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Clinical Trial News

Bond University Researchers Trial Stem Cell Therapy to Restore Vision in Animals

  • Researchers at Bond University are trialing stem cell therapies in pigs to treat blindness, specifically targeting age-related macular degeneration and retinitis pigmentosa.
  • The stem cell technology aims to efficiently create cells needed for vision restoration, potentially lowering production costs and increasing accessibility for patients.
  • Animal studies involving retinal cell transplantation into blind pigs are planned to assess the treatment's efficacy and safety over approximately 18 months.
  • Bond University is collaborating with national and international partners, bidding for a Cooperative Research Centre to advance stem cell therapies in Australia.

Digital Technologies Transform Cognitive Assessment in Schizophrenia Drug Trials

  • Digital assessment tools are revolutionizing cognitive drug trials for schizophrenia, offering automated, standardized testing that reduces inter-rater variability and patient burden compared to traditional pen-and-paper methods.
  • Advanced technologies like smartphone-based assessments and wearable devices enable more frequent cognitive measurements outside clinical settings, potentially increasing trial sensitivity and providing deeper insights into treatment efficacy.
  • Novel digital approaches, including CANTAB cognitive assessments, are enabling better patient stratification and more precise identification of treatment effects in schizophrenia patients with baseline cognitive deficits.

SSY Group Receives China Approval for Cycloserine Capsules and Lornoxicam Injection

  • SSY Group has received approval from China's National Medical Products Administration for Cycloserine Capsules.
  • Cycloserine Capsules are indicated for the treatment of tuberculosis and urinary tract infections, addressing critical medical needs.
  • Lornoxicam injection, also approved, targets acute pain management, expanding SSY Group's pain relief portfolio.
  • These approvals represent a significant advancement in SSY Group's product development and market presence in China.

Bacteria-Assisted Aptamer-Drug Conjugates Enhance Pancreatic Cancer Therapy

  • Researchers developed a novel approach using bacteria-assisted delivery of aptamer-drug conjugates (ApDCs) to target and treat pancreatic cancer.
  • The ApDC, Sgc8c-MMAE, targets PTK7, which is highly expressed in pancreatic cancer cells, and the bacteria VNP20009 enhances drug penetration into the tumor microenvironment.
  • In vivo studies demonstrated that VNP@Sgc8c-MMAE significantly improved tumor regression, increased immune cell infiltration, and prolonged survival in mouse models of pancreatic cancer.
  • This synergistic therapy shows promise by improving drug delivery, modulating the tumor microenvironment, and enhancing antitumor immunity in pancreatic cancer.

Ultragenyx's UX111 Gene Therapy Shows Promise in Sanfilippo Syndrome Type A

• UX111 (rebisufligene etisparvovec) is an investigational gene therapy for Sanfilippo syndrome type A (MPS IIIA), a rare and fatal lysosomal storage disease. • The therapy uses an AAV9 vector to deliver a functional copy of the SGSH gene, addressing the underlying enzyme deficiency that causes heparan sulfate accumulation. • UX111 has received multiple designations, including Regenerative Medicine Advanced Therapy and Orphan Drug status, highlighting its potential impact on this unmet medical need.

Clarity Pharmaceuticals' 64Cu-SAR-bisPSMA Phase III Trial for Prostate Cancer Receives Positive FDA Guidance

  • Clarity Pharmaceuticals' AMPLIFY trial, a Phase III study of 64Cu-SAR-bisPSMA for prostate cancer biochemical recurrence, received positive feedback from the FDA.
  • The AMPLIFY trial will enroll approximately 220 patients with rising PSA levels after initial prostate cancer treatment, with recruitment expected to begin in early 2025.
  • Previous trials (COBRA and PROPELLER) showed 64Cu-SAR-bisPSMA had higher lesion uptake and detection compared to standard imaging, supporting the AMPLIFY trial design.

Study on Olaparib Plus Abiraterone as First-line Therapy in Men With Metastatic Castration-resistant Prostate Cancer

NCT03732820Active, Not RecruitingPhase 3
AstraZeneca
Posted 10/31/2018

Positron Emission Tomography (PET) Imaging of Participants With Confirmed Prostate Cancer Using 64Cu-SAR-bisPSMA (PROPELLER)

NCT04839367CompletedPhase 1
Clarity Pharmaceuticals Ltd
Posted 7/13/2021

64Cu-SAR-bisPSMA for Identification of Participants With Recurrence of Prostate Cancer (COBRA)

NCT05249127CompletedPhase 1
Clarity Pharmaceuticals Ltd
Posted 4/11/2022

Positron Emission Tomography Using 64Cu-SAR-bisPSMA in Participants With High-risk Prostate Cancer Prior to Radical Prostatectomy

NCT06056830RecruitingPhase 3
Clarity Pharmaceuticals Ltd
Posted 12/21/2023

Oxytocin Receptor Agonists Show Promise in Promoting Hair Growth

  • Non-peptidic oxytocin receptor (OXTR) agonists, LIT001 and WAY267464, demonstrate hair growth-promoting effects, suggesting potential as novel treatments for alopecia.
  • Bioinformatic analysis reveals low OXTR expression in dermal papilla (DP) cells from alopecia patients, with OXTR knockdown suppressing hair growth-related gene expression.
  • In vitro studies show that LIT001 and WAY267464 increase hair growth-related gene expression in DP cells and accelerate hair follicle elongation in hair follicloids.
  • The findings suggest that targeting OXTR with specific agonists could offer a new therapeutic avenue for hair loss, warranting further investigation in organ culture and animal models.

UK Trials to Assess Cannabis-Based Medicines for Childhood Epilepsy

  • Two new trials in the UK will investigate the benefits of cannabis-based medicines for children and adults with refractory epilepsy, addressing a critical need for evidence-based treatment options.
  • The trials, funded by the National Institute for Healthcare Research and conducted by University College London, will involve 500 NHS patients and assess the safety and efficacy of these medicines in reducing seizures.
  • Researchers will evaluate the impact of cannabis-based medicines on various factors, including learning, sleep, behavior, quality of life, and anxiety, potentially leading to NHS approval if proven clinically and cost-effective.
  • The studies aim to provide a standardized, safe, and reliable treatment option for patients with uncontrolled epilepsy, reducing the financial burden on families who currently rely on costly private prescriptions.

CAR T-cell Therapy Offers Hope for Children with Aggressive T-ALL

  • Great Ormond Street Hospital launches a clinical trial for children with relapsed or resistant T-cell acute lymphoblastic leukemia (T-ALL) using CAR T-cell therapy.
  • The CAR T-cell therapy targets the CCR9 protein found on cancerous T cells, aiming to eliminate leukemia while preserving healthy T cells.
  • The study will run a child and adult study at the same time, potentially accelerating access to targeted therapies for children.
  • Researchers hope this approach will establish a precedent for including children in early-phase clinical studies, ensuring timely access to innovative treatments.

UK Trials Launch to Evaluate Cannabis-Based Medicines for Epilepsy

  • Two new trials in the UK will assess the safety and efficacy of cannabis-based medicines for treating epilepsy in adults and children who have not responded to standard treatments.
  • The trials, funded by the National Institute for Healthcare Research, will involve 500 NHS patients and are led by researchers from University College London and Great Ormond Street Hospital.
  • Researchers will examine the impact of cannabis-based medicines on seizure frequency, learning, sleep, behavior, quality of life, and anxiety in participants with various forms of epilepsy.
  • The data from these trials could potentially lead to the licensing of cannabis-based medicines by the MHRA and their approval for use within the NHS, providing a new treatment option.

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