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Clinical Trial News

FDA Approves Arimoclomol for Niemann-Pick Disease Type C After Multiple Failed Trials

  • Arimoclomol, previously developed by Orphazyme, receives FDA approval for treating Niemann-Pick disease type C, marking a significant milestone after 13 years of development.
  • The drug's approval offers a new therapeutic option for patients with this rare and progressive genetic disorder, addressing a critical unmet medical need.
  • Despite earlier setbacks in clinical trials, arimoclomol's eventual approval underscores the FDA's commitment to providing treatments for rare diseases.
  • This approval highlights the importance of continued research and regulatory flexibility in bringing potentially life-changing therapies to patients with limited options.

Vicebio Secures $100 Million to Advance RSV and hMPV Combination Vaccine

  • Vicebio Ltd. has secured $100 million in Series B funding to advance its next-generation vaccines for respiratory illnesses, including RSV and hMPV.
  • The funding will support Phase I testing of VXB-241, a bivalent vaccine targeting RSV and hMPV, with initial readouts expected in mid-2025.
  • Vicebio will also use the funds to develop VXB-251, a trivalent vaccine targeting RSV, hMPV, and parainfluenza virus 3 (PIV3).
  • Vicebio's Molecular Clamp platform stabilizes viral glycoproteins, enhancing the immune response and offering broad applicability to various viruses.

A Study of RSV-HMPV Bivalent Vaccine VXB-241 in Older Adults

NCT06556147Active, Not RecruitingPhase 1
Vicebio Australia Proprietary Limited
Posted 8/13/2024

Aptar's N-Sorb Nitrosamine Mitigation Solution Accepted into FDA's Emerging Technology Program

  • Aptar's N-Sorb, utilizing the 3-Phase Activ-Polymer™ platform, has been accepted into the FDA's Emerging Technology Program, signaling a significant advancement in pharmaceutical safety.
  • N-Sorb addresses N-nitrosamine impurities in drugs, classified as probable human carcinogens, by integrating into packaging to react with precursors and scavenge impurities.
  • This active packaging intervention could eliminate the need for drug reformulation, supporting compliance with FDA and EMA regulations and potentially accelerating drug development.
  • The FDA's selective program acceptance highlights N-Sorb's promise in modernizing pharmaceutical manufacturing and quality control, offering a potential pathway for expedited regulatory review.

DBV Technologies Concludes VITESSE Trial Screening for Viaskin Peanut Patch

  • DBV Technologies completed screening for its VITESSE Phase 3 trial, surpassing recruitment goals for peanut-allergic children aged 4-7.
  • The VITESSE trial, evaluating the Viaskin Peanut Patch, is the largest immunotherapy study for this patient group, spanning 86 global sites.
  • Topline data from the VITESSE trial, assessing the safety and efficacy of Viaskin Peanut Patch, is anticipated in Q4 2025.
  • DBV Technologies is focusing on advancing the Viaskin Peanut Patch program to support a Biologic License Application submission.

Ruxolitinib Plus TKI Therapy Shows Promise in Chronic Myeloid Leukemia

  • Adding ruxolitinib to tyrosine kinase inhibitors (TKIs) significantly increased deep molecular response rates in chronic-phase chronic myeloid leukemia (CP-CML) patients.
  • The combination therapy led to a higher proportion of patients meeting criteria for treatment discontinuation compared to TKI therapy alone.
  • The SWOG S1712 clinical trial demonstrated that ruxolitinib may sensitize leukemic stem cells to TKIs, potentially eradicating measurable residual disease.
  • Toxicity profiles were similar between the two arms, with a slightly higher incidence of grade 1/2 anemia in the ruxolitinib arm.

Active Breaks Show Promise in Boosting Academic Performance for Children with and without ADHD

  • A new study, Break4Brain, investigates the impact of physical activity (PA) on academic achievement in children, focusing on those with and without ADHD.
  • The study employs a two-phase design, including a crossover study and a cluster randomized controlled trial (RCT), to assess acute and chronic PA effects.
  • Researchers will evaluate changes in brain function, academic performance, working memory, and attention through various experimental conditions and interventions.
  • The Break4Brain project anticipates that PA will enhance cognitive and academic outcomes, potentially offering a novel approach to support children with ADHD.

UC Davis Orthopaedic Surgery Enhances Support for Pediatric Cancer Patients

  • UC Davis Comprehensive Cancer Center provides advanced care for pediatric bone and soft tissue cancers, including osteosarcoma and Ewing’s sarcoma.
  • The center offers access to cutting-edge clinical trials and comprehensive supportive programs, focusing on both treatment and long-term care for young patients.
  • Advanced surgical techniques, including limb-sparing procedures and innovative bone reconstruction, are employed to improve outcomes and quality of life.
  • Early detection through awareness of symptoms like unexplained pain and lumps is emphasized to improve treatment success in pediatric sarcoma cases.

AAVantgarde Bio Doses First Patient in Phase 1/2 Trial of AAVB-081 for Usher Syndrome Type 1B

  • AAVantgarde Bio has dosed the first patient in its Phase 1/2 LUCE-1 trial, evaluating AAVB-081 gene therapy for Usher Syndrome Type 1B (USH1B).
  • AAVB-081 is a dual hybrid AAV8 vector designed to deliver a functional copy of the Myo7A gene via subretinal administration.
  • The open-label, dose-escalation LUCE-1 trial will enroll 15 patients aged 18-50 with USH1B, assessing safety and efficacy endpoints over 61 months.
  • Researchers are also exploring gene-editing approaches for other forms of Usher syndrome, such as Usher syndrome type 2A (USH2A), to address hearing and vision loss.

Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa

NCT06591793RecruitingPhase 1
AAVantgarde Bio Srl
Posted 7/2/2024

Watchful Waiting Shows Non-Inferiority to BCG in High-Grade T1 Bladder Cancer After Second TUR

  • A phase 3 trial (JCOG1019) reveals watchful waiting is non-inferior to BCG for relapse-free survival in high-grade T1 bladder cancer patients with no residual tumor after a second TUR.
  • The study demonstrated similar overall survival and metastasis-free survival between the watchful waiting and BCG groups, suggesting comparable long-term outcomes.
  • Watchful waiting may reduce the need for BCG treatment in select patients, potentially alleviating BCG shortage issues and decreasing treatment-related adverse events.
  • Findings suggest watchful waiting could be a new standard of care for high-grade T1 bladder cancer patients with no residual tumor after a second TUR.

Transdermal Estradiol Shows Promise as Alternative to LHRH Agonists in High-Risk Prostate Cancer

  • A phase 3 study presented at ESMO 2024 indicates transdermal estradiol (tE2) is as effective as LHRH agonists for androgen suppression in high-risk, non-metastatic prostate cancer.
  • tE2 offers a different side effect profile, reducing hot flushes but increasing gynecomastia, providing patients with more personalized treatment options.
  • The study found no significant difference in metastasis-free survival or overall survival between patients treated with tE2 and those treated with LHRH agonists.
  • Researchers suggest tE2 should be considered a standard of care androgen deprivation therapy option for patients with non-metastatic prostate cancer.

Prostate Adenocarcinoma TransCutaneous Hormones

NCT00303784Unknown StatusPhase 3
University College, London
Posted 3/1/2006

Systemic Therapy in Advancing or Metastatic Prostate Cancer: Evaluation of Drug Efficacy

NCT00268476Active, Not RecruitingPhase 2
Medical Research Council
Posted 7/8/2005

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