Neurology

RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 T stem cells in patients living with amyotrophic lateral sclerosis (pwALS).

An Intermediate Expanded Access Protocol (EAP) with CNM-Au8 for Amyotrophic Lateral Sclerosis for NIH Grant RFA-NS-23-012

The primary purpose of this phase 3b study is to assess the efficacy of a modified regimen of ublituximab as measured by T1 Gadolinium (Gd)-enhancing lesions and pharmacokinetics in participants with Relapsing Multiple Sclerosis (RMS). The study consists of 2 parts: Part A is single-armed and open-label and Part B is randomized, double-blind, placebo-controlled.

This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).

The purpose of this randomized, double-blind, placebo-controlled, parallel group study is to determine the efficacy of frexalimab in delaying the disability progression and the safety up to 36 months double-blind administration of study intervention compared to placebo in male and female participants with nrSPMS (aged 18 to 60 years at the time of enrollment). People diagnosed with nrSPMS are eligible for enrollment as long as they meet all the inclusion criteria and none of the exclusion criteria. Study details include:

* This event-driven study will end when the target number of 6-month cCDP events is achieved, and the study is expected to last 43 months from randomization of the first participant to the common study end.
* The number of scheduled visits will be up to 25 (including 3 follow-up visits) with a visit frequency of every month for the first 6 months and then every 3 months.

The purpose of each study is to independently measure the annualized relapse rate (ARR) with administration of frexalimab compared to a daily oral dose of teriflunomide in male and female participants with relapsing forms of multiple sclerosis (aged 18 to 55 years at the time of enrollment). People diagnosed with relapsing forms of multiple sclerosis are eligible for enrollment as long as they meet all the inclusion criteria and none of the exclusion criteria.

Study details include:

* This event-driven study will have variable duration of approximately 40 months for the first participant being randomized and approximately 20 months for the last participant randomized.
* The study intervention duration will vary ranging from approximately 12 to 40 months.
* The assessment of scheduled visits will include 1 common end of study \[EOS\] visit and 3 follow-up visits) with a visit frequency of every 4 weeks for the first 6 months and then every 3 months.

This phase III trial tests whether continuous or intermittent zanubrutinib after achieving a complete remission (CR) with rituximab works in older adult patients with mantle cell lymphoma (MCL) who have not received treatment in the past (previously untreated). Rituximab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. Zanubrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. When zanubrutinib is used in MCL, the current standard of care is to continue administering the drug indefinitely until disease progression. This continuous treatment comes with clinical as well as financial toxicity, which could be especially detrimental in older patients. For patients who achieve a CR after initial zanubrutinib plus rituximab therapy, it may be safe and equally effective to stop treatment and restart zanubrutinib upon disease progression rather than continuing indefinitely in previously untreated older adult patients with MCL.

The purpose of this study is to assess the efficacy, safety, PK, and PD of satralizumab in participants with NMDAR and LGI1 encephalitis.