clinical trials

Tecfidera (dimethyl fumarate) demonstrated strong efficacy in Japanese and other East Asian patients with relapsing-remitting multiple sclerosis (RRMS), a Phase 3 clinical trial shows. These results are consistent with previous clinical trials, which included mostly white MS patients, and show that Tecfidera can also be effective across various other patient demographics. Findings of the trial were reported in the study, “A randomized placebo-controlled trial of delayed-release dimethyl fumarate in patients with relapsing-remitting multiple sclerosis from East Asia and other countries,” in the journal BMC Neurology. Tecfidera, a delayed-release dimethyl fumarate capsule, marketed by Biogen, is an oral therapy approved in many parts of the world for the treatment of RRMS. In previous Phase 3 clinical studies, DEFINE (NCT00420212), and CONFIRM (NCT00451451), Tecfidera showed substantial effectiveness on clinical and neuroradiological measures in RRMS patients. The study participants were predominantly white (79% in DEFINE and 84% in CONFIRM), and there were 10% or fewer East Asian patients. In general, very little data is available on Tecfidera's effectiveness in East Asian MS patients. In this APEX Part 1 (NCT01838668) trial, researchers from the Kansai Medical University in Japan and Biogen evaluated the safety and efficacy of Tecfidera over 24 weeks (six months) in the treatment of RRMS patients from East Asia and other countries. Participants with active MS between the ages of 18 and 55, with ethnic origins in Japan, South Korea, or Taiwan were included. To compare East Asian and white MS patients, study enrollment was expanded to patients from Eastern Europe (Czech Republic and Poland). In all, the six-month, double-blind, placebo-controlled study recruited 225 patients, 142 of whom were East Asian (63.4%). It was completed by 213 participants. Patients were randomly assigned to receive Tecfidera, (240 mg, twice daily) or a matching placebo for six months. They were assessed at the beginning of the study, at three months, and again at six months. They underwent MRI (magnetic resonance imaging) scans for neurological examination, in addition to routine health checks. The primary objective of the study was the total number of new inflammatory lesions on brain MRI scans from three to six months. Secondary goals included the number of specific new, or newly enlarging T2 hyperintense lesions — lesions reflective of damage to nerve cell connections — from the beginning of the study to six months. Tertiary goals included standard safety measurements, and annualized relapse rate over six months. “We chose radiological measures to serve as primary and secondary endpoints, due to the ability of MRI to detect lesions that might not produce clinical manifestations in the short-term,” the researchers wrote. Results showed that Tecfidera treatment significantly reduced (84%) the total number of new MRI lesions from weeks 12 to 24 (primary objective), compared with placebo — specifically by 85% in the Japanese subgroup, 81% in the total East Asian subgroup, and 87% in the Eastern European subgroup. Regarding the trial's secondary objective, the total number of new MRI lesions from the beginning of the study up to six months was reduced by 75% in the Tecfidera group (78% in the Japanese, 76% in the East Asian, and 73% in the Eastern European subgroups), and the mean number of new/newly enlarging T2 hyperintense lesions was reduced by 63% in the Japanese, and 58% in the East Asian subgroups, compared with placebo. Most patients reported one or more adverse events (77% in the placebo group and 86% in the Tecfidera group). Most adverse events were mild or moderate in severity, and the ones affecting patients taking Tecfidera either related mainly to flushing symptoms or to gastrointestinal problems. The team concluded that the "results suggest that the strong efficacy and favorable benefit-risk profile of [Tecfidera] extends to Japanese and other East Asian patients with MS." The second part of the ongoing clinical trial, APEX Part 2, is an open-label extension trial — where both the researchers and participants know which treatment they are getting — designed to further examine the long-term safety and tolerability of Tecfidera in East Asian MS patients.

