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Clinical Trial News

RSV Vaccine Development Surges with New Approvals and Diverse Strategies

  • Recent advancements in understanding hRSV structure and immune responses have led to new vaccine approvals targeting older adults, pregnant women, and infants.
  • Several vaccine candidates are in clinical trials, employing live-attenuated, recombinant-vector, subunit, particle-based, and mRNA approaches to stimulate immunity.
  • Monoclonal antibody treatments like Nirsevimab offer immediate protection for infants, complementing vaccine strategies and addressing the high disease burden of hRSV.
  • Combination vaccines and mRNA modalities are being explored to broaden protection and improve vaccine delivery, though challenges remain in optimizing these new approaches.

Ultrasound-Localized Tuina and Nerve Blocks Show Promise for Cervicogenic Headache

  • A randomized controlled trial is set to investigate the efficacy of combining ultrasound-localized meridian sinew tuina with greater and third occipital nerve injections for cervicogenic headache (CEH).
  • The study will compare this integrative approach to ultrasound-guided nerve injections alone, assessing improvements in pain scores, headache intensity, and cervical spine mobility.
  • Medical infrared thermography will be used as an objective tool to evaluate treatment efficacy by measuring temperature variations related to nerve compression and inflammation.
  • This trial aims to provide a safe and effective manual therapy protocol, offering insights for future research and clinical management of CEH, a prevalent and challenging headache disorder.

Tolebrutinib Shows Promise in Slowing Disability Progression in Non-Relapsing Secondary Progressive MS

  • Tolebrutinib significantly delayed the onset of 6-month confirmed disability progression by 31% compared to placebo in patients with nrSPMS.
  • The proportion of patients experiencing confirmed disability improvement nearly doubled with tolebrutinib compared to placebo, indicating potential for functional gains.
  • Safety data showed liver enzyme elevations as a notable adverse event, but most cases resolved without intervention, emphasizing the need for careful monitoring.
  • HERCULES trial results support tolebrutinib as a potential treatment for nrSPMS, addressing a significant unmet need in managing disability accumulation.

Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 1)

NCT04410978CompletedPhase 3
Sanofi
Posted 6/30/2020

Nonrelapsing Secondary Progressive Multiple Sclerosis (NRSPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168)

NCT04411641CompletedPhase 3
Sanofi
Posted 9/24/2020

Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 2)

NCT04410991CompletedPhase 3
Sanofi
Posted 6/11/2020

Primary Progressive Multiple Sclerosis (PPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (PERSEUS)

NCT04458051Active, Not RecruitingPhase 3
Sanofi
Posted 8/13/2020

A Study to Investigate the Efficacy of Fenebrutinib in Relapsing Multiple Sclerosis (RMS)

NCT05119569Active, Not RecruitingPhase 2
Hoffmann-La Roche
Posted 3/1/2022

Arrowhead's Plozasiran Receives FDA Breakthrough Therapy Designation for Familial Chylomicronemia Syndrome

  • Arrowhead Pharmaceuticals' plozasiran, an investigational RNAi therapeutic, has received Breakthrough Therapy Designation from the FDA for familial chylomicronemia syndrome (FCS).
  • Clinical trials showed plozasiran significantly reduced triglycerides by 80% and decreased the risk of acute pancreatitis by 83% in FCS patients.
  • Arrowhead plans to submit a New Drug Application to the FDA by the end of 2024, seeking approval for plozasiran as a treatment for FCS, where currently no approved therapies exist.
  • The FDA's designation aims to expedite the development and review process, potentially bringing a new treatment option to patients with this rare genetic disease.

Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)

NCT05089084Active, Not RecruitingPhase 3
Arrowhead Pharmaceuticals
Posted 1/11/2022

Innovent's Mazdutide Shows Superior Efficacy in Type 2 Diabetes and Obesity Trials

  • Mazdutide demonstrated superior blood sugar control and weight loss compared to dulaglutide in the Phase 3 DREAMS-2 trial for type 2 diabetes.
  • In the GLORY-1 trial, mazdutide led to significant weight reduction in overweight or obese patients, with over 50% achieving at least a 15% body weight reduction.
  • Mazdutide also showed improvements in cardiometabolic risk factors, including blood pressure, lipids, and liver fat content, across multiple studies.
  • Innovent has submitted two New Drug Applications (NDAs) to China's NMPA for mazdutide for chronic weight management and glycemic control.

