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A Proof of Concept Clinical Trial to Investigate OTL-300 in Transfusion-Dependent Beta-Thalassemia Patients

Trial Profile

A Proof of Concept Clinical Trial to Investigate OTL-300 in Transfusion-Dependent Beta-Thalassemia Patients

Recruiting
Phase of Trial: Phase I

Latest Information Update: 22 Jan 2019

At a glance

  • Drugs OTL 300 (Primary)
  • Indications Beta-thalassaemia
  • Focus Adverse reactions; Proof of concept; Therapeutic Use
  • Most Recent Events

    • 22 Jan 2019 According to an Orchard Therapeutics media release, this trial with the clinical coordination of doctor Sarah Marktel of SR-Tiget, has involved other Italian centers specializing in thalassemia and the cooperation of patient organizations.
    • 22 Jan 2019 According to an Orchard Therapeutics media release, the company anticipates reporting data from all nine patients in this proof of concept trial later this year.
    • 22 Jan 2019 According to an Orchard Therapeutics media release, interim analysis of long-term efficacy outcomes in seven of the nine treated patients with more than one year of follow up from this ongoing trial were published in Nature Medicine Journal.
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