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Clinical Trial News

Beacon Therapeutics' AGTC-501 Shows Sustained Efficacy in XLRP Phase 2 Trial

  • Beacon Therapeutics announced positive 24-month data from the Phase 2 SKYLINE trial of AGTC-501 for X-linked retinitis pigmentosa (XLRP).
  • The high-dose cohort of AGTC-501 demonstrated a 57% response rate in retinal sensitivity improvement, the trial's primary endpoint.
  • AGTC-501 was generally safe and well-tolerated, with no clinically significant safety events associated with the treatment.
  • Pivotal Phase 2/3 VISTA and open-label Phase 2 DAWN trials are ongoing, supporting further development of AGTC-501 for XLRP.

A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)

NCT06275620Enrolling by InvitationPhase 2
Beacon Therapeutics
Posted 11/14/2023

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

NCT04850118Active, Not RecruitingPhase 2
Beacon Therapeutics
Posted 3/14/2024

Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

NCT03316560Active, Not RecruitingPhase 1
Beacon Therapeutics
Posted 4/16/2018

ACCESS-GC Trial Integrates Genetic Counselors into Primary Care in British Columbia

  • The ACCESS-GC trial aims to integrate genetic counselors into primary care settings in British Columbia to address healthcare inequities.
  • Led by Dr. Jehannine (J9) Austin, the trial seeks to provide personalized genetic care directly within communities, enhancing precision medicine.
  • The trial will assess the effectiveness of this integrated approach for conditions with a genetic component, such as diabetes and psychiatric disorders.
  • Funded by Genome BC, the initiative aims to improve patient outcomes and evaluate the economic benefits of embedding genetic counselors in primary care.

Narmafotinib Receives FDA Fast Track Designation for Advanced Pancreatic Cancer

  • The FDA has granted Fast Track Designation to Amplia Therapeutics' narmafotinib for advanced pancreatic cancer, expediting its development and review.
  • Narmafotinib, a FAK inhibitor, may offer an advantage over existing therapies, potentially qualifying it for Accelerated Approval and Priority Review.
  • The ACCENT trial, evaluating narmafotinib with gemcitabine and Abraxane, is ongoing in Australia, South Korea, and planned for the US, showing promising early results.
  • Fast Track Designation allows Amplia Therapeutics to work more closely with the FDA, accelerating the clinical program for this devastating disease.

ACCENT: AMP945 in Combination with Nab-paclitaxel and Gemcitabine for Treatment of Pancreatic Cancer

NCT05355298Active, Not RecruitingPhase 1
Amplia Therapeutics Limited
Posted 5/31/2022

Sidney Hecht Honored for Pioneering Work Linking Chemistry and Biology to Combat Disease

• Sidney Hecht received the Arizona Bioscience Pioneer Award for Lifetime Achievement for merging biology and chemistry to improve human health. • Hecht's research at ASU's Biodesign Center focuses on mitochondrial disorders, particularly Friedreich’s ataxia, a genetic neuromuscular disease. • Hecht played a key role in developing Hycamtin, a chemotherapy drug for ovarian and lung cancer, and studied the anti-tumor agent Bleomycin. • His center has developed compounds that redirect electrons in dysfunctional respiratory chains, with one in clinical trials for Friedreich’s ataxia.

AngioDynamics Launches RECOVER-AV Trial for AlphaVac System in Pulmonary Embolism Treatment

  • AngioDynamics has initiated the RECOVER-AV trial to assess the AlphaVac F1885 System's safety and efficacy in treating acute, intermediate-risk pulmonary embolism (PE).
  • The multi-center, multi-national trial will enroll patients across Europe, evaluating the reduction in right ventricular/left ventricular (RV/LV) ratio post-procedure.
  • The study follows the APEX-AV trial in the U.S., which demonstrated the AlphaVac system's safety and effectiveness in improving right ventricular function and reducing clot burden.
  • The AlphaVac F1885 System received CE Mark approval in Europe, allowing AngioDynamics to expand its reach in treating PE, where prevalence is higher than in the U.S.

Mantle Cell Lymphoma Pipeline Shows Promise with Novel Targeted Therapies

• The Mantle Cell Lymphoma (MCL) pipeline is robust, featuring over 20 companies developing more than 22 therapies. • Key players like AbbVie and BeiGene are advancing novel drugs, including BTK inhibitors and Bcl-2 inhibitors, to improve MCL treatment. • Clinical trials are evaluating promising therapies such as Venetoclax, ADI-001, and Orelabrutinib across various phases. • Recent data highlights potential for combination therapies and novel mechanisms like BTK degradation in MCL treatment.

