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Clinical Trial News

AMX0035 Slows Functional Decline in Amyotrophic Lateral Sclerosis

  • A new study reveals that AMX0035 significantly slows the progression of functional decline in patients with amyotrophic lateral sclerosis (ALS).
  • The trial demonstrated a statistically significant difference in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) scores between the AMX0035 and placebo groups.
  • AMX0035, a combination of sodium phenylbutyrate and taurursodiol, represents a promising therapeutic option for ALS, addressing a critical unmet need.
  • The findings support the potential of AMX0035 to improve the quality of life and extend survival for individuals affected by this devastating neurodegenerative disease.

AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT03127514CompletedPhase 2
Amylyx Pharmaceuticals Inc.
Posted 6/22/2017

Christie Hospital Launches Initiative to Boost Cancer Trial Access and Diversity

  • The Christie NHS Foundation Trust partners with Innovative Trials to enhance cancer clinical trial recruitment, focusing on improving access for patients in six key UK regions.
  • The initiative specifically targets underrepresented black, Asian, and minority ethnic communities, aiming to increase trial participation through culturally appropriate outreach materials.
  • A network of cancer centers will be established across the UK to enable local trial participation, addressing the fact that fewer than 50% of cancer patients recall discussing clinical trials post-diagnosis.

Real-World Evidence Gains Momentum: FDA Approvals and Cross-Industry Collaborations Mark Turning Point

  • The FDA's approval of Ibrance's expanded indication for male breast cancer patients, supported by real-world evidence, marks a significant milestone in RWE adoption in regulatory decision-making.
  • Cross-industry collaboration through the Friends of Cancer Research study demonstrated consistency between real-world endpoints and clinical trial outcomes in lung cancer research.
  • Industry experts predict a 30-50% increase in regulatory decisions incorporating RWE in 2020, with particular focus on single-arm trials using real-world data as comparator cohorts.

Clinical Trials Evolution: New Data Management and Design Approaches Set to Transform Drug Development in 2020

• Clinical trial designs are evolving to incorporate broader patient populations, with new approaches including longitudinal studies, synthetic data integration, and combined results from both physical and virtual sites.
• Up to 90% of US clinical trials face significant enrollment delays, prompting the adoption of hybrid trials, community-based sites, and digital engagement strategies to improve patient recruitment.
• The industry is moving towards reusable data models, combining EHR data with trial information to create comprehensive repositories, while synthetic data emerges as a solution for trial efficiency and GDPR compliance.

Novel Neural Biomarker ERNA Shows Promise for Optimizing Parkinson's Disease Deep Brain Stimulation

  • Researchers have identified evoked resonant neural activity (ERNA) as a reliable biomarker that could improve deep brain stimulation electrode placement and programming for Parkinson's disease patients.
  • ERNA demonstrates key advantages including large amplitude signals, localization to the dorsal subthalamic nucleus, and persistence under general anesthesia, making it potentially valuable for both awake and asleep DBS procedures.
  • Clinical studies show ERNA amplitude correlates with therapeutic benefits, suggesting its utility for optimizing electrode configurations and potentially enabling more precise, personalized DBS treatment approaches.

Rare Disease Drug Development Faces Complex Challenges Despite Growing Pipeline

  • Despite affecting 30 million Europeans, approximately 90% of the 7,000 known rare diseases still lack effective treatments, highlighting a significant unmet medical need.
  • Orphan Drug Designations have declined by 49% at EMA and 8% at FDA between 2014-2019, while pricing pressures and clinical trial complexities continue to challenge development.
  • Industry remains committed to rare disease research with nearly one-third of pipeline drugs targeting orphan conditions, supported by patient advocacy groups and regulatory incentives.

Psychedelics and Psychedelic-Assisted Psychotherapy: A Review of Clinical Applications

This article reviews the clinical application of psychedelic drugs in psychiatric disorders, focusing on MDMA, psilocybin, LSD, and ayahuasca. It highlights the resurgence of research into their therapeutic potential, supported by organizations like MAPS and the Heffter Research Institute, and discusses their efficacy in treating conditions such as PTSD, depression, and anxiety.

Roche and Bicycle Therapeutics Forge $1.7B Cancer Immunotherapy Alliance

• Roche's Genentech unit partners with Bicycle Therapeutics in a $1.7 billion deal to leverage innovative bicyclic peptide platform for cancer immunotherapy development, with a $30 million upfront payment.
• Bicycle's proprietary technology combines antibody-like targeting with small-molecule drug properties, offering unique advantages in manufacturing, dosing, and tissue penetration for cancer treatment.
• The collaboration focuses on discovering novel cancer immunotherapy targets, with Bicycle handling early development and Roche taking over clinical-stage programs.

Clinical Trial Sponsors Face Mandate to Publish Decade of Missing Data

  • A federal judge has ruled that government research agencies misinterpreted a law requiring clinical trial data publication, creating a 10-year gap.
  • Universities, drug companies, and medical device manufacturers may need to release previously unpublished data from trials of unapproved drugs and devices.
  • The ruling affects trials conducted between 2007 and 2017, potentially impacting hundreds to over a thousand noncompliant trials.
  • Experts assert the decision enhances public access to clinical trial results, making it harder to conceal unfavorable outcomes.

NIH Initiates Clinical Trial of Remdesivir for COVID-19 in the U.S.

  • The National Institute of Health (NIH) has begun a clinical trial of Gilead Sciences' remdesivir to treat COVID-19 in U.S. hospitals.
  • The first participant is an American patient at the Nebraska Medical Center, who was quarantined from the Diamond Princess cruise ship.
  • Clinical trials are also underway in China to evaluate remdesivir's efficacy against SARS-CoV-2, the virus causing COVID-19.
  • U.S. health officials are preparing for potential significant disruptions due to the expected spread of COVID-19.

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