According to Ractigen Therapeutics, this study is an open-label, single-arm, dose-escalation study designed to evaluate the safety, tolerance, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of RAG-18 in pediatric patients with Duchenne Muscular Dystrophy (DMD).
The study will enroll approximately 12 subjects into four cohorts to assess the safety and tolerability of ascending intravenous doses. Secondary objectives include characterizing the pharmacokinetics (PK)/pharmacodynamics (PD) profile and assessing exploratory efficacy through changes in muscle biomarkers, muscle composition, cardiac/pulmonary function, and motor performance. The decision to escalate to the next dose level will be based on a comprehensive safety evaluation of the preceding cohort. Read More: Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing saRNA Therapeutic for Duchenne Muscular Dystrophy
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What is RAG-18 saRNA?
RAG-18 is a first of its kind saRNA candidate designed to specifically target and activate UTRN gene expression in muscle cells via RNAa mechanism. The utrophin protein encoded by the UTRN gene is structurally and functionally similar to dystrophin, and its upregulation could potentially serve as a functional replacement for the missing dystrophin in DMD muscle cells, providing treatment for all DMD patients regardless of the specific mutation location.
RAG-18 saRNA Early Phase 1 – NCT07282652
An Investigator-Initiated Study to Evaluate the Safety/Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy.
Study Start (Estimated): 2025-12
Primary Completion (Estimated): 2026-11
Study Completion (Estimated): 2026-11
Number of Participants (Estimated): 12
Study Type: Interventional
Location: China
Ages Eligible for Study: 4 Years to 15 Years (Child)
Principal inclusion and exclusion criteria. – NCT07282652 –
How to Participate in the RAG-18 saRNA Clinical Trial
To participate in the RAG-18 saRNA Early Phase 1 clinical trial, you can use the contact information below. Learn More: How to Participate in Clinical Trials for Duchenne (DMD)?
Name: Yi Dai
Phone Number: +86 138 111 77 531
Email: [email protected]
Read More: Clinical Trials for Duchenne (List of All Researches)
Merhaba dmd kas hastası Halil kerim ve mert eymen için bizde denemelere katılmak istiyoruz gerekli incelemenin acilen yapılması önemle az olunur