BrainStorm Cell Therapeutics announced that the production of its therapy NurOwn will be expanded to support upcoming clinical trials, namely a Phase 3 trial in amyotrophic lateral sclerosis (ALS) and a Phase 2 trial in progressive multiple sclerosis (MS). BrainStorm’s proprietary, stem cell-based technology called…

Biogen announced the start of a global Phase 3b clinical trial to evaluate the efficacy and safety of extended interval dosing (EID) with Tysabri (natalizumab) in patients with relapsing-remitting multiple sclerosis (RRMS). Results of the six-week dosing interval will be compared with the approved standard interval dosing…

Because multiple sclerosis (MS) presentation and progression course can be very different between people of African ancestry and Caucasians, the recruitment of minorities to Phase 3 clinical trials is of particular importance. Researchers in the MS field and the general MS community should make a greater effort to improve…

Treatment with autologous hematopoietic stem cell transplant (aHSCT) led to a sustained decrease in disability and almost no clinical relapses in patients with relapsing-remitting multiple sclerosis (RRMS) who had failed to respond to prior immunosuppressive therapies, an Australian Phase 2 trial shows. Trial findings were published in the study, “Prospective phase…

OWC Pharmaceutical Research is planning new clinical studies to evaluate the activity and safety of its cannabinoid-enriched sublingual soluble tablet. The company announced that an Institutional Review Board (IRB) in Israel approved its request to conduct a safety and tolerability trial there. The company also is asking to amend the…

Treatment with Ampyra (dalfampridine) for 24 weeks leads to sustained and clinically meaningful improvements in walking ability as reported by multiple sclerosis (MS) patients with gait difficulties, according to a study analyzing results from a Phase 3 trial. The study, “Assessment of Clinically Meaningful Improvements in Self-Reported…

The U.S. Food and Drug Administration (FDA) has approved BrainStorm Cell Therapeutics‘ request to open a Phase 2 clinical trial testing the safety and effectiveness of its proprietary NurOwn mesenchymal stem cell (MSC) treatment in progressive multiple sclerosis (MS) patients. The request was in the form of Investigational New Drug…

Lipoic acid (LA), an over-the-counter antioxidant supplement, helps to mildly delay a decline in walking speed in secondary progressive multiple sclerosis (SPMS) patients, particularly those with lesser disability, analysis of a small, two-year clinical study reports. No improvements were seen in balance among…

Artificial Intelligence May Help Reduce Dosage of Gadolinium in MRIs, Researchers Suggest There’s been concern recently about the accumulation of gadolinium in the brains of people who’ve had MRIs. Gadolinium is the dye that’s used to provide the “contrast” that can highlight lesions in the brain that might…

Atara Biotherapeutics’ investigational ATA190, a cell therapy that wipes out immune B-cells infected with the Epstein-Barr virus (EBV), led to neurological improvements and reduced symptoms in patients with primary and secondary progressive multiple sclerosis (MS), a Phase 1 trial shows. The trial results were published in the Journal…

BrainStorm Cell Therapeutics is planning to launch a Phase 2 clinical trial in the United States to evaluate the safety and activity of its lead cell therapy candidate, NurOwn, in people with progressive multiple sclerosis (MS). The company announced that has submitted an Investigational New Drug (IND) application to…

Treating primary progressive multiple sclerosis (PPMS) patients with Ocrevus (ocrelizumab) can help to preserve strength and function in their hands and the arms, analysis of data from a Phase 3 trial found. The research, “Ocrelizumab reduces progression of upper extremity impairment in patients with primary progressive…

A Phase 3 clinical trial intending to confirm the potential of MD1003, a high-dose biotin, in treating progressive multiple sclerosis (MS) patients is completely enrolled, MedDay Pharmaceuticals, the investigational therapy’s developer, announced. The randomized, double-blind, and placebo-controlled study (NCT02936037), called SPI2, follows the previous Phase 3 trial (NCT02220933),…

Fast Forward, a nonprofit subsidiary of the National Multiple Sclerosis Society, will invest up to $330,000 to advance the clinical development of an antibody that was shown to lessen inflammation and nerve cell damage in a multiple sclerosis (MS) mouse model. The funding will help develop the antibody (a protein…

Treatment with autologous hematopoietic stem cell transplant (aHSCT) led to a safe and rapid lessening of disability and no clinical relapses in patients with aggressive multiple sclerosis (MS), according to a new study. The research, “The use of autologous hematopoietic stem cell transplantation as a…

Merck KGaA announced that evobrutinib, its oral candidate for relapsing multiple sclerosis (MS), was able to safely and significantly reduce active brain lesions over 24 weeks of treatment, according to results of a Phase 2 study sponsored by the company.

