MedPath

Clinical Trial News

Ractigen's RAG-18 Receives FDA Orphan Drug Designation for Duchenne and Becker Muscular Dystrophy

• Ractigen Therapeutics' RAG-18, a small activating RNA (saRNA) therapy, has been granted Orphan Drug Designation by the FDA for treating Duchenne and Becker muscular dystrophy. • RAG-18 previously received Rare Pediatric Disease Designation, making it the first saRNA therapy to achieve both designations, highlighting its potential for treating rare genetic conditions. • The therapy aims to increase utrophin production by targeting the UTRN gene, offering a functional substitute for the deficient dystrophin protein in DMD and BMD patients. • Preclinical data presented at OTS 2023 demonstrated RAG-18's ability to induce utrophin expression and ameliorate muscle damage in a mouse model of DMD.

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

NCT04998396RecruitingPhase 1
Aspa Therapeutics
Posted 9/8/2021

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

NCT06100276RecruitingPhase 1
UniQure Biopharma B.V.
Posted 8/1/2024

Infusion of Autologous T Cells Engineered to Target FSH Receptor in Recurrent Ovarian Cancer

NCT05316129RecruitingPhase 1
H. Lee Moffitt Cancer Center and Research Institute
Posted 4/28/2022

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

NCT04884815RecruitingPhase 1
Ultragenyx Pharmaceutical Inc
Posted 9/27/2021

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830Active, Not RecruitingPhase 1
Intellia Therapeutics
Posted 12/10/2021

A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants with Pi*ZZ AATD

NCT06405633RecruitingPhase 1
Wave Life Sciences Ltd.
Posted 7/19/2024

A Clinical Study of ONCT-808 in Subjects With Relapsed or Refractory B-Cell Malignancies

NCT05588440TerminatedPhase 1
Oncternal Therapeutics, Inc
Posted 5/9/2023

A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease

NCT05956626RecruitingNot Applicable
Ocugen
Posted 8/25/2023

Study of NXC-201 CAR-T in Patients With Light Chain (AL) Amyloidosis

NCT06097832RecruitingPhase 1
Nexcella Inc.
Posted 6/5/2024

Descartes-15 for Patients With Relapsed/Refractory Multiple Myeloma

NCT06304636Active, Not RecruitingPhase 1
Cartesian Therapeutics
Posted 6/19/2024

Safety Study for a Gamma Delta T Cell Product Used with Low Dose Radiotherapy in Patients with Locally Advanced or Metastatic NSCLC or Solid Tumors with Bone Metastases

NCT06069570RecruitingPhase 1
Kiromic BioPharma Inc.
Posted 11/7/2023

P-BCMA-ALLO1 Allogeneic CAR-T Cells in the Treatment of Subjects With Multiple Myeloma

NCT04960579RecruitingPhase 1
Poseida Therapeutics, Inc.
Posted 5/5/2022

A Phase 1 Study of ADI-001 in Autoimmune Disease

NCT06375993RecruitingPhase 1
Adicet Therapeutics
Posted 11/10/2024

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

NCT05836259RecruitingPhase 1
Tenaya Therapeutics
Posted 8/10/2023

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

NCT04783181RecruitingPhase 1
Adrenas Therapeutics Inc
Posted 7/1/2021

Proact: A Study of REACT in Subjects With Type 2 Diabetes Mellitus and Chronic Kidney Disease

NCT05099770RecruitingPhase 3
Prokidney
Posted 1/5/2022

T-Cell Therapy (EB103) in Adults With Relapsed/Refractory B-Cell Non-Hodgkin's Lymphoma (NHL)

NCT06343311RecruitingPhase 1
Estrella Biopharma, Inc.
Posted 6/1/2024

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224CompletedPhase 1
Sangamo Therapeutics
Posted 7/23/2019

Clinical Trial Evaluating Safety and Immunogenicity of Bird Flu Vaccines

  • A clinical trial is underway to assess the safety and immunogenicity of influenza A/H5 vaccines in healthy adults.
  • The study involves administering two doses of an egg-derived vaccine, spaced approximately three weeks apart, to participants.
  • Participants aged 18-64 or 65 and older with a BMI under 35 kg/m2 are eligible to enroll in the study.
  • The trial provides study-related care at no cost to participants, and health insurance is not required.

ArsenalBio Secures $325 Million in Series C Funding to Advance CAR T-Cell Therapies for Solid Tumors

  • Arsenal Biosciences has raised $325 million in Series C funding to progress its CAR T-cell therapies targeting solid tumors, demonstrating strong investor confidence.
  • The funding will support the advancement of ArsenalBio's pipeline, including AB-1015 for ovarian cancer and AB-2100 for kidney cancer, both currently in Phase I clinical trials.
  • ArsenalBio's T-cell engineering platform utilizes synthetic biology to genetically program T cells, enhancing their ability to target and destroy solid tumors.
  • The investment will also enable ArsenalBio to scale up manufacturing capabilities and explore new innovations in T-cell medicine, expanding its therapeutic reach.

