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Clinical Trial News

Study Finds No Short-Term Benefit of Adding Revascularization to Medical Therapy for Carotid Stenosis

  • A multicenter randomized trial reveals no advantage in adding revascularization to optimized medical therapy for patients with carotid stenosis in the first two years of treatment.
  • The study included patients with both asymptomatic and symptomatic carotid stenosis of 50% or greater who had a five-year predicted ipsilateral stroke risk below 20%.
  • Researchers are continuing follow-up to five years after randomization to determine if revascularization might provide benefits over a longer timeframe.

SciSparc Advances Cannabinoid-Based Therapies for CNS Disorders, Including Autism

  • SciSparc has enrolled the first patient in its clinical trial for SCI-210, a cannabinoid-based treatment for Autism Spectrum Disorder (ASD) in children.
  • The company's pipeline includes SCI-110 for Tourette Syndrome and Alzheimer's agitation, and SCI-160 for pain management, all leveraging cannabinoid pharmaceuticals.
  • SciSparc has expanded its intellectual property portfolio with recent patents in the U.S., Canada, and Europe, enhancing its competitive position.
  • Through MitoCareX Bio, SciSparc is also involved in cancer drug discovery, targeting mitochondrial SLC25 proteins.

Organon Expands Dermatology Portfolio with $1.2 Billion Acquisition of Dermavant

• Organon is set to acquire Dermavant for up to $1.2 billion, gaining Vtama (tapinarof) cream, a commercialized plaque psoriasis drug. • Vtama, a non-steroidal topical cream, is under FDA review for atopic dermatitis, with a decision expected in Q4, potentially triggering a $75 million milestone payment. • Organon aims to leverage its global reach to maximize Vtama's commercial potential, combining Dermavant's U.S. presence with Organon's market access and regulatory expertise. • Roivant's sale of Dermavant aligns with its strategy to focus on specialty drug candidates with blockbuster potential, following previous sales and new subsidiary launches.

Subclinical HSV-1 Reactivation Common in Solid Organ Transplant Recipients

  • A study of solid organ transplant (SOT) recipients found frequent subclinical Herpes Simplex Virus-1 (HSV-1) reactivation, highlighting potential risks.
  • Researchers observed that 80% of SOT recipients experienced HSV-1 shedding, with most episodes occurring without visible lesions.
  • Longer shedding episodes correlated with higher viral titers, suggesting a potential link to adverse patient outcomes post-transplantation.
  • The findings underscore the need for further investigation into the impact of subclinical HSV-1 reactivation in immunocompromised individuals.

Stirred-Tank Bioreactors Enhance CAR-T Cell Efficacy in Tumor Microenvironments

• A novel CAR-T cell manufacturing process using stirred-tank bioreactors (STBs) demonstrates improved efficacy in simulated tumor microenvironments. • STB-produced CAR-T cells exhibit a higher proportion of CD8 T cells, crucial for directly killing tumor cells, compared to static cultures. • Lower CD4/CD8 ratios in STB-manufactured CAR-T cells correlate with enhanced patient responsiveness to CAR-T cell treatment. • The optimized bioprocessing method shows potential for improving CAR-T cell therapies targeting HER2-positive breast cancer.

FDA to Decide on Govorestat for Classic Galactosemia by November 28 After Advisory Committee Not Required

  • The FDA has completed its late-cycle review meeting for govorestat, a treatment for Classic Galactosemia, and an advisory committee meeting will not be required.
  • The Prescription Drug User Fee Act (PDUFA) target action date for govorestat remains on track for November 28, 2024, indicating a decision is expected by then.
  • Govorestat, a CNS penetrant Aldose Reductase Inhibitor, has shown clinical benefits in children with Galactosemia, improving daily living activities and reducing plasma galactitol levels.
  • Applied Therapeutics is preparing for the potential approval of govorestat with a focus on patient access, physician awareness, and payor engagement.

BRIUMVI Rapid Infusion Shows Promise in Multiple Sclerosis Treatment

  • Phase 3b ENHANCE study reveals that 30-minute infusions of BRIUMVI (ublituximab-xiiy) are well-tolerated in patients with relapsing forms of multiple sclerosis (RMS).
  • Data suggest patients switching from prior anti-CD20 therapies can eliminate the initial BRIUMVI infusion, streamlining the treatment process.
  • Rapid infusions were completed without interruptions, with infusion-related reactions being mild (Grade 1) and resolving completely, enhancing patient convenience.
  • These findings support ongoing efforts to optimize BRIUMVI dosing regimens, potentially leading to label-enabling studies and improved patient outcomes.

Study to Assess the Efficacy and Safety of Ublituximab in Participants With Relapsing Forms of Multiple Sclerosis (RMS) ( ULTIMATE 1 )

NCT03277261CompletedPhase 3
TG Therapeutics, Inc.
Posted 9/19/2017

Study to Assess the Efficacy and Safety of Ublituximab in Participants With Relapsing Forms of Multiple Sclerosis (RMS)

NCT03277248CompletedPhase 3
TG Therapeutics, Inc.
Posted 8/25/2017

Govorestat NDA for Classic Galactosemia Faces No FDA Advisory Committee, PDUFA Date on Track

  • Applied Therapeutics' govorestat, a CNS penetrant Aldose Reductase Inhibitor, is under NDA review for treating Classic Galactosemia, a rare metabolic disorder.
  • The FDA has communicated that an Advisory Committee meeting, previously scheduled for October 9, 2024, will no longer be required for the govorestat NDA review.
  • The FDA's Priority Review of the govorestat NDA is proceeding as planned, with alignment on post-marketing requirements expected in October 2024.
  • The PDUFA target action date for the FDA's decision on govorestat remains on track for November 28, 2024, signaling potential approval by the end of the year.

Pomalidomide Shows Promise in Reducing Bleeding for Hereditary Hemorrhagic Telangiectasia

  • A Phase II clinical trial (PATH-HHT) demonstrated that pomalidomide significantly reduces the severity of nosebleeds in patients with hereditary hemorrhagic telangiectasia (HHT).
  • The study, published in The New England Journal of Medicine, showed a notable improvement in the HHT-specific quality of life for patients treated with pomalidomide.
  • Pomalidomide was generally well-tolerated, although some patients experienced side effects such as neutropenia, constipation, and rash, leading to dose adjustments.
  • These findings suggest pomalidomide may offer a new therapeutic avenue for HHT, a bleeding disorder with limited FDA-approved treatment options.

Pomalidomide for the Treatment of Bleeding in HHT

NCT03910244CompletedPhase 2
The Cleveland Clinic
Posted 10/17/2019

Lenvatinib Plus Pembrolizumab Demonstrates Significant Antitumor Activity in Advanced Endometrial Cancer

  • Lenvatinib plus pembrolizumab shows substantial antitumor activity in patients with stage III/IV recurrent endometrial carcinoma, according to the ENGOT-en9/LEAP-001 study.
  • The combination therapy achieved an objective response rate of 55.7% in all patients and 50.6% in the proficient mismatch repair (pMMR) population.
  • Median duration of response was 16.1 months for all-comers and 23.2 months for the pMMR population, indicating deep and durable responses.
  • Adverse events were manageable with dose modifications and supportive care, supporting the clinical utility of this combination in advanced endometrial cancer.

Pembrolizumab (MK-3475) Plus Lenvatinib (E7080/MK-7902) Versus Chemotherapy for Endometrial Carcinoma (ENGOT-en9 / MK-7902-001)

NCT03884101CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 4/11/2019

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