Additional analysis of clinical data from Celgene’s investigational agent ozanimod continues to demonstrate its potential to benefit patients with relapsing multiple sclerosis (MS). Findings from the Phase 3 SUNBEAM and RADIANCE Part B trials were discussed at the 34th Congress of the European Committee for Treatment and…

Data from two European trials — a real-life study in Italy, and a long-term Czech trial looking at impact on cognition — supported the efficacy of a widely approved cannabinoid spray in treating moderate to severe spasticity (muscular stiffness or involuntary spasms) in multiple sclerosis (MS) patients who…

Treatment of multiple sclerosis (MS) patients with rituximab leads to lower-than-reported mortality rates, according to a large real-world study in the U.S. and Sweden. The findings also revealed no deaths due to infusion reactions or to systemic inflammation. The study, “Mortality rates in large US and…

Patients with relapsing-remitting multiple sclerosis (RRMS) who switch to Tysabri (natalizumab) after relapses on first-line treatment with other medications show greater relapse reduction and less disability progression than those switching to Gilenya (fingolimod), according to a real-world study. The research, “Comparative effectiveness of switching…

The Accelerated Cure Project (ACP) for Multiple Sclerosis and EMD Serono— a unit of Merck KGaA —  will jointly incorporate the perspectives of U.S. and Canadian multiple sclerosis (MS) patients into the design and implementation of clinical trials. The ACP, a nonprofit group based in Waltham, Massachusetts, strives to accelerate…

Clinical data and real-world results support the long-term efficacy of Biogen’s medications for multiple sclerosis (MS), according to scientific presentations being released by the company. Specifically, findings support the effectiveness of Tecfidera (dimethyl fumarate) and Tysabri (natalizumab) used early in the disease’s course, as well as the…

A small group of multiple sclerosis (MS) patients with aggressive disease, who were treated with hematopoietic stem cell transplant in a clinical trial, reported a drop in their fatigue levels that researchers suggested was likely due to lesser inflammation. The study, “Autologous hematopoietic stem cell transplantation improves…

Emerald Health Pharmaceuticals announced that it has begun enrolling healthy volunteers for a Phase 1 clinical trial evaluating the safety and tolerability of EHP-101, a potential cannabidiol treatment for multiple sclerosis (MS) and scleroderma. The randomized, double-blind, and placebo-controlled study (ACTRN12618001390279p) will  investigate the safety and pharmacokinetics (how…

A 48-week treatment of relapsing multiple sclerosis (MS) with TG Therapeutics’ investigational compound ublituximab led to a marked reduction of brain and spinal cord lesions, massive depletion of relapse-associated immune B-cells, and significantly halted disability progression, according to results from a Phase 2 clinical trial. The data…

Treatment with Rapacan (rapamycin) decreased the size and volume of brain lesions in patients with multiple sclerosis (MS), an Iranian study reports. The study, “Promising effect of rapamycin on multiple sclerosis,” was published in the journal Multiple Sclerosis and Related Disorders. Rapamycin, or sirolimus, is an immunosuppressive…

GeNeuro announced it has reacquired from Servier the worldwide rights to commercialize and develop the investigational humanized antibody GNbAC1 for the treatment of multiple sclerosis (MS). The decision came after Servier, a European company which, together with GeNeuro, developed the GNbAC1 program, declined to continue developing the therapy due to…

Progressive multiple sclerosis patients — with primary or secondary progressive disease — treated with high doses of oral ibudilast in a Phase 2 clinical trial showed a 48 percent slowing in the progression of brain atrophy, or shrinkage, relative to those given a placebo, study data show. What this…

Two years of treatment with oral Gilenya (fingolimod) significantly reduced the rate of relapses when compared to Avonex (interferon beta-1a) intramuscular injections in children and adolescents with relapsing forms of multiple sclerosis (RMS), according to Phase 3 clinical trial results. Additionally, Gilenya (marketed by Novartis) decreased the number of central nervous…