacy, safety, and tolerability of CYB003, a deuterated psilocin analog, as an additional treatment for adults with Major Depressive Disorder (MDD). This Phase III, multi-center, double-blind, randomized controlled study compares two active doses of CYB003 against a placebo in patients experiencing moderate to severe depression who have not adequately responded to stable antidepressant treatment. Participants will receive either one of two doses of CYB003 or a placebo, along with manualized psychological support provided by a facilitator. The study includes a screening period, a dosing period, and follow-up assessments. The psychological support sessions are standardized to assist participants during the trial. During the study, participants will be assessed using the Montgomery-Asberg Depression Scale (MADRS) at multiple time points including screening, baseline, and several days during treatment up to the trial's end at Day 84. Researchers will monitor symptoms of depression, safety, and tolerability throughout the trial. Participants will also undergo various evaluations to ensure adherence and safety during the study period, which spans approximately 12 weeks from screening through the final assessment.

Researchers are evaluating the safety, tolerability, and effectiveness of VLS-01 buccal film (VLS-01-BU) as a short-term treatment for adults with treatment resistant Major Depressive Disorder (TRD). This Phase 2, multicenter, double-blind, randomized, placebo-controlled trial aims to compare antidepressant effects of VLS-01-BU against placebo, focusing on the onset and durability of these effects. About 142 participants with TRD will be randomly assigned in equal groups to receive two doses of either VLS-01-BU or placebo via buccal transmucosal administration, spaced two weeks apart. Following this placebo-controlled period, symptoms will be monitored for 12 weeks. Then, all participants will be re-randomized to receive a single additional double-blind dose of VLS-01-BU at one of two dose strengths during a non-placebo-controlled treatment phase. Safety and efficacy will be assessed two weeks after the third dose. Participants will be closely monitored throughout the study, including during the 12-week follow-up after the second dose and after the final treatment. Researchers will measure changes in depression severity using the Montgomery-Åsberg Depression Rating Scale (MADRS) from baseline to Day 29. Safety evaluations and tolerability assessments will also be conducted to understand the effects and duration of VLS-01-BU treatment.

Researchers are studying the effects of diroximel fumarate (DRF), also known as VUMERITY4, when taken during pregnancy by people with multiple sclerosis (MS). MS is a condition where the immune system attacks nerves in the brain and spinal cord, causing damage that affects body function. The study is observational and aims to collect health information from three groups: those who took DRF during pregnancy, those who took other MS drugs but not DRF, and those who did not take any MS drugs during pregnancy. The main focus is to find out how many babies are born with major congenital malformations (MCMs), which are serious birth defects. Participants include people with relapsing forms of MS who become pregnant between October 29, 2019, and July 31, 2030. The study gathers information without changing medical care and compares pregnancy and birth outcomes among the groups. Researchers will monitor important outcomes such as loss of the baby before and after 20 weeks of pregnancy, premature births, babies smaller than expected for their age, and live births. Participants remain in the study until the end of their pregnancy, with their babies followed for up to one year after birth. Medical records will be reviewed twice: halfway through and at the end of the study, which is planned to finish by April 30, 2031. The main outcome measured is the number of major congenital malformations in babies up to 52 weeks after birth, along with other pregnancy and birth health measures.

Researchers are conducting a retrospective, observational, longitudinal study across multiple countries to gather real-world information about patients with ATTR amyloidosis. The study aims to describe patient characteristics such as demographics, family history, key comorbidities, and humanistic outcomes including quality of life, neuropathy impairment, and activities of daily living. It also seeks to understand treatment patterns and disease outcomes, as well as healthcare resource use before and after diagnosis. This study involves analyzing existing data from patients diagnosed with ATTR amyloidosis without introducing any interventions. Patients will be followed from diagnosis throughout the study period to assess various clinical and economic outcomes. No treatments or procedures are administered as part of this study, as it focuses solely on observing and collecting secondary data. Participants' health care use will be evaluated, including outpatient visits, emergency department visits, hospitalizations, and transplants, up to 12 years post-diagnosis. Researchers will also assess clinical measures such as neuropathy impairment scores and new disease manifestations. The study will track mortality and healthcare costs to better understand the disease journey and its impact over time.