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The Safety and Efficacy Evaluation of RM-001 for the Treatment of Transfusions Dependent β-Thalassaemia Major in Clinical Trials

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Trial Profile

The Safety and Efficacy Evaluation of RM-001 for the Treatment of Transfusions Dependent β-Thalassaemia Major in Clinical Trials

Status: Recruiting
Phase of Trial: Phase 0

Latest Information Update: 26 Sep 2024

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At a glance

  • Drugs RM 001 Reforgene Medicine (Primary)
  • Indications Beta-thalassaemia
  • Focus First in man; Therapeutic Use

    • Most Recent Events

    • 12 Dec 2023 Results (n=7, As of July 31, 2023) from ChiCTR2100053406 and ChiCTR2100052858 assessing safety and efficacy results from 7 patients that have been dosed with RM-001 were presented at the 65th American Society of Hematology Annual Meeting and Exposition.
    • 07 Aug 2023 New trial record
    • 15 Jun 2023 Results assessing safety and efficacy from patients that received RM-001 infusion from two ongoing clinical trials (ChiCTR2100053406 & ChiCTR2100052858), including a recent treated transfusion-dependent beta -thalassemia patient who also carries two alpha-globin genes deletion presented at the 28th Congress of the European Haematology Association.

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