Cybin Advances CYB003 and CYB004 Clinical Programs for Mental Health Disorders

  • Cybin is set to initiate a Phase 3 study of CYB003 for Major Depressive Disorder (MDD) following a productive meeting with the FDA.
  • Twelve-month efficacy data from the Phase 2 study of CYB003 in MDD is expected in early Q4 2024, offering insights into long-term benefits.
  • Topline efficacy and safety results for CYB004 in Generalized Anxiety Disorder (GAD) are anticipated by year-end 2024 or early Q1 2025.
  • Cybin has strengthened its R&D team with the addition of Dr. Atul R. Mahableshwarkar and Dr. Tom Macek to lead the CYB003 and CYB004 programs, respectively.

FDA Clears Epitomee Capsule for Weight Management

  • The FDA has cleared the Epitomee capsule as an adjunct to lifestyle modification for weight management in adults with a BMI of 25 to 40 kg/m2.
  • The Epitomee capsule expands in the stomach to create a feeling of fullness, later disintegrating in the intestines for natural elimination.
  • Clinical trial data demonstrated significantly greater weight reduction with Epitomee compared to placebo (P < .0001) over 24 weeks.
  • A significant 55.5% of patients taking Epitomee achieved at least 5% body weight loss, demonstrating its effectiveness in weight management.

The Effect of Extended Use of the EPITOMEE CAPSULE (ELECT)

NCT04994769CompletedNot Applicable
Epitomee medical
Posted 8/31/2021

The Effect of Epitomee Capsule on Body Weight in Patients With Overweight and Obesity

NCT04222322CompletedNot Applicable
Epitomee medical
Posted 9/15/2020

GENFIT Reports Positive Financial Results in H1 2024 Driven by Iqirvo's U.S. Launch

• GENFIT reported €61.6 million in cash and equivalents as of June 30, 2024, excluding a €48.7 million milestone from Ipsen for Iqirvo's U.S. launch. • The company's revenues reached €59.0 million, boosted by the €48.7 million milestone payment invoiced in June 2024. • A positive opinion from the EMA for Iqirvo anticipates a final decision in H2 2024, potentially triggering a €26.5 million milestone upon pricing approval. • Phase 2 trial for VS-01 in Acute on-Chronic Liver Failure (ACLF) is progressing with protocol modifications to address recruitment challenges.

ESMO 2024: Key Breast Cancer Advances Highlighted

  • Final KEYNOTE-522 data confirmed pembrolizumab's benefit in neoadjuvant chemotherapy for early-stage triple-negative breast cancer, improving 5-year overall survival.
  • NATALEE trial's 4-year update showed ribociclib plus aromatase inhibition significantly improved invasive disease-free survival in high-risk, hormone receptor-positive early breast cancer.
  • DESTINY-Breast12 trial demonstrated trastuzumab deruxtecan's significant intracranial activity in HER2-positive metastatic breast cancer patients with brain metastases.
  • ICARUS-BREAST01 trial revealed promising objective response rates and progression-free survival with patritumab deruxtecan in HER3-positive, hormone receptor-positive, HER2-negative metastatic breast cancer.

Study of Pembrolizumab (MK-3475) Versus Placebo After Complete Resection of High-Risk Stage III Melanoma (MK-3475-054/1325-MG/KEYNOTE-054)

NCT02362594Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 7/16/2015

Phase III Radium 223 mCRPC-PEACE III

NCT02194842Active, Not RecruitingPhase 3
European Organisation for Research and Treatment of Cancer - EORTC
Posted 10/1/2015

A Clinical Trial Investigating the Safety, Tolerability, and Therapeutic Effects of BNT113 in Combination With Pembrolizumab Versus Pembrolizumab Alone for Patients With a Form of Head and Neck Cancer Positive for Human Papilloma Virus 16 and Expressing the Protein PD-L1

NCT04534205RecruitingPhase 2
BioNTech SE
Posted 1/7/2021

Study of Pembrolizumab (MK-3475) Plus Chemotherapy vs Placebo Plus Chemotherapy as Neoadjuvant Therapy and Pembrolizumab vs Placebo as Adjuvant Therapy in Participants With Triple Negative Breast Cancer (TNBC) (MK-3475-522/KEYNOTE-522)

NCT03036488Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 3/7/2017

Hypofractionated vs Standard Radiotherapy in Breast Cancer With an Indication for Regional Lymph Node Irradiation About Lymphedema Occurrence

NCT03127995Active, Not RecruitingNot Applicable
UNICANCER
Posted 9/1/2016

Chemotherapy Before Surgery and Radiation Therapy or Surgery and Radiation Therapy Alone in Treating Patients With Nasal and Paranasal Sinus Cancer That Can Be Removed by Surgery