AB-1015, an Integrated Circuit T (ICT) Cell Therapy in Patients With Platinum Resistant Epithelial Ovarian Cancer

NCT05617755Active, Not RecruitingPhase 1
Arsenal Biosciences, Inc.
Posted 11/29/2022

AB-2100, an Integrated Circuit T Cell Therapy in Patients With Recurrent Clear-cell Renal Cell Carcinoma (ccRCC)

NCT06245915RecruitingPhase 1
Arsenal Biosciences, Inc.
Posted 2/26/2024

Eli Lilly's Weekly Insulin Efsitora Shows Promise in Phase 3 Trials

  • Eli Lilly's efsitora, a weekly insulin injection, demonstrated effectiveness comparable to daily insulin in Phase 3 trials for type 2 diabetes.
  • In the QWINT-1 trial, patients new to insulin saw similar A1C reductions with efsitora compared to daily insulin after 52 weeks.
  • The QWINT-3 trial showed efsitora also effectively lowered A1C levels in patients previously on daily insulin, with results after 26 weeks.
  • Efsitora offers the potential for 313 fewer injections per year, aiming to simplify insulin therapy and improve patient adherence.

FDA Clears IND for Medigene's TCR-T Therapy MDG1015 in Advanced Solid Tumors

  • Medigene receives FDA clearance for its Investigational New Drug (IND) application for MDG1015, a T-cell receptor engineered T-cell (TCR-T) therapy.
  • A Phase I clinical trial, EPITOME1015-I, is planned to evaluate MDG1015's safety, feasibility, and preliminary efficacy in patients with advanced solid tumors.
  • MDG1015 targets NY-ESO-1/LAGE-1a and incorporates a PD1-41BB costimulatory switch protein to enhance anti-tumor activity and persistence.
  • Medigene anticipates launching the trial by the end of 2024 and expects a data readout from EPITOME1015-I by the end of 2025, pending additional financing.

FDA Grants Fast Track Designation to VMT01 for MC1R-Positive Melanoma

  • The FDA has granted Fast Track designation to VMT01, a targeted alpha-particle therapy, for unresectable or metastatic melanoma expressing melanocortin 1 receptor (MC1R).
  • VMT01, developed by Perspective Therapeutics, delivers the alpha-emitting isotope 212Pb to tumor sites with MC1R overexpression, offering a novel approach to treatment.
  • A Phase 1/2a study (NCT05655312) is currently evaluating VMT01, with plans to explore a VMT01/anti-PD-1 combination cohort pending IRB approval.
  • The Fast Track designation aims to expedite VMT01's development and review, potentially offering accelerated approval and priority review for this therapy.

MC1R-targeted Alpha-particle Therapy Trial in Adults With Advanced Melanoma

NCT05655312RecruitingPhase 1
Perspective Therapeutics
Posted 6/1/2023

Balloon Angioplasty Plus Medical Management Shows Promise for Intracranial Artery Stenosis

  • A randomized clinical trial evaluated balloon angioplasty plus aggressive medical management for severe symptomatic intracranial atherosclerotic stenosis (sICAS).
  • The study found a significantly lower incidence of stroke or death within 30 days, or ischemic stroke or revascularization through 12 months, in the angioplasty group.
  • Balloon angioplasty plus aggressive medical management may be an effective treatment for sICAS, but the early risk of stroke or death should be considered.
  • The trial challenges previous studies on sICAS, which did not demonstrate the superiority of endovascular stenting over medical management.

Balloon Angioplasty for Symptomatic Intracranial Artery Stenosis

NCT03703635Active, Not RecruitingNot Applicable
Ministry of Science and Technology of the People´s Republic of China
Posted 10/17/2018

RAPID_REVIVE Trial to Evaluate Vidofludimus Calcium for Post-COVID Syndrome

  • The RAPID_REVIVE study, led by Frankfurt University Hospital, is a phase 2 trial investigating treatments for post-COVID syndrome (PCS).
  • The trial will assess vidofludimus calcium (IMU-838), a drug that activates Nurr1 and inhibits DHODH, potentially reducing inflammation and viral infection.
  • The adaptive, randomized, placebo-controlled, double-blind study will enroll 376 patients across eleven NAPKON locations in Germany.
  • Researchers aim to identify prognostic biomarkers to tailor PCS treatment strategies and improve patient outcomes.

Underrepresentation of Minorities Found in Pediatric Ophthalmology Clinical Trials

  • A cross-sectional analysis of 41 clinical studies from 1997-2022 revealed underrepresentation of Hispanic, Black, and Asian participants in pediatric ophthalmology research.
  • White participants were overrepresented in Pediatric Eye Disease Investigator Group (PEDIG) studies, while female participants were proportionally represented.
  • Enrollment trends indicate a decrease in White and Asian participant enrollment and an increase in Hispanic participant enrollment over the study period.
  • The findings highlight disparities in clinical study enrollment and suggest a need for improved diversity in pediatric ophthalmology research.

Hoth Therapeutics Expands HT-001 Phase 2a Trial for EGFRi-Induced Skin Toxicities

  • Hoth Therapeutics has received IRB approval from Montefiore Medical Center and Dana-Farber Cancer Institute for its Phase 2a trial of HT-001.
  • The Phase 2a trial will assess the efficacy, safety, and tolerability of topical HT-001 for skin toxicities related to EGFR inhibitor treatments.
  • This First-in-Human trial aims to provide a new therapeutic option for cancer patients suffering from EGFRi-associated skin toxicities.
  • Hoth Therapeutics reports a strong cash position with over $9 million and no planned capital raise for the remainder of 2024.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.