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Clinical Trial News

Jaguar Health Launches Gelclair for Oral Mucositis in Cancer Patients

  • Jaguar Health has launched Gelclair, an FDA-approved prescription product, marking its entry into cancer supportive care.
  • Gelclair provides rapid, lasting pain relief for oral mucositis without stinging, addressing a significant need for cancer patients.
  • Oral mucositis affects up to 90% of head and neck cancer patients undergoing chemotherapy and radiotherapy, causing treatment interruptions.
  • The launch includes a dedicated sales team and digital marketing, targeting oncology professionals and patients to improve awareness.

UK's MHRA Aims for New Clinical Trial Regulation by January 2026

  • The MHRA anticipates implementing new UK clinical trial regulations by January 2026, following endorsement from the Labour Government.
  • The new framework focuses on patient safety and aims to streamline clinical trial approvals, fostering innovation and international trial participation.
  • Key changes include a single application route, coordinated regulatory and ethics review, and updated review timelines for clinical trial applications.
  • The regulation will also enhance research transparency, mandating trial registration and results publication, while aligning with international GCP standards.

European Commission Grants Orphan Drug Designation to Astria's Navenibart for Hereditary Angioedema

  • The European Commission has granted Orphan Medicinal Product Designation (OMPD) to navenibart for treating hereditary angioedema (HAE).
  • Phase 1b/2 ALPHA-STAR trial results showed navenibart had a favorable safety profile and reduced monthly HAE attack rates by 90-96% over six months.
  • Astria Therapeutics plans to advance navenibart to a Phase 3 trial in Q1 2025, supported by OMPD incentives for rare disease drug development.
  • The OMPD status complements the FDA's orphan drug designation, highlighting the global unmet need and potential impact of navenibart on HAE treatment.

VCN-01 Receives Orphan Drug Designation from European Commission for Retinoblastoma Treatment

  • Theriva Biologics' VCN-01, an oncolytic adenovirus, has been granted orphan medicinal product designation by the European Commission for retinoblastoma treatment.
  • This designation emphasizes the urgent need for new treatment options for retinoblastoma, a rare and life-threatening eye cancer in children.
  • VCN-01 previously received orphan drug and rare pediatric disease designations from the U.S. FDA for retinoblastoma, highlighting its potential.
  • The European Commission's decision provides Theriva with benefits like market exclusivity and reduced regulatory fees to support VCN-01's development.

Evaluate Safety and the Oncolitic Adenovirus VCN-01 Activity in Patients With Refractory Retinoblastoma

NCT03284268CompletedNot Applicable
Fundació Sant Joan de Déu
Posted 9/6/2017

UConn Stroke Drug Advances Towards Clinical Trials with NIH Grant

  • UConn's experimental small-molecule drug has shown promise in reducing damage and restoring function after stroke in animal models.
  • A follow-up NIH grant of over $2 million will further advance testing of the anti-inflammatory therapy that targets the P2X4 receptor.
  • The drug aims to reduce brain damage caused by ischemic stroke by blocking the P2X4 receptor, potentially improving short- and long-term recovery.
  • Researchers plan to seek FDA approval for Investigational New Drug status, paving the way for first-in-human clinical trials.

Cytokinetics Advances Cardiac Myosin Modulation Programs; Aficamten NDA Submitted

  • Cytokinetics submitted a New Drug Application (NDA) to the FDA for aficamten in Q3, marking a significant step toward potential U.S. approval.
  • Phase 3 COMET-HF trial of omecamtiv mecarbil and Phase 2 AMBER-HFpEF trial of CK-586 are expected to begin in Q4, expanding the pipeline.
  • The company is preparing for the global commercial launch of aficamten, potentially in 2025, focusing on market access and patient experience.
  • An unbranded disease awareness campaign, "HCM Beyond The Heart," has been launched to educate healthcare professionals about hypertrophic cardiomyopathy (HCM).

Apretude Shows Over 99% Effectiveness in Real-World HIV Prevention Studies

  • Real-world studies demonstrate Apretude (cabotegravir long-acting injectable) has over 99% effectiveness in preventing HIV, reinforcing its potential as a PrEP option.
  • The PILLAR implementation study reveals that Apretude reduces PrEP-related stigma and anxiety among men who have sex with men and transgender men, compared to daily oral PrEP.
  • Participants in the PILLAR trial reported high acceptability of Apretude, citing convenience and reduced worry about missing doses, despite some injection site reactions.
  • Flexible clinic scheduling, transportation assistance, and virtual appointments were identified as valuable facilitators of adherence to Apretude, enhancing its real-world impact.

Sonire Therapeutics' HIFU System Receives FDA Breakthrough Device Designation for Pancreatic Cancer

  • Sonire Therapeutics' Suizenji high-intensity focused ultrasound (HIFU) system has been granted Breakthrough Device designation by the FDA for pancreatic cancer treatment.
  • The Suizenji HIFU system uses cavitation ultrasound to deliver thermal therapy with lower acoustic energy and enhanced image clarity for targeted treatment.
  • The system is currently undergoing evaluation in a Phase 2 trial for patients with unresectable pancreatic cancer who are refractory or intolerant to initial chemotherapy.
  • The FDA designation aims to expedite the development and review of innovative technologies that may offer more effective treatment for life-threatening conditions.

Cessatech's CT001 Pediatric Pain Study Reaches Halfway Recruitment Milestone

  • Cessatech A/S has reached the halfway point in patient recruitment for its Paediatric Study 0202, with 75 children now included in the trial.
  • Study 0202 is the final clinical study required to evaluate the safety and efficacy of CT001, a nasal spray for acute pain in children.
  • The company anticipates completing recruitment by the end of 2024, with ongoing recruitment at sites in Spain and the UK.
  • CT001's development aligns with the European Medicines Agency's (EMA) Paediatric Committee guidelines, aiming to provide easy-to-administer pain relief.

AI Tools Revolutionize Cancer Diagnosis and Treatment Precision, Expert Says

  • AI technology is transforming cancer care by integrating patient factors like tumor microenvironment, demographics, and family history to deliver more precise diagnoses and treatment predictions.
  • Advanced AI tools can differentiate between visually similar prostate cancers by analyzing multiple factors, enabling more accurate prediction of disease progression and treatment outcomes.
  • The role of pathologists has evolved from purely diagnostic to becoming integral members of patient care teams, utilizing AI to provide comprehensive patient profiles for targeted therapies.

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