A Study of IBI362 in Participants With Type 2 Diabetes

NCT05606913CompletedPhase 3
Innovent Biologics (Suzhou) Co. Ltd.
Posted 1/6/2023

A Study of IBI362 in Participants With Obesity or Overweight

NCT05607680CompletedPhase 3
Innovent Biologics (Suzhou) Co. Ltd.
Posted 11/14/2022

Toripalimab Shows Promise in Advanced Solid Tumors with POLE/POLD1 Mutations

  • A Phase II trial of toripalimab shows an overall response rate of 21.4% in advanced solid tumors with polymerase epsilon (POLE) or POLD1 mutations.
  • Patients with POLE exonuclease domain mutations (EDMs) demonstrated a higher response rate of 66.7% compared to non-EDM mutations.
  • Co-mutation of PBRM1 with POLE/POLD1 was associated with higher tumor mutational burden (TMB) and improved immunotherapy response signatures.
  • Monitoring circulating tumor DNA (ctDNA) dynamics, specifically POLE variant allele frequency (VAF), may provide insights into tumor progression during treatment.

Stepped Care Model Shows Promise in Enhancing Adherence to Internet-Based CBT for Depression

  • A recent study investigated the efficacy of a stepped care model combined with internet-based cognitive behavioral therapy (i-CBT) for adults with major depressive disorder (MDD).
  • The research found that while i-CBT effectively reduced depressive symptoms, the addition of stepped care did not significantly improve symptom reduction compared to i-CBT alone.
  • Participants in the stepped care group completed significantly more therapy sessions on average, suggesting the model's potential to improve treatment adherence.
  • The study highlights the need for further research to explore specific stepped care interventions and their long-term effects on diverse populations with depression.

Incorporating Stepped Care Approach Into e-CBT for Depression

NCT04747873CompletedNot Applicable
Dr. Nazanin Alavi
Posted 4/7/2021

Roche's PiaSky Approved in EU as First Monthly Self-Administered Treatment for Paroxysmal Nocturnal Haemoglobinuria

Monoclonal AntibodyRare DiseaseDrug Approval
  • The European Commission has approved PiaSky (crovalimab), the first monthly subcutaneous treatment for paroxysmal nocturnal haemoglobinuria (PNH), offering patients the option to self-administer following training.
  • PiaSky utilizes innovative recycling antibody technology developed by Chugai Pharmaceutical, allowing it to bind to C5 protein multiple times and remain effective longer with a small volume of medicine.
  • Clinical trials demonstrated PiaSky's non-inferiority to eculizumab, the current standard of care, while potentially reducing treatment burden through less frequent administration and eliminating the need for regular clinic visits.

A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors

NCT04432584Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 9/30/2020

A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

NCT04861259Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 10/22/2021

A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

NCT04958265Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 11/17/2021

A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors

NCT04434092Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 10/8/2020

CATHEDRAL-HF Trial Explores Carvedilol Monotherapy in Heart Failure with Improved Ejection Fraction

  • The CATHEDRAL-HF trial investigated carvedilol as a single maintenance therapy for heart failure (HF) patients with improved ejection fraction (EF).
  • Findings suggest that discontinuing other HF treatments while maintaining carvedilol may be safe regarding left ventricular EF, mortality, and hospitalization.
  • Exploratory outcomes at 52 weeks showed no significant differences in hospitalizations, deaths, or ventricular arrhythmias between the control and intervention groups.
  • Experts emphasize the potential to reduce polypharmacy in chronic HF patients, though further studies with larger samples are needed to confirm these findings.

Closed-Loop tACS Shows Promise for Individualized Depression Treatment

• A pilot study explored individualized closed-loop transcranial alternating current stimulation (tACS) for depression, adjusting stimulation frequency to each participant’s individual alpha frequency (IAF). • The study found that repeated sessions of IAF-tACS reduced alpha frequency brain activity and improved mood, suggesting a potential cumulative effect via homeostatic mechanisms. • Key questions remain regarding parameter optimization, safety of repeated treatments, and comparative efficacy with other stimulation modalities like tDCS. • This research opens avenues for exploring individualized tES interventions, potentially enhancing therapeutic outcomes for depression and other clinical indications.

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