NCT03493425RecruitingPhase 2
ECOG-ACRIN Cancer Research Group
Posted 3/12/2019

A Clinical Study of the Safety and Effectiveness of an Investigational Cell Therapy Given With and Without an Investigational RNA-based Vaccine in Patients With Organ Tumors

NCT04503278RecruitingPhase 1
BioNTech Cell & Gene Therapies GmbH
Posted 9/16/2020

Neoadjuvant mFolfirinox With or Without Preoperative Concomitant Chemoradiotherapy in Patients With Borderline Resectable Pancreatic Carcinoma (PANDAS-PRODIGE 44)

NCT02676349Active, Not RecruitingPhase 2
Institut de Cancérologie de Lorraine
Posted 10/13/2016

Study to Compare Tivozanib in Combination With Nivolumab to Tivozanib Monotherapy in Subjects With Renal Cell Carcinoma

NCT04987203Active, Not RecruitingPhase 3
AVEO Pharmaceuticals, Inc.
Posted 9/9/2021

Study of Pembrolizumab (MK-3475) in Combination With Adjuvant Chemotherapy With or Without Radiotherapy in Participants With Newly Diagnosed Endometrial Cancer After Surgery With Curative Intent (MK-3475-B21 / KEYNOTE-B21 / ENGOT-en11 / GOG-3053)

NCT04634877Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 1/10/2021

A Study in Ovarian Cancer Patients Evaluating Rucaparib and Nivolumab as Maintenance Treatment Following Response to Front-Line Platinum-Based Chemotherapy

NCT03522246Active, Not RecruitingPhase 3
pharmaand GmbH
Posted 5/14/2018

Carboplatin-paclitaxel With Retifanlimab or Placebo in Participants With Locally Advanced or Metastatic Squamous Cell Anal Carcinoma (POD1UM-303/InterAACT 2).

NCT04472429Active, Not RecruitingPhase 3
Incyte Corporation
Posted 1/12/2021

ONC-392 and Pembrolizumab in Platinum Resistant Ovarian Cancer

NCT05446298Active, Not RecruitingPhase 2
OncoC4, Inc.
Posted 12/22/2022

Capivasertib+Paclitaxel as First Line Treatment for Patients With Locally Advanced or Metastatic TNBC

NCT03997123Active, Not RecruitingPhase 3
AstraZeneca
Posted 6/25/2019

A Trial to Evaluate Efficacy and Safety of Ribociclib With Endocrine Therapy as Adjuvant Treatment in Patients With HR+/HER2- Early Breast Cancer

NCT03701334Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 12/7/2018

Durvalumab+ Gemcitabine/Cisplatin (Neoadjuvant Treatment) and Durvalumab (Adjuvant Treatment) in Patients With MIBC

NCT03732677Active, Not RecruitingPhase 3
AstraZeneca
Posted 11/16/2018

Study of Chemoradiotherapy With or Without Pembrolizumab (MK-3475) For The Treatment of Locally Advanced Cervical Cancer (MK-3475-A18/KEYNOTE-A18/ENGOT-cx11/GOG-3047)

NCT04221945Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 5/12/2020

A Study of T-DXd in Participants With or Without Brain Metastasis Who Have Previously Treated Advanced or Metastatic HER2 Positive Breast Cancer

NCT04739761Active, Not RecruitingPhase 3
AstraZeneca
Posted 6/22/2021

Patritumab Deruxtecan (U3-1402) in Unresectable Locally Advanced or Metastatic Breast Cancer

NCT04965766Active, Not RecruitingPhase 2
Gustave Roussy, Cancer Campus, Grand Paris
Posted 5/11/2021

Pre-operative Trial for Breast Cancer With Nivolumab in Combination With Novel IO

NCT03815890RecruitingPhase 2
The Netherlands Cancer Institute
Posted 10/4/2019

A Randomized Trial to Assess the Role of Imaging During Follow Up After Radical Surgery of High Risk Melanoma

NCT03116412RecruitingNot Applicable
Uppsala University
Posted 6/8/2017

Safety and Efficacy of Lenvatinib (E7080/MK-7902) With Pembrolizumab (MK-3475) in Combination With Transarterial Chemoembolization (TACE) in Participants With Incurable/Non-metastatic Hepatocellular Carcinoma (MK-7902-012/E7080-G000-318/LEAP-012)

NCT04246177Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 5/22/2020

HARE-40: HPV Anti-CD40 RNA vaccinE

NCT03418480CompletedPhase 1
University of Southampton
Posted 4/11